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Congenital, familial and genetic disorders – page 11

  • 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

Adrenogenital syndrome

Recruiting
InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:ATUMELNANT
  • Sponsor:Crinetics Pharmaceuticals Inc.

Ataxia telangiectasia

Recruiting
Investigational
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:L-Acetylleucine
  • Sponsor:Intrabio Limited

Epidermolysis bullosa

Recruiting
Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:H-GLY-LYS-TYR-GLY-PHE-TYR-THR-HIS-VAL-PHE-ARG-LEU-LYS-LYS-TRP-ILE-GLN-LYS-VAL-ILE-ASP-GLN-PHE-GLY-GLU-OH
  • Sponsor:Xinnate AB

Epidermolysis bullosa

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:AUTOLOGOUS KERATINOCYTES AND FIBROBLASTS TRANSDUCED WITH A RETROVIRAL VECTOR ENCODING COL7A1 CDNA
  • Sponsor:Institut National De La Sante Et De La Recherche Medicale

Duchenne muscular dystrophy

Recruiting
InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:SGT-003
  • Sponsor:Solid Biosciences Inc.

Prader-Willi syndrome

Recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Sponsor:Harmony Biosciences LLC

Factor IX deficiency

Recruiting
Registered drugNo placebo
Countries:BulgariaBulgaria
  • Participants:18–64 years · 65+ years
  • Substances:Etranacogene Dezaparvovec
  • Sponsor:CSL Behring LLC

Sickle cell disease

Recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Etavopivat
  • Sponsor:Novo Nordisk A/S

Fibrodysplasia ossificans progressiva

Recruiting
Registered drugNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years
  • Sponsor:Amsterdam UMC Stichting

Spina bifida

Recruiting
Registered drug
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:OXYBUTYNIN HYDROCHLORIDE
  • Sponsor:CHRU De Nancy

Tuberous sclerosis complex

Recruiting
Investigational
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Cannabidiol
  • Sponsor:Oils4cure S.L.

Naevus flammeus

Recruiting
Registered drug
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Sirolimus
  • Sponsor:Aft Pharmaceuticals Limited

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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