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Congenital, familial and genetic disorders – page 42

  • 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

Glycogen storage disease type II

Not recruiting

Long-Term Safety Study of Avalglucosidase Alfa for Patients with Pompe Disease in France

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Avalglucosidase Alfa
  • Sponsor:Sanofi Winthrop Industrie

Myotonic dystrophy

Not recruiting

Study on AOC 1001 for Treating Myotonic Dystrophy Type 1 in Patients

Investigational
Countries:DenmarkDenmark
  • Participants:0–17 years · 18–64 years
  • Substances:Humanised Igg1 Monoclonal Antibody Against Tfr1 Conjugated To Double Stranded Sirna Oligonucleotide Against Dmpk Via A Non-Cleavable Linker
  • Sponsor:Avidity Biosciences Inc.

Spinal muscular atrophy

Not recruiting

Long-term Safety Study of Onasemnogene Abeparvovec for Patients with Spinal Muscular Atrophy

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Onasemnogene Abeparvovec
  • Sponsor:Novartis Pharma AG

Amyotrophic lateral sclerosis+2

Not recruiting

Study of Tofersen for Adults with Presymptomatic Amyotrophic Lateral Sclerosis (ALS) Due to SOD1 Gene Mutation

Investigational
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Tofersen
  • Sponsor:Biogen Idec Research Limited

Asplenia

Not recruiting

Study on the Safety and Immune Response of Meningococcal B Vaccines in Adults with Asplenia: Bexsero, Trumenba, and a Drug Combination

Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Neisseria Meningitidis Group B Fhbp Protein Subfamily A
  • Sponsor:Assistance Publique Hopitaux De Paris

Colorectal cancer metastatic+2

Not recruiting

Study on the Safety and Efficacy of CR6086 and Balstilimab for Patients with Pretreated Metastatic Colorectal and Other GI Cancers

InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:18–64 years · 65+ years
  • Substances:Balstilimab
  • Sponsor:Rottapharm Biotech S.r.l.

Pyruvate kinase deficiency anaemia

Not recruiting

Study on the Effectiveness and Safety of Mitapivat for Children with Pyruvate Kinase Deficiency Receiving Regular Blood Transfusions

Investigational
Countries:CzechiaCzechia
  • Participants:0–17 years
  • Substances:Mitapivat
  • Sponsor:Agios Pharmaceuticals Inc.

Familial hypertriglyceridaemia+1

Not recruiting

Study on the Effects of ISIS 678354 in Patients with Familial Chylomicronemia Syndrome (FCS)

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:ISIS 678354 sodium salt
  • Sponsor:Ionis Pharmaceuticals Inc.

Sickle cell anaemia

Not recruiting

Study on Voxelotor for Reducing Hemolysis in Patients with Sickle Cell Disease

Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Voxelotor
  • Sponsor:Assistance Publique Hopitaux De Paris

Wolfram syndrome

Not recruiting

Study on Tirzepatide for Improving Insulin Production in Patients with Wolfram Syndrome Type 1

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Tirzepatide
  • Sponsor:Ospedale San Raffaele S.r.l.

Stargardt's disease

Not recruiting

Study on the Long-Term Safety of SAR422459 for Patients with Stargardt's Disease

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Lentiviral vector containing the human ABCA4 gene
  • Sponsor:Sanofi-Aventis Recherche & Developpement

Hereditary neuropathic amyloidosis

Not recruiting

Study on NTLA-2001 for Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Ziclumeran
  • Sponsor:Intellia 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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