Clinical Trials
| Topic | Trial name of investigational drug | Clinical Trial ID(s) | MTG(s) | Status | OrphaCode | Link |
|---|---|---|---|---|---|---|
| Diabetes Mellitus | ASCEND: A Study of Cardiovascular Events in Diabetes |
| 3 |
|
| |
| Type 1 Diabetes Mellitus | Study of Safety and Efficacy of CFZ533 in Type 1 Diabetes Pediatric and Young Adult Subjects (CCFZ533X2207) |
| 3 |
|
| |
| Type 1 Diabetes Mellitus | KidsAP02 |
| 3 |
|
| |
| Type 1 Diabetes Mellitus | Initiative pour la Promotion de la Qualité et Epidémiologie chez les Enfants et Adolescents atteints du Diabète sucré (IPQE-EAD |
| 3 |
|
| |
| Type 1 Diabetes Mellitus | 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). |
| 3 |
|
| |
| Type 1 Diabetes Mellitus | Evaluation of the Dreamed Advisor Pro for Automated Insulin Pump Setting Adjustments in Children and Adolescents With Type 1 Diabetes- The Advice4U Pro Study |
| 3 |
|
| |
| Type 1 Diabetes Mellitus | 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 |
| 3 |
|
| |
| Type 1 Diabetes Mellitus | Diagnode 2 - GAD-Alum (Diamyd) Administered Into Lymph Nodes in Combination With Vitamin D in Type 1 Diabetes |
| 3 |
|
| |
| Type 1 Diabetes Mellitus | 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 |
| 3 |
|
| |
| Type 1 Diabetes Mellitus | A Study Comparing LY900014 to Insulin Lispro (Humalog) in Children and Adolescents With Type 1 Diabetes (PRONTO-Peds) |
| 3 |
|
| |
| Type 1 Diabetes Mellitus | The Artificial Pancreas in Very Young Children With T1D (KidsAP02) |
| 3 |
|
| |
| Type 1 Diabetes Mellitus | 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) |
| 3 |
|
| |
| Type 2 Diabetes Mellitus | Diabetes Study of Linagliptin and Empagliflozin in Children and Adolescents (DINAMO)TM |
| 3 |
|
| |
| Type 2 Diabetes Mellitus | 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 |
| 3 |
|
| |
| Bacterial risk | Bacteremia Antibiotic Length Actually Needed for Clinical Effectiveness: Randomized Controlled Trial |
|
| |||
| Cluster Headache - Episodic and Chronic | NOVARTIS SOM B2412 roll over |
|
| |||
| Idiopathic Short Stature | A 4 Year Combination Therapy of Growth Hormone and (GnRH) Agonist in Children With a Short Predicted Height (ZomaTrip) |
| 5 |
| ||
| Metabolic Acidosis | 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 |
| ERKNetMetabERN |
| ||
| Natriuretic Peptides,Obesity | The Diurnal Rhythm in Natriuretic Peptide Levels and Relationship With Nocturnal Blood Pressure |
| 3 |
| ||
| Small for Gestational Age | REAL 5 |
| 5 |
| ||
| Small for Gestational Age | Effect of Insulin Sensitization on IGF-1 Response to Growth Hormone in SGA Children |
| 5 |
| ||
| Biobanking In cancer patients | Personalize My Treatment (PMT) Registry (PMT) |
|
| |||
| Graves’ hyperthyroidism | Selenium supplementation for patients with Graves’ hyperthyroidism (the GRASS trial) |
| 8 |
| ||
| Bardet Biedl Syndrome (BBS) Alström Syndrome (AS) | 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 |
| 56 |
|
| |
| Genetic Obesity | Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity |
| 56 |
|
| |
| Behçet's Disease | 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 |
| BloodNET ERN ?? |
|
| |
| Neuroendocrine Tumours | Efficacy and Safety Study in Pancreatic or Midgut Neuroendocrine Tumours Having Progressed Radiologically While Previously Treated With Lanreotide Autogel® 120 mg (CLARINET FORTE) |
| 4 |
|
| |
| Neuroendocrine Tumors | Efficacy and Safety of Lanreotide Autogel/ Depot 120 mg vs. Placebo in Subjects With Lung Neuroendocrine Tumors (SPINET) |
| 4 |
|
| |
| Neuroendocrine Tumors | Efficacy and Safety of Lanreotide Autogel (ATG) in Combination With Temozolomide in Subjects With Thoracic Neuroendocrine Tumors. (ATLANT) |
| 4 |
|
| |
| Chronic Adrenal Insufficiency | A European Multi-Centre, Post-Authorization, Observation Study (Registry) of Patients with Chronic Adrenal Insufficiency |
| 1 |
|
| |
