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Congenital, familial and genetic disorders – page 3

  • 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

Vascular malformation

Recruiting
Registered drugNo placebo
Countries:SloveniaSlovenia
  • Participants:18–64 years · 65+ years
  • Substances:Bleomycin Sulfate Ph. Eur.
  • Sponsor:Institute Of Oncology Ljubljana

Stargardt's disease

Recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Tinlarebant
  • Sponsor:Belite Bio Inc.

Friedreich's ataxia

Recruiting
Registered drugNo placebo
Countries:CzechiaCzechia
  • Participants:18–64 years · 65+ years · Healthy volunteers
  • Substances:Omaveloxolone
  • Sponsor:Fakultni Nemocnice V Motole

Dementia+3

Recruiting
InvestigationalNo placebo
Countries:SpainSpain
  • Participants:18–64 years · 65+ years · Healthy volunteers
  • Substances:[18F]DED
  • Sponsor:Barcelonabeta Brain Research Center

Duchenne muscular dystrophy

Recruiting
InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years
  • Sponsor:Biomarin Pharmaceutical Inc.

Angelman's syndrome

Recruiting
InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years
  • Substances:2'-O-(2-Methoxyethyl) Modified Antisense Oligonucleotide Targeting Ube3A Antisense Transcript Rna
  • Sponsor:Ionis Pharmaceuticals Inc.

Hepato-lenticular degeneration

Recruiting
Registered drug
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years · Healthy volunteers
  • Substances:Copper (64Cu) Chloride
  • Sponsor:Region Midtjylland

Congenital cystic kidney disease+1

Recruiting
Registered drugNo placebo
Countries:SpainSpain
  • Participants:18–64 years · 65+ years
  • Substances:Ursodeoxycholic Acid
  • Sponsor:Asociacion Instituto Biogipuzkoa

Hereditary angioedema

Recruiting
InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Donidalorsen
  • Sponsor:Ionis Pharmaceuticals Inc.

K-ras gene mutation+1

Recruiting
Registered drugNo placebo
Countries:FinlandFinland
  • Participants:18–64 years · 65+ years
  • Substances:Carboplatin
  • Sponsor:Merck Sharp & Dohme LLC

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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