Clinical Trials & Studies

Clinical Trials and Studies

Filter list of Clinical Trials and Studies

Trial/Study NameTopicStudy TypeMTGORPHACodeTrial/study IDLink
From New List: Study of Safety and Efficacy of CFZ533 in Type 1 Diabetes Pediatric and Young Adult Subjects (CCFZ533X2207)Type 1 Diabetes MellitusclinicalMTG3
  • ORPHA181371
NCT04129528
Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial (OTBB3-FU)Prader-Willi SyndromeclinicalMTG5
  • ORPHA739
Number EudraCT 2020-006161-11
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 SyndromeclinicalMTG5
  • ORPHA739
Number EudraCT 2019-002385- 12
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 SyndromePrader-Willi SyndromeclinicalMTG5
  • ORPHA739
NCT05032326
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 tumorsclinicalMTG1 MTG4
  • ORPHA573163
  • ORPHA97253
NCT04924075, MK6482- PN015
An Open-label, Ascending Multiple-dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis ImperfectaOsteogenesis Imperfectaclinical
  • ORPHA666
NCT04545554
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 TumorsclinicalMTG4
  • ORPHA100092
  • ORPHA100075
  • ORPHA506052
NCT03049189, ITM-LET-01-COMPETE
PRIME Precision Radiotherapy Using MR-linac for Pancreatic Neuroendocrine Tumours in MEN1 PatientsMultiple Endocrine Neoplasia Type 1clinicalMTG4
  • OPRPHA652
NCT05037461
Libretto-531 studyMedullary Thyroid CancerclinicalMTG8
  • ORPHA1332
2019-001978-28, J2G-MC-JZJB
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 TumorclinicalMTG4
  • ORPHA100092
NCT03972488, CAAA601A22301/NETTER-2
DSDCare: Standardized center-centric care of DSD across the lifespanDSDclinicalMTG7
  • ORPHA90771
  • ORPHA2982
  • ORPHA98085
  • ORPHA325546
DRKS00022521
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)clinicalMTG3
  • ORPHA181371
  • ORPHA181376
NCT04277598, MARSYAS II, APO-2
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 RecurrenceAdrenocortical CarcinomaclinicalMTG1
  • ORPHA1501
NCT03723941
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 TTPAcquired Thrombotic Thrombocytopenic PurpuraclinicalEudraCT Number: 2017-001117-86 S
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 RelacorilantCushing SyndromeclinicalMTG1 MTG6
  • ORPHA99892
  • ORPHA96253
  • ORPHA99889
NCT03697109, CORT125134-455,NL68116.078.18
GRACE-2Cushing SyndromeclinicalMTG1 MTG6
  • ORPHA99892
  • ORPHA96253
  • ORPHA99889
NCT03604198, CORT125134-452, 2018-001616-30
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 TreatmentCushing Disease and AcromegalyclinicalMTG6
  • ORPHA963
NCT01794793
Europea Observational study in Acromegaly – Acronis StudyAcromegalyclinicalMTG6
  • ORPHA963
CSOM230CIC05
A Trial to Assess the Long-term Safety of Octreotide Subcutaneous Depot in Patients With AcromegalyAcromegalyclinicalMTG6
  • ORPHA963
NCT04125836 (HS-19-647)
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.AcromegalyclinicalMTG6
  • ORPHA963
NCT04076462 (HS-18-633)
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)AcromegalyclinicalMTG6
  • ORPHA963
NCT03792555
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)AcromegalyclinicalMTG6
  • ORPHA963
NCT03789656
Efficacy and Safety of Octreotide Capsules (MYCAPSSA) in AcromegalyAcromegalyclinicalMTG6
  • ORPHA963
NCT03252353
Comparison of Oral Octreotide Capsules to Injectable Somatostatin Analogs in Acromegaly (MPOWERED).AcromegalyclinicalMTG6
  • ORPHA963
NCT02685709
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 diseaseCushing DiseaseclinicalMTG6
  • ORPHA96253
NCT03708900
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 DiseaseCushing DiseaseclinicalMTG6
