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Congenital, familial and genetic disorders – page 16

  • 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

Patent ductus arteriosus

Recruiting
Registered drug
Countries:CzechiaCzechia
  • Participants:0–17 years
  • Substances:Paracetamol
  • Sponsor:University College Dublin

Craniosynostosis

Recruiting
Registered drugNo placebo
Countries:HungaryHungary
  • Participants:0–17 years
  • Substances:Rocuronium Bromide
  • Sponsor:University Of Debrecen

Myotonic dystrophy

Recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Mexiletine Hydrochloride
  • Sponsor:Lupin Atlantis Holdings SA

Duchenne muscular dystrophy

Recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Givinostat
  • Sponsor:Italfarmaco S.p.A.

HER2 gene amplification

Recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Zongertinib
  • Sponsor:Boehringer Ingelheim Espana S.A.

Osteochondrodysplasia

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Infigratinib
  • Sponsor:Qed Therapeutics Inc.

Glycogen storage disease type I

Recruiting
InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Pariglasgene Brecaparvovec
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Fabry's disease

Recruiting
InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Pegunigalsidase Alfa
  • Sponsor:Chiesi Farmaceutici S.p.A.

Cystic fibrosis

Recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Deutivacaftor
  • Sponsor:Vertex Pharmaceuticals Inc.

Glycogen storage disease type II

Recruiting
InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:18–64 years
  • Substances:Vanglusagene Ensiparvovec
  • Sponsor:Genentech Inc.

Univentricular heart+1

Recruiting
Registered drug
Countries:PolandPoland
  • Participants:18–64 years · 65+ years
  • Substances:Sildenafil
  • Sponsor:Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego Panstwowy Instytut Badawczy

Fibrodysplasia ossificans progressiva

Recruiting
Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Incb000928
  • Sponsor:Incyte Corp.

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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