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Congenital, familial and genetic disorders – page 66

  • 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

Polycystic liver disease

Not recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Octreotide Hydrochloride
  • Sponsor:Camurus AB

Haemophilia B with anti factor IX+1

Not recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Sponsor:Apcintex Limited

Osteogenesis imperfecta

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Neridronate Sodium
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Isodicentric chromosome 15 syndrome+3

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

Angelman's syndrome

Not recruiting
InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Rugonersen
  • Sponsor:F. Hoffmann-La Roche AG

Cystic fibrosis

Not recruiting
Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years
  • Substances:Elexacaftor
  • Sponsor:Vertex Pharmaceuticals Inc.

Cystic fibrosis

Not recruiting
Registered drugNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years
  • Substances:Elexacaftor
  • Sponsor:Vertex Pharmaceuticals Inc.

Primary hypercholesterolaemia

Not recruiting
Registered drugNo placebo
Countries:HungaryHungary
  • Participants:18–64 years · 65+ years
  • Substances:Inclisiran
  • Sponsor:Semmelweis University

Heritable pulmonary arterial hypertension+2

Not recruiting
Registered drug
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Dapagliflozin
  • Sponsor:Region Hovedstaden

Ataxia telangiectasia

Not recruiting
InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years · 18–64 years
  • Substances:Dexamethasone Sodium Phosphate
  • Sponsor:Quince Therapeutics S.p.A.

Breast cancer+5

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Fluoroestradiol F-18
  • Sponsor:GE Healthcare Limited

Adrenoleukodystrophy

Not recruiting
Investigational
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Leriglitazone
  • Sponsor:Minoryx Therapeutics S.L.

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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