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Congenital, familial and genetic disorders – page 40

  • 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

Alpha-1 antitrypsin deficiency

Not recruiting

Study on the Safety and Tolerability of WVE-006 for Patients with Alpha-1 Antitrypsin Deficiency (AATD) Pi*ZZ

InvestigationalNo placebo
Countries:FinlandFinland
  • Participants:18–64 years · 65+ years
  • Substances:WVE-006
  • Sponsor:Wave Life Sciences Ireland Limited

MELAS syndrome

Not recruiting

Study on the Effects of TTI-0102 for Patients with MELAS Syndrome

Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Mercaptamine-Pantetheine Disulfide Acetate
  • Sponsor:Thiogenesis Therapeutics

Polycystic liver disease

Not recruiting

Study on Leuprorelin Acetate for Slowing Disease Progression in Premenopausal Women with Severe Polycystic Liver Disease

Registered drugNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years
  • Substances:Leuprorelin Acetate
  • Sponsor:Universitair Medisch Centrum Groningen

Bile acid synthesis disorder

Not recruiting

Long-term Safety Study of Cholic Acid for Patients with Bile Acid Synthesis Defects

Registered drugNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Cholic Acid
  • Sponsor:Amsterdam UMC Stichting

Anaplastic lymphoma kinase gene mutation

Not recruiting

Study Comparing Alectinib and Crizotinib for Patients with ALK-Positive Advanced Non-Small Cell Lung Cancer

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:18–64 years · 65+ years
  • Substances:Alectinib
  • Sponsor:F. Hoffmann-La Roche AG

Angelman's syndrome

Not recruiting

Study on the Long-Term Safety of GTX-102 for Patients with Angelman Syndrome

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Apazunersen
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Porphyria non-acute

Not recruiting

Study on the Safety and Tolerability of Oral Dersimelagon for Patients with Erythropoietic Protoporphyria or X-Linked Protoporphyria

InvestigationalNo placebo
Countries:BulgariaBulgaria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Dersimelagon Phosphate
  • Sponsor:Tanabe Pharma America Inc.

Sickle cell disease

Not recruiting

Study on Improving Treatment for Sickle Cell Disease Patients Using Hydroxycarbamide

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Hydroxycarbamide
  • Sponsor:Les Hopitaux Universitaires De Strasbourg

Adrenoleukodystrophy

Not recruiting

Long-term Safety and Efficacy Study of Elivaldogene Autotemcel for Patients with Cerebral Adrenoleukodystrophy

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Elivaldogene Autotemcel
  • Sponsor:Genetix Biotherapeutics Inc.

Duchenne muscular dystrophy

Not recruiting

Study on the Effects of Casimersen and Golodirsen for Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Casimersen
  • Sponsor:Sarepta Therapeutics Inc.

Mucopolysaccharidosis III

Not recruiting

Study on the Safety and Initial Effects of AAV9-CAG-coh-SGSH in Children with Sanfilippo A Syndrome

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years
  • Substances:Adeno-Associated Viral Vector Serotype 9 Containing The Human Sulfamidase Gene
  • Sponsor:Esteve Pharmaceuticals S.A.

Mucopolysaccharidosis I

Not recruiting

Study on Gene Therapy with Autologous Stem Cells and Drug Combination for Children with Mucopolysaccharidosis Type I Hurler Variant

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Busulfan
  • Sponsor:Orchard Therapeutics (Europe) Limited

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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