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Congenital, familial and genetic disorders – page 53

  • 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

Hypertrophic cardiomyopathy

Not recruiting
Investigational
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Mavacamten
  • Sponsor:Bristol-Myers Squibb Services Unlimited Company

K-ras gene mutation+1

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Sotorasib
  • Sponsor:Institut Gustave Roussy

Fibrodysplasia ossificans progressiva

Not recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:(R)‑Tetrahydrofuran‑3‑Yl 4‑(6‑(5‑(4‑Ethoxy‑1‑Isopropylpiperidin‑4‑Yl)Pyridin-2-Yl)Pyrrolo[1,2-B]Pyridazin-4-Yl)Piperazine-1-Carboxylate Sesquisuccinate
  • Sponsor:Clementia Pharmaceuticals Inc. An Ipsen Company

Gaucher's disease type III+1

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Eliglustat Tartrate
  • Sponsor:Genzyme Corp.

Ataxia telangiectasia

Not recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Dexamethasone Sodium Phosphate
  • Sponsor:Quince Therapeutics S.p.A.

Gaucher's disease type I

Not recruiting
InvestigationalNo placebo
Countries:SpainSpain
  • Participants:18–64 years · 65+ years
  • Substances:Adeno-Associated Viral Vector Serotype S3 Containing Codon-Optimised Expression Cassette Encoding Human Beta-Glucocerebrosidase Variant
  • Sponsor:Spur Therapeutics Limited

Congenital cystic kidney disease

Not recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Tolvaptan
  • Sponsor:Otsuka Pharmaceutical Development & Commercialization Inc.

Cystic fibrosis

Not recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Dirocaftor
  • Sponsor:Universitair Medisch Centrum Utrecht

Osteochondrodysplasia

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

Hereditary angioedema

Not recruiting
InvestigationalNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Garadacimab
  • Sponsor:CSL Behring LLC

Metachromatic leukodystrophy

Not recruiting
Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Atidarsagene Autotemcel
  • Sponsor:Orchard Therapeutics (Europe) Limited

Fragile X syndrome

Not recruiting
InvestigationalNo placebo
Countries:IrelandIreland
  • Participants:0–17 years · 18–64 years
  • Substances:Cannabidiol
  • Sponsor:Harmony Biosciences Management 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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