| Medullary Thyroid Carcinoma | A Study of Two Different Doses of Cabozantinib (XL184) in Progressive, Metastatic Medullary Thyroid Cancer (EXAMINER) |
| 8 |
|
| |
| Medullary Thyroid Carcinoma | 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) |
| 8 |
|
| |
| Thyroid Carcinoma | 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) |
| 8 |
|
| |
| Anaplastic Thyroid Carcinoma | 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). |
| 8 |
|
| |
| Thyroid Carcinoma | Lenvatinib |
| 8 |
|
| |
| Thyroid Carcinoma | Focus on Values to Stimulate Shared Decisions in Patients With Thyroid Cancer: A Multifaceted COMmunication BOoster (COMBO)s |
| 8 |
|
| |
| Thyroid Neoplasms | FDG-PET/CT in Evaluation of Cytological Indeterminate Thyroid Nodules to Prevent Unnecessary Surgery (EfFECTS) (EfFECTS) |
| 8 |
|
| |
| Achondroplasia | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia |
| 5 |
|
| |
| Adrenocortical carcinoma | Efficacy of Adjuvant Mitotane Treatment (ADIUVO) (ADIUVO) |
| 1 |
|
| |
| Diabetes Insipidus | CARGOx - Use of Copeptin Measurement After Arginine Infusion for the Differential Diagnosis of Diabetes Insipidus - the CARGOx Study (CARGOx) |
| 6 |
|
| |
| Genetic Obesity | Setmelanotide for the Treatment of LEPR Deficiency Obesity |
| 56 |
|
| |
| Type 1 Diabetes Mellitus | LA Study Comparing LY900014 to Insulin Lispro (Humalog) in Adults With Type 1 Diabetes Using Insulin Pump Therapy (PRONTO-Pump-2) |
| 3 |
|
| |
| Hypoparathyroidism | A Clinical Study Investigating the Safety, Tolerability, PK and PD of PCO371 in Patients With Hypoparathyroidism - PCO 104 UG |
| 2 |
|
| |
| Hypoparathyroidism | A Study to Learn if Recombinant Human Parathyroid Hormone [rhPTH(1-84)] Can Improve Symptoms and Metabolic Control in Adults With Hypoparathyroidism (BALANCE) |
| 2 |
|
| |
| Hypoparathyroidism | A Randomized, Double-Blind, Multiple Ascending Oral Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of PCO371 in Patients With Hypoparathyroidism |
| 2 |
|
| |
| Primary Aldosteronism | DP13 - A Phase II Study in Patients With Primary Aldosteronism |
| 1 |
|
| |
| Pheochromocytoma | PRESCRIPT - Phenoxybenzamine Versus Doxazosin in PCC Patients |
| 14 |
|
| |
| Pheochromocytoma and Paraganglioma | Multicenter Pheochromocytoma and Paraganglioma Evaluation (MUPPET) |
| 14 |
|
| |
| Pheochromocytoma and Paraganglioma | FIRSTMAPPP, Genetic variants associated with increased susceptibility to thyroid cancer |
| 14 |
|
| |
| Pheochromocytoma/Paraganglioma, Pancreatic Neuroendocrine Tumours | A study of MK-6482 Monotherapy in advanced Pheochromocytoma/Paraganglioma (PPGL) and pancreatic neuroendocrine tumors (pNET) |
| 14 |
|
| |
| Prolactinoma | 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 |
| 6 |
|
| |
| PHTS tumour syndrome | INSPECT (Investigation into PHTS tumor syndrome) |
| 4 |
|
| |
| Wolfram Syndrome | 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 |
| 3 |
|
| |
| Familial Hypercholesterolemia | 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 |
| GuardHeart?? |
|
| |
| Congenital Adrenal Hyperplasia | PREDEX |
| 1 |
|
| |
| Congenital Adrenal Hyperplasia | A Phase III study of efficacy, safety and tolerability of Chronocort® compared with standard glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasia |
| 1 |
|
| |
| Congenital Adrenal Hyperplasia | Open-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapy (RESTORE) |
| 1 |
|
| |
| Congenital Adrenal Hyperplasia | 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 |
| 1 |
|
| |
| Congenital Adrenal Hyperplasia | 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 |
| 1 |
|
| |
| Congenital Adrenal Hyperplasia | DIUR-006 : A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAH Diurnal Limited |
| 1 |
|
| |
| Congenital Adrenal Hyperplasia | 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 |
| 1 |
|
| |
