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Congenital, familial and genetic disorders – page 55

  • 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

Retinitis pigmentosa

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Atropine Sulfate
  • Sponsor:eyeDNA Therapeutics

Huntington's disease

Not recruiting
Investigational
Countries:AustriaAustria
  • Participants:18–64 years
  • Substances:Tominersen
  • Sponsor:F. Hoffmann-La Roche AG

Neurofibromatosis+1

Not recruiting
Registered drug
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Selumetinib
  • Sponsor:AstraZeneca AB

Non-small cell lung cancer+1

Not recruiting
Registered drugNo placebo
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:Alectinib
  • Sponsor:F. Hoffmann-La Roche AG

Non-Hodgkin's lymphoma+13

Not recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Tazemetostat
  • Sponsor:Epizyme Inc.

Congenital cystic kidney disease

Not recruiting
Registered drug
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years
  • Substances:Amiloride
  • Sponsor:Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

Adrenogenital syndrome

Not recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Crinecerfont
  • Sponsor:Neurocrine Biosciences Inc.

Glycogen storage disease type I

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Codeine Phosphate
  • Sponsor:Moderna Therapeutics Inc.

Fatty acid oxidation disorder

Not recruiting
Registered drugNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years
  • Substances:Doconexent
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Corneal dystrophy

Not recruiting
Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Ripasudil
  • Sponsor:Kowa Research Institute Inc.

Alexander disease

Not recruiting
Investigational
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years
  • Substances:2'-O-(2-Methoxyethyl)-D-Ribose Antisense Oligonucleotide Targeting Glial Fibrillary Acidic Protein Messenger Ribonucleic Acid
  • Sponsor:Ionis Pharmaceuticals Inc.

Methylmalonic acidaemia

Not recruiting
Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Modified Mrna Encoding Human Methylmalonyl-Coenzyme A Mutase Containing A Polymorphism At Position 671
  • Sponsor:Moderna Therapeutics Inc.

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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