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Congenital, familial and genetic disorders – page 59

  • 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

Chronic granulomatous disease

Not recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Autologous Cd34+ Cells Transduced With The G1Xcgd Lentiviral Vector Containing The Human Cybb Gene
  • Sponsor:Genethon

Hereditary motor and sensory neuropathy

Not recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Nmd670
  • Sponsor:NMD Pharma A/S

Adrenogenital syndrome

Not recruiting
Investigational
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:Crinecerfont
  • Sponsor:Neurocrine Biosciences Inc.

Primary immunodeficiency syndrome+2

Not recruiting
Registered drugNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:0–17 years · 18–64 years
  • Substances:Human Normal Immunoglobulin
  • Sponsor:Universitair Medisch Centrum Utrecht

Glycogen storage disease type II

Not recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Cipaglucosidase Alfa
  • Sponsor:Amicus Therapeutics Inc.

Primary immunodeficiency syndrome

Not recruiting
InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Human Normal Immunoglobulin
  • Sponsor:Octapharma Pharmazeutika Produktionsgesellschaft mbH

Fabry's disease

Not recruiting
InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:18–64 years
  • Substances:Isaralgagene Civaparvovec
  • Sponsor:Sangamo Therapeutics Inc.

Short stature+3

Not recruiting
Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Somapacitan
  • Sponsor:Novo Nordisk A/S

Phenylketonuria

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Codeine Phosphate
  • Sponsor:Moderna Therapeutics Inc.

Hereditary angioedema with C1 esterase inhibitor deficiency

Not recruiting
Registered drug
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Deucrictibant
  • Sponsor:Pharvaris Netherlands B.V.

Sickle cell disease

Not recruiting
Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Etavopivat
  • Sponsor:Novo Nordisk A/S

Colour blindness

Not recruiting
InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Adeno-Associated Viral Vector Serotype 8 Containing The Human Cnga3 Gene Under The Control Of A Cone Arrestin Promoter
  • Sponsor:Universitaetsklinikum Tuebingen AöR

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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