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Congenital, familial and genetic disorders – page 14

  • 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

Alpha-1 antitrypsin deficiency

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

Sickle cell anaemia

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

Thalassaemia

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

Study on the Effectiveness of Ambroxol and Clenbuterol in Children and Adults with Gaucher Disease Type 3

Recruiting
Registered drug
Countries:The NetherlandsThe Netherlands
  • Participants:0–17 years · 18–64 years
  • Substances:Ambroxol Hydrochloride
  • Sponsor:Amsterdam UMC Stichting

Hereditary angioedema

Recruiting
Registered drugNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Berotralstat
  • Sponsor:Biocryst Pharmaceuticals Inc.

Crigler-Najjar syndrome

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Amoxicillin Sodium
  • Sponsor:Genethon

Arteriovenous malformation

Recruiting
Registered drugNo placebo
Countries:NorwayNorway
  • Participants:18–64 years · 65+ years
  • Substances:Apixaban
  • Sponsor:Oslo University Hospital HF

Wiskott-Aldrich syndrome

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Etuvetidigene Autotemcel
  • Sponsor:Genethon

Rectal cancer+2

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Dostarlimab
  • Sponsor:Centre Hospitalier Universitaire De Dijon

Heart disease congenital+1

Recruiting
Registered drug
Countries:The NetherlandsThe Netherlands
  • Participants:0–17 years
  • Substances:Allopurinol Sodium
  • Sponsor:Universitair Medisch Centrum Utrecht

Squamous cell carcinoma of the oral cavity+4

Recruiting
Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Afatinib
  • Sponsor:Fundacio Institut De Recerca De L'Hospital De La Santa Creu I Sant Pau

Congenital hypotransferrinaemia+5

Recruiting
InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years
  • Substances:Human Apotransferrin
  • Sponsor:Prothya Biosolutions Netherlands B.V.

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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