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Congenital, familial and genetic disorders – page 52

  • 803 clinical trials
  • 311 recruiting
  • 375 conditions
  • 27 countries

Conditions in this area

375 conditions with trials, the busiest first

Duchenne muscular dystrophy28Factor VIII deficiency25Sickle cell disease25Cystic fibrosis24Hereditary angioedema22Hypertrophic cardiomyopathy17Spinal muscular atrophy17Type IIa hyperlipidaemia15Glycogen storage disease type II14Factor IX deficiency13Huntington's disease13Sickle cell anaemia13Congenital cystic kidney disease12Myotonic dystrophy12Non-small cell lung cancer12Osteochondrodysplasia12Adrenogenital syndrome11Alpha-1 antitrypsin deficiency11K-ras gene mutation11Retinitis pigmentosa11Severe myoclonic epilepsy of infancy11Thalassaemia11Epidermolysis bullosa10Fabry's disease10+351

Clinical trials in Congenital, familial and genetic disorders

803 trials across Europe, recruiting first

Type of trial
Recruitment status
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803 clinical trials

Hypertrophic cardiomyopathy

Not recruiting

Study on the Effects of MYK-224 for Patients with Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction

InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:18–64 years · 65+ years
  • Substances:BMS-986435
  • Sponsor:Myokardia Inc.

PIK3CA related overgrowth spectrum

Not recruiting

Study on the Long-Term Safety of Alpelisib for Patients with PIK3CA-Related Overgrowth Spectrum (PROS)

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Alpelisib
  • Sponsor:Novartis Pharma AG

Huntington's disease

Not recruiting

Safety and Tolerability Study of AB-1001 Brain Injection in Adults with Early Huntington's Disease

Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Gadoteric Acid
  • Sponsor:AskBio France

Spinal muscular atrophy

Not recruiting

Study on Long-Term Safety and Effects of Apitegromab in Patients with Type 2 and TYPE 3 Spinal Muscular Atrophy Who Completed Previous Trials

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Apitegromab
  • Sponsor:Scholar Rock Inc.

Alternating hemiplegia of childhood

Not recruiting

Study on Oxygen and Nitrous Oxide for Treating Dystonic and Plegic Attacks in Children with Alternating Hemiplegia

Registered drug
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Nitrous Oxide
  • Sponsor:Assistance Publique Hopitaux De Paris

Hypophosphatasia

Not recruiting

Study on ALXN1850 for Adolescents and Adults with Hypophosphatasia Not Previously Treated with Asfotase Alfa

Investigational
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:ALXN1850
  • Sponsor:Alexion Pharmaceuticals Inc.

Congenital myopathy

Not recruiting

Study on Salbutamol for Improving Muscle Strength in Patients with Congenital Myopathy

Registered drugNo placebo
Countries:SwedenSweden
  • Participants:0–17 years · 18–64 years
  • Substances:Salbutamol
  • Sponsor:Vaestra Goetalandsregionen

Sickle cell anaemia+1

Not recruiting

Study on Crizanlizumab for Adolescents and Adults with Sickle Cell Disease Experiencing Vaso-Occlusive Crises

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Crizanlizumab
  • Sponsor:Novartis Pharma AG

Sickle cell anaemia+1

Not recruiting

A Phase 4 Study of Crizanlizumab Treatment for Patients with Sickle Cell Disease Who Previously Participated in Novartis Clinical Trials

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Crizanlizumab
  • Sponsor:Novartis Pharma AG

Cardiovascular disorder+1

Not recruiting

Study on Long-Term Effects of Lerodalcibep for Lowering Cholesterol in Patients with Familial Hypercholesterolemia or Cardiovascular Disease Risks

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Lerodalcibep
  • Sponsor:Lib Therapeutics LLC

Basal cell naevus syndrome

Not recruiting

Study on Patidegib Gel for Reducing Basal Cell Carcinomas in Adults with Gorlin Syndrome

Investigational
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Patidegib Hydrochloride Isopropanolate
  • Sponsor:Sol-Gel Technologies Ltd.

Phenylketonuria

Not recruiting

Study Comparing Sepiapterin and Sapropterin for Treating Phenylketonuria in Patients Aged 2 Years and Older

Registered drugNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:(S)-2-Amino-6-(2-Hydroxypropanoyl)-7,8-Dihydropteridin-4(3H)-One
  • Sponsor:PTC Therapeutics 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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