  • ORPHA96253
NCT02180217
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 diseaseCushing DiseaseclinicalMTG6
  • ORPHA96253
NCT01331239
Efficacy and Safety Evaluation of Osilodrostat in Cushing’s Disease (LINC-4)Cushing DiseaseclinicalMTG6
  • ORPHA96253
NCT02697734
Registre belge des enfants traités par hormone de croissance BELGROWGrowth Hormone DeficiencyclinicalMTG6
  • ORPHA95488
  • ORPHA314795
  • ORPHA364526
BELGROW
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 DEFICIENCYGrowth Hormone DeficiencyclinicalMTG6
  • ORPHA95488
NCT03831880
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 DeficiencyclinicalMTG6
  • ORPHA95488
NCT03811535
TransCon hGH 301-CT Ext (1) Enlighten (weekly GH/TransconGrowth Hormone DeficiencyclinicalMTG6
  • ORPHA95488
NCT03344458
Safety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient ChildrenGrowth Hormone DeficiencyclinicalMTG6
  • ORPHA95488
NCT02968004
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 DeficiencyclinicalMTG6
  • ORPHA95488
NCT02781727
A phase 3 trial evaluating the effect of long acting GH (MOD-4023) in patients with GHD. CP-4-005Growth Hormone DeficiencyclinicalMTG6
  • ORPHA95488
NCT01909479
A Phase II, Dose and Frequency Finding Study of MOD-4023 in Growth Hormone Deficient Adults (GHDA)Growth Hormone DeficiencyclinicalMTG6
  • ORPHA95488
MOD-4023 (2) - OLE
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 DeficiencyclinicalMTG6
  • ORPHA95488
NCT04615273 (TCH-306)
Safety and Efficacy Study of MOD-4023 to Treat Children With Growth Hormone DeficiencyGrowth Hormone DeficiencyclinicalMTG6
  • ORPHA95488
NCT03874013
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 DeficiencyGrowth Hormone DeficiencyclinicalMTG6
  • ORPHA95488
NCT02616562
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-CL207X-linked hypophosphatemiaclinicalMTG2
  • ORPHA89936
NCT04188964
X linked Hypof (XLH) Registry for Patients With X-Linked Hypophosphatemia (XLH Registry)X-Linked HypophosphatemiaclinicalMTG2
  • ORPHA89936
NCT03193476
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 TumoursclinicalMTG4
  • ORPHA877
  • ORPHA100092
  • ORPHA100101
NCT02955069
Optimising Steroid Replacement in Patients With Adrenal Insufficiency (Plenadren)Adrenal InsufficiencyclinicalMTG1
  • ORPHA85138
  • ORPHA199296
  • ORPHA
  • 199299
NCT03282487
Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome (OTBB3)Prader-Willi SyndromeclinicalMTG5
  • ORPHA739
NCT04283578
ZEPHYR Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome (ZEPHYRPrader-Willi SyndromeclinicalMTG5
  • ORPHA739
NCT03790865
Effect of Liraglutide for Weight Management in Paediatric Subjects With Prader-Willi SyndromePrader-Willi SyndromeclinicalMTG5
  • ORPHA739
NCT02527200
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 ObesityclinicalMTG5 MTG6
  • ORPHA71529
NCT04963231
Long Term Extension Trial of SetmelanotideGenetic ObesityclinicalMTG5 MTG6
  • ORPHA71526
NCT 03013543
Setmelanotide for the Treatment of Early-Onset POMC Deficiency ObesityGenetic ObesityclinicalMTG5 MTG6
  • ORPHA71526
NCT 02896192
An Open-Label Multiple-Dose Study of RZ358 in Patients with Congenital Hyperinsulinism, Protocol RZ358-606Congenital HyperinsulinismclinicalMTG3
  • ORPHA657
NCT04538989,RZ358-606
Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital HyperinsulinismCongenital HyperinsulinismclinicalMTG3
  • ORPHA657
NCT04172441
An Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon for the Treatment of Children with Congenital HyperinsulinismCongenital HyperinsulinismclinicalMTG3
  • ORPHA657
NCT03941236
