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Congenital, familial and genetic disorders – page 5

  • 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

Myotonic dystrophy

Recruiting
Registered drug
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Substances:ADS-019
  • Sponsor:Sarepta Therapeutics Inc.

Alpha-1 antitrypsin deficiency

Recruiting
InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Human Alpha1-Proteinase Inhibitor
  • Sponsor:Grifols Therapeutics LLC

Severe myoclonic epilepsy of infancy

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · Healthy volunteers
  • Substances:4-Fluoro-N-[2-[4-(2-Methoxyphenyl)Piperazin-1-Yl]Ethyl]-N-Pyridin-2-Ylbenzamide
  • Sponsor:Hospices Civils De Lyon

Sickle cell anaemia with crisis+2

Recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Lidocaine Hydrochloride
  • Sponsor:CHU Saint Pierre

Hereditary optic atrophy

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Sponsor:Gensight Biologics

Porphyria non-acute

Recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Sponsor:Disc Medicine Inc.

Hereditary spastic paraplegia

Recruiting
InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years
  • Sponsor:Azienda Socio Sanitaria Territoriale Papa Giovanni Xxiii

MELAS syndrome+2

Recruiting
Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years
  • Sponsor:Khondrion B.V.

Fragile X syndrome

Recruiting
Investigational
Countries:SpainSpain
  • Participants:18–64 years
  • Substances:2-[4-[3-(METHYLAMINO)-1-PHENYLPROPOXY]PHENYL]ETHANOL HYDROCHLORIDE
  • Sponsor:Connecta Therapeutics S.L.

Sickle cell disease

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Imatinib
  • Sponsor:Assistance Publique Hopitaux De Paris

Netherton's syndrome

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Sponsor:Biocryst 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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