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Congenital, familial and genetic disorders – page 15

  • 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

Hereditary neuropathic amyloidosis

Recruiting
InvestigationalNo placebo
Countries:CyprusCyprus
  • Participants:18–64 years · 65+ years
  • Substances:Eplontersen
  • Sponsor:Ionis Pharmaceuticals Inc.

Facioscapulohumeral muscular dystrophy

Recruiting
Registered drug
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Ads-010
  • Sponsor:Sarepta Therapeutics Inc.

Retinitis pigmentosa

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Adeno-Associated Viral Vector Serotype 2.7M8 Containing The Chrimsonr-Tdtomato Gene
  • Sponsor:Gensight Biologics

Atrial septal defect

Recruiting
Registered drugNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years
  • Substances:Acetylsalicylic Acid
  • Sponsor:Sint Antonius Ziekenhuis Stichting

Sickle cell disease

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Hydroxycarbamide
  • Sponsor:Theravia

BRAF V600E mutation positive+1

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Cobicistat
  • Sponsor:Fore Biotherapeutics Inc.

Von Willebrand's disease

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Octocog Alfa
  • Sponsor:Takeda Development Center Americas Inc.

Neurofibromatosis

Recruiting
Registered drugNo placebo
Countries:PolandPoland
  • Participants:0–17 years
  • Substances:Crizotinib
  • Sponsor:Medical University Of Warsaw

Factor VIII deficiency

Recruiting
Registered drugNo placebo
Countries:BulgariaBulgaria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Emicizumab
  • Sponsor:Emory University

Angelman's syndrome

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

Congenital aplastic anaemia

Recruiting
InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years
  • Substances:Autologous Cd34+Enriched Cells From Patients With Fanconi Anemia Subtype A Transduced Ex Vivo With Lentiviral Vector Carrying The Fanconi Anemia Complementation Group A 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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