A Two-Period, Open-label Trial Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children with Congenital HyperinsulinismCongenital HyperinsulinismclinicalMTG3
  • ORPHA657
NCT03777176
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 HyperinsulinismclinicalMTG3
  • ORPHA657
EudraCT 2016-004186-83,CODIGO RZ358-606. RZ358 - 606
Triac Trial II in MCT8 Deficiency PatientsMCT8 DeficiencyclinicalMTG8
  • ORPHA59
NCT02396459
Thyroid Hormone Analog Therapy in MCT8 Deficiency: Triac Trial PatientsMCT8 DeficiencyclinicalMTG8
  • ORPHA59
NCT02060474
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 SyndromeMcCune- Albright SyndromeclinicalMTG1 MTG2 MTG6 MTG8
  • ORPHA562
NCT00278915
An Open-Label Extension Study of Levoketoconazole (2S,4R-ketoconazole) in the Treatment of Endogenous Cushing’s SyndromeCushing SyndromeclinicalMTG1 MTG6
  • ORPHA553
  • ORPHA99892
  • ORPHA99893
NCT03621280, COR-2017-OLE
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 OsilodrostatCushing SyndromeclinicalMTG1 MTG6
  • ORPHA553
  • ORPHA99892
  • ORPHA99893
NCT03606408
LOGICS: A Study to Assess the Safety and Efficacy of Levoketoconazole in the Treatment of Endogenous Cushing’s Syndrome (Levoketoconazole).Cushing SyndromeclinicalMTG1 MTG6
  • ORPHA553
  • ORPHA99892
  • ORPHA99893
NCT03277690
Efficacy and Safety of Exenatide in the Treatment of Hypothalamic Obesity After Craniopharyngioma Therapy (CRANIOEXE)Hypothalamic ObesityclinicalMTG6
  • ORPHA54595
NCT02860923
GRADIENT; A phase 3, randomized, double-blind, placebo-controlled study of the efficacy and safety of RelacorilantAdrenal TumoursclinicalMTG1
  • ORPHA443287
  • ORPHA553
NCT04308590
Structured Evaluation of adRENal Tumors Discovered Incidentally – Prospectively Investigating the Testing Yield (SERENDIPITY)Adrenal TumoursclinicalMTG1
  • ORPHA443287
  • ORPHA1501
NCT02324647
Combined 18F-Fluorodeoxyglucose (FDG) Positron Emission Tomography (PET) and 123I-Iodometomidate (123I-IMTO) Imaging for Adrenal Neoplasia.Adrenal TumoursclinicalMTG1
  • ORPHA443287
  • ORPHA1501
NCT02010957
FAMAINAdrenal TumoursclinicalMTG1
  • ORPHA443287
  • ORPHA1501
EudraCT Number: 2012-003604-13
CHIRACIC -Surgery of Subclinical Cortisol Secreting Adrenal Incidentalomas (CHIRACIC)Adrenal TumoursclinicalMTG1
  • ORPHA443287
NCT02364089
Growing up With the Young Endocrine Support System (YESS!) (YESS)Transition,paediatric chronic endocrine disordersclinicalMTG1 MTG5 MTG6 MTG7 MTG8
  • ORPHA418
  • ORPHA174590
  • ORPHA881
  • ORPHA95488
  • ORPHA85138
  • ORPHA754
  • ORPHA9571
  • ORPHA95494
NCT04252001
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 HyperplasiaCongenital Adrenal HyperplasiaclinicalMTG1
  • ORPHA418
NCT04544410
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 HyperplasiaclinicalMTG1
  • ORPHA418
NCT04490915,NBI-74788-CAH3003
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 HyperplasiaCongenital Adrenal HyperplasiaclinicalMTG1
  • ORPHA418
NCT04457336
DIUR-006 : A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAH Diurnal LimitedCongenital Adrenal HyperplasiaclinicalMTG1
  • ORPHA418
NCT03062280,EudraCT Number: 2015-005448-32
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/AOUBoCongenital Adrenal HyperplasiaclinicalMTG1
  • ORPHA418
NCT04806451, NBI-74788-CAH2006
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 HyperplasiaCongenital Adrenal HyperplasiaclinicalMTG1
  • ORPHA418
NCT04045145,NBI-74788
Open-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapy (RESTORE)Congenital Adrenal HyperplasiaclinicalMTG1
  • ORPHA418
NCT03532022
A Phase III study of efficacy, safety and tolerability of Chronocort® compared with standard glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasiaCongenital Adrenal HyperplasiaclinicalMTG1
