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Congenital, familial and genetic disorders – page 64

  • 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

Urea cycle disorder+3

Not recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Hhalpc
  • Sponsor:Cellaïon SA

Factor VIII deficiency

Not recruiting
InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Efanesoctocog Alfa
  • Sponsor:Swedish Orphan Biovitrum AB (publ)

Short stature+3

Not recruiting
InvestigationalNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:0–17 years
  • Substances:Somapacitan
  • Sponsor:Novo Nordisk A/S

Retinitis pigmentosa

Not recruiting
Registered drug
Countries:AustriaAustria
  • Participants:18–64 years
  • Substances:Acetylcysteine
  • Sponsor:Johns Hopkins University

Adrenogenital syndrome

Not recruiting
Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Hydrocortisone
  • Sponsor:Spruce Biosciences Inc.

Adrenogenital syndrome

Not recruiting
Registered drug
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Hydrocortisone
  • Sponsor:Spruce Biosciences Inc.

Netherton's syndrome

Not recruiting
Investigational
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Spesolimab
  • Sponsor:Boehringer Ingelheim Espana S.A.

Friedreich's ataxia

Not recruiting
Registered drug
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Dimethyl Fumarate
  • Sponsor:Universita' Degli Studi Di Napoli Federico II

Congenital generalised lipodystrophy

Not recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Metreleptin
  • Sponsor:Amryt Pharmaceuticals Designated Activity Company

Tourette's disorder

Not recruiting
Investigational
Countries:BulgariaBulgaria
  • Participants:0–17 years · 18–64 years
  • Substances:Ecopipam Hydrochloride
  • Sponsor:Emalex Biosciences Inc.

Factor IX deficiency

Not recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Substances:Fidanacogene Elaparvovec
  • Sponsor:Pfizer Inc.

Haemophilia

Not recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Fitusiran
  • Sponsor:Sanofi-Aventis Recherche & Developpement

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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