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Congenital, familial and genetic disorders – page 7

  • 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

Hypochondroplasia

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Vosoritide
  • Sponsor:Biomarin Pharmaceutical Inc.

Adenomatous polyposis coli

Recruiting
Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Sirolimus
  • Sponsor:Biodexa Limited

Hereditary angioedema

Recruiting
Investigational
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:ADX-324
  • Sponsor:Adarx Pharmaceuticals Inc.

K-ras gene mutation+2

Recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Budesonide
  • Sponsor:F. Hoffmann-La Roche AG

Fabry's disease

Recruiting
Registered drugNo placebo
Countries:PolandPoland
  • Participants:18–64 years · 65+ years
  • Substances:Prednisone
  • Sponsor:Medical University Of Lodz

Spinal muscular atrophy

Recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Apitegromab
  • Sponsor:Scholar Rock Inc.

PIK3CA related overgrowth spectrum

Recruiting
InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Alpelisib
  • Sponsor:Novartis Pharma AG

Familial dilated cardiomyopathy

Recruiting
Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:AAV9-CTNT-COBAG3
  • Sponsor:Alexion Pharmaceuticals Inc.

Hypochondroplasia

Recruiting
Registered drug
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Vosoritide
  • Sponsor:Biomarin Pharmaceutical Inc.

Asplenia

Recruiting
Registered drugNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years · Healthy volunteers
  • Substances:Pneumococcal Polysaccharide Serotype 1 Conjugated To Crm197 Adsorbed On Aluminium Phosphate
  • Sponsor:Leids Universitair Medisch Centrum (LUMC)

Adrenogenital syndrome

Recruiting
InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Substances:Crinecerfont

Myelodysplastic syndrome+1

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Momelotinib Dihydrochloride Monohydrate
  • Sponsor:Groupe Francophone Des Myelodysplasies

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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