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Congenital, familial and genetic disorders – page 63

  • 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

K-ras gene mutation

Not recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Adagrasib
  • Sponsor:Bristol-Myers Squibb Services Unlimited Company

PIK3CA related overgrowth spectrum+1

Not recruiting
InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Miransertib
  • Sponsor:Merck Sharp & Dohme LLC

Heart disease congenital

Not recruiting
Registered drugNo placebo
Countries:NorwayNorway
  • Participants:0–17 years · 18–64 years
  • Substances:Theophylline
  • Sponsor:Oslo University Hospital HF

Glycogen storage disease type V+2

Not recruiting
Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Edg-5506
  • Sponsor:Edgewise Therapeutics Inc.

Study on the Safety of Migalastat for Patients with Fabry Disease and Severe Kidney Problems on Dialysis

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Migalastat
  • Sponsor:Amicus Therapeutics Inc.

Becker's muscular dystrophy

Not recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Substances:Edg-5506
  • Sponsor:Edgewise Therapeutics Inc.

Ornithine transcarbamoylase deficiency

Not recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Avalotcagene Ontaparvovec
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Spinal muscular atrophy

Not recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Risdiplam
  • Sponsor:UZ Leuven

Prader-Willi syndrome

Not recruiting
Investigational
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Pbf-999
  • Sponsor:Palo Biofarma S.L.

Hereditary ataxia

Not recruiting
Investigational
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Trehalose Dihydrate (Ph.eur.)
  • Sponsor:Seelos Therapeutics Inc.

Sickle cell disease

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Famotidine
  • Sponsor:Assistance Publique Hopitaux De Paris

Pyruvate kinase deficiency anaemia

Not recruiting
InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Autologous Cd34+ Hematopoietic Stem Cells Transduced Ex Vivo With A Lentiviral Vector Encoding The Codon-Optimized Version Of Pklr Gene
  • Sponsor:Rocket Pharmaceuticals 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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