Clinical Trials

ERN Clinical Trials Repository

Filters list of Clinical Trials

Legend
Name of StudyERNsTypeStatusOrphaCodeDiseasePhaseDateLink
OLE-IEDAT: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 StudyClinical trialOngoing
  • Ataxia-telangiectasia
  • 3
2018-09-20
An Open Label, 1-Year Trial, Including a Double-Blind Placebo-Controlled Withdrawal Period, of Setmelanotide (RM-493), a Melanocortin 4 Receptor (MC4R) Agonist, in Early Onset Leptin Receptor (LEPR) Deficiency Obesity Due to Bi-Allelic Loss-of-Function LEPR Genetic MutationClinical trialOngoing
  • Obesity due to leptin receptor gene deficiency
  • 3
2018-01-30
ADVANCE: A randomized, double-blind, placebo-controlled, multinational, multicenter study with open-label treatment extension to assess the effect of MIN-102 on the progression of adrenomyeloneuropathy in male patients with X-linked adrenoleukodystrophyClinical trialOngoing
  • X-linked adrenoleukodystrophy
  • 2
  • 3
2017-10-26
COMPETE: Efficacy and Safety of 177Lu-edotreotide PRRT in GEP-NET PatientsClinical trialOngoing
  • Gastroenteropancreatic neuroendocrine neoplasm
  • 3
2017-02-02
A multicenter open-label study to evaluate safety and dosimetry of Lutathera in adolescent patients with somatostatin receptor positive gastroenteropancreatic neuroendocrine (GEP-NET) tumors, pheochromocytoma and paragangliomas - NETTER-PClinical trialOngoing
  • Gastroenteropancreatic neuroendocrine neoplasm
  • Pheochromocytoma-paraganglioma
  • 2
2021-09-30
A Phase I/IIa Study of Intra-tumoral BT-001 (TG6030) Administered Alone and in Combination With Pembrolizumab in Patients With Cutaneous or, Subcutaneous Lesions or Easily Injectable Lymph Nodes of Metastatic/Advanced Solid Tumors.Clinical trialOngoing
  • Non-Hodgkin lymphoma
  • Soft tissue sarcoma
  • Cutaneous neuroendocrine carcinoma
  • 1
  • 2
2021-02-25
A One Year, Randomized, Double-Blind, Placebo-Controlled Study of TBR-760 in Adult Patients with Non-Functioning Pituitary AdenomasClinical trialOngoing
  • Non-functioning pituitary adenoma
  • 2
2020-07-31
NEXUS: An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)Clinical trialOngoing
  • X-linked cerebral adrenoleukodystrophy
  • 2
2019-09-13
An open-label, multi-center, roll-over study to assess long term safety inpatients with endogenous Cushing's syndrome who have completed a priorNovartis-sponsored osilodrostat (LCI699) study and are judged by theinvestigator to benefit from continued treatment with osilodrostat. - NAClinical trialOngoing
  • Cushing disease
  • 2
2018-10-05
MERKLIN 2: A phase II, open label study to investigate the efficacy and safety of domatinostat in combination with avelumab in patients with advanced unresectable/metastatic Merkel Cell Carcinoma progressing on anti-PD(L)1 antibody therapyClinical trialOngoing
  • Cutaneous neuroendocrine carcinoma
  • 2
2020-11-04
PATHFNDR-1: A Randomized, Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Paltusotine in Subjects With Acromegaly Treated With Long-acting Somatostatin Receptor LigandsClinical trialOngoing
  • Acromegaly
  • 3
2021-05-12
An Open-Label Multiple-Dose Study of RZ358 in Patients with Congenital HyperinsulinismClinical trialTerminated
  • Congenital isolated hyperinsulinism
  • 2
2020-03-04
An Open-Label Phase 2 Study of Surufatinib in Patients with Neuroendocrine Tumours in EuropeClinical trialOngoing
  • Neuroendocrine neoplasm
  • 2
2021-09-03
An Open Label, 1-Year Trial, including a Double-Blind Placebo-Controlled Withdrawal Period, of Setmelanotide (RM-493), a Melanocortin 4 Receptor (MC4R) Agonist, in Early Onset POMC Deficiency Obesity due to Bi-Allelic Loss-of-Function POMC or PCSK1 Genetic MutationClinical trialTerminated
  • Obesity due to pro-opiomelanocortin deficiency
  • 3
2018-06-04
