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Congenital, familial and genetic disorders – page 60

  • 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

Huntington's disease

Not recruiting
Investigational
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:VOTOPLAM
  • Sponsor:Novartis Pharma AG

Huntington's disease

Not recruiting
InvestigationalNo placebo
Countries:SpainSpain
  • Participants:18–64 years
  • Substances:Mbf-015
  • Sponsor:Medibiofarma S.L.

Facioscapulohumeral muscular dystrophy

Not recruiting
Registered drug
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Satralizumab
  • Sponsor:Centre Hospitalier Universitaire De Nice

Severe myoclonic epilepsy of infancy

Not recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Fenfluramine Hydrochloride
  • Sponsor:UCB Biosciences Inc.

Osteochondrodysplasia+1

Not recruiting
InvestigationalNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years
  • Sponsor:Sanofi-Aventis Recherche & Developpement

Type IIa hyperlipidaemia

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

Ichthyosis+1

Not recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Deucravacitinib
  • Sponsor:Centre Hospitalier Universitaire De Nice

Adrenogenital syndrome

Not recruiting
Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Abiraterone Acetate
  • Sponsor:Crinetics Pharmaceuticals Inc.

Cystic fibrosis

Not recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Substances:Ivacaftor
  • Sponsor:Vertex Pharmaceuticals Inc.

Osteogenesis imperfecta

Not recruiting
Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Calcium
  • Sponsor:Amgen Inc.

Hypertrophic cardiomyopathy

Not recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Mavacamten
  • Sponsor:Myokardia Inc.

Hereditary angioedema with C1 esterase inhibitor deficiency+1

Not recruiting
InvestigationalNo placebo
Countries:BulgariaBulgaria
  • Participants:18–64 years · 65+ years
  • Substances:Star-0215
  • Sponsor:Astria Therapeutics 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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