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Congenital, familial and genetic disorders – page 12

  • 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

Study on How Nusinersen is Processed in Adults and Children with Spinal Muscular Atrophy Using the ThecaFlex DRx System

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Nusinersen
  • Sponsor:Biogen Idec Research Limited

Myotonic dystrophy

Recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Mexiletine
  • Sponsor:Lupin Atlantis Holdings SA

Osteogenesis imperfecta

Recruiting
Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Hydrochlorothiazide
  • Sponsor:Istituto Ortopedico Rizzoli

Type IIa hyperlipidaemia

Recruiting
Registered drug
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Inclisiran
  • Sponsor:Novartis Pharma AG

Hypertrophic cardiomyopathy

Recruiting
Registered drug
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Sotagliflozin
  • Sponsor:Lexicon Pharmaceuticals Inc.

Study on the Effectiveness and Safety of Nizubaglustat in Patients with Niemann-Pick Type C, GM1, or GM2 Gangliosidosis

Recruiting
Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:NIZUBAGLUSTAT
  • Sponsor:Azafaros B.V.

Tuberous sclerosis complex+1

Recruiting
Registered drug
Countries:PolandPoland
  • Participants:0–17 years
  • Substances:Sirolimus
  • Sponsor:Instytut Pomnik Centrum Zdrowia Dziecka

Hereditary angioedema

Recruiting
InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Deucrictibant
  • Sponsor:Pharvaris Netherlands B.V.

Tuberous sclerosis complex+1

Recruiting
Registered drug
Countries:PolandPoland
  • Participants:0–17 years · 18–64 years
  • Substances:Sirolimus
  • Sponsor:Instytut Pomnik Centrum Zdrowia Dziecka

Congenital cystic kidney disease

Recruiting
Registered drugNo placebo
Countries:ItalyItaly
  • Participants:18–64 years
  • Substances:Metformin
  • Sponsor:University Of Bari Aldo Moro

Endometrial cancer+18

Recruiting
InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Bay 2927088
  • Sponsor:Bayer AG

Dent's disease

Recruiting
Registered drugNo placebo
Countries:ItalyItaly
  • Participants:18–64 years
  • Substances:Alpelisib
  • Sponsor:Ospedale Pediatrico Bambino Gesu

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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