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Congenital, familial and genetic disorders – page 35

  • 803 clinical trials
  • 311 recruiting
  • 375 conditions
  • 27 countries

Conditions in this area

375 conditions with trials, the busiest first

Duchenne muscular dystrophy28Factor VIII deficiency25Sickle cell disease25Cystic fibrosis24Hereditary angioedema22Hypertrophic cardiomyopathy17Spinal muscular atrophy17Type IIa hyperlipidaemia15Glycogen storage disease type II14Factor IX deficiency13Huntington's disease13Sickle cell anaemia13Congenital cystic kidney disease12Myotonic dystrophy12Non-small cell lung cancer12Osteochondrodysplasia12Adrenogenital syndrome11Alpha-1 antitrypsin deficiency11K-ras gene mutation11Retinitis pigmentosa11Severe myoclonic epilepsy of infancy11Thalassaemia11Epidermolysis bullosa10Fabry's disease10+351

Clinical trials in Congenital, familial and genetic disorders

803 trials across Europe, recruiting first

Type of trial
Recruitment status
Quick filters

803 clinical trials

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.

Primary hyperoxaluria

Not recruiting

Study of nedosiran for patients with primary hyperoxaluria type 1 and severe kidney impairment with or without dialysis

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years
  • Substances:Nedosiran
  • Sponsor:Dicerna Pharmaceuticals Inc.

Hypophosphatasia

Not recruiting

Study of ilofotase alfa effects on biomarkers in adults with hypophosphatasia

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Ilofotase Alfa
  • Sponsor:AM-Pharma B.V.

Duchenne muscular dystrophy

Not recruiting

Study of SAT-3247 Compared to Placebo for Patients with Duchenne Muscular Dystrophy Who Can Walk

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:SAT-3247 oxalate
  • Sponsor:Satellos Bioscience Inc.

Primary immunodeficiency syndrome

Not recruiting

Long-term safety and effectiveness study of leniolisib for patients with primary immunodeficiency and immune system problems

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Leniolisib Phosphate
  • Sponsor:Pharming Technologies B.V.

Type IIa hyperlipidaemia

Not recruiting

A study testing how well zodasiran works and how safe it is for teenagers and adults with homozygous familial hypercholesterolemia

Investigational
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Zodasiran
  • Sponsor:Arrowhead Pharmaceuticals Inc.

Factor VIII deficiency+1

Not recruiting

A study looking at the safety of switching from emicizumab to denecimig in adults and adolescents with haemophilia A with or without inhibitors

InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Sponsor:Novo Nordisk A/S

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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