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Congenital, familial and genetic disorders – page 4

  • 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

Hepato-lenticular degeneration

Recruiting

A study comparing different zinc doses for the treatment of Wilson disease in healthy participants.

Registered drug
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years · Healthy volunteers
  • Substances:Copper (64CU) chloride
  • Sponsor:Region Midtjylland

Congenital cystic kidney disease+1

Recruiting

A study to evaluate the effectiveness and safety of ursodeoxycholic acid in patients with hepatorenal polycystic diseases.

Registered drugNo placebo
Countries:SpainSpain
  • Participants:18–64 years · 65+ years
  • Substances:Ursodeoxycholic Acid
  • Sponsor:Asociacion Instituto Biogipuzkoa

Hereditary angioedema

Recruiting

A study of donidalorsen for children with hereditary angioedema

InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Donidalorsen
  • Sponsor:Ionis Pharmaceuticals Inc.

K-ras gene mutation+1

Recruiting

A study of pembrolizumab, cetuximab, and MK-1084 for patients with advanced or metastatic non-small cell lung cancer with KRAS G12C mutations

Registered drugNo placebo
Countries:FinlandFinland
  • Participants:18–64 years · 65+ years
  • Substances:Carboplatin
  • Sponsor:Merck Sharp & Dohme LLC

Canavan disease

Recruiting

A study to evaluate the effectiveness of ciracigene golparvovec in children with Canavan disease

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Sponsor:Myrtelle Inc.

Osteogenesis imperfecta

Recruiting

A safety study of romosozumab in children and adolescents with osteogenesis imperfecta

Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Romosozumab
  • Sponsor:Amgen Inc.

Propionic acidaemia

Recruiting

A study to evaluate the long-term safety and effectiveness of mRNA-3927 in patients with Propionic Acidemia

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Modified Messenger Ribonucleic Acid Encoding Human Propionyl-Coenzyme A Carboxylase Alpha And Beta Subunits Encapsulated Into Lipid Nanoparticles
  • Sponsor:Moderna Inc.

K-ras gene mutation+1

Recruiting

Evaluation of Safety, Pharmacokinetics, and Efficacy of RO7782493 and Panitumumab in Advanced Solid Tumors with KRAS G12D Mutation

Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Panitumumab
  • Sponsor:Genentech Inc.

Thalassaemia beta+1

Recruiting

A Study of Gene Therapy Using Modified Stem Cells for Patients with Transfusion-Dependent Beta-Thalassemia Who Need Regular Blood Transfusions

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years
  • Substances:Busulfan
  • Sponsor:Fondazione Telethon Ets

Glycogen storage disease type II

Recruiting

A Study of S-606001 Added to Enzyme Replacement Therapy for Patients with Late-Onset Pompe Disease

Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Sponsor:Shionogi B.V.

Von Willebrand's disease

Recruiting

A Study of VGA039 to Prevent Bleeding in Teenagers and Adults with von Willebrand Disease Who Have Not Used Regular Preventive Treatment Before

InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:VGA039
  • Sponsor:Vega Therapeutics Inc.

Hypertrophic cardiomyopathy

Recruiting

Comparing hydrochlorothiazide and valsartan for treating high blood pressure in patients with hypertrophic obstructive cardiomyopathy

Registered drugNo placebo
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:Hydrochlorothiazide
  • Sponsor:Medical University Of Graz

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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