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Congenital, familial and genetic disorders – page 4

  • 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

Osteogenesis imperfecta

Recruiting
Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Romosozumab
  • Sponsor:Amgen Inc.

Propionic acidaemia

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Modified Messenger Ribonucleic Acid Encoding Human Propionyl-Coenzyme A Carboxylase Alpha And Beta Subunits Encapsulated Into Lipid Nanoparticles
  • Sponsor:Moderna Therapeutics Inc.

K-ras gene mutation+1

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Panitumumab
  • Sponsor:Genentech Inc.

Thalassaemia beta+1

Recruiting
Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years
  • Substances:Busulfan
  • Sponsor:Fondazione Telethon Ets

Von Willebrand's disease

Recruiting
InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:VGA039
  • Sponsor:Vega Therapeutics Inc.

Hypertrophic cardiomyopathy

Recruiting
Registered drugNo placebo
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:Hydrochlorothiazide
  • Sponsor:Medical University Of Graz

Sickle cell anaemia+1

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Etavopivat
  • Sponsor:Novo Nordisk A/S

Adrenogenital syndrome

Recruiting
Investigational
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:ATUMELNANT
  • Sponsor:Crinetics Pharmaceuticals Inc.

Type IIa hyperlipidaemia

Recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Sponsor:Merck Sharp & Dohme LLC

Osteochondrodysplasia

Recruiting
Investigational
Countries:NorwayNorway
  • Participants:0–17 years
  • Substances:Infigratinib
  • Sponsor:Qed 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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