Clinical Trials

Clinical Trials

Filter list of Clinical Trials

TopicTrial name of investigational drugClinical Trial ID(s)MTG(s)StatusOrphaCodeLink
Diabetes MellitusASCEND: A Study of Cardiovascular Events in Diabetes
  • NCT00135226
3
  • ongoing
  • ORPHA101952
Type 1 Diabetes MellitusStudy of Safety and Efficacy of CFZ533 in Type 1 Diabetes Pediatric and Young Adult Subjects (CCFZ533X2207)
  • NCT04129528
3
  • ongoing
  • recruitment
  • ORPHA181371
Type 1 Diabetes MellitusKidsAP02
  • KIDSAP consortium
3
  • ongoing
  • ORPHA181371
Type 1 Diabetes MellitusInitiative pour la Promotion de la Qualité et Epidémiologie chez les Enfants et Adolescents atteints du Diabète sucré (IPQE-EAD
  • IPQE-EAD
  • IKEKAD
3
  • ongoing
  • ORPHA181371
Type 1 Diabetes MellitusA 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).
  • NCT02825251
3
  • completed
  • ORPHA181371
Type 1 Diabetes MellitusEvaluation of the Dreamed Advisor Pro for Automated Insulin Pump Setting Adjustments in Children and Adolescents With Type 1 Diabetes- The Advice4U Pro Study
  • NCT03003806
3
  • completed
  • ORPHA181371
Type 1 Diabetes MellitusFLAIR- 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
  • NCT03040414
3
  • completed
  • ORPHA181371
Type 1 Diabetes MellitusDiagnode 2 - GAD-Alum (Diamyd) Administered Into Lymph Nodes in Combination With Vitamin D in Type 1 Diabetes
  • NCT03345004
3
  • concluded
  • ORPHA181371
Type 1 Diabetes MellitusPhase 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
  • NCT03667053
3
  • completed
  • ORPHA181371
Type 1 Diabetes MellitusA Study Comparing LY900014 to Insulin Lispro (Humalog) in Children and Adolescents With Type 1 Diabetes (PRONTO-Peds)
  • NCT03740919
3
  • concluded
  • ORPHA181371
Type 1 Diabetes MellitusThe Artificial Pancreas in Very Young Children With T1D (KidsAP02)
  • NCT03784027
3
  • completed
  • ORPHA181371
Type 1 Diabetes MellitusA 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)
  • NCT03875729
  • PRV-031-001
3
  • ongoing
  • ORPHA181371
Type 2 Diabetes MellitusDiabetes Study of Linagliptin and Empagliflozin in Children and Adolescents (DINAMO)TM
  • NCT03429543
3
  • ongoing
  • ORPHA181376
Type 2 Diabetes MellitusA 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
  • NCT04029480
  • MK8835-059
3
  • ongoing
  • recruiting
  • ORPHA181376
Bacterial riskBacteremia Antibiotic Length Actually Needed for Clinical Effectiveness: Randomized Controlled Trial
  • NCT03005145
  • ongoing
Cluster Headache - Episodic and ChronicNOVARTIS SOM B2412 roll over
  • NCT02619617
  • completed
Idiopathic Short StatureA 4 Year Combination Therapy of Growth Hormone and (GnRH) Agonist in Children With a Short Predicted Height (ZomaTrip)
  • NCT00840944
5
  • ongoing
Metabolic AcidosisA 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
  • 2018-001303-36
ERKNetMetabERN
  • ongoing
Natriuretic Peptides,ObesityThe Diurnal Rhythm in Natriuretic Peptide Levels and Relationship With Nocturnal Blood Pressure
  • NCT03834168
3
  • ongoing
Small for Gestational AgeREAL 5
  • 2018-000232-10
5
  • ongoing
Small for Gestational AgeEffect of Insulin Sensitization on IGF-1 Response to Growth Hormone in SGA Children
  • NCT02931591
5
  • completed
Biobanking In cancer patientsPersonalize My Treatment (PMT) Registry (PMT)
  • NCT02355171
  • ongoing
Graves’ hyperthyroidismSelenium supplementation for patients with Graves’ hyperthyroidism (the GRASS trial)
  • NCT01611896
8
  • concluded
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
  • NCT03746522
56
  • concluded
  • OPRHA240371
  • OPRHA64
  • ORPHA110
Genetic ObesitySetmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity
  • NCT03651765
56
  • ongoing
  • recruiting
  • OPRHA240371
  • OPRHA64
  • ORPHA110
Behçet's DiseaseStudio 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
  • NCT02307513
BloodNET ERN ??