| Congenital Adrenal Hyperplasia | 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, |
| 1 |
|
| |
| Congenital Adrenal Hyperplasia | 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 |
| 1 |
|
| |
| Transition,paediatric chronic endocrine disorders | Growing up With the Young Endocrine Support System (YESS!) (YESS) |
| 15678 |
|
| |
| Adrenal Tumours | CHIRACIC -Surgery of Subclinical Cortisol Secreting Adrenal Incidentalomas (CHIRACIC) |
| 1 |
|
| |
| Adrenal Tumours | FAMAIN |
| 1 |
|
| |
| Adrenal Tumours | Combined 18F-Fluorodeoxyglucose (FDG) Positron Emission Tomography (PET) and 123I-Iodometomidate (123I-IMTO) Imaging for Adrenal Neoplasia. |
| 1 |
|
| |
| Adrenal Tumours | Structured Evaluation of adRENal Tumors Discovered Incidentally - Prospectively Investigating the Testing Yield (SERENDIPITY) |
| 1 |
|
| |
| Adrenal Tumours | GRADIENT; A phase 3, randomized, double-blind, placebo-controlled study of the efficacy and safety of Relacorilant |
| 1 |
|
| |
| Hypothalamic Obesity | Efficacy and Safety of Exenatide in the Treatment of Hypothalamic Obesity After Craniopharyngioma Therapy (CRANIOEXE) |
| 6 |
|
| |
| Cushing Syndrome | LOGICS: A Study to Assess the Safety and Efficacy of Levoketoconazole in the Treatment of Endogenous Cushing's Syndrome (Levoketoconazole). |
| 16 |
|
| |
| Cushing Syndrome | 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 |
| 16 |
|
| |
| Cushing Syndrome | An Open-Label Extension Study of Levoketoconazole (2S,4R-ketoconazole) in the Treatment of Endogenous Cushing’s Syndrome |
| 16 |
|
| |
| McCune- Albright Syndrome | 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 |
| 1268 |
|
| |
| MCT8 Deficiency | Thyroid Hormone Analog Therapy in MCT8 Deficiency: Triac Trial Patients |
| 8 |
|
| |
| MCT8 Deficiency | Triac Trial II in MCT8 Deficiency Patients |
| 8 |
|
| |
| Congenital Hyperinsulinism | 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. |
| 3 |
|
| |
| Congenital Hyperinsulinism | A Two-Period, Open-label Trial Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism |
| 3 |
|
| |
| Congenital Hyperinsulinism | An Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism |
| 3 |
|
| |
| Congenital Hyperinsulinism | Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism |
| 3 |
|
| |
| Congenital Hyperinsulinism | An Open-Label Multiple-Dose Study of RZ358 in Patients with Congenital Hyperinsulinism, Protocol RZ358-606 |
| 3 |
|
| |
| Genetic Obesity | Setmelanotide for the Treatment of Early-Onset POMC Deficiency Obesity |
| 56 |
|
| |
| Genetic Obesity | Long Term Extension Trial of Setmelanotide |
| 56 |
|
| |
| Genetic Obesity | 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) |
| 56 |
|
| |
| Prader-Willi Syndrome | Effect of Liraglutide for Weight Management in Paediatric Subjects With Prader-Willi Syndrome |
| 5 |
|
| |
| Prader-Willi Syndrome | ZEPHYR Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome (ZEPHYR |
| 5 |
|
| |
| Prader-Willi Syndrome | Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome (OTBB3) |
| 5 |
|
| |
| Adrenal Insufficiency | Optimising Steroid Replacement in Patients With Adrenal Insufficiency (Plenadren) |
| 1 |
|
| |
| Neuroendocrine Tumours | 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) |
| 4 |
|
| |
| X-Linked Hypophosphatemia | X linked Hypof (XLH) Registry for Patients With X-Linked Hypophosphatemia (XLH Registry) |
| 2 |
|
| |
| X-linked hypophosphatemia | 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 |
| 2 |
|
| |
| Growth Hormone Deficiency | 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 |
| 6 |
|
| |
| Growth Hormone Deficiency | Safety and Efficacy Study of MOD-4023 to Treat Children With Growth Hormone Deficiency |
| 6 |
|
| |
| Growth Hormone Deficiency | 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) |
| 6 |
|
| |
| Growth Hormone Deficiency | A Phase II, Dose and Frequency Finding Study of MOD-4023 in Growth Hormone Deficient Adults (GHDA) |
