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Congenital, familial and genetic disorders – page 32

  • 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

Not yet recruiting

Study on the Effects of Mitapivat on Brain Blood Flow and Oxygen Use in Patients with Sickle Cell Anemia

InvestigationalNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years
  • Substances:Mitapivat
  • Sponsor:Amsterdam UMC Stichting

Arteriovenous malformation

Not yet recruiting

Study on the Safety and Effectiveness of Regorafenib for Patients with Arterio-Venous Malformations Unresponsive to Standard Treatment

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Regorafenib
  • Sponsor:Cliniques Universitaires Saint-Luc

Biotinidase deficiency

Not yet recruiting

Study on the Effectiveness of Biotin and Biotin Sodium for Children with Metabolic Disorders

Registered drugNo placebo
Countries:SpainSpain
  • Participants:0–17 years
  • Substances:Biotin
  • Sponsor:Fundacion Publica Galega Instituto De Investigacion Sanitaria De Santiago De Compostela

Retinitis pigmentosa

Not yet recruiting

Study on the Effectiveness and Safety of Ultevursen for Patients with Retinitis Pigmentosa Due to USH2A Gene Mutations

Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Ultevursen
  • Sponsor:Laboratoires Thea

Duchenne muscular dystrophy

Not yet recruiting

Study on the Safety and Effectiveness of DT-DEC01 for Patients with Duchenne Muscular Dystrophy

InvestigationalNo placebo
Countries:PolandPoland
  • Participants:0–17 years
  • Sponsor:Dystrogen Therapeutics Technology Polska Sp. z o.o.

Congenital arterial malformation

Not yet recruiting

Study on Sirolimus and Prednisolone for Treating Superficial Arteriovenous Malformations in Patients

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Sirolimus
  • Sponsor:Centre Hospitalier Universitaire Amiens Picardie

Muscular dystrophy

Not yet recruiting

Study on the Effectiveness of Deflazacort in Treating LMNA-related Congenital Muscular Dystrophy in Adults and Children

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years
  • Substances:Deflazacort
  • Sponsor:Azienda Ospedaliero Universitaria Pisana

Sickle cell disease

Not yet recruiting

Study of GBT021601 for Adults and Children with Sickle Cell Disease

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:(S)-2-Hydroxy-6-((4-(2-(2-Hydroxyethyl)Nicotinoyl)Morpholin-3-Yl)Methoxy)Benzaldehyde
  • Sponsor:Global Blood Therapeutics Inc.

Heart disease congenital

Not yet recruiting

Study Comparing Custodiol-N and Custodiol for Heart Surgery in Children with Congenital Heart Defects

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Substances:Alanine
  • Sponsor:Dr. Franz Koehler Chemie GmbH

Neurofibromatosis

Not yet recruiting

Study on Trametinib for Patients with Neurofibromatosis Type 1 and Symptomatic Plexiform Neurofibromas

Registered drugNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years
  • Substances:Trametinib
  • Sponsor:Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

Adenomatous polyposis coli

Not yet recruiting

Study on the Safety of Sirolimus for Children with Familial Adenomatous Polyposis

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Sirolimus
  • Sponsor:Centre Hospitalier Universitaire De Toulouse

Duchenne muscular dystrophy+1

Not yet recruiting

Study on the Safety and Effectiveness of Delandistrogene Moxeparvovec for Patients with Duchenne Muscular Dystrophy

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Delandistrogene Moxeparvovec
  • Sponsor:Sarepta 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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