  • concluded
  • ORPHA:117
Neuroendocrine TumoursEfficacy and Safety Study in Pancreatic or Midgut Neuroendocrine Tumours Having Progressed Radiologically While Previously Treated With Lanreotide Autogel® 120 mg (CLARINET FORTE)
  • NCT02651987
4
  • concluded
  • ORPHA100092
Neuroendocrine TumorsEfficacy and Safety of Lanreotide Autogel/ Depot 120 mg vs. Placebo in Subjects With Lung Neuroendocrine Tumors (SPINET)
  • NCT02683941
4
  • completed
  • ORPHA100101
Neuroendocrine TumorsEfficacy and Safety of Lanreotide Autogel (ATG) in Combination With Temozolomide in Subjects With Thoracic Neuroendocrine Tumors. (ATLANT)
  • NCT02698410
4
  • completed
  • ORPHA100101
Chronic Adrenal InsufficiencyA European Multi-Centre, Post-Authorization, Observation Study (Registry) of Patients with Chronic Adrenal Insufficiency
  • NCT01661387
1
  • Terminated
  • ORPHA101959
Medullary Thyroid CarcinomaA Study of Two Different Doses of Cabozantinib (XL184) in Progressive, Metastatic Medullary Thyroid Cancer (EXAMINER)
  • NCT01896479
8
  • completed
  • ORPHA1332
Medullary Thyroid CarcinomaA 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)
  • NCT04211337
  • EUDRACT: 2019-001978-28
8
  • ongoing
  • recruiting
  • ORPHA1332
Thyroid CarcinomaPhase 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)
  • NCT03037385
8
  • ongoing
  • ORPHA1332
Anaplastic Thyroid CarcinomaNEO 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).
  • NCT01099436
8
  • recruiting
  • ORPHA142
Thyroid CarcinomaLenvatinib
  • 2014-005199-27
8
  • Results
  • ORPHA146
Thyroid CarcinomaFocus on Values to Stimulate Shared Decisions in Patients With Thyroid Cancer: A Multifaceted COMmunication BOoster (COMBO)s
  • NCT03905369
8
  • ongoing
  • ORPHA146
Thyroid NeoplasmsFDG-PET/CT in Evaluation of Cytological Indeterminate Thyroid Nodules to Prevent Unnecessary Surgery (EfFECTS) (EfFECTS)
  • NCT02208544
8
  • completed
  • ORPHA146
AchondroplasiaA Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
  • NCT03197766
5
  • completed
  • ORPHA15
Adrenocortical carcinomaEfficacy of Adjuvant Mitotane Treatment (ADIUVO) (ADIUVO)
  • NCT00777244
1
  • ongoing
  • ORPHA1501
Diabetes InsipidusCARGOx - Use of Copeptin Measurement After Arginine Infusion for the Differential Diagnosis of Diabetes Insipidus - the CARGOx Study (CARGOx)
  • NCT03572166
6
  • ongoing
  • recruiting
  • ORPHA178029
Genetic ObesitySetmelanotide for the Treatment of LEPR Deficiency Obesity
  • NCT03287960
56
  • concluded
  • ORPHA179494
Type 1 Diabetes MellitusLA Study Comparing LY900014 to Insulin Lispro (Humalog) in Adults With Type 1 Diabetes Using Insulin Pump Therapy (PRONTO-Pump-2)
  • NCT03830281
3
  • completed
  • ORPHA181371
HypoparathyroidismA Clinical Study Investigating the Safety, Tolerability, PK and PD of PCO371 in Patients With Hypoparathyroidism - PCO 104 UG
  • NCT 04209179
2
  • Terminated
  • ORPHA181405
HypoparathyroidismA Study to Learn if Recombinant Human Parathyroid Hormone [rhPTH(1-84)] Can Improve Symptoms and Metabolic Control in Adults With Hypoparathyroidism (BALANCE)
  • NCT03324880
2
  • ongoing
  • recruiting
  • ORPHA181405
HypoparathyroidismA Randomized, Double-Blind, Multiple Ascending Oral Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of PCO371 in Patients With Hypoparathyroidism
  • NCT04209179
2
  • Terminated
  • ORPHA181405
Primary AldosteronismDP13 - A Phase II Study in Patients With Primary Aldosteronism
  • NCT04007406
1
  • completed
