ERN Clinical Trials Repository
Name of Study | ERNs | Type | Status | OrphaCode | Disease | Phase | Date | Link |
---|---|---|---|---|---|---|---|---|
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 Study | Clinical trial | Ongoing |
|
| 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 Mutation | Clinical trial | Ongoing |
|
| 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 adrenoleukodystrophy | Clinical trial | Ongoing |
|
| 2017-10-26 | |||
COMPETE: Efficacy and Safety of 177Lu-edotreotide PRRT in GEP-NET Patients | Clinical trial | Ongoing |
|
| 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-P | Clinical trial | Ongoing |
|
| 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 trial | Ongoing |
|
| 2021-02-25 | |||
A One Year, Randomized, Double-Blind, Placebo-Controlled Study of TBR-760 in Adult Patients with Non-Functioning Pituitary Adenomas | Clinical trial | Ongoing |
|
| 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 trial | Ongoing |
|
| 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. - NA | Clinical trial | Ongoing |
|
| 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 therapy | Clinical trial | Ongoing |
|
| 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 Ligands | Clinical trial | Ongoing |
|
| 2021-05-12 | |||
An Open-Label Multiple-Dose Study of RZ358 in Patients with Congenital Hyperinsulinism | Clinical trial | Terminated |
|
| 2020-03-04 | |||
An Open-Label Phase 2 Study of Surufatinib in Patients with Neuroendocrine Tumours in Europe | Clinical trial | Ongoing |
|
| 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 Mutation | Clinical trial | Terminated |
|
| 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 pathway | Clinical trial | Ongoing |
|
| 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 Disease | Clinical trial | Ongoing |
|
| 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 Relacorilant | Clinical trial | Ongoing |
|
| 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 Disorders | Clinical trial | Ongoing |
|
| 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/Paraganglioma | Clinical trial | Ongoing |
|
| 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 Syndrome | Clinical trial | Ongoing |
|
| 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 Mutation | Clinical trial | Ongoing |
|
| 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 Syndrome | Clinical trial | Ongoing |
|
| 2018-02-23 | |||
OPTICS: An Open-label Extension Study of Levoketoconazole (2S,4R-ketoconazole) in the Treatment of Endogenous Cushing's Syndrome | Clinical trial | Ongoing |
|
| 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 trial | Ongoing |
|
| 2018-09-13 | |||
PODIUM 201: A Phase 2 Study of INCMGA00012 in Participants With Metastatic Merkel Cell Carcinoma | Clinical trial | Ongoing |
|
| 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 trial | Terminated |
|
| 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 Hyperplasia | Clinical trial | Ongoing |
|
| 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 Acromegaly | Clinical trial | Terminated |
|
| 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 Obesity | Clinical trial | Terminated |
|
| 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 Hyperplasia | Clinical trial | Ongoing |
|
| 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 Deficiency | Clinical trial | Ongoing |
|
| 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 Studies | Observational study | Ongoing |
|
| 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 study | Ongoing |
|
| 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 acromegaly | Clinical trial | Ongoing |
|
| 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 Cancer | Clinical trial | Ongoing |
|
| 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 Deficiency | Clinical trial | Ongoing |
|
| 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 Relacorilant | Clinical trial | Ongoing |
|
| 2020-11-05 | |||
A Pivotal, International, Randomised, Double-blind, Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome | Clinical trial | Ongoing |
|
| 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 Origin | Clinical trial | Ongoing |
|
| 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 trial | Ongoing |
|
| 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 Care | Clinical trial | Terminated |
|
| 2019-11-13 | |||
SPINET: A Phase 3, Prospective, Randomized, Double-blind, Multi-center Study of the Efficacy and Safety of Lanreotide Autogel/Depot 120 mg Plus BSC vs. Placebo Plus BSC for Tumor Control in Subjects With Well Differentiated, Metastatic and/or Unresectable, Typical or Atypical, Lung Neuroendocrine Tumors | Clinical trial | Ongoing |
|
| 2017-03-06 | |||
MOSAIC: A Phase 1/2 Study of ARQ 092 (Miransertib) in Subjects with PIK3CA-related Overgrowth Spectrum and Proteus Syndrome | Clinical trial | Terminated |
|
| 2017-05-16 | |||
VASE: Phase III multicentric study evaluating the efficacy and safety of sirolimus in Vascular Anomalies that are refractory to standard care | Clinical trial | Ongoing |
|
| 2016-01-25 | |||
A Randomised, Placebo Controlled, Double Blind, Multicentre Proof of Concept Study to Assess the Safety and Efficacy of Two Doses of VAD044 in Patients With Hereditary Hemorrhagic Telangiectasia (HHT) | Clinical trial | Ongoing |
|
| 2022-07-18 | |||
Effectiveness of Somatostatin Analogues in Patients with hereditary hemorrhagic telangiectasia and symptomatic gastrointestinal bleeding, the SAIPAN-trial: a multicenter, randomized, open-label, parallelgroup, superiority trial. | Clinical trial | Ongoing |
|
| 2020-11-26 | |||
NF1-EXCEL: The Effect of Lamotrigine on Cognitive Deficits Associated With Neurofibromatosis Type 1: a Phase II Randomized Controlled Multi-centre Trial |
| Clinical trial | Ongoing |
|
| 2014-10-01 | ||
A Phase 2, Randomized, Double-blind, Placebo-controlled Evaluation of the Safety and Efficacy of BMS-986165 With Background Treatment in Subjects With Lupus Nephritis |
| Clinical trial | Ongoing |
|
| 2019-10-02 | ||
SENSCISTM- ON: An open-label extension trial to assess the long term safety of nintedanib in patients with Systemic Sclerosis associated Interstitial Lung Disease (SSc-ILD) |
| Clinical trial | Ongoing |
|
| 2018-08-01 | ||
NOVESA: A Phase 2a Randomized, Double-blind, Placebo-controlled, Multi-center Study to Evaluate the Efficacy, Safety, and Tolerability of Orally Administered GLPG1690 for 24 Weeks in Subjects With Systemic Sclerosis |
| Clinical trial | Ongoing |
|
| 2019-01-14 | ||
A Randomized, Placebo-Controlled, Double-Blind, Multicenter Study to Assess the Efficacy and Safety of Branebrutinib Treatment in Subjects With Active Systemic Lupus Erythematosus or Primary Sjögren's Syndrome, or Branebrutinib Treatment Followed by Open-label Abatacept Treatment in Subjects With Active Rheumatoid Arthritis |
| Clinical trial | Ongoing |
|
| 2020-06-19 | ||
NECESSITY: NEw Clinical Endpoints in Patients With Primary Sjögren's Syndrome (pSS): an Interventional Trial Based on stratifYing Patients |
| Clinical trial | Ongoing |
|
| 2022-01-20 | ||
DETERMINE: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial to Evaluate Efficacy and Safety of Lenabasum in Dermatomyositis |
| Clinical trial | Terminated |
|
| 2019-11-20 | ||
The RECLAIIM Study: A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of IgPro20 (Subcutaneous Immunoglobulin, Hizentra®) in Adults with Dermatomyositis (DM |
| Clinical trial | Ongoing |
|
| 2019-10-21 | ||
COVID Vaccination in Rare and Complex Connective Tissue Diseases: ERN ReCONNET Multicentre Prospective Cohort Study |
| Observational Study | Ongoing |
|
| 2021-02-01 | ||
Name of Study | ERNs | Type | Status | OrphaCode | Disease | Phase | Date | Link |
Last update: November 28, 2024 on 11:19
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. |