| 6 |
|
| |
| Growth Hormone Deficiency | A phase 3 trial evaluating the effect of long acting GH (MOD-4023) in patients with GHD. CP-4-005 |
| 6 |
|
| |
| Growth Hormone Deficiency | A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD) |
| 6 |
|
| |
| Growth Hormone Deficiency | Safety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children |
| 6 |
|
| |
| Growth Hormone Deficiency | TransCon hGH 301-CT Ext (1) Enlighten (weekly GH/Transcon |
| 6 |
|
| |
| Growth Hormone Deficiency | A trial comparing the effect and safety of once weekly dosing of somapacitan with daily Norditropin® in children with growth hormone deficiency (REAL 4) |
| 6 |
|
| |
| Growth Hormone Deficiency | 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 |
| 6 |
|
| |
| Growth Hormone Deficiency | Registre belge des enfants traités par hormone de croissance BELGROW |
| 6 |
|
| |
| Cushing Disease | Efficacy and Safety Evaluation of Osilodrostat in Cushing's Disease (LINC-4) |
| 6 |
|
| |
| Cushing Disease | 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 |
| 6 |
|
| |
| Cushing Disease | 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 |
| 6 |
|
| |
| Cushing Disease | 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 |
| 6 |
|
| |
| Acromegaly | Comparison of Oral Octreotide Capsules to Injectable Somatostatin Analogs in Acromegaly (MPOWERED). |
| 6 |
|
| |
| Acromegaly | Efficacy and Safety of Octreotide Capsules (MYCAPSSA) in Acromegaly |
| 6 |
|
| |
| Acromegaly | 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) |
| 6 |
|
| |
| Acromegaly | 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) |
| 6 |
|
| |
| Acromegaly | 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. |
| 6 |
|
| |
| Acromegaly | A Trial to Assess the Long-term Safety of Octreotide Subcutaneous Depot in Patients With Acromegaly |
| 6 |
|
| |
| Acromegaly | Europea Observational study in Acromegaly - Acronis Study |
| 6 |
|
| |
| Cushing Disease and Acromegaly | 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 |
| 6 |
|
| |
| Cushing Syndrome | GRACE-2 |
| 16 |
|
| |
| Cushing Syndrome | 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 |
| 16 |
|
| |
| Acquired Thrombotic Thrombocytopenic Purpura | 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 |
| BloodNET ERN ?? |
| ||
| Adrenocortical Carcinoma | 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 |
| 1 |
|
| |
| Diabetic Foot Ulcer (DFU) | 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) |
| 3 |
|
| |
| DSD | DSDCare: Standardized center-centric care of DSD across the lifespan |
| 7 |
|
| |
| Gastro-enteropancreatic Neuroendocrine Tumor | 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) |
| 4 |
|
| |
| Medullary Thyroid Cancer | Libretto-531 study |
| 8 |
|
| |
| Multiple Endocrine Neoplasia Type 1 | PRIME Precision Radiotherapy Using MR-linac for Pancreatic Neuroendocrine Tumours in MEN1 Patients |
| 4 |
|
| |
| Neuroendocrine Tumors | 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). |
| 4 |
|
| |
| Osteogenesis Imperfecta | An Open-label, Ascending Multiple-dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis Imperfecta |
| ERN BOND |
|
| |
| Pheochromocytoma/Paraganglioma, pancreatic neuroendocrine tumors | 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) |
| 14 |
|
| |
| Prader-Willi Syndrome | 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 |
| 5 |
|
| |
| Prader-Willi Syndrome | 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) |
| 5 |
|
| |
| Prader-Willi Syndrome | Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial (OTBB3-FU) |
| 5 |
|
| |
| Type 1 Diabetes Mellitus | Study of Safety and Efficacy of CFZ533 in Type 1 Diabetes Pediatric and Young Adult Subjects (CCFZ533X2207) |
| 3 |
|
| |
| Topic | Trial name of investigational drug | Clinical Trial ID(s) | MTG(s) | Status | OrphaCode | Link |
Last update: May 19, 2022 on 11:02