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Congenital, familial and genetic disorders – page 62

  • 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

Gaucher's disease type III+1

Not recruiting
Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years
  • Substances:Imiglucerase
  • Sponsor:Genzyme Corp.

Becker's muscular dystrophy

Not recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Edg-5506
  • Sponsor:Edgewise Therapeutics Inc.

Hypertrophic cardiomyopathy

Not recruiting
Registered drug
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Aficamten
  • Sponsor:Cytokinetics Inc.

Factor VIII deficiency

Not recruiting
InvestigationalNo placebo
Countries:BulgariaBulgaria
  • Participants:18–64 years · 65+ years
  • Substances:Peboctocogene Camaparvovec
  • Sponsor:Bayer AG

K-ras gene mutation+3

Not recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Cetuximab
  • Sponsor:Novartis Pharma AG

Cystic fibrosis

Not recruiting
Investigational
Countries:FranceFrance
  • Participants:18–64 years
  • Substances:3-Amino-N-{[5-[4-[Bis[(2S,3R,4R,5R)-2,3,4,5,6-Pentahydroxyhexyl]Amino]Piperidine-1-Carbonyl]-1,3-Diethyl-Benzimidazol-1-Ium-2-Yl]Methyl}-5H-Pyrrolo[2,3-B]Pyrazine-2-Carboxamide Dihydrochloride Chloride
  • Sponsor:Enterprise Therapeutics Limited

Activated PI3 kinase delta syndrome

Not recruiting
InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Sponsor:Pharming Technologies B.V.

Tuberous sclerosis complex+1

Not recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Radiprodil
  • Sponsor:Grin Therapeutics Inc.

Phenylketonuria

Not recruiting
Registered drug
Countries:DenmarkDenmark
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Esomeprazole
  • Sponsor:Synlogic Inc.

Hypophosphatasia

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Asfotase Alfa
  • Sponsor:Alexion Pharmaceuticals Inc.

Retinitis pigmentosa

Not recruiting
InvestigationalNo placebo
Countries:SwedenSweden
  • Participants:18–64 years · 65+ years
  • Substances:Cpk850
  • Sponsor:Novartis Pharma AG

Neoplasm malignant+1

Not recruiting
InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Larotrectinib Sulfate
  • Sponsor:Bayer AG

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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