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Congenital, familial and genetic disorders – page 51

  • 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
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796 clinical trials

Porphyria non-acute

Not recruiting

Study on the Effects of Dersimelagon Phosphate for Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria

Investigational
Countries:BulgariaBulgaria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Dersimelagon Phosphate
  • Sponsor:Tanabe Pharma America Inc.

Factor VIII deficiency

Not recruiting

Safety Study of Damoctocog Alfa Pegol for Children Aged 7 to 11 with Severe Hemophilia A

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Damoctocog Alfa Pegol
  • Sponsor:Bayer AG

K-ras gene mutation+1

Not recruiting

Study of adagrasib in elderly patients (70 years or older) or patients with poor performance status who have KRAS G12C mutation-positive non-small cell lung cancer

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Adagrasib
  • Sponsor:ETOP IBCSG Partners Foundation

Osteochondrodysplasia

Not recruiting

Study on the Effectiveness and Safety of Infigratinib for Children Aged 3 to 17 with Achondroplasia

Investigational
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Infigratinib
  • Sponsor:Qed Therapeutics Inc.

Hereditary angioedema with C1 esterase inhibitor deficiency+1

Not recruiting

Study on Oral PHA-022121 for Preventing Angioedema Attacks in Patients with Hereditary Angioedema

Investigational
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:(S)-N-(1-Deutero-1-(3-Chloro-5-Fluoro-2-((2-Methyl-4-(1-Methyl-1H-1,2,4-Triazol-5-Yl)Quinolin-8-Yloxy)Methyl)Phenyl)Ethyl)-2-(Difluoromethoxy)Acetamide
  • Sponsor:Pharvaris Netherlands B.V.

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

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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