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Congenital, familial and genetic disorders – page 58

  • 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

Spinal muscular atrophy

Not recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Onasemnogene Abeparvovec
  • Sponsor:Novartis Pharma AG

Sickle cell disease

Not recruiting
Registered drug
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Decitabine
  • Sponsor:Novo Nordisk A/S

Study of Nusinersen for Patients with Spinal Muscular Atrophy Previously Treated with Onasemnogene Abeparvovec

Not recruiting
Registered drugNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Substances:Nusinersen
  • Sponsor:Biogen Idec Research Limited

Congenital pseudarthrosis

Not recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Autologous Adipose-Derived Stem Cells
  • Sponsor:Novadip Biosciences

Type V hyperlipidaemia

Not recruiting
InvestigationalNo placebo
Countries:HungaryHungary
  • Participants:18–64 years · 65+ years
  • Substances:Synthetic Double-Stranded Sirna Oligonucleotide Directed Against Apolipoprotein C-Iii Mrna And Covalently Linked To A Ligand Containing Three N-Acetylgalactosamine Residues
  • Sponsor:Arrowhead Pharmaceuticals Inc.

Phenylketonuria

Not recruiting
Registered drugNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Substances:Pegvaliase
  • Sponsor:Biomarin Pharmaceutical Inc.

Pyruvate kinase deficiency anaemia

Not recruiting
Investigational
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Mitapivat
  • Sponsor:Agios Pharmaceuticals Inc.

Glycogen storage disease type II

Not recruiting
Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Avalglucosidase Alfa
  • Sponsor:Sanofi-Aventis Recherche & Developpement

Duchenne muscular dystrophy

Not recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Fragment Antibody Targeting Human Tfr1 Conjugated To Phosphorodiamidate Morpholino Oligomer
  • Sponsor:Dyne Therapeutics Inc.

Spinal muscular atrophy

Not recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Taldefgrobep Alfa
  • Sponsor:Biohaven Pharmaceuticals Inc.

Von Hippel-Lindau disease

Not recruiting
InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Belzutifan
  • Sponsor:Peloton Therapeutics Inc.

Duchenne muscular dystrophy

Not recruiting
Registered drug
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Eculizumab
  • Sponsor:Pfizer 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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