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Congenital, familial and genetic disorders – page 15

  • 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

Heart disease congenital+1

Recruiting

Study on Allopurinol Sodium for Brain Injury Prevention in Newborns with Critical Congenital Heart Disease Undergoing Heart Surgery

Registered drug
Countries:The NetherlandsThe Netherlands
  • Participants:0–17 years
  • Substances:Allopurinol Sodium
  • Sponsor:Universitair Medisch Centrum Utrecht

Squamous cell carcinoma of the oral cavity+4

Recruiting

Study on the Safety and Effectiveness of Afatinib for Fanconi Anemia Patients with Advanced Squamous Cell Carcinoma in the Oral Cavity, Oropharynx, Hypopharynx, or Larynx

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Afatinib
  • Sponsor:Fundacio Institut De Recerca De L Hospital De La Santa Creu I Sant Pau

Congenital hypotransferrinaemia+5

Recruiting

Study on the Use of Human Apotransferrin for Treating Patients with Atransferrinemia

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years
  • Substances:Human Apotransferrin
  • Sponsor:Prothya Biosolutions Netherlands B.V.

Hereditary neuropathic amyloidosis

Recruiting

Long-Term Safety and Efficacy Study of Eplontersen in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

InvestigationalNo placebo
Countries:CyprusCyprus
  • Participants:18–64 years · 65+ years
  • Substances:Eplontersen
  • Sponsor:Ionis Pharmaceuticals Inc.

Facioscapulohumeral muscular dystrophy

Recruiting

Study of ADS-010 for Adults with Facioscapulohumeral Muscular Dystrophy Type 1

Registered drug
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:ADS-010
  • Sponsor:Sarepta Therapeutics Inc.

Retinitis pigmentosa

Recruiting

Study of GS030 gene therapy safety and tolerability in patients with Retinitis Pigmentosa

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Adeno-Associated Viral Vector Serotype 2.7M8 Containing The Chrimsonr-Tdtomato Gene
  • Sponsor:Gensight Biologics

Atrial septal defect

Recruiting

Study on Blood Thinners Rivaroxaban, Clopidogrel, and Aspirin for Patients with Heart Defects Undergoing Closure Procedures

Registered drugNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years
  • Substances:Acetylsalicylic Acid
  • Sponsor:Sint Antonius Ziekenhuis Stichting

Cystic fibrosis

Recruiting

Study on the Safety and Effects of SPL84 for Patients with Cystic Fibrosis

Investigational
Countries:CzechiaCzechia
  • Participants:18–64 years · 65+ years
  • Sponsor:Splisense Ltd.

Sickle cell disease

Recruiting

Study on Hydroxycarbamide Tablets for Children with Sickle Cell Disease

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Hydroxycarbamide
  • Sponsor:Theravia

BRAF V600E mutation positive+1

Recruiting

Study on the Effectiveness and Safety of Plixorafenib (FORE8394) for Patients with Cancer Having BRAF Alterations

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Cobicistat
  • Sponsor:Fore Biotherapeutics Inc.

Von Willebrand's disease

Recruiting

Study on Preventing Bleeding in Children with Severe von Willebrand Disease Using Vonicog Alfa and Octocog Alfa

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Octocog Alfa
  • Sponsor:Takeda Development Center Americas Inc.

Neurofibromatosis

Recruiting

Study on Crizotinib for Treating Severe Neurofibromatosis Type 2 in Children Not Eligible for Surgery or Radiotherapy

Registered drugNo placebo
Countries:PolandPoland
  • Participants:0–17 years
  • Substances:Crizotinib
  • Sponsor:Medical University Of Warsaw

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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