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Congenital, familial and genetic disorders – page 61

  • 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

Hereditary angioedema

Not recruiting
InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Substances:Garadacimab
  • Sponsor:CSL Behring LLC

Netherton's syndrome

Not recruiting
InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:18–64 years
  • Substances:Sxr1096
  • Sponsor:Sixera Pharma AB

Cerebral palsy+5

Not recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Valbenazine Ditosylate
  • Sponsor:Neurocrine Biosciences Inc.

Glycogen storage disease type II

Not recruiting
InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Substances:Cipaglucosidase Alfa
  • Sponsor:Amicus Therapeutics Inc.

Muscular dystrophy

Not recruiting
Registered drugNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Abiraterone Acetate
  • Sponsor:Atamyo Therapeutics

Spinal muscular atrophy

Not recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Risdiplam
  • Sponsor:F. Hoffmann-La Roche AG

Alport's syndrome

Not recruiting
Investigational
Countries:AustriaAustria
  • Participants:18–64 years
  • Substances:Setanaxib
  • Sponsor:Calliditas Therapeutics Suisse S.A.

Bronchiectasis+1

Not recruiting
Registered drug
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Bi 1291583
  • Sponsor:Boehringer Ingelheim Espana S.A.

Severe myoclonic epilepsy of infancy+1

Not recruiting
InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Soticlestat
  • Sponsor:Takeda Development Center Americas Inc.

Tuberous sclerosis complex+2

Not recruiting
Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Cannabidiol
  • Sponsor:Jazz Pharmaceuticals Research UK Limited

Neoplasm malignant+2

Not recruiting
InvestigationalNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years · 18–64 years
  • Substances:Larotrectinib Sulfate
  • Sponsor:Bayer AG

Spinal muscular atrophy

Not recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Risdiplam
  • Sponsor:F. Hoffmann-La Roche 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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