Long Term Extension Trial of setmelanotide (RM-493) for patients who have completed a trial of Setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathwayClinical trialOngoing
  • Obesity due to congenital leptin deficiency
  • Obesity due to pro-opiomelanocortin deficiency
  • Obesity due to melanocortin 4 receptor deficiency
  • 2
  • 3
2018-07-15
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 DiseaseClinical trialOngoing
  • Endogenous Cushing syndrome
  • 2
2021-04-28
GRACE: Glucocorticoid Receptor Antagonism in the Treatment of Cushing Syndrome: A Phase 3, Double-Blind, Placebo-Controlled, Randomized-Withdrawal Study of the Efficacy and Safety of RelacorilantClinical trialOngoing
  • Endogenous Cushing syndrome
  • 3
2018-10-16
A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle DisordersClinical trialOngoing
  • Hyperinsulinism-hyperammonemia syndrome
  • 4
2018-02-20
SPENCER: A Phase 1/2 Trial of a Novel Therapeutic Vaccine (EO2401) in Combination With Immune Check Point Blockade, for Treatment of Patients With Locally Advanced or Metastatic Adrenocortical Carcinoma, or Malignant Pheochromocytoma/ParagangliomaClinical trialOngoing
  • Adrenocortical carcinoma
  • Sporadic pheochromocytoma/secreting paraganglioma
  • 1
  • 2
2020-08-06
ZEPHYR: A Phase 2b/3 Study to Evaluate the Safety, Tolerability, and Effects of Livoletide (AZP-531), an Unacylated Ghrelin Analog, on Food-related Behaviors in Patients With Prader-Willi SyndromeClinical trialOngoing
  • Prader-Willi syndrome
  • 2
  • 3
2019-03-26
The KHENERGYZE Study: A Phase IIb Double-blind, Randomised, Placebo-controlled, Multi-centre, Confirmative Three-way Cross-over Study on Cognitive Function With Two Doses of KH176 in Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeu(UUR) m.3243A>G MutationClinical trialOngoing
  • MELAS
  • Maternally-inherited diabetes and deafness
  • 2
2019-10-30
A Double-blind, Placebo-Controlled, Randomized Withdrawal Following Open Label Therapy Study to Assess the Safety and Efficacy of Levoketoconazole (2S, 4R-ketoconazole) in the Treatment of Endogenous Cushing's SyndromeClinical trialOngoing
  • Cushing disease
  • 3
2018-02-23
OPTICS: An Open-label Extension Study of Levoketoconazole (2S,4R-ketoconazole) in the Treatment of Endogenous Cushing's SyndromeClinical trialOngoing
  • Cushing disease
  • 3
2019-03-25
A Double Blind, Placebo-Controlled, Phase 2 Study to Assess the Safety, Tolerability, and Efficacy of ISIS 766720 (IONIS GHR-LRX, an Antisense Inhibitor of the Growth Hormone Receptor) Administered Once Every 28 Days for 16 Weeks in Patients with Acromegaly Being Treated with Long-acting Somatostatin Receptor Ligands (SRL)Clinical trialOngoing
  • Acromegaly
  • 2
2018-09-13
PODIUM 201: A Phase 2 Study of INCMGA00012 in Participants With Metastatic Merkel Cell CarcinomaClinical trialOngoing
  • Cutaneous neuroendocrine carcinoma
  • 2
2019-02-25
BALANCE: A Randomized, Double-blind, Placebo-controlled, Adaptive Study to Evaluate Symptom Improvement and Metabolic Control Among Adult Subjects With Symptomatic Hypoparathyroidism Treated With Recombinant Human Parathyroid Hormone [rhPTH(1-84)]Clinical trialTerminated
  • Familial isolated hypoparathyroidism due to agenesis of parathyroid gland
  • Syndrome with hypoparathyroidism
  • 4
2018-04-10
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 HyperplasiaClinical trialOngoing
  • Classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency
  • 2
2021-01-07
A Phase 3, Randomized, Double-blind, Placebo-controlled, Multi-center Trial to Assess Efficacy and Safety of Octreotide Subcutaneous Depot (CAM2029) in Patients With AcromegalyClinical trialTerminated
  • Acromegaly
  • 3
2020-12-11
A Phase 3 Trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe ObesityClinical trialTerminated
  • Alström syndrome
  • Bardet-Biedl syndrome
  • 3
2019-08-27
