Clinical Trials and Studies
| Trial/Study Name | Topic | Study Type | MTG | ORPHACode | Trial/study ID | Link |
|---|---|---|---|---|---|---|
| From New List: Study of Safety and Efficacy of CFZ533 in Type 1 Diabetes Pediatric and Young Adult Subjects (CCFZ533X2207) | Type 1 Diabetes Mellitus | clinical | MTG3 |
| NCT04129528 | Info |
| Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial (OTBB3-FU) | Prader-Willi Syndrome | clinical | MTG5 |
| Number EudraCT 2020-006161-11 | Info |
| OXYTOCIN TREATMENT IN NEONATES AND INFANTS AGED FROM 0 TO 3 MONTHS WITH PRADER-WILLI SYNDROME: A STUDY OF THE SAFETY AND EFFICACY ON ORAL AND SOCIAL SKILLS AND FEEDING BEHAVIOR OF INTRANASAL ADMINISTRATIONS OF OXYTOCIN VS. PLACEBO (PHASE III CLINICAL TRIAL) | Prader-Willi Syndrome | clinical | MTG5 |
| Number EudraCT 2019-002385- 12 | Info |
| OTBB3 Long-term Interventional Follow-up Study up to 4 Years of Age of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial and Comparison With an Untreated Cohort of Children With Prader-Willi Syndrome | Prader-Willi Syndrome | clinical | MTG5 |
| NCT05032326 | Info |
| A Phase 2 Study to Evaluate the Efficacy and Safety of Belzutifan (MK-6482, Formerly PT2977) Monotherapy in Participants With Advanced Pheochromocytoma/Paraganglioma (PPGL) or Pancreatic Neuroendocrine Tumor (pNET) | Pheochromocytoma/Paraganglioma, pancreatic neuroendocrine tumors | clinical | MTG1 MTG4 |
| NCT04924075, MK6482- PN015 | Info |
| An Open-label, Ascending Multiple-dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis Imperfecta | Osteogenesis Imperfecta | clinical |
| NCT04545554 | Info | |
| A prospective, randomised, Controlled, Open-label, Multicentre phase III study to evaluate efficacy and safety of Peptide Receptor Radionuclide Therapy (PRRT) with 177Lu-Edotreotide compared to targeted molecular therapy with Everolimus in patients with inoperable, progressive, somato-statin receptor-positive (SSTR+), neuroendocrine tumours of gastroenteric or pancreatic origin (GEP-NET). | Neuroendocrine Tumors | clinical | MTG4 |
| NCT03049189, ITM-LET-01-COMPETE | Info |
| PRIME Precision Radiotherapy Using MR-linac for Pancreatic Neuroendocrine Tumours in MEN1 Patients | Multiple Endocrine Neoplasia Type 1 | clinical | MTG4 |
| NCT05037461 | Info |
| Libretto-531 study | Medullary Thyroid Cancer | clinical | MTG8 |
| 2019-001978-28, J2G-MC-JZJB | Info |
| A phase III multi-center, randomized, open-label study to evaluate the efficacy and safety of Lutathera in patients with, Grade 2 and Grade 3 advanced GEP-NET (NETTER-2 study) | Gastro-enteropancreatic Neuroendocrine Tumor | clinical | MTG4 |
| NCT03972488, CAAA601A22301/NETTER-2 | Info |
| DSDCare: Standardized center-centric care of DSD across the lifespan | DSD | clinical | MTG7 |
| DRKS00022521 | Info |
| A randomized, placebo-controlled, double-blind study to evaluate safety and dose dependent clinical efficacy of APO-2 at three different doses in patients with diabetic foot ulcer (MARSYAS II) | Diabetic Foot Ulcer (DFU) | clinical | MTG3 |
| NCT04277598, MARSYAS II, APO-2 | Info |
| ACC: ADIUVO II o ACACIA: Mitotane With or Without Cisplatin and Etoposide After Surgery in Treating Participants With Stage I-III Adrenocortical Cancer With High Risk of Recurrence | Adrenocortical Carcinoma | clinical | MTG1 |
| NCT03723941 | Info |
| A phase IV, prospective, randomised single-blind UK multicentre non-inferiority trial of low-dose versus standard dose rituximab for prevention of relapses in acquired TTP | Acquired Thrombotic Thrombocytopenic Purpura | clinical | EudraCT Number: 2017-001117-86 S | Info | ||
| Glucocorticoid Receptor Antagonism in the Treatment of Cushing Syndrome (GRACE): A Phase 3, Double-Blind, Placebo-Controlled, Randomized-Withdrawal Study of the Efficacy and Safety of Relacorilant | Cushing Syndrome | clinical | MTG1 MTG6 |
| NCT03697109, CORT125134-455,NL68116.078.18 | Info |
| GRACE-2 | Cushing Syndrome | clinical | MTG1 MTG6 |
| NCT03604198, CORT125134-452, 2018-001616-30 | Info |
| An Open Label, Multi-center Pasireotide Roll-over Protocol for Patients Who Have Completed a Previous Novartis-sponsored Pasireotide Study and Are Judged by the Investigator to Benefit From Continued Pasireotide Treatment | Cushing Disease and Acromegaly | clinical | MTG6 |
| NCT01794793 | Info |
| Europea Observational study in Acromegaly – Acronis Study | Acromegaly | clinical | MTG6 |
| CSOM230CIC05 | Info |
| A Trial to Assess the Long-term Safety of Octreotide Subcutaneous Depot in Patients With Acromegaly | Acromegaly | clinical | MTG6 |
| NCT04125836 (HS-19-647) | Info |
| A Phase 3, randomized, double-blind, placebo-controlled, multi-center trial to assess efficacy and safety of octreotide subcutaneous depot(CAM2029) in patients with acromegaly. | Acromegaly | clinical | MTG6 |
| NCT04076462 (HS-18-633) | Info |
| A double-blind, placebo-controlled, randomized withdrawal study to evaluate the safety, pharmacokinetics and efficacy of CRN00808 in patients with acromegaly that are responders to octreotide lar or lanreotide depot (ACROBAT EVOLVE) | Acromegaly | clinical | MTG6 |
| NCT03792555 | Info |
| An open label exploratory study to evaluate the safety, pharmacokinetics and efficacy of CRN00808 in patients with acromegaly treated with somatostatin analogue based treatment regimens (ACROBAT EDGE) | Acromegaly | clinical | MTG6 |
| NCT03789656 | Info |
| Efficacy and Safety of Octreotide Capsules (MYCAPSSA) in Acromegaly | Acromegaly | clinical | MTG6 |
| NCT03252353 | Info |
| Comparison of Oral Octreotide Capsules to Injectable Somatostatin Analogs in Acromegaly (MPOWERED). | Acromegaly | clinical | MTG6 |
| NCT02685709 | Info |
| A phase II, multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing’s disease | Cushing Disease | clinical | MTG6 |
| NCT03708900 | Info |
| Phase III, Multi-center, Double-blind, Randomized Withdrawal Study of LCI699 Following a 24 Week, Single-arm, Open-label Dose Titration and Treatment Period to Evaluate the Safety and Efficacy of LCI699 for the Treatment of Patients With Cushing’s Disease | Cushing Disease | clinical | MTG6 |
| NCT02180217 | Info |
| A proof of concept, open-label, forced titration, multi-center study to assess the safety/tolerability and efficacy of 10-weeks treatment of LCI699 followed by a 12-week treatment period in patients with Cushing’s disease | Cushing Disease | clinical | MTG6 |
| NCT01331239 | Info |
| Efficacy and Safety Evaluation of Osilodrostat in Cushing’s Disease (LINC-4) | Cushing Disease | clinical | MTG6 |
| NCT02697734 | Info |
| Registre belge des enfants traités par hormone de croissance BELGROW | Growth Hormone Deficiency | clinical | MTG6 |
| BELGROW | Info |
| A PHASE 3, RANDOMIZED, MULTICENTER, OPEN-LABEL, CROSSOVER STUDY ASSESSING SUBJECT PERCEPTION OF TREATMENT BURDEN WITH USE OF WEEKLY GROWTH HORMONE (SOMATROGON) VERSUS DAILY GROWTH HORMONE (GENOTROPIN (REGISTERED)) INJECTIONS IN CHILDREN WITH GROWTH HORMONE DEFICIENCY | Growth Hormone Deficiency | clinical | MTG6 |
| NCT03831880 | Info |
| A trial comparing the effect and safety of once weekly dosing of somapacitan with daily Norditropin® in children with growth hormone deficiency (REAL 4) | Growth Hormone Deficiency | clinical | MTG6 |
| NCT03811535 | Info |
| TransCon hGH 301-CT Ext (1) Enlighten (weekly GH/Transcon | Growth Hormone Deficiency | clinical | MTG6 |
| NCT03344458 | Info |
| Safety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children | Growth Hormone Deficiency | clinical | MTG6 |
| NCT02968004 | Info |
| A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD) | Growth Hormone Deficiency | clinical | MTG6 |
| NCT02781727 | Info |
| A phase 3 trial evaluating the effect of long acting GH (MOD-4023) in patients with GHD. CP-4-005 | Growth Hormone Deficiency | clinical | MTG6 |
| NCT01909479 | Info |
| A Phase II, Dose and Frequency Finding Study of MOD-4023 in Growth Hormone Deficient Adults (GHDA) | Growth Hormone Deficiency | clinical | MTG6 |
| MOD-4023 (2) - OLE | Info |
| A Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency (foresiGHt) | Growth Hormone Deficiency | clinical | MTG6 |
| NCT04615273 (TCH-306) | Info |
| Safety and Efficacy Study of MOD-4023 to Treat Children With Growth Hormone Deficiency | Growth Hormone Deficiency | clinical | MTG6 |
| NCT03874013 | Info |
| Investigating Efficacy and Safety of Once-weekly NNC0195-0092 (Somapacitan) Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency | Growth Hormone Deficiency | clinical | MTG6 |
| NCT02616562 | Info |
| A phase 1/2, open-label, multicenter, non-randomized study to assess the safety, tolerability, pharmacokinetics and efficacy of Burosumab in pediatric patients from birth to less than 1 year of age with X-linked hypophosphatemia (XLH). BUR-CL207 | X-linked hypophosphatemia | clinical | MTG2 |
| NCT04188964 | Info |
| X linked Hypof (XLH) Registry for Patients With X-Linked Hypophosphatemia (XLH Registry) | X-Linked Hypophosphatemia | clinical | MTG2 |
| NCT03193476 | Info |
| Study of Efficacy and Safety of PDR001 in Patients With Advanced or Metastatic, Well-differentiated, Non-functional Neuroendocrine Tumors of Pancreatic, Gastrointestinal (GI), or Thoracic Origin or Poorly-differentiated Gastroenteropancreatic Neuroendocrine Carcinoma (GEP-NEC) | Neuroendocrine Tumours | clinical | MTG4 |
| NCT02955069 | Info |
| Optimising Steroid Replacement in Patients With Adrenal Insufficiency (Plenadren) | Adrenal Insufficiency | clinical | MTG1 |
| NCT03282487 | Info |
| Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome (OTBB3) | Prader-Willi Syndrome | clinical | MTG5 |
| NCT04283578 | Info |
| ZEPHYR Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome (ZEPHYR | Prader-Willi Syndrome | clinical | MTG5 |
| NCT03790865 | Info |
| Effect of Liraglutide for Weight Management in Paediatric Subjects With Prader-Willi Syndrome | Prader-Willi Syndrome | clinical | MTG5 |
| NCT02527200 | Info |
| RM-493-22 (A Phase 2, Two-Stage (Open-Label Run-in Followed by Randomized Withdrawal), Double-Blind, Placebo-Controlled Study of Setmelanotide in Patients With Specific Gene Defects in the Melanocortin-4 Receptor Pathway) | Genetic Obesity | clinical | MTG5 MTG6 |
| NCT04963231 | Info |
| Long Term Extension Trial of Setmelanotide | Genetic Obesity | clinical | MTG5 MTG6 |
| NCT 03013543 | Info |
| Setmelanotide for the Treatment of Early-Onset POMC Deficiency Obesity | Genetic Obesity | clinical | MTG5 MTG6 |
| NCT 02896192 | Info |
| An Open-Label Multiple-Dose Study of RZ358 in Patients with Congenital Hyperinsulinism, Protocol RZ358-606 | Congenital Hyperinsulinism | clinical | MTG3 |
| NCT04538989,RZ358-606 | Info |
| Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism | Congenital Hyperinsulinism | clinical | MTG3 |
| NCT04172441 | Info |
| An Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism | Congenital Hyperinsulinism | clinical | MTG3 |
| NCT03941236 | Info |
| A Two-Period, Open-label Trial Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism | Congenital Hyperinsulinism | clinical | MTG3 |
| NCT03777176 | Info |
| An Open-Label Multiple-Dose Study of RZ358 in Patients with Congenital Hyperinsulinism”. Estudio abierto de dosis múltiples de RZ358 en pacientes con hiperinsulinismo congénito. | Congenital Hyperinsulinism | clinical | MTG3 |
| EudraCT 2016-004186-83,CODIGO RZ358-606. RZ358 - 606 | Info |
| Triac Trial II in MCT8 Deficiency Patients | MCT8 Deficiency | clinical | MTG8 |
| NCT02396459 | Info |
| Thyroid Hormone Analog Therapy in MCT8 Deficiency: Triac Trial Patients | MCT8 Deficiency | clinical | MTG8 |
| NCT02060474 | Info |
| MCRN007 (D6992C00044) – An Open-Label, Non-Comparative Trial To Evaluate The Safety, Efficacy And Pharmacokinetics Of FASLODEX™ (Fulvestrant) In Girls With Progressive Precocious Puberty Associated With Mccune Albright Syndrome | McCune- Albright Syndrome | clinical | MTG1 MTG2 MTG6 MTG8 |
| NCT00278915 | Info |
| An Open-Label Extension Study of Levoketoconazole (2S,4R-ketoconazole) in the Treatment of Endogenous Cushing’s Syndrome | Cushing Syndrome | clinical | MTG1 MTG6 |
| NCT03621280, COR-2017-OLE | Info |
| An Open-label, Multi-center, Roll-over Study to Assess Long Term Safety in Patients With Endogenous Cushing’s Syndrome Who Have Completed a Prior Novartis-sponsored Osilodrostat (LCI699) Study and Are Judged by the Investigator to Benefit From Continued Treatment With Osilodrostat | Cushing Syndrome | clinical | MTG1 MTG6 |
| NCT03606408 | Info |
| LOGICS: A Study to Assess the Safety and Efficacy of Levoketoconazole in the Treatment of Endogenous Cushing’s Syndrome (Levoketoconazole). | Cushing Syndrome | clinical | MTG1 MTG6 |
| NCT03277690 | Info |
| Efficacy and Safety of Exenatide in the Treatment of Hypothalamic Obesity After Craniopharyngioma Therapy (CRANIOEXE) | Hypothalamic Obesity | clinical | MTG6 |
| NCT02860923 | Info |
| GRADIENT; A phase 3, randomized, double-blind, placebo-controlled study of the efficacy and safety of Relacorilant | Adrenal Tumours | clinical | MTG1 |
| NCT04308590 | Info |
| Structured Evaluation of adRENal Tumors Discovered Incidentally – Prospectively Investigating the Testing Yield (SERENDIPITY) | Adrenal Tumours | clinical | MTG1 |
| NCT02324647 | Info |
| Combined 18F-Fluorodeoxyglucose (FDG) Positron Emission Tomography (PET) and 123I-Iodometomidate (123I-IMTO) Imaging for Adrenal Neoplasia. | Adrenal Tumours | clinical | MTG1 |
| NCT02010957 | Info |
| FAMAIN | Adrenal Tumours | clinical | MTG1 |
| EudraCT Number: 2012-003604-13 | Info |
| CHIRACIC -Surgery of Subclinical Cortisol Secreting Adrenal Incidentalomas (CHIRACIC) | Adrenal Tumours | clinical | MTG1 |
| NCT02364089 | Info |
| Growing up With the Young Endocrine Support System (YESS!) (YESS) | Transition,paediatric chronic endocrine disorders | clinical | MTG1 MTG5 MTG6 MTG7 MTG8 |
| NCT04252001 | Info |
| A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects With Classic Congenital Adrenal Hyperplasia | Congenital Adrenal Hyperplasia | clinical | MTG1 |
| NCT04544410 | Info |
| CAHtalyst study – A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects with Classic Congenital Adrenal Hyperplasia, | Congenital Adrenal Hyperplasia | clinical | MTG1 |
| NCT04490915,NBI-74788-CAH3003 | Info |
| A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects With Classic Congenital Adrenal Hyperplasia | Congenital Adrenal Hyperplasia | clinical | MTG1 |
| NCT04457336 | Info |
| DIUR-006 : A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAH Diurnal Limited | Congenital Adrenal Hyperplasia | clinical | MTG1 |
| NCT03062280,EudraCT Number: 2015-005448-32 | Info |
| A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment – Protocollo: NBI- 74788-CAH2006 – Codice EudraCT: 2020-004381-19 – Promosso da: Neurocrine Biosciences, Inc.- riferimento pratica CE: 589/2021/Farm/AOUBo | Congenital Adrenal Hyperplasia | clinical | MTG1 |
| NCT04806451, NBI-74788-CAH2006 | Info |
| A Phase 2, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia | Congenital Adrenal Hyperplasia | clinical | MTG1 |
| NCT04045145,NBI-74788 | Info |
| Open-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapy (RESTORE) | Congenital Adrenal Hyperplasia | clinical | MTG1 |
| NCT03532022 | Info |
| A Phase III study of efficacy, safety and tolerability of Chronocort® compared with standard glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasia | Congenital Adrenal Hyperplasia | clinical | MTG1 |
| NCT02716818 | Info |
| PREDEX | Congenital Adrenal Hyperplasia | clinical | MTG1 |
| 15554888 | Info |
| A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia | Familial Hypercholesterolemia | clinical |
| EudraCT Number: 2011-005400-15 | Info | |
| Treatment Trial (in preparation): An international, randomised, double-blind, efficacy and safety trial of GLP-1 receptor agonist (e.g. Liraglutide) treatment in paediatric and adult patients with Wolfram Syndrome to control neurodegeneration and hyperglycaemia | Wolfram Syndrome | clinical | MTG3 |
| NCT03717909 | Info |
| INSPECT (Investigation into PHTS tumor syndrome) | PHTS tumour syndrome | clinical | MTG4 |
| 2018-5056 | Info |
| PROLACT A Phase III study of efficacy, safety and tolerability of Chronocort® compared with standard glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasia | Prolactinoma | clinical | MTG6 |
| NCT04107480 | Info |
| A study of MK-6482 Monotherapy in advanced Pheochromocytoma/Paraganglioma (PPGL) and pancreatic neuroendocrine tumors (pNET) | Pheochromocytoma/Paraganglioma, Pancreatic Neuroendocrine Tumours | clinical | MTG1 MTG4 |
| MK6482-PN015 | Info |
| FIRSTMAPPP, Genetic variants associated with increased susceptibility to thyroid cancer | Pheochromocytoma and Paraganglioma | clinical | MTG1 MTG4 |
| NCT01371201 | Info |
| Multicenter Pheochromocytoma and Paraganglioma Evaluation (MUPPET) | Pheochromocytoma and Paraganglioma | clinical | MTG1 MTG4 |
| NCT03344016 | Info |
| PRESCRIPT – Phenoxybenzamine Versus Doxazosin in PCC Patients | Pheochromocytoma | clinical | MTG1 MTG4 |
| NCT01379898 | Info |
| DP13 – A Phase II Study in Patients With Primary Aldosteronism | Primary Aldosteronism | clinical | MTG1 |
| NCT04007406 | Info |
| A Randomized, Double-Blind, Multiple Ascending Oral Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of PCO371 in Patients With Hypoparathyroidism | Hypoparathyroidism | clinical | MTG2 |
| NCT04209179 | Info |
| A Study to Learn if Recombinant Human Parathyroid Hormone [rhPTH(1-84)] Can Improve Symptoms and Metabolic Control in Adults With Hypoparathyroidism (BALANCE) | Hypoparathyroidism | clinical | MTG2 |
| NCT03324880 | Info |
| A Clinical Study Investigating the Safety, Tolerability, PK and PD of PCO371 in Patients With Hypoparathyroidism – PCO 104 UG | Hypoparathyroidism | clinical | MTG2 |
| NCT 04209179 | Info |
| LA Study Comparing LY900014 to Insulin Lispro (Humalog) in Adults With Type 1 Diabetes Using Insulin Pump Therapy (PRONTO-Pump-2) | Type 1 Diabetes Mellitus | clinical | MTG3 |
| NCT03830281 | Info |
| Setmelanotide for the Treatment of LEPR Deficiency Obesity | Genetic Obesity | clinical | MTG5 MTG6 |
| NCT03287960 | Info |
| CARGOx – Use of Copeptin Measurement After Arginine Infusion for the Differential Diagnosis of Diabetes Insipidus – the CARGOx Study (CARGOx) | Diabetes Insipidus | clinical | MTG6 |
| NCT03572166 | Info |
| Efficacy of Adjuvant Mitotane Treatment (ADIUVO) (ADIUVO) | Adrenocortical carcinoma | clinical | MTG1 |
| NCT00777244 | Info |
| A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | Achondroplasia | clinical | MTG5 |
| NCT03197766 | Info |
| FDG-PET/CT in Evaluation of Cytological Indeterminate Thyroid Nodules to Prevent Unnecessary Surgery (EfFECTS) (EfFECTS) | Thyroid Neoplasms | clinical | MTG8 |
| NCT02208544 | Info |
| Focus on Values to Stimulate Shared Decisions in Patients With Thyroid Cancer: A Multifaceted COMmunication BOoster (COMBO)s | Thyroid Carcinoma | clinical | MTG8 |
| NCT03905369 | Info |
| Lenvatinib | Thyroid Carcinoma | clinical | MTG8 |
| 2014-005199-27 | Info |
| NEO adjuvant Treatment in Anaplastic Cancer of the Thyroid (NEO-TACT study) (in preparation) Clinical Study: Focus on values to stimulate shared decisions in patients with thyroid cancer: A multifaceted COMmunication Booster (COMBO). | Anaplastic Thyroid Carcinoma | clinical | MTG8 |
| NCT01099436 | Info |
| Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Patients With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors (ARROW) | Thyroid Carcinoma | clinical | MTG8 |
| NCT03037385 | Info |
| A Multicenter, Randomized, Open-label, Phase 3 Trial Comparing Selpercatinib to Physicians Choice of Cabozantinib or Vandetanib in Patients With Progressive, Advanced, Kinase Inhibitor Naïve, RET-Mutant Medullary Thyroid Cancer (LIBRETTO-531) | Medullary Thyroid Carcinoma | clinical | MTG8 |
| NCT04211337,EUDRACT: 2019-001978-28 | Info |
| A Study of Two Different Doses of Cabozantinib (XL184) in Progressive, Metastatic Medullary Thyroid Cancer (EXAMINER) | Medullary Thyroid Carcinoma | clinical | MTG8 |
| NCT01896479 | Info |
| A European Multi-Centre, Post-Authorization, Observation Study (Registry) of Patients with Chronic Adrenal Insufficiency | Chronic Adrenal Insufficiency | clinical | MTG1 |
| NCT01661387 | Info |
| Efficacy and Safety of Lanreotide Autogel (ATG) in Combination With Temozolomide in Subjects With Thoracic Neuroendocrine Tumors. (ATLANT) | Neuroendocrine Tumors | clinical | MTG4 |
| NCT02698410 | Info |
| Efficacy and Safety of Lanreotide Autogel/ Depot 120 mg vs. Placebo in Subjects With Lung Neuroendocrine Tumors (SPINET) | Neuroendocrine Tumors | clinical | MTG4 |
| NCT02683941 | Info |
| Efficacy and Safety Study in Pancreatic or Midgut Neuroendocrine Tumours Having Progressed Radiologically While Previously Treated With Lanreotide Autogel® 120 mg (CLARINET FORTE) | Neuroendocrine Tumours | clinical | MTG4 |
| NCT02651987 | Info |
| Studio multicentrico, randomizzato, a bracci paralleli, controllato con placebo (in doppio cieco) e con controllo attivo (in aperto) per confrontare l’efficacia e la sicurezza di lonapegsomatropina una volta alla settimana con il placebo e un prodotto a base di somatropina una volta al giorno in adulti con deficit dell’ormone della crescita / A Phase 3 Randomized, Double-blind Study to Evaluate the Efficacy and Safety of Apremilast (CC-10004) in Subjects With Active Behçet’s Disease | Behçet's Disease | clinical |
| NCT02307513 | Info | |
| Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity | Genetic Obesity | clinical | MTG5 MTG6 |
| NCT03651765 | Info |
| Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity Phase 3 | Bardet Biedl Syndrome (BBS) Alström Syndrome (AS) | clinical | MTG5 MTG6 |
| NCT03746522 | Info |
| Selenium supplementation for patients with Graves’ hyperthyroidism (the GRASS trial) | Graves’ hyperthyroidism | clinical | MTG8 | NCT01611896 | Info | |
| Personalize My Treatment (PMT) Registry (PMT) | Biobanking In cancer patients | clinical | NCT02355171 | Info | ||
| Effect of Insulin Sensitization on IGF-1 Response to Growth Hormone in SGA Children | Small for Gestational Age | clinical | MTG5 | NCT02931591 | Info | |
| REAL 5 | Small for Gestational Age | clinical | MTG5 | 2018-000232-10 | Info | |
| The Diurnal Rhythm in Natriuretic Peptide Levels and Relationship With Nocturnal Blood Pressure | Natriuretic Peptides,Obesity | clinical | MTG3 | NCT03834168 | Info | |
| A Phase 3b, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of TRC101 in Delaying Chronic Kidney Disease Progression in Subjects with Metabolic Acidosis | Metabolic Acidosis | clinical | 2018-001303-36 | Info | ||
| A 4 Year Combination Therapy of Growth Hormone and (GnRH) Agonist in Children With a Short Predicted Height (ZomaTrip) | Idiopathic Short Stature | clinical | MTG5 | NCT00840944 | Info | |
| NOVARTIS SOM B2412 roll over | Cluster Headache - Episodic and Chronic | clinical | NCT02619617 | Info | ||
| Bacteremia Antibiotic Length Actually Needed for Clinical Effectiveness: Randomized Controlled Trial | Bacterial risk | clinical | NCT03005145 | Info | ||
| A Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled Clinical Study to Evaluate the Safety and Efficacy of Ertugliflozin (MK-8835/PF-04971729) in Pediatric Participants (ages 10 to 17 years, inclusive) with Type 2 Diabetes Mellitus | Type 2 Diabetes Mellitus | clinical | MTG3 |
| NCT04029480,MK8835-059 | Info |
| Diabetes Study of Linagliptin and Empagliflozin in Children and Adolescents (DINAMO)TM | Type 2 Diabetes Mellitus | clinical | MTG3 |
| NCT03429543 | Info |
| A Phase 3, Randomized, Double-Blind, Multinational, Placebo-Controlled Study to Evaluate Efficacy and Safety of Teplizumab (PRV-031), a Humanized, FcR Non-Binding, anti-CD3 Monoclonal Antibody, in Children and Adolescents with Newly Diagnosed Type 1 Diabetes (T1D) | Type 1 Diabetes Mellitus | clinical | MTG3 |
| NCT03875729,PRV-031-001 | Info |
