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Congenital, familial and genetic disorders – page 34

  • 796 clinical trials
  • 308 recruiting
  • 374 conditions
  • 27 countries

Conditions in this area

374 conditions with trials, the busiest first

Duchenne muscular dystrophy28Factor VIII deficiency25Sickle cell disease25Cystic fibrosis24Hereditary angioedema22Spinal muscular atrophy17Hypertrophic cardiomyopathy16Type IIa hyperlipidaemia15Glycogen storage disease type II14Factor IX deficiency13Sickle cell anaemia13Congenital cystic kidney disease12Huntington's disease12Myotonic dystrophy12Non-small cell lung cancer12Osteochondrodysplasia12Adrenogenital syndrome11Alpha-1 antitrypsin deficiency11K-ras gene mutation11Retinitis pigmentosa11Severe myoclonic epilepsy of infancy11Thalassaemia11Fabry's disease10Hereditary angioedema with C1 esterase inhibitor deficiency10+350

Clinical trials in Congenital, familial and genetic disorders

796 trials across Europe, recruiting first

Type of trial
Recruitment status
Quick filters

796 clinical trials

Hereditary angioedema with C1 esterase inhibitor deficiency+1

Not recruiting

Study of Navenibart to prevent attacks in adults and adolescents with Hereditary Angioedema

Investigational
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:NAVENIBART
  • Sponsor:Astria Therapeutics Inc.

Familial hypertriglyceridaemia+1

Not recruiting

Study of ISIS 678354 for Patients with Severe High Triglycerides

Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Isis 678354 Sodium Salt
  • Sponsor:Ionis Pharmaceuticals Inc.

Primary immunodeficiency syndrome

Not recruiting

Study on the Effects of TAK-881 and Hyaluronidase in Adults with Primary Immunodeficiency Diseases

Registered drugNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Human Normal Immunoglobulin
  • Sponsor:Takeda Development Center Americas Inc.

Primary immunodeficiency syndrome

Not recruiting

Study on Long-term Safety of TAK-881 (Human Normal Immunoglobulin, Hyaluronidase) for Patients with Primary Immunodeficiency Diseases

InvestigationalNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Human Normal Immunoglobulin
  • Sponsor:Baxalta Innovations GmbH

Familial hypertriglyceridaemia+1

Not recruiting

Study of Olezarsen (ISIS 678354) in Patients with Severe Hypertriglyceridemia: Testing Effects on Triglyceride Levels

Investigational
Countries:BulgariaBulgaria
  • Participants:18–64 years · 65+ years
  • Substances:Isis 678354 Sodium Salt
  • Sponsor:Ionis Pharmaceuticals Inc.

Hereditary ataxia

Not recruiting

Safety and tolerability of intrathecal S233107 in participants with spinocerebellar ataxia type 3: a Phase 1b/2a first‑in‑human randomized study

Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years
  • Sponsor:Institut De Recherches Internationales Servier IRIS

Cystic fibrosis

Not recruiting

Evaluation of Safety, Tolerability, and Biodistribution of Inhaled RCT2100 in Cystic Fibrosis: A Phase 1/2 Multicenter Study

Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years
  • Substances:Ivacaftor
  • Sponsor:Recode Therapeutics Inc.

Congenital absence of bile ducts

Not recruiting

Efficacy and Safety Evaluation of Odevixibat in Pediatric Biliary Atresia Post-Kasai Hepatoportoenterostomy: A Double-Blind, Randomized, Placebo-Controlled Trial

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Odevixibat
  • Sponsor:Ipsen Pharma

Thalassaemia beta

Not recruiting

Evaluation of the Efficacy, Safety, and Tolerability of Subcutaneous REGN7999 in Adults with Iron Overload Due to Non-Transfusion Dependent Beta-Thalassemia

Investigational
Countries:GreeceGreece
  • Participants:18–64 years
  • Substances:REGN7999
  • Sponsor:Regeneron Pharmaceuticals Inc.

A study to evaluate how the body processes AZ-3102 in healthy male subjects

Not recruiting
InvestigationalNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years · Healthy volunteers
  • Substances:(2S,3R,4R,5S)-1-[5-(2-FLUORO-BIPHENYL-4-YLMETHOXY)-PENTYL]-2-HYDROXYMETHYL-PIPERIDINE-3,4,5-TRIOL
  • Sponsor:Azafaros B.V.

Cystic fibrosis

Not recruiting

Bioequivalence Study of Ivacaftor 150 mg Film‑Coated Tablets in Healthy Adults for Cystic Fibrosis

Registered drugNo placebo
Countries:RomaniaRomania
  • Participants:18–64 years · Healthy volunteers
  • Substances:Ivacaftor
  • Sponsor:Sun Pharmaceutical Industries Limited

Angelman's syndrome

Not recruiting

A Study Testing the Safety and How Well GTX-102 Works in Adults and Children with Angelman Syndrome

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:APAZUNERSEN
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Substances studied in this area

The therapies tested most often in Congenital, familial and genetic disorders trials.

Sirolimus21 clinical trialsSodium Chloride21 clinical trialsPrednisolone12 clinical trialsPlerixafor11 clinical trialsBusulfan10 clinical trialsIvacaftor9 clinical trialsMitapivat9 clinical trialsParacetamol8 clinical trialsTezacaftor8 clinical trials
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