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Congenital, familial and genetic disorders – page 56

  • 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

Osteochondrodysplasia

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Vosoritide
  • Sponsor:Biomarin Pharmaceutical Inc.

Von Willebrand's disease

Not recruiting
Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Octocog Alfa
  • Sponsor:Baxalta Innovations GmbH

Osteogenesis imperfecta

Not recruiting
Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Setrusumab
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Propionic acidaemia

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Codeine Phosphate
  • Sponsor:Moderna Therapeutics Inc.

Study on the Safety and Effectiveness of SRP-9003 and Prednisone for Patients with Limb-Girdle Muscular Dystrophy Type 2E/R4

Not recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Prednisone
  • Sponsor:Sarepta Therapeutics Inc.

Type IIa hyperlipidaemia

Not recruiting
Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Sponsor:Merck Sharp & Dohme LLC

Haemophilia

Not recruiting
InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Fitusiran
  • Sponsor:Genzyme Corp.

Mucopolysaccharidosis II

Not recruiting
InvestigationalNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:0–17 years · 18–64 years
  • Substances:Iduronate-2-Sulfatase Fused To A Fc Polypeptide That Binds To The Human Transferrin Receptor
  • Sponsor:Denali Therapeutics Inc.

Mitochondrial DNA depletion

Not recruiting
InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Doxecitine
  • Sponsor:UCB Biosciences Inc.

Immune-mediated enterocolitis+1

Not recruiting
Registered drug
Countries:CzechiaCzechia
  • Participants:0–17 years · 18–64 years
  • Substances:Ciclosporin
  • Sponsor:Novartis Pharma AG

Facioscapulohumeral muscular dystrophy

Not recruiting
InvestigationalNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years
  • Substances:Losmapimod
  • Sponsor:Fulcrum Therapeutics Inc.

Factor IX deficiency

Not recruiting
Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Etranacogene Dezaparvovec
  • Sponsor:CSL Behring LLC

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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