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Congenital, familial and genetic disorders – page 24

  • 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
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796 clinical trials

Trisomy 21+1

Recruiting

Study on Sleep and Temperature Changes in Alzheimer’s Disease for Down Syndrome Patients Using Izaflortaucipir (18F)

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:18–64 years
  • Substances:Izaflortaucipir (18F)
  • Sponsor:Fundacio Institut De Recerca De L'Hospital De La Santa Creu I Sant Pau

Glycogen storage disease type II

Recruiting

Study on the Effects of Avalglucosidase Alfa Enzyme Replacement Therapy in Patients with Pompe Disease

Registered drugNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Avalglucosidase Alfa
  • Sponsor:Region Hovedstaden

ABO haemolytic disease of newborn

Recruiting

Study of Nipocalimab in Pregnant Women at Risk of Severe Hemolytic Disease of the Fetus and Newborn (HDFN)

Investigational
Countries:AustriaAustria
  • Participants:18–64 years
  • Substances:Nipocalimab
  • Sponsor:Janssen Cilag International

Glycogen storage disease type II

Recruiting

Study on Gene Therapy with AAV9.LAMP2B for Male Patients with Danon Disease

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Eculizumab
  • Sponsor:Rocket Pharmaceuticals Inc.

Neonatal alloimmune thrombocytopenia

Recruiting

Study on Nipocalimab for Reducing Risk of Fetal and Neonatal Alloimmune Thrombocytopenia in At-risk Pregnancies

Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Substances:Nipocalimab
  • Sponsor:Janssen Cilag International

Factor VIII deficiency+1

Recruiting

Study on Long-Term Safety and Effects of Marstacimab for Patients with Severe Hemophilia A or B, With or Without Inhibitors

InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Marstacimab
  • Sponsor:Pfizer Inc.

PIK3CA related overgrowth spectrum

Recruiting

Study on the Effects and Safety of Alpelisib for Children and Adults with Megalencephaly-Capillary Malformation Polymicrogyria Syndrome

Registered drug
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Alpelisib
  • Sponsor:Centre Hospitalier Universitaire De Dijon

Congenital aplastic anaemia

Recruiting

Long-Term Study on the Safety and Effects of RP-L102 Infusion for Patients with Fanconi Anemia Subtype A

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Autologous Cd34+Enriched Cells From Patients With Fanconi Anemia Subtype A Transduced Ex Vivo With Lentiviral Vector Carrying The Fanconi Anemia Complementation Group A Gene
  • Sponsor:Rocket Pharmaceuticals Inc.

Congenital absence of bile ducts

Recruiting

Study on Long-term Effects and Safety of Odevixibat in Children with Biliary Atresia

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Odevixibat
  • Sponsor:Ipsen Pharma

Duchenne muscular dystrophy

Recruiting

Study on the Safety and Effects of SQY51 for Children and Adults with Duchenne Muscular Dystrophy

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Sqy51
  • Sponsor:Sqy Therapeutics

Factor VIII deficiency+1

Recruiting

Study on Marstacimab for Children with Severe Hemophilia A or B, With or Without Inhibitors

InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Marstacimab
  • Sponsor:Pfizer Inc.

Alpha-1 antitrypsin deficiency

Recruiting

Study on Fazirsiran for Patients with Alpha-1 Antitrypsin Deficiency-Related Liver Disease and Fibrosis

Registered drug
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:Fazirsiran
  • Sponsor:Takeda Development Center Americas 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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