  • ORPHA181415
PheochromocytomaPRESCRIPT - Phenoxybenzamine Versus Doxazosin in PCC Patients
  • NCT01379898
14
  • concluded
  • ORPHA276621
  • ORPHA29072
Pheochromocytoma and ParagangliomaMulticenter Pheochromocytoma and Paraganglioma Evaluation (MUPPET)
  • NCT03344016
14
  • ongoing
  • ORPHA276621
  • ORPHA29072
Pheochromocytoma and ParagangliomaFIRSTMAPPP, Genetic variants associated with increased susceptibility to thyroid cancer
  • NCT01371201
14
  • ongoing
  • ORPHA276621
  • ORPHA29072
Pheochromocytoma/Paraganglioma, Pancreatic Neuroendocrine TumoursA study of MK-6482 Monotherapy in advanced Pheochromocytoma/Paraganglioma (PPGL) and pancreatic neuroendocrine tumors (pNET)
  • MK6482-PN015
14
  • ongoing
  • recruiting
  • ORPHA276621
  • ORPHA29072
ProlactinomaPROLACT A Phase III study of efficacy, safety and tolerability of Chronocort® compared with standard glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasia
  • NCT04107480
6
  • ongoing
  • recruiting
  • ORPHA2965
PHTS tumour syndromeINSPECT (Investigation into PHTS tumor syndrome)
  • 2018-5056
4
  • ongoing
  • ORPHA306498
Wolfram SyndromeTreatment 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
  • NCT03717909
3
  • ongoing
  • ORPHA3463
Familial HypercholesterolemiaA 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
  • EudraCT Number: 2011-005400-15
GuardHeart??
  • ongoing
  • ORPHA391665
Congenital Adrenal HyperplasiaPREDEX
  • 15554888
1
  • recruiting
  • ORPHA418
Congenital Adrenal HyperplasiaA Phase III study of efficacy, safety and tolerability of Chronocort® compared with standard glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasia
  • NCT02716818
1
  • concluded
  • ORPHA418
Congenital Adrenal HyperplasiaOpen-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapy (RESTORE)
  • NCT03532022
1
  • ongoing
  • ORPHA418
Congenital Adrenal HyperplasiaA 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
  • NCT04045145
  • NBI-74788
1
  • starting
  • recruiting
  • ORPHA418
Congenital Adrenal HyperplasiaA 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
  • NCT04806451
  • NBI-74788-CAH2006
1
  • Ongoing
  • ORPHA418
Congenital Adrenal HyperplasiaDIUR-006 : A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAH Diurnal Limited
  • NCT03062280
  • EudraCT Number: 2015-005448-32
1
  • ongoing
  • ORPHA418
Congenital Adrenal HyperplasiaA 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
  • NCT04457336
1
  • ongoing
  • ORPHA418
Congenital Adrenal HyperplasiaCAHtalyst 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,
  • NCT04490915
  • NBI-74788-CAH3003
1
  • ongoing
  • recruiting
  • ORPHA418
Congenital Adrenal HyperplasiaA 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
  • NCT04544410
1
  • ongoing
  • ORPHA418
Transition,paediatric chronic endocrine disordersGrowing up With the Young Endocrine Support System (YESS!) (YESS)
  • NCT04252001
15678
  • ongoing
  • ORPHA418
  • ORPHA174590
  • ORPHA881
  • ORPHA95488
  • ORPHA85138
  • ORPHA754
  • ORPHA9571
  • ORPHA95494
Adrenal TumoursCHIRACIC -Surgery of Subclinical Cortisol Secreting Adrenal Incidentalomas (CHIRACIC)
  • NCT02364089
1
  • ongoing
  • recruiting
  • ORPHA443287
Adrenal TumoursFAMAIN
  • EudraCT Number: 2012-003604-13
1
  • ongoing
  • ORPHA443287
  • ORPHA1501
Adrenal TumoursCombined 18F-Fluorodeoxyglucose (FDG) Positron Emission Tomography (PET) and 123I-Iodometomidate (123I-IMTO) Imaging for Adrenal Neoplasia.
