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Congenital, familial and genetic disorders – page 66

  • 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

Sickle cell anaemia with crisis+1

Not recruiting

Study on the Safety and Effects of Crovalimab for Treating Vaso-Occlusive Episodes in Sickle Cell Disease Patients

Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Crovalimab
  • Sponsor:F. Hoffmann-La Roche AG

Corneal dystrophy

Not recruiting

Study on the Safety and Effectiveness of Ripasudil Eye Drops for Patients with Fuchs Dystrophy After Cataract Surgery

Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Ripasudil
  • Sponsor:Kowa Research Institute Inc.

Factor VIII deficiency+3

Not recruiting

Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Sponsor:Apcintex Limited

Facioscapulohumeral muscular dystrophy

Not recruiting

Study on AOC 1020 for Adults with Facioscapulohumeral Muscular Dystrophy (FSHD)

Investigational
Countries:ItalyItaly
  • Participants:18–64 years · 65+ years
  • Substances:Humanised Igg1 Monoclonal Antibody Against Tfr1 Conjugated To Double Stranded Sirna Oligonucleotide Against Dux4 Mrna Via A Non-Cleavable Linker
  • Sponsor:Avidity Biosciences Inc.

Early onset familial Alzheimer's disease

Not recruiting

Study on the Safety and Effects of ABBV-916 and MK-6240 for Patients with Early Alzheimer's Disease

Investigational
Countries:ItalyItaly
  • Participants:18–64 years · 65+ years
  • Substances:Florquinitau (18F)
  • Sponsor:AbbVie Deutschland GmbH & Co. KG

Patent ductus arteriosus

Not recruiting

Study on the Use of Ibuprofen and Paracetamol for Treating Patent Ductus Arteriosus in Extremely Premature Infants

Registered drug
Countries:IrelandIreland
  • Participants:0–17 years
  • Substances:Ibuprofen
  • Sponsor:Royal College Of Surgeons In Ireland

Tourette's disorder

Not recruiting

Study on the Long-Term Safety of Ecopipam Hydrochloride Tablets for Children, Adolescents, and Adults with Tourette's Disorder

InvestigationalNo placebo
Countries:BulgariaBulgaria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Ecopipam Hydrochloride
  • Sponsor:Emalex Biosciences Inc.

Polycystic liver disease

Not recruiting

Study on Octreotide for Patients with Symptomatic Polycystic Liver Disease

Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Octreotide Hydrochloride
  • Sponsor:Camurus AB

Haemophilia B with anti factor IX+1

Not recruiting

Study on the Safety and Effectiveness of SerpinPC for Patients with Hemophilia B with Inhibitors

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Sponsor:Apcintex Limited

Osteogenesis imperfecta

Not recruiting

Study Comparing Setrusumab and Bisphosphonates for Children with Osteogenesis Imperfecta

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Neridronate Sodium
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Isodicentric chromosome 15 syndrome+3

Not recruiting

Study on Long-Term Safety of Soticlestat for Adults and Children with Developmental Epileptic Encephalopathies

InvestigationalNo placebo
Countries:PolandPoland
  • Participants:0–17 years · 18–64 years
  • Substances:Soticlestat
  • Sponsor:Takeda Development Center Americas Inc.

Angelman's syndrome

Not recruiting

Study on the Safety and Tolerability of Rugonersen in Patients with Angelman Syndrome

InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Rugonersen
  • Sponsor:F. Hoffmann-La Roche AG

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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