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 HyperplasiaClinical trialOngoing
  • Classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency
  • 2
2021-02-23
A Phase 3, Open-label, Randomized, Multicenter, 12 Months, Efficacy and Safety Study of Weekly MOD-4023 Compared to Daily Genotropin - Therapy in Pre-pubertal Children With Growth Hormone DeficiencyClinical trialOngoing
  • Non-acquired isolated growth hormone deficiency
  • Non-acquired combined pituitary hormone deficiency
  • 3
2017-10-31
The Etiology and Extent of Impaired Quality of Life, Fatigue and Cognitive, Affective and Emotional Dysfunction in Patients With Cushing's Syndrome - Prospective StudiesObservational studyOngoing
  • Endogenous Cushing syndrome
  • NA
2017-05-01
A Prospective, Multicenter, Non-interventional Study to Investigate the Disease Characteristics of Adult Patients With Long Chain Fatty Acid Oxidation Disorders (FAOD)Observational studyOngoing
  • Long chain 3-hydroxyacyl-CoA dehydrogenase deficiency
  • NA
2021-01-27
A Phase 3, open-label, single-arm, multi-center trial to assess the long term safety of octreotide subcutaneous depot (CAM2029) in patients with acromegalyClinical trialOngoing
  • Acromegaly
  • 3
2020-11-12
LIBRETTO-531: 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 CancerClinical trialOngoing
  • Familial medullary thyroid carcinoma
  • 3
2020-03-10
REAL4: A Trial Comparing the Effect and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® in Children With Growth Hormone DeficiencyClinical trialOngoing
  • Non-acquired isolated growth hormone deficiency
  • 3
2019-05-20
GRADIENT: Glucocorticoid Receptor Antagonism in the Treatment of Hypercortisolism in Patients with Cortisol-Secreting Adrenal Adenomas or Hyperplasia: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of RelacorilantClinical trialOngoing
  • Endogenous Cushing syndrome
  • 3
2020-11-05
A Pivotal, International, Randomised, Double-blind, Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram SyndromeClinical trialOngoing
  • Wolfram syndrome
  • 2
2020-10-09
COMPOSE: A Prospective, Randomized, Controlled, Open-label, Multicenter Trial to Evaluate [...] Lutetium (177Lu) Edotreotide [...] in Patients with Well-differentiated Aggressive Grade 2 and Grade 3, Somatostatin receptor-positive (SSTR+), Neuroendocrine Tumors of GastroEnteric or Pancreatic OriginClinical trialOngoing
  • Gastroenteropancreatic neuroendocrine neoplasm
  • 3
2021-10-29
BALANCE: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA-APOCIII-LRX Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)Clinical trialOngoing
  • Familial chylomicronemia syndrome
  • 3
2021-05-20
UNLOCKED: A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of CareClinical trialTerminated
  • Hyperinsulinism-hyperammonemia syndrome
  • 2
2019-11-13
Name of StudyERNsTypeStatusOrphaCodeDiseasePhaseDateLink
Last update: April 15, 2024 on 08:44

Legend

1) Name of Study: Official title of the study.
2) ERN: European Reference Networks (ERNs) involved to study. Full list of ERNs can be found here: European Reference Networks.
3) Type: Type of study:
Clinical trial: Interventional study, evaluating a drug, a medical device, a protocol, a gene therapy, a cell therapy or a vaccine.
Observational study: Clinical research project designed to study the course of a disease
(observe the effects of an intervention/exposure or follow a cohort of patients over a period of time).
4) Status: Current status of the study (Ongoing or Terminated).
5) OrphaCode: Target disease(s) (OrphaCode) that is being studied, OrphaCodes available here.
6) Disease: Disease(s) that is being studied.
7) Phase: Stage of the Clinical Trial (not applicable "NA" for observational study).
8) Date: Start date of the study.
9) Link: Direct Expert Link to the study on the Orphanet.