  • ORPHA418
NCT02716818
PREDEXCongenital Adrenal HyperplasiaclinicalMTG1
  • ORPHA418
15554888
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 HypercholesterolemiaFamilial Hypercholesterolemiaclinical
  • ORPHA391665
EudraCT Number: 2011-005400-15
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 hyperglycaemiaWolfram SyndromeclinicalMTG3
  • ORPHA3463
NCT03717909
INSPECT (Investigation into PHTS tumor syndrome)PHTS tumour syndromeclinicalMTG4
  • ORPHA306498
2018-5056
PROLACT A Phase III study of efficacy, safety and tolerability of Chronocort® compared with standard glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasiaProlactinomaclinicalMTG6
  • ORPHA2965
NCT04107480
A study of MK-6482 Monotherapy in advanced Pheochromocytoma/Paraganglioma (PPGL) and pancreatic neuroendocrine tumors (pNET)Pheochromocytoma/Paraganglioma, Pancreatic Neuroendocrine TumoursclinicalMTG1 MTG4
  • ORPHA276621
  • ORPHA29072
MK6482-PN015
FIRSTMAPPP, Genetic variants associated with increased susceptibility to thyroid cancerPheochromocytoma and ParagangliomaclinicalMTG1 MTG4
  • ORPHA276621
  • ORPHA29072
NCT01371201
Multicenter Pheochromocytoma and Paraganglioma Evaluation (MUPPET)Pheochromocytoma and ParagangliomaclinicalMTG1 MTG4
  • ORPHA276621
  • ORPHA29072
NCT03344016
PRESCRIPT – Phenoxybenzamine Versus Doxazosin in PCC PatientsPheochromocytomaclinicalMTG1 MTG4
  • ORPHA276621
  • ORPHA29072
NCT01379898
DP13 – A Phase II Study in Patients With Primary AldosteronismPrimary AldosteronismclinicalMTG1
  • ORPHA181415
NCT04007406
A Randomized, Double-Blind, Multiple Ascending Oral Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of PCO371 in Patients With HypoparathyroidismHypoparathyroidismclinicalMTG2
  • ORPHA181405
NCT04209179
A Study to Learn if Recombinant Human Parathyroid Hormone [rhPTH(1-84)] Can Improve Symptoms and Metabolic Control in Adults With Hypoparathyroidism (BALANCE)HypoparathyroidismclinicalMTG2
  • ORPHA181405
NCT03324880
A Clinical Study Investigating the Safety, Tolerability, PK and PD of PCO371 in Patients With Hypoparathyroidism – PCO 104 UGHypoparathyroidismclinicalMTG2
  • ORPHA181405
NCT 04209179
LA Study Comparing LY900014 to Insulin Lispro (Humalog) in Adults With Type 1 Diabetes Using Insulin Pump Therapy (PRONTO-Pump-2)Type 1 Diabetes MellitusclinicalMTG3
  • ORPHA181371
NCT03830281
Setmelanotide for the Treatment of LEPR Deficiency ObesityGenetic ObesityclinicalMTG5 MTG6
  • ORPHA179494
NCT03287960
CARGOx – Use of Copeptin Measurement After Arginine Infusion for the Differential Diagnosis of Diabetes Insipidus – the CARGOx Study (CARGOx)Diabetes InsipidusclinicalMTG6
  • ORPHA178029
NCT03572166
Efficacy of Adjuvant Mitotane Treatment (ADIUVO) (ADIUVO)Adrenocortical carcinomaclinicalMTG1
  • ORPHA1501
NCT00777244
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaAchondroplasiaclinicalMTG5
  • ORPHA15
NCT03197766
FDG-PET/CT in Evaluation of Cytological Indeterminate Thyroid Nodules to Prevent Unnecessary Surgery (EfFECTS) (EfFECTS)Thyroid NeoplasmsclinicalMTG8
  • ORPHA146
NCT02208544
Focus on Values to Stimulate Shared Decisions in Patients With Thyroid Cancer: A Multifaceted COMmunication BOoster (COMBO)sThyroid CarcinomaclinicalMTG8
  • ORPHA146
NCT03905369
LenvatinibThyroid CarcinomaclinicalMTG8
  • ORPHA146
2014-005199-27
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 CarcinomaclinicalMTG8
  • ORPHA142
NCT01099436
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 CarcinomaclinicalMTG8
  • ORPHA1332
NCT03037385
