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Congenital, familial and genetic disorders – page 17

  • 796 clinical trials
  • 308 recruiting
  • 374 conditions
  • 27 countries

Conditions in this area

374 conditions with trials, the busiest first

Duchenne muscular dystrophy28Factor VIII deficiency25Sickle cell disease25Cystic fibrosis24Hereditary angioedema22Spinal muscular atrophy17Hypertrophic cardiomyopathy16Type IIa hyperlipidaemia15Glycogen storage disease type II14Factor IX deficiency13Sickle cell anaemia13Congenital cystic kidney disease12Huntington's disease12Myotonic dystrophy12Non-small cell lung cancer12Osteochondrodysplasia12Adrenogenital syndrome11Alpha-1 antitrypsin deficiency11K-ras gene mutation11Retinitis pigmentosa11Severe myoclonic epilepsy of infancy11Thalassaemia11Fabry's disease10Hereditary angioedema with C1 esterase inhibitor deficiency10+350

Clinical trials in Congenital, familial and genetic disorders

796 trials across Europe, recruiting first

Type of trial
Recruitment status
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796 clinical trials

Combined immunodeficiency

Recruiting

Study on Gene Therapy for Severe Combined Immunodeficiency (SCID) Using ARTEGENE in Patients with Artemis Gene Mutations

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Autologous Cd34+ Haematopoietic Stem And Progenitor Cells Transduced With A Lentiviral Vector Containing The Human Dclre1C Gene
  • Sponsor:Assistance Publique Hopitaux De Paris

Neurofibromatosis

Recruiting

Imaging Study with 89Zr-Bevacizumab for Predicting Treatment Effects in Patients with NF2-Related Schwannomatosis

InvestigationalNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years
  • Substances:Bevacizumab Zirconium Zr-89
  • Sponsor:Leids Universitair Medisch Centrum (LUMC)

Urethral valves

Recruiting

Study on Early Oxybutynin Treatment for Boys with Posterior Urethral Valves

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Oxybutynin
  • Sponsor:Centre Hospitalier Universitaire De Bordeaux

Cystic fibrosis

Recruiting

Study on Improving Bone Health in Children with Cystic Fibrosis Using Cholecalciferol

Registered drugNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years
  • Substances:Colecalciferol
  • Sponsor:Fakultni Nemocnice V Motole

Staphylococcal infection+1

Recruiting

Study on the Safety of Inhaled Teicoplanin for Treating Staphylococcus aureus Infections in Cystic Fibrosis Patients

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years
  • Substances:Teicoplanin
  • Sponsor:Azienda Ospedaliera Universitaria Integrata Verona

Duchenne muscular dystrophy

Recruiting

Study on the Safety and Effects of Givinostat in Young Children with Duchenne Muscular Dystrophy (Ages 2 to Less Than 6 Years)

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Givinostat
  • Sponsor:Italfarmaco S.p.A.

Myotonic dystrophy

Recruiting

Long-Term Safety and Efficacy Study of Mexiletine Hydrochloride for Patients with Myotonic Dystrophy Type 1 and Type 2

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Mexiletine Hydrochloride
  • Sponsor:Lupin Atlantis Holdings SA

Hydrocele+1

Recruiting

Study on Sclerotherapy for Hydrocele and Spermatocele Using Lauromacrogol 400 and Ethanol in Adult Patients

Registered drugNo placebo
Countries:SwedenSweden
  • Participants:18–64 years · 65+ years
  • Substances:Ethanol, Anhydrous
  • Sponsor:Region Jamtland Harjedalen

Hereditary hypophosphataemic rickets

Recruiting

Study of KK8123 for Adults with X-linked Hypophosphatemia

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years
  • Substances:Kk8123
  • Sponsor:Kyowa Kirin Inc.

Hereditary ataxia+1

Recruiting

Study on the Safety of VO659 for Patients with Spinocerebellar Ataxia Types 1, