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Congenital, familial and genetic disorders – page 2

  • 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
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796 clinical trials

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

Recruiting
InvestigationalNo placebo
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.

Duchenne muscular dystrophy

Recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Sponsor:Avidity Biosciences Inc.

Type IIa hyperlipidaemia

Recruiting
Investigational
Countries:NorwayNorway
  • Participants:0–17 years
  • Substances:Lerodalcibep
  • Sponsor:Lib Therapeutics LLC

Osteochondrodysplasia

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

Myotonic dystrophy

Recruiting
Registered drug
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Humanised Igg1 Kappa Fragment Antibody Targeting Tfr1 Conjugated To P125 Oligonucleotide
  • Sponsor:Dyne Therapeutics Inc.

A study to assess the safety of Anumigilimab in adults with sickle cell disease

Recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:ANUMIGILIMAB
  • Sponsor:CSL Behring LLC

Homocystinuria

Recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Sponsor:Travere Therapeutics Inc.

Hereditary haemochromatosis

Recruiting
Investigational
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:VAMIFEPORT TRIHYDROCHLORIDE
  • Sponsor:CSL Behring LLC

Factor VIII deficiency+1

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years
  • Substances:Fidanacogene Elaparvovec
  • Sponsor:Pfizer Inc.

Hypochondroplasia

Recruiting
InvestigationalNo placebo
Countries:NorwayNorway
  • Participants:0–17 years
  • Substances:Infigratinib
  • Sponsor:Qed Therapeutics Inc.

Rett syndrome

Recruiting
Registered drug
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Fenfluramine Hydrochloride
  • Sponsor:UCB Biosciences Inc.

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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