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 CarcinomaclinicalMTG8
  • ORPHA1332
NCT04211337,EUDRACT: 2019-001978-28
A Study of Two Different Doses of Cabozantinib (XL184) in Progressive, Metastatic Medullary Thyroid Cancer (EXAMINER)Medullary Thyroid CarcinomaclinicalMTG8
  • ORPHA1332
NCT01896479
A European Multi-Centre, Post-Authorization, Observation Study (Registry) of Patients with Chronic Adrenal InsufficiencyChronic Adrenal InsufficiencyclinicalMTG1
  • ORPHA101959
NCT01661387
Efficacy and Safety of Lanreotide Autogel (ATG) in Combination With Temozolomide in Subjects With Thoracic Neuroendocrine Tumors. (ATLANT)Neuroendocrine TumorsclinicalMTG4
  • ORPHA100101
NCT02698410
Efficacy and Safety of Lanreotide Autogel/ Depot 120 mg vs. Placebo in Subjects With Lung Neuroendocrine Tumors (SPINET)Neuroendocrine TumorsclinicalMTG4
  • ORPHA100101
NCT02683941
Efficacy and Safety Study in Pancreatic or Midgut Neuroendocrine Tumours Having Progressed Radiologically While Previously Treated With Lanreotide Autogel® 120 mg (CLARINET FORTE)Neuroendocrine TumoursclinicalMTG4
  • ORPHA100092
NCT02651987
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 DiseaseBehçet's Diseaseclinical
  • ORPHA:117
NCT02307513
Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe ObesityGenetic ObesityclinicalMTG5 MTG6
  • OPRHA240371
  • OPRHA64
  • ORPHA110
NCT03651765
Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity Phase 3Bardet Biedl Syndrome (BBS) Alström Syndrome (AS)clinicalMTG5 MTG6
  • OPRHA240371
  • OPRHA64
  • ORPHA110
NCT03746522
Selenium supplementation for patients with Graves’ hyperthyroidism (the GRASS trial)Graves’ hyperthyroidismclinicalMTG8NCT01611896
Personalize My Treatment (PMT) Registry (PMT)Biobanking In cancer patientsclinicalNCT02355171
Effect of Insulin Sensitization on IGF-1 Response to Growth Hormone in SGA ChildrenSmall for Gestational AgeclinicalMTG5NCT02931591
REAL 5Small for Gestational AgeclinicalMTG52018-000232-10
The Diurnal Rhythm in Natriuretic Peptide Levels and Relationship With Nocturnal Blood PressureNatriuretic Peptides,ObesityclinicalMTG3NCT03834168
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 AcidosisMetabolic Acidosisclinical2018-001303-36
A 4 Year Combination Therapy of Growth Hormone and (GnRH) Agonist in Children With a Short Predicted Height (ZomaTrip)Idiopathic Short StatureclinicalMTG5NCT00840944
NOVARTIS SOM B2412 roll overCluster Headache - Episodic and ChronicclinicalNCT02619617
Bacteremia Antibiotic Length Actually Needed for Clinical Effectiveness: Randomized Controlled TrialBacterial riskclinicalNCT03005145
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 MellitusType 2 Diabetes MellitusclinicalMTG3
  • ORPHA181376
NCT04029480,MK8835-059
Diabetes Study of Linagliptin and Empagliflozin in Children and Adolescents (DINAMO)TMType 2 Diabetes MellitusclinicalMTG3
  • ORPHA181376
NCT03429543
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 MellitusclinicalMTG3
  • ORPHA181371
NCT03875729,PRV-031-001
The Artificial Pancreas in Very Young Children With T1D (KidsAP02)Type 1 Diabetes MellitusclinicalMTG3
  • ORPHA181371
NCT03784027
A Study Comparing LY900014 to Insulin Lispro (Humalog) in Children and Adolescents With Type 1 Diabetes (PRONTO-Peds)Type 1 Diabetes MellitusclinicalMTG3
  • ORPHA181371
NCT03740919
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 InsulinType 1 Diabetes MellitusclinicalMTG3
  • ORPHA181371
NCT03667053
Diagnode 2 – GAD-Alum (Diamyd) Administered Into Lymph Nodes in Combination With Vitamin D in Type 1 DiabetesType 1 Diabetes MellitusclinicalMTG3
  • ORPHA181371
NCT03345004