| The Artificial Pancreas in Very Young Children With T1D (KidsAP02) | Type 1 Diabetes Mellitus | clinical | MTG3 |
| NCT03784027 | Info |
| A Study Comparing LY900014 to Insulin Lispro (Humalog) in Children and Adolescents With Type 1 Diabetes (PRONTO-Peds) | Type 1 Diabetes Mellitus | clinical | MTG3 |
| NCT03740919 | Info |
| Phase 3, Randomized, Double-blind, Placebo/Active-controlled, Parallel-arm Trial to Assess Efficacy, Safety, and Pharmacokinetics of Dasiglucagon Relative to Placebo/GlucaGen® as Rescue Therapy for Severe Hypoglycemia in Children With T1DM Treated With Insulin | Type 1 Diabetes Mellitus | clinical | MTG3 |
| NCT03667053 | Info |
| Diagnode 2 – GAD-Alum (Diamyd) Administered Into Lymph Nodes in Combination With Vitamin D in Type 1 Diabetes | Type 1 Diabetes Mellitus | clinical | MTG3 |
| NCT03345004 | Info |
| FLAIR- Fuzzy Logic Automated Insulin Regulation: 3 A Crossover Study Comparing Two Automated Insulin Delivery System 4 Algorithms (PID vs. PID + Fuzzy Logic) in Individuals with Type 1 Diabetes | Type 1 Diabetes Mellitus | clinical | MTG3 |
| NCT03040414 | Info |
| Evaluation of the Dreamed Advisor Pro for Automated Insulin Pump Setting Adjustments in Children and Adolescents With Type 1 Diabetes- The Advice4U Pro Study | Type 1 Diabetes Mellitus | clinical | MTG3 |
| NCT03003806 | Info |
| A randomized, multicentre trial evaluating the efficacy and safety of fast-acting insulin aspart in continuous subcutaneous insulin infusion in adults with type 1 diabetes (onset 5). | Type 1 Diabetes Mellitus | clinical | MTG3 |
| NCT02825251 | Info |
| Initiative pour la Promotion de la Qualité et Epidémiologie chez les Enfants et Adolescents atteints du Diabète sucré (IPQE-EAD | Type 1 Diabetes Mellitus | clinical | MTG3 |
| IPQE-EAD,IKEKAD | Info |
| KidsAP02 | Type 1 Diabetes Mellitus | clinical | MTG3 |
| KIDSAP consortium | Info |
| Study of Safety and Efficacy of CFZ533 in Type 1 Diabetes Pediatric and Young Adult Subjects (CCFZ533X2207) | Type 1 Diabetes Mellitus | clinical | MTG3 |
| NCT04129528 | Info |
| ASCEND: A Study of Cardiovascular Events in Diabetes | Diabetes Mellitus | clinical | MTG3 |
| NCT00135226 | Info |
| Prevalence of overweight and obesity in children with X-linked hypophosphatemia | X-linked hypophosphataemia | observational | MTG2 |
| DOI:10.1530/EC-19-0481 | Info |
| Impact of Thyroid autoimmunity on Fertility outcome in intrauterine insemination (IUI) patients | Thyroid Autoimmunity | observational | MTG8 |
| 10.1093/humrep/dex033 | Info |
| Risk of malignancy of the thyroid nodule evaluated by scintigraphy: a single centre retrospective study | Thyroid Nodule | observational | MTG8 |
| 10.1530/endoabs.64.009 | Info |
| Validation of the diagnostic accuracy of the electronic nose in the detection of thyroid cancer. | Thyroid carcinoma | observational | MTG8 |
| NCT04883294 | Info |
| Impact of thyroid autoimmunity on cumulative delivery rates in in vitro fertilization/intracytoplasmic sperm injection patients | Thyroid Autoimmunity | observational | MTG8 |
| 10.1016/j.fertnstert.2016.03.011 | Info |
| Long-term safety and efficacy of Omnitrope®, a somatropin biosimilar, in children requiring growth hormone treatment: PATRO Children study | Somatropin treatment | observational | MTG5 |
| PMCID: PMC3593298 | Info |
| Genetic testing in inherited endocrine disorders: joint position paper of the European reference network on rare endocrine conditions (Endo-ERN) | Rare genetic endocrine disorders | observational | MTG1 MTG2 MTG3 MTG4 MTG5 MTG6 MTG7 MTG8 |
| 10.1186/s13023-020-01420-w | Info |
| COVID-19 Infections In People With Rare Endocrine Conditions | Rare Endocrine Conditions, COVID-19 | observational | MTG1 MTG2 MTG3 MTG4 MTG5 MTG6 MTG7 MTG8 |
| 10.1530/endoabs.73.OC14.5 | Info |
| Ensat-HT | Primary Aldosteronism, Pheochromocytoma/Paraganglioma, Cushing Syndrome | observational | MTG1 MTG6 |
| ens@t-ht, European Union’s Horizon 2020 - grant agreement No 633983 | Info |
| Circulating microRNAs in primary aldosteronism | Primary Aldosteronism | observational | MTG1 |
| DOI: 10.1530/endoabs.63.GP198 | Info |
| Optimizing mineralocorticoid replacement therapy in patients with congenital adrenal hyperplasia and Addison’s disease | Primary Adrenal Insufficiency | observational | MTG1 |
| DOI: 10.1530/endoabs.63.GP197 | Info |
| Prolactin levels in PWS | Prader-Willi Syndrome | observational | MTG5 |
| 10.3390/jcm10163613 | Info |
| Efficacy and Safety of Norditropin® (Somatropin) in Children With Prader-Willi Syndrome (PWS) | Prader-Willi Syndrome | observational | MTG5 |
| NCT00705172 | Info |
| The Molecular Registry of Pituitary Adenomas (REMAH): A bet of Spanish Endocrinology for the future of individualized medicine and translational research. | Pituitary Adenoma | observational | MTG6 |
| doi: 10.1016/j.endonu.2016.03.001. | Info |
