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Congenital, familial and genetic disorders – page 13

  • 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

Type IIa hyperlipidaemia

Recruiting
Registered drug
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Inclisiran
  • Sponsor:Novartis Pharma AG

Rett syndrome

Recruiting
Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years
  • Substances:Mirtazapine
  • Sponsor:University Of Trieste

Basal cell naevus syndrome

Recruiting
Registered drugNo placebo
Countries:ItalyItaly
  • Participants:18–64 years · 65+ years
  • Substances:Sonidegib
  • Sponsor:Fondazione GONO Plus

Congenital cystic kidney disease

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years
  • Substances:Rotigotine
  • Sponsor:Centre Hospitalier Universitaire Rouen

Alpha-1 antitrypsin deficiency

Recruiting
Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Human Alpha1-Proteinase Inhibitor
  • Sponsor:Grifols S.A.

Pelizaeus-Merzbacher disease

Recruiting
Registered drugNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:0–17 years
  • Substances:Deferiprone
  • Sponsor:Amsterdam UMC Stichting

Severe myoclonic epilepsy of infancy

Recruiting
Investigational
Countries:HungaryHungary
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Clemizole Hydrochloride
  • Sponsor:Epygenix Therapeutics Inc.

Chronic obstructive pulmonary disease+1

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

Neonatal alloimmune thrombocytopenia

Recruiting
Registered drugNo placebo
Countries:AustriaAustria
  • Participants:18–64 years
  • Substances:Nipocalimab
  • Sponsor:Janssen Cilag International

Alpha-1 antitrypsin deficiency

Recruiting
InvestigationalNo placebo
Countries:IrelandIreland
  • Participants:18–64 years
  • Substances:Gr0015
  • Sponsor:Beam 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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