  • NCT02010957
1
  • concluded
  • ORPHA443287
  • ORPHA1501
Adrenal TumoursStructured Evaluation of adRENal Tumors Discovered Incidentally - Prospectively Investigating the Testing Yield (SERENDIPITY)
  • NCT02324647
1
  • completed
  • ORPHA443287
  • ORPHA1501
Adrenal TumoursGRADIENT; A phase 3, randomized, double-blind, placebo-controlled study of the efficacy and safety of Relacorilant
  • NCT04308590
1
  • ongoing
  • ORPHA443287
  • ORPHA553
Hypothalamic ObesityEfficacy and Safety of Exenatide in the Treatment of Hypothalamic Obesity After Craniopharyngioma Therapy (CRANIOEXE)
  • NCT02860923
6
  • concluded
  • ORPHA54595
Cushing SyndromeLOGICS: A Study to Assess the Safety and Efficacy of Levoketoconazole in the Treatment of Endogenous Cushing's Syndrome (Levoketoconazole).
  • NCT03277690
16
  • concluded
  • ORPHA553
  • ORPHA99892
  • ORPHA99893
Cushing SyndromeAn 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
  • NCT03606408
16
  • ongoing
  • ORPHA553
  • ORPHA99892
  • ORPHA99893
Cushing SyndromeAn Open-Label Extension Study of Levoketoconazole (2S,4R-ketoconazole) in the Treatment of Endogenous Cushing’s Syndrome
  • NCT03621280
  • COR-2017-OLE
16
  • ongoing
  • ORPHA553
  • ORPHA99892
  • ORPHA99893
McCune- Albright SyndromeMCRN007 (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
  • NCT00278915
1268
  • ongoing
  • ORPHA562
MCT8 DeficiencyThyroid Hormone Analog Therapy in MCT8 Deficiency: Triac Trial Patients
  • NCT02060474
8
  • completed
  • ORPHA59
MCT8 DeficiencyTriac Trial II in MCT8 Deficiency Patients
  • NCT02396459
8
  • ongoing
  • recruiting
  • ORPHA59
Congenital HyperinsulinismAn Open-Label Multiple-Dose Study of RZ358 in Patients with Congenital Hyperinsulinism”. Estudio abierto de dosis múltiples de RZ358 en pacientes con hiperinsulinismo congénito.