| A Multicenter Epidemiological Study on Second Malignancy in Non-Syndromic Pheochromocytoma/Paraganglioma Patients in Italy | Non-Syndromic Pheochromocytoma/Paraganglioma | observational | MTG1 |
| 10.3390/cancers13225831 | Info |
| Development and Progression of Carcinoid Heart Disease in a Cohort of Adult Patients With Neuroendocrine Tumors Carcinoid Heart Disease (CHD): An Observational French Multi-Centre Cohort Study CRUSOE – NETs (CaRdiac UltraSonic OutcomEs – NETs) | Neuroendocrine Tumors | observational | MTG4 |
| NCT03498040 | Info |
| MEN 1 data dase / Impact of Delay in Diagnosis in Outcomes in MEN1: Results From the Dutch MEN1 Study Group | MEN1 | observational | MTG4 |
| DutchMEN study group (DMSG)/ doi.org/10.1210/clinem/dgaa800 | Info |
| Hypopituitarism ENEA Rare etiologies Observational Study (HEROS) | Hypopituitarism, Histiocytosis X, Sarcoidosis, Hypophysitis, Idiopatic Diabetes Insipidus | observational | MTG6 |
| HEROS | Info |
| An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP) | Hypophosphatasia (HPP) | observational | MTG2 |
| NCT02306720 | Info |
| A European Network for the Investigation of Gender Incongruence (ENIGI) | Hormonal Therapy in Transsexual Persons | observational | MTG7 |
| NCT01072825 | Info |
| NSEuroNet database | Genetic Short Stature | observational | MTG5 |
| NSEuroNet | Info |
| RIFTOS MKI – Radioactive Iodine reFractory Asymptomatic Patients in Differentiated Thyroid Cancer – an Observational Study to Assess the Use of Multikinase Inhibitors | Differentiated Thyroid Carcinoma | observational | MTG8 |
| NCT02303444 | Info |
| Low iodine diet study (LID) | Differentiated Thyroid Carcinoma | observational | MTG8 |
| DOI: 10.1210/clinem/dgab691 | Info |
| Long-Term Effects of Radioiodine Treatment on Female Fertility in Survivors of Childhood Differentiated Thyroid Carcinoma | Differentiated Thyroid Carcinoma | observational | MTG8 |
| doi: 10.1089/thy.2019.0560 | Info |
| Gonadectomy in conditions affecting sex development: a registry-based cohort study | Differences of Sex Development | observational | MTG7 |
| 10.1530/eje-20-1058, PMID: 33780351 | Info |
| Personalized medicine in diabetes: design and characterization of a high-performance genotyping tool that allows the classification of different diabetes | Diabetes Mellitus | observational | MTG3 |
| PID2019-107513RB-I00 | Info |
| The etiology and extent of impaired quality of life, fatigue and cognitive, affective and emotional dysfunction in patients with Cushing’s syndrome | Cushing Syndrome | observational | MTG1 MTG6 |
| NCT03211624 | Info |
| Non-interventional Study for the Generation of Long Term Safety and Efficacy Data of Pasireotide s.c. in Patients With Cushing’s Disease (Post-Authorization Safety Study) (SOM2030) | Cushing Disease | observational | MTG6 |
| NCT02310269 | Info |
| Clinical characteristics of children with Congenital Hyperinsulinism: Results from the European Registries for Rare Endocrine Conditions (EuRRECA)Project | Congential Hyperinsulinism | observational | MTG3 |
| ESPE & EuRRECa programme (https://eurreca.net/) | Info |
| Maximising the Utilisation of the Hyperinsulinism Global Registry (HIGR) [Max HIGR] | Congenital Hyperinsulinism | observational | MTG3 |
| MDBR-21-102-CHI | Info |
| International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia: data from the I-CAH registry | Congenital Adrenal Hyperplasia | observational | MTG1 |
| 10.1530/eje-20-1249, PMID: 33460392 | Info |
| CHRODIS PLUS, | Chronic disease | observational |
| CHRODIS+ | Info | |
| A European Post-Authorisation Observational Study (Registry) of Patients With Chronic Adrenal Insufficiency (AI) – EU-AIR | Chronic Adrenal Insufficiency | observational | MTG1 |
| NCT01661387 | Info |
| Follow-up of pubertal development and fertility in boys who underwent testicular tissue biopsy for fertility preservation | Chromosomal DSD | observational | MTG7 |
| Info | |
| Identification of selective androgen receptor co-regulators on the basis of an in vitro model of androgen insensitivity syndrome | Androgen Insensitivity Syndrome | observational | MTG7 |
| HO 6028/2-1 | Info |
| Structured Evaluation of adRENal tumors Discovered Incidentally – Prospectively Investigating the Testing Yield- Serendipity Protocol NTR 4799 | Adrenal Tumours | observational | MTG1 |
| Serendipity Protocol NTR 4799 | Info |
| SERENDIPITY – Structured Evaluation of adRENal Tumors Discovered Incidentally – Prospectively Investigating the Testing Yield (SERENDIPITY) | Adrenal Tumours | observational | MTG1 |
| NCT02324647 | Info |
| ENSAT registry study | Adrenal tumours | observational | MTG1 |
| ENS@T | Info |
| A National Multi-Centre study on etiology and long term outcome of Primary Adrenal Insufficiency in children | Adrenal Insufficiency | observational | MTG1 |
