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Congenital, familial and genetic disorders

  • 795 clinical trials
  • 307 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 hyperlipidaemia15Factor IX deficiency13Glycogen storage disease type II13Sickle 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

795 trials across Europe, recruiting first

Type of trial
Recruitment status
Quick filters

795 clinical trials

Hereditary angioedema with C1 esterase inhibitor deficiency+1

Recruiting
InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:NAVENIBART
  • Sponsor:Astria Therapeutics Inc.

Huntington's disease

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

Von Willebrand's disease+1

Recruiting
Investigational
Countries:GermanyGermany
  • Participants:18–64 years
  • Substances:RNA, (UM-SP-(2′-DEOXY-2′-FLUORO)A-SP-GM-AM-AM-(2′-DEOXY-2′-FLUORO)A-CM-UM-CM-AM-UM-AM-GM-(2′-DEOXY-2′-FLUORO)C-GM-(2′-DEOXY-2′-FLUORO)A-UM-UM-GM-CM-AM-SP-CM-SP-AM), COMPLEX WITH RNA (UM-SP-GM-SP-CM-AM-AM-UM-(2′-DEOXY-2′-FLUORO)C-GM-(2′-DEOXY-2′-FLUORO)C-(2′-DEOXY-2′-FLUORO)U-(2′-DEOXY-2′-FLUORO)A-UM-GM-AM-GM-UM-UM-UM-CM-UM-AM) 3′-[[(2S,4R)-1-[29-[[2-(ACETYLAMINO)-2-DEOXY-Β-D-GALACTOPYRANOSYL]OXY]-14,14-BIS[[3-[[3-[[5-[[2-(ACETYLAMINO)-2-DEOXY-Β-D-GALACTOPYRANOSYL]OXY]-1-OXOPENTYL]AMINO]PROPYL]AMINO]-3-OXOPROPOXY]METHYL]-1,12,19,25-TETRAOXO-16-OXA-13,20,24-TRIAZANONACOS-1-YL]-4-HYDROXY-2-PYRROLIDINYL]METHYL HYDROGEN PHOSPHATE] (1:1)
  • Sponsor:Alnylam Pharmaceuticals Inc.

Hereditary angioedema

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

Ornithine transcarbamoylase deficiency

Recruiting
Investigational
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Adeno-Associated Virus Serotype Rh79 Containing The Human Otc Gene
  • Sponsor:Iecure Inc.

Duchenne muscular dystrophy+1

Recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Sponsor:Entrada Therapeutics Inc.

Factor VIII deficiency+1

Recruiting
Registered drug
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:Efanesoctocog Alfa
  • Sponsor:GWT-Tud GmbH

Glycogen storage disease type II

Recruiting
Investigational
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Sponsor:Denali Therapeutics Inc.

Factor VIII deficiency

Recruiting
Registered drug
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Emicizumab
  • Sponsor:F. Hoffmann-La Roche AG

Factor VIII deficiency

Recruiting
Registered drug
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Ro7589655
  • Sponsor:F. Hoffmann-La Roche AG

Alpha-1 antitrypsin deficiency

Recruiting
Registered drug
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Human Alpha1-Proteinase Inhibitor
  • Sponsor:CSL Behring LLC

Long-Term Safety and Efficacy of KL1333 in Adults with Primary Mitochondrial Disease

Recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:2-Isopropyl-3H-Naphtho(1,2-D)Imidazole-4,5-Dione
  • Sponsor:Pharming Technologies B.V.

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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