  • EudraCT 2016-004186-83
  • CODIGO RZ358-606. RZ358 - 606
3
  • ongoing
  • results
  • ORPHA657
Congenital HyperinsulinismA Two-Period, Open-label Trial Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism
  • NCT03777176
3
  • completed
  • ORPHA657
Congenital HyperinsulinismAn Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism
  • NCT03941236
3
  • ongoing
  • ORPHA657
Congenital HyperinsulinismTrial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism
  • NCT04172441
3
  • completed
  • ORPHA657
Congenital HyperinsulinismAn Open-Label Multiple-Dose Study of RZ358 in Patients with Congenital Hyperinsulinism, Protocol RZ358-606
  • NCT04538989
  • RZ358-606
3
  • ongoing
  • recruiting
  • ORPHA657
Genetic ObesitySetmelanotide for the Treatment of Early-Onset POMC Deficiency Obesity
  • NCT 02896192
56
  • concluded
  • ORPHA71526
Genetic ObesityLong Term Extension Trial of Setmelanotide
  • NCT 03013543
56
  • ongoing
  • ORPHA71526
Genetic ObesityRM-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)
  • NCT04963231
56
  • concluding
  • ORPHA71529
Prader-Willi SyndromeEffect of Liraglutide for Weight Management in Paediatric Subjects With Prader-Willi Syndrome
  • NCT02527200
5
  • ongoing
  • ORPHA739
Prader-Willi SyndromeZEPHYR Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome (ZEPHYR
  • NCT03790865
5
  • Terminated
  • ORPHA739
Prader-Willi SyndromeOxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome (OTBB3)
  • NCT04283578
5
  • ongoing
  • ORPHA739
Adrenal InsufficiencyOptimising Steroid Replacement in Patients With Adrenal Insufficiency (Plenadren)
  • NCT03282487
1
  • completed
  • ORPHA85138
  • ORPHA199296
  • ORPHA
  • 199299
Neuroendocrine TumoursStudy 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)
  • NCT02955069
4
  • completed
  • ORPHA877
  • ORPHA100092
  • ORPHA100101
X-Linked HypophosphatemiaX linked Hypof (XLH) Registry for Patients With X-Linked Hypophosphatemia (XLH Registry)
  • NCT03193476
2
  • ongoing
  • ORPHA89936
X-linked hypophosphatemiaA 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
  • NCT04188964
2
  • ongoing
  • ORPHA89936
Growth Hormone DeficiencyInvestigating 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
  • NCT02616562
6
  • ongoing
  • recruiting
  • ORPHA95488
Growth Hormone DeficiencySafety and Efficacy Study of MOD-4023 to Treat Children With Growth Hormone Deficiency
  • NCT03874013
6
  • completed
  • ORPHA95488
Growth Hormone DeficiencyA 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)
  • NCT04615273 (TCH-306)
6
  • ongoing
  • recruiting
  • ORPHA95488
Growth Hormone DeficiencyA Phase II, Dose and Frequency Finding Study of MOD-4023 in Growth Hormone Deficient Adults (GHDA)
  • MOD-4023 (2) - OLE
6
  • completed
  • ORPHA95488
Growth Hormone DeficiencyA phase 3 trial evaluating the effect of long acting GH (MOD-4023) in patients with GHD. CP-4-005
  • NCT01909479
6
  • completed
  • ORPHA95488
Growth Hormone DeficiencyA Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)
  • NCT02781727
6
  • completed
  • ORPHA95488
Growth Hormone DeficiencySafety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children
  • NCT02968004
6
  • ongoing
  • ORPHA95488
Growth Hormone DeficiencyTransCon hGH 301-CT Ext (1) Enlighten (weekly GH/Transcon
  • NCT03344458
6
  • ongoing
  • ORPHA95488
Growth Hormone DeficiencyA trial comparing the effect and safety of once weekly dosing of somapacitan with daily Norditropin® in children with growth hormone deficiency (REAL 4)
  • NCT03811535
6
  • ongoing
  • ORPHA95488
Growth Hormone DeficiencyA 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
  • NCT03831880
6
  • completed
  • ORPHA95488
Growth Hormone DeficiencyRegistre belge des enfants traités par hormone de croissance BELGROW
  • BELGROW
6
  • ongoing
  • ORPHA95488
  • ORPHA314795
  • ORPHA364526
Cushing DiseaseEfficacy and Safety Evaluation of Osilodrostat in Cushing's Disease (LINC-4)
  • NCT02697734
6
  • completed
  • ORPHA96253
Cushing DiseaseA 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
  • NCT01331239
6
  • completed
  • ORPHA96253
Cushing DiseasePhase 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
  • NCT02180217
6
  • completed
  • ORPHA96253
Cushing DiseaseA 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
  • NCT03708900
6
  • ongoing
  • recruiting
  • ORPHA96253
AcromegalyComparison of Oral Octreotide Capsules to Injectable Somatostatin Analogs in Acromegaly (MPOWERED).