| https://doi.org/10.1186/1472-6823-14-40 | Info |
| Primary Adrenal Insufficiency in Childhood: Data From a Large Nationwide Cohort/ A National Multicentre study on the frequence of adrenal crisis in children with adrenal insufficiency <6 years of age | Adrenal Insufficiency | observational | MTG1 |
| 10.1210/clinem/dgaa881 | Info |
| EOLIA | Acromegaly | observational | MTG6 |
| CSMS995BFR13 | Info |
| Development of ACRODAT®, a new software medical device to assess disease activity in patients with acromegaly. | Acromegaly | observational | MTG6 |
| DOI: 10.1007/s11102-017-0835-5 | Info |
| LAS. Liège Acromegaly Survey. A large prospective observational registry of patients with acromegaly. | Acromegaly | observational | MTG6 |
| doi: 10.1530/ERC-17-0253 | Info |
| A European observational study in patients with uncontrolled acromegaly who are being treated with long acting pasireotide | Acromegaly | observational | MTG6 |
| 10.1530/endoabs.56.GP8 | Info |
| Lifetime Impact of Achondroplasia Study in Europe-LIAISE, (NCT03449368) | Achondroplasia | observational | MTG5 |
| NCT03449368 | Info |
| SF1 next | 46,XY DSD | observational | MTG7 |
| Study ID 201810_GS | Info |
| Puberty in Females with PAIS | 46,XY DSD | observational | MTG7 |
| Study ID 201402_GGF | Info |
| Prevalence of overweight and obesity in children with X-linked hypophosphatemia | X-linked hypophosphataemia | observational | MTG2 MTG5 |
| 10.1530/EC-19-0481 | Info |
| International Multicentre Validation Study of SAGIT® Instrument in Acromegaly | Acromegaly | observational | MTG6 |
| NCT02539927 | Info |
| Registry for Patients With X-linked Hypophosphatemia (XLH Registry) | X-linked Hypophosphatemia | observational | MTG2 |
| NCT03193476 | Info |
| Diabetes-related antibody-testing is a valuable screening tool for diagnosing monogenic diabetes – a survey from the worldwide SWEET registry | Type 1 Diabetes | observational | MTG3 |
| NCT04427189 | Info |
| An Innovative Approach Towards Understanding and Arresting Type 1 Diabetes (INNODIA) (INNODIA) | Type 1 Diabetes | observational | MTG3 |
| NCT03936634 | Info |
| THETA: cycling from hypoThyroidism to Hyperthyroidism during Early treatment of differentiated Thyroid carcinomA | Thyroid carcinoma | observational | MTG8 |
| Trial NL7228 (NTR7427) | Info |
| Low iodine diet study (LID) | Thyroid cancer | observational | MTG8 |
| doi.org/10.1210/clinem/dgab691 | Info |
| Adrenal tropism of SARS-CoV-2 and adrenal findings in a post-mortem case series of patients with severe fatal COVID-19 | SARS-CoV-2, Adrenal | observational | MTG1 |
| doi.org/10.1038/s41467-022-29145-3 | Info |
| Oberar : Rare Obesity Cohorts With Food Behavioral Disorders : Better Diagnosis for Better Treatment | Obesity | observational | MTG5 |
| NCT04604626 | Info |
| MEN 1 data dase / Impact of Delay in Diagnosis in Outcomes in MEN1: Results From the Dutch MEN1 Study Group | MEN1 | observational | MTG4 |
| doi.org/10.1210/clinem/dgaa800 | Info |
| PARADIGHM (Physicians Advancing Disease Knowledge in Hypoparathyroidism): A Registry for Patients With Chronic Hypoparathyroidism | Hypoparathyroidism | observational | MTG2 |
| NCT01922440 | Info |
| Safety and effectiveness of replacement with biosimilar growth hormone in adults with growth hormone deficiency: results from an international, post-marketing surveillance study (PATRO Adults) | Growth Hormone Deficiency | observational | MTG5 |
| doi.org/10.1007/s11102-021-01139-2 | Info |
| Long term effects of radioiodine treatment in suvivors | Differentiated Thyroid Carcinoma | observational | MTG8 |
| 10.1089/thy.2019.0560 | Info |
| The etiology and extent of impaired quality of life, fatigue and cognitive, affective and emotional dysfunction in patients with Cushing’s syndrome | Cushing's Syndrome | observational | MTG1 MTG6 |
| BRAIN | Info |
| Complicushing: Cushing’s Disease Complications | Cushing's Disease | observational | MTG6 |
| NCT02568982 | Info |
| Clinical characteristics of children with Congenital Hyperinsulinism: Results from the European Registries for Rare Endocrine Conditions (EuRRECa) Project | Congenital Hyperinsulism | observational | MTG3 |
| doi.org/10.1159/000518849 | Info |
| Pathogenic and low-frequency variants in children with Central Precocious Puberty | Central Precocious Puberty | observational | MTG6 |
| doi.org/10.3389/fendo.2021.745048 | Info |
| ENSAT registry-based randomized clinical trials for adrenocortical carcinoma | Adrenal Tumours | observational | MTG1 |
| 10.1530/EJE-20-0800 | Info |
| Omics Signature in the Diagnosis of Hypertension (ENSAT-HT) | Hypertension | observational | MTG1 MTG4 |
| NCT02772315, P17-284/2018-4231 | Info |
| Trial/Study Name | Topic | Study Type | MTG | ORPHACode | Trial/study ID | Link |