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 DiabetesType 1 Diabetes MellitusclinicalMTG3
  • ORPHA181371
NCT03040414
Evaluation of the Dreamed Advisor Pro for Automated Insulin Pump Setting Adjustments in Children and Adolescents With Type 1 Diabetes- The Advice4U Pro StudyType 1 Diabetes MellitusclinicalMTG3
  • ORPHA181371
NCT03003806
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 MellitusclinicalMTG3
  • ORPHA181371
NCT02825251
Initiative pour la Promotion de la Qualité et Epidémiologie chez les Enfants et Adolescents atteints du Diabète sucré (IPQE-EADType 1 Diabetes MellitusclinicalMTG3
  • ORPHA181371
IPQE-EAD,IKEKAD
KidsAP02Type 1 Diabetes MellitusclinicalMTG3
  • ORPHA181371
KIDSAP consortium
Study of Safety and Efficacy of CFZ533 in Type 1 Diabetes Pediatric and Young Adult Subjects (CCFZ533X2207)Type 1 Diabetes MellitusclinicalMTG3
  • ORPHA181371
NCT04129528
ASCEND: A Study of Cardiovascular Events in DiabetesDiabetes MellitusclinicalMTG3
  • ORPHA101952
NCT00135226
Prevalence of overweight and obesity in children with X-linked hypophosphatemiaX-linked hypophosphataemiaobservationalMTG2
  • ORPHA89936
DOI:10.1530/EC-19-0481
Impact of Thyroid autoimmunity on Fertility outcome in intrauterine insemination (IUI) patientsThyroid  AutoimmunityobservationalMTG8
  • 5A03.2
  • 5A02.0
10.1093/humrep/dex033
Risk of malignancy of the thyroid nodule evaluated by scintigraphy: a single centre retrospective studyThyroid NoduleobservationalMTG8
  • ORPHA146
10.1530/endoabs.64.009
Validation of the diagnostic accuracy of the electronic nose in the detection of thyroid cancer.Thyroid carcinomaobservationalMTG8
  • ORPHA146
NCT04883294
Impact of thyroid autoimmunity on cumulative delivery rates in in vitro fertilization/intracytoplasmic sperm injection patientsThyroid AutoimmunityobservationalMTG8
  • 5A03.2
  • 5A02.0
10.1016/j.fertnstert.2016.03.011
Long-term safety and efficacy of Omnitrope®, a somatropin biosimilar, in children requiring growth hormone treatment: PATRO Children studySomatropin treatmentobservationalMTG5
  • ORPHA631
  • ORPHA739
  • ORPHA881
PMCID: PMC3593298
Genetic testing in inherited endocrine disorders: joint position paper of the European reference network on rare endocrine conditions (Endo-ERN)Rare genetic endocrine disordersobservationalMTG1 MTG2 MTG3 MTG4 MTG5 MTG6 MTG7 MTG8
  • ORPHA97978
10.1186/s13023-020-01420-w
COVID-19 Infections In People With Rare Endocrine ConditionsRare Endocrine Conditions, COVID-19observationalMTG1 MTG2 MTG3 MTG4 MTG5 MTG6 MTG7 MTG8
  • ORPHA97978
10.1530/endoabs.73.OC14.5
Ensat-HTPrimary Aldosteronism, Pheochromocytoma/Paraganglioma, Cushing SyndromeobservationalMTG1 MTG6
  • ORPHA181415
  • ORPHA573163
  • ORPHA553
ens@t-ht, European Union’s Horizon 2020 - grant agreement No 633983
Circulating microRNAs in primary aldosteronismPrimary AldosteronismobservationalMTG1
  • ORPHA181415
DOI: 10.1530/endoabs.63.GP198
Optimizing mineralocorticoid replacement therapy in patients with congenital adrenal hyperplasia and Addison’s diseasePrimary Adrenal InsufficiencyobservationalMTG1
  • ORPHA418
  • ORPHA85138
DOI: 10.1530/endoabs.63.GP197
Prolactin levels in PWSPrader-Willi SyndromeobservationalMTG5
  • ORPHA739
10.3390/jcm10163613
Efficacy and Safety of Norditropin® (Somatropin) in Children With Prader-Willi Syndrome (PWS)Prader-Willi SyndromeobservationalMTG5
  • ORPHA739
NCT00705172
The Molecular Registry of Pituitary Adenomas (REMAH): A bet of Spanish Endocrinology for the future of individualized medicine and translational research.Pituitary AdenomaobservationalMTG6
  • ORPHA99408
doi: 10.1016/j.endonu.2016.03.001.
A Multicenter Epidemiological Study on Second Malignancy in Non-Syndromic Pheochromocytoma/Paraganglioma Patients in ItalyNon-Syndromic Pheochromocytoma/ParagangliomaobservationalMTG1
  • ORPHA573163
10.3390/cancers13225831
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 TumorsobservationalMTG4
  • ORPHA877
NCT03498040
MEN 1 data dase / Impact of Delay in Diagnosis in Outcomes in MEN1: Results From the Dutch MEN1 Study GroupMEN1observationalMTG4
  • ORPHA652
DutchMEN study group (DMSG)/ doi.org/10.1210/clinem/dgaa800
Hypopituitarism ENEA Rare etiologies Observational Study (HEROS)Hypopituitarism, Histiocytosis X, Sarcoidosis, Hypophysitis, Idiopatic Diabetes InsipidusobservationalMTG6
  • ORPHA101957
  • ORPHA389
  • ORPHA797
  • ORPHA95506
  • ORPHA95626
HEROS
An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)Hypophosphatasia (HPP)observationalMTG2 
  • ORPHA436
NCT02306720
A European Network for the Investigation of Gender Incongruence (ENIGI)Hormonal Therapy in Transsexual PersonsobservationalMTG7
  • HA6Z
NCT01072825
NSEuroNet databaseGenetic Short StatureobservationalMTG5
  • ORPHA98733
NSEuroNet
RIFTOS MKI – Radioactive Iodine reFractory Asymptomatic Patients in Differentiated Thyroid Cancer – an Observational Study to Assess the Use of Multikinase InhibitorsDifferentiated Thyroid CarcinomaobservationalMTG8
  • ORPHA146
NCT02303444
Low iodine diet study (LID)Differentiated Thyroid CarcinomaobservationalMTG8
  • ORPHA146
DOI: 10.1210/clinem/dgab691
Long-Term Effects of Radioiodine Treatment on Female Fertility in Survivors of Childhood Differentiated Thyroid CarcinomaDifferentiated Thyroid CarcinomaobservationalMTG8
  • ORPHA146
doi: 10.1089/thy.2019.0560
Gonadectomy in conditions affecting sex development: a registry-based cohort studyDifferences of Sex DevelopmentobservationalMTG7
  • ORPHA90771
10.1530/eje-20-1058, PMID: 33780351
Personalized medicine in diabetes: design and characterization of a high-performance genotyping tool that allows the classification of different diabetesDiabetes MellitusobservationalMTG3
  • 5A14
PID2019-107513RB-I00
The etiology and extent of impaired quality of life, fatigue and cognitive, affective and emotional dysfunction in patients with Cushing’s syndromeCushing SyndromeobservationalMTG1 MTG6
  • ORPHA553
NCT03211624
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 DiseaseobservationalMTG6
  • ORPHA96253
NCT02310269
Clinical characteristics of children with Congenital Hyperinsulinism: Results from the European Registries for Rare Endocrine Conditions (EuRRECA)ProjectCongential HyperinsulinismobservationalMTG3
  • ORPHA657
ESPE & EuRRECa programme (https://eurreca.net/)
Maximising the Utilisation of the Hyperinsulinism Global Registry (HIGR) [Max HIGR]Congenital HyperinsulinismobservationalMTG3
  • ORPHA657
MDBR-21-102-CHI
International practice of corticosteroid replacement therapy in congenital adrenal hyperplasia: data from the I-CAH registryCongenital Adrenal HyperplasiaobservationalMTG1
  • ORPHA418
10.1530/eje-20-1249, PMID: 33460392
CHRODIS PLUS,Chronic diseaseobservational
  • 5A14
CHRODIS+
A European Post-Authorisation Observational Study (Registry) of Patients With Chronic Adrenal Insufficiency (AI) – EU-AIRChronic Adrenal InsufficiencyobservationalMTG1
  • ORPHA101959
NCT01661387
Follow-up of pubertal development and fertility in boys who underwent testicular tissue biopsy for fertility preservationChromosomal DSDobservationalMTG7
  • ORPHA325546
Identification of selective androgen receptor co-regulators on the basis of an in vitro model of androgen insensitivity syndromeAndrogen Insensitivity SyndromeobservationalMTG7
  • ORPHA754
HO 6028/2-1
Structured Evaluation of adRENal tumors Discovered Incidentally – Prospectively Investigating the Testing Yield- Serendipity Protocol NTR 4799Adrenal TumoursobservationalMTG1
  • ORPHA100091
Serendipity Protocol NTR 4799
SERENDIPITY – Structured Evaluation of adRENal Tumors Discovered Incidentally – Prospectively Investigating the Testing Yield (SERENDIPITY)Adrenal TumoursobservationalMTG1
  • ORPHA100091
NCT02324647
ENSAT registry studyAdrenal tumoursobservationalMTG1
  • ORPHA100091
ENS@T
A National Multi-Centre study on etiology and long term outcome of Primary Adrenal Insufficiency in childrenAdrenal InsufficiencyobservationalMTG1
  • ORPHA101958
  • ORPHA418
https://doi.org/10.1186/1472-6823-14-40
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 ageAdrenal InsufficiencyobservationalMTG1
  • ORPHA101958
10.1210/clinem/dgaa881
EOLIAAcromegalyobservationalMTG6
  • ORPHA963
CSMS995BFR13
Development of ACRODAT®, a new software medical device to assess disease activity in patients with acromegaly.AcromegalyobservationalMTG6
  • ORPHA963
DOI: 10.1007/s11102-017-0835-5
LAS. Liège Acromegaly Survey. A large prospective observational registry of patients with acromegaly.AcromegalyobservationalMTG6
  • ORPHA963
doi: 10.1530/ERC-17-0253
A European observational study in patients with uncontrolled acromegaly who are being treated with long acting pasireotideAcromegalyobservationalMTG6
  • ORPHA963
10.1530/endoabs.56.GP8
Lifetime Impact of Achondroplasia Study in Europe-LIAISE, (NCT03449368)AchondroplasiaobservationalMTG5 
  • ORPHA15
NCT03449368
SF1 next46,XY DSDobservationalMTG7
  • ORPHA325351
Study ID 201810_GS
Puberty in Females with PAIS46,XY DSDobservationalMTG7
  • ORPHA90797
Study ID 201402_GGF
Prevalence of overweight and obesity in children with X-linked hypophosphatemiaX-linked hypophosphataemiaobservationalMTG2 MTG5
  • ORPHA89936
10.1530/EC-19-0481
International Multicentre Validation Study of SAGIT® Instrument in AcromegalyAcromegalyobservationalMTG6
  • ORPHA963
NCT02539927
Registry for Patients With X-linked Hypophosphatemia (XLH Registry)X-linked HypophosphatemiaobservationalMTG2
  • ORPHA89936
NCT03193476
Diabetes-related antibody-testing is a valuable screening tool for diagnosing monogenic diabetes – a survey from the worldwide SWEET registryType 1 DiabetesobservationalMTG3
  • ORPHA181371
NCT04427189
An Innovative Approach Towards Understanding and Arresting Type 1 Diabetes (INNODIA) (INNODIA)Type 1 DiabetesobservationalMTG3
  • ORPHA181371
NCT03936634
THETA: cycling from hypoThyroidism to Hyperthyroidism during Early treatment of differentiated Thyroid carcinomAThyroid carcinomaobservationalMTG8
  • ORPHA146
Trial NL7228 (NTR7427)
Low iodine diet study (LID)Thyroid cancerobservationalMTG8
  • ORPHA146
doi.org/10.1210/clinem/dgab691
Adrenal tropism of SARS-CoV-2 and adrenal findings in a post-mortem case series of patients with severe fatal COVID-19SARS-CoV-2, AdrenalobservationalMTG1
  • XA0NE9
doi.org/10.1038/s41467-022-29145-3
Oberar : Rare Obesity Cohorts With Food Behavioral Disorders : Better Diagnosis for Better TreatmentObesityobservationalMTG5
  • ORPHA739
NCT04604626
MEN 1 data dase / Impact of Delay in Diagnosis in Outcomes in MEN1: Results From the Dutch MEN1 Study GroupMEN1observationalMTG4
  • ORPHA652
doi.org/10.1210/clinem/dgaa800
PARADIGHM (Physicians Advancing Disease Knowledge in Hypoparathyroidism): A Registry for Patients With Chronic HypoparathyroidismHypoparathyroidismobservationalMTG2
  • ORPHA181405
NCT01922440
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 DeficiencyobservationalMTG5
  • ORPHA101957
doi.org/10.1007/s11102-021-01139-2
Long term effects of radioiodine treatment in suvivorsDifferentiated Thyroid CarcinomaobservationalMTG8
  • ORPHA146
10.1089/thy.2019.0560
The etiology and extent of impaired quality of life, fatigue and cognitive, affective and emotional dysfunction in patients with Cushing’s syndromeCushing's SyndromeobservationalMTG1 MTG6
  • ORPHA553
BRAIN
Complicushing: Cushing’s Disease ComplicationsCushing's DiseaseobservationalMTG6
  • ORPHA96253
NCT02568982
Clinical characteristics of children with Congenital Hyperinsulinism: Results from the European Registries for Rare Endocrine Conditions (EuRRECa) ProjectCongenital HyperinsulismobservationalMTG3
  • ORPHA657
doi.org/10.1159/000518849
Pathogenic and low-frequency variants in children with Central Precocious PubertyCentral Precocious PubertyobservationalMTG6
  • ORPHA759
doi.org/10.3389/fendo.2021.745048
ENSAT registry-based randomized clinical trials for adrenocortical carcinomaAdrenal TumoursobservationalMTG1
  • ORPHA1501
10.1530/EJE-20-0800
Omics Signature in the Diagnosis of Hypertension (ENSAT-HT)HypertensionobservationalMTG1 MTG4
  • ORPHA573163
  • ORPHA97253
  • ORPHA181415
  • ORPHA99893
NCT02772315, P17-284/2018-4231
Trial/Study NameTopicStudy TypeMTGORPHACodeTrial/study IDLink