  • NCT02685709
6
  • completed
  • ORPHA963
AcromegalyEfficacy and Safety of Octreotide Capsules (MYCAPSSA) in Acromegaly
  • NCT03252353
6
  • ongoing
  • ORPHA963
AcromegalyAn 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)
  • NCT03789656
6
  • concluded
  • ORPHA963
AcromegalyA 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)
  • NCT03792555
6
  • concluded
  • ORPHA963
AcromegalyA Phase 3, randomized, double-blind, placebo-controlled, multi-center trial to assess efficacy and safety of octreotide subcutaneous depot(CAM2029) in patients with acromegaly.
  • NCT04076462 (HS-18-633)
6
  • ongoing
  • recruiting
  • ORPHA963
AcromegalyA Trial to Assess the Long-term Safety of Octreotide Subcutaneous Depot in Patients With Acromegaly
  • NCT04125836 (HS-19-647)
6
  • ongoing
  • recruiting
  • ORPHA963
AcromegalyEuropea Observational study in Acromegaly - Acronis Study
  • CSOM230CIC05
6
  • recruiting
  • ORPHA963
Cushing Disease and AcromegalyAn 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
  • NCT01794793
6
  • ongoing
  • ORPHA963
Cushing SyndromeGRACE-2
  • NCT03604198
  • CORT125134-452
  • 2018-001616-30
16
  • ongoing
  • ORPHA99892
  • ORPHA96253
  • ORPHA99889
Cushing SyndromeGlucocorticoid 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
  • NCT03697109
  • CORT125134-455
  • NL68116.078.18
16
  • ongoing
  • ORPHA99892
  • ORPHA96253
  • ORPHA99889
Acquired Thrombotic Thrombocytopenic PurpuraA 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
  • EudraCT Number: 2017-001117-86 S
BloodNET ERN ??
  • ongoing
Adrenocortical CarcinomaACC: 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
  • NCT03723941
1
  • ongoing
  • ORPHA1501
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)
  • NCT04277598
  • MARSYAS II
  • APO-2
3
  • ongoing
  • ORPHA181371
  • ORPHA181376
DSDDSDCare: Standardized center-centric care of DSD across the lifespan
  • DRKS00022521
7
  • ongoing
  • ORPHA90771
  • ORPHA2982
  • ORPHA98085
  • ORPHA325546
Gastro-enteropancreatic Neuroendocrine TumorA 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)
  • NCT03972488
  • CAAA601A22301/NETTER-2
4
  • ongoing
  • ORPHA100092
Medullary Thyroid CancerLibretto-531 study
  • 2019-001978-28
  • J2G-MC-JZJB
8
  • ongoing
  • ORPHA1332
Multiple Endocrine Neoplasia Type 1PRIME Precision Radiotherapy Using MR-linac for Pancreatic Neuroendocrine Tumours in MEN1 Patients
  • NCT05037461
4
  • starting
  • OPRPHA652
Neuroendocrine TumorsA 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).
  • NCT03049189
  • ITM-LET-01-COMPETE
4
  • ongoing
  • ORPHA100092
  • ORPHA100075
  • ORPHA506052
Osteogenesis ImperfectaAn Open-label, Ascending Multiple-dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
  • NCT04545554
ERN BOND
  • Ongoing
  • ORPHA666
Pheochromocytoma/Paraganglioma, pancreatic neuroendocrine tumorsA 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)
  • NCT04924075
  • MK6482- PN015
14
  • Ongoing
  • ORPHA573163
  • ORPHA97253
Prader-Willi SyndromeOTBB3 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
  • NCT05032326
5
  • Recrutiing
  • ORPHA739
Prader-Willi SyndromeOXYTOCIN 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)
  • Number EudraCT 2019-002385- 12
5
  • Active
  • ORPHA739
Prader-Willi SyndromeLong-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial (OTBB3-FU)
  • Number EudraCT 2020-006161-11
5
  • Active
  • recruiting
  • ORPHA739
Type 1 Diabetes MellitusStudy of Safety and Efficacy of CFZ533 in Type 1 Diabetes Pediatric and Young Adult Subjects (CCFZ533X2207)
  • NCT04129528
3
  • ongoing
  • recruitment
  • ORPHA181371
TopicTrial name of investigational drugClinical Trial ID(s)MTG(s)StatusOrphaCodeLink