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Congenital, familial and genetic disorders – page 54

  • 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

Gaucher's disease type I

Not recruiting

Study on the Long-term Safety of FLT201 for Patients with Gaucher Disease Type 1

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:18–64 years · 65+ years
  • Substances:Adeno-Associated Viral Vector Serotype S3 Containing Codon-Optimised Expression Cassette Encoding Human Beta-Glucocerebrosidase Variant
  • Sponsor:Spur Therapeutics Limited

Congenital cystic kidney disease

Not recruiting

Study on the Safety and Effects of Tolvaptan for Infants and Young Children with Autosomal Recessive Polycystic Kidney Disease

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Tolvaptan
  • Sponsor:Otsuka Pharmaceutical Development & Commercialization Inc.

Cystic fibrosis

Not recruiting

Study on the Effects of Dirocaftor, Posenacaftor, and Nesolicaftor for Adults with Cystic Fibrosis

Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Dirocaftor
  • Sponsor:Universitair Medisch Centrum Utrecht

Osteochondrodysplasia

Not recruiting

Study on the Long-Term Safety and Effectiveness of Vosoritide for Children with Achondroplasia

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Substances:Vosoritide
  • Sponsor:Biomarin Pharmaceutical Inc.

Hereditary angioedema

Not recruiting

Long-term safety study of garadacimab (CSL312) for prevention of hereditary angioedema attacks

InvestigationalNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Garadacimab
  • Sponsor:CSL Behring LLC

Metachromatic leukodystrophy

Not recruiting

Study on the Effects of Atidarsagene Autotemcel for Patients with Late Juvenile Metachromatic Leukodystrophy

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

Fragile X syndrome

Not recruiting

Study on Long-Term Safety of ZYN002 Gel for Children, Adolescents, and Young Adults with Fragile X Syndrome

InvestigationalNo placebo
Countries:IrelandIreland
  • Participants:0–17 years · 18–64 years
  • Substances:Cannabidiol
  • Sponsor:Harmony Biosciences Management Inc.

Duchenne muscular dystrophy gene carrier

Not recruiting

Study to Determine the Best Dose of Vesleteplirsen for Patients with Duchenne Muscular Dystrophy Suitable for Exon 51-Skipping Treatment

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Vesleteplirsen
  • Sponsor:Sarepta Therapeutics Inc.

Huntington's disease

Not recruiting

Study on the Safety and Effects of PTC518 for Patients with Huntington's Disease

Investigational
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:PTC518
  • Sponsor:PTC Therapeutics Inc.

Cerebral palsy+1

Not recruiting

Study on the Effectiveness and Safety of NT 201 for Treating Lower Limb Spasticity in Children and Adolescents with Cerebral Palsy

Registered drug
Countries:LatviaLatvia
  • Participants:0–17 years
  • Substances:Clostridium Botulinum Neurotoxin Type A (150Kd), Free Of Complexing Proteins
  • Sponsor:Merz Pharmaceuticals GmbH

Tuberous sclerosis complex

Not recruiting

Study on Cannabidiol as Add-on Therapy for Seizures in Patients Aged 1-65 with Tuberous Sclerosis Complex

Registered drugNo placebo
Countries:PolandPoland
  • Participants:0–17 years · 18–64 years
  • Substances:Cannabidiol
  • Sponsor:Jazz Pharmaceuticals Research UK Limited

Neurofibromatosis

Not recruiting

Study on Selumetinib for Children Aged 1 to 6 with Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Substances:Selumetinib
  • Sponsor:AstraZeneca AB

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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