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Congenital, familial and genetic disorders – page 6

  • 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

Haemophilia

Recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Fitusiran
  • Sponsor:Sanofi-Aventis Recherche & Developpement

Hypophosphatasia

Recruiting
Investigational
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years · Healthy volunteers
  • Substances:ALE1
  • Sponsor:Alesta Therapeutics B.V.

Neurofibromatosis+1

Recruiting
InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Sponsor:Merck Healthcare KGaA

Acute myeloid leukaemia+1

Recruiting
Registered drugNo placebo
Countries:GreeceGreece
  • Participants:18–64 years · 65+ years
  • Substances:Gilteritinib
  • Sponsor:Hellenic Society Of Hematology

Trisomy 21

Recruiting
Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:1-((3S,8S,9S,10R,13S,14S,17S)3-BENZYLOXY-10,13,17-TRIMETHYL-2,3,4,7,8,9,10,11,12,13,14,15,16,17-TETRADECAHYDRO-1HCYCLOPENTA[A]PHENANTHRENE-17-YL)ETHAN-1-ONE
  • Sponsor:Aelis Farma

Leukodystrophy

Recruiting
InvestigationalNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:0–17 years · 18–64 years
  • Substances:Guanabenz Acetate
  • Sponsor:Amsterdam UMC Stichting

Hypertrophic cardiomyopathy+1

Recruiting
Investigational
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Sponsor:Imbria Pharmaceuticals Inc.

Endometrial cancer+12

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Zanidatamab
  • Sponsor:Jazz Pharmaceuticals Ireland Limited

Congenital cystic kidney disease

Recruiting
Registered drug
Countries:AustriaAustria
  • Participants:18–64 years
  • Substances:Dapagliflozin
  • Sponsor:University Of Cologne

Facioscapulohumeral muscular dystrophy

Recruiting
Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Humanised Igg1 Monoclonal Antibody Against Tfr1 Conjugated To Double Stranded Sirna Oligonucleotide Against Dux4 Mrna Via A Non-Cleavable Linker
  • Sponsor:Avidity Biosciences Inc.

Phenylketonuria

Recruiting
Investigational
Countries:CzechiaCzechia
  • Participants:18–64 years · 65+ years
  • Sponsor:Otsuka Pharmaceutical Development & Commercialization Inc.

Sickle cell disease

Recruiting
Registered drug
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Sodium Chloride
  • Sponsor:Centre Hospitalier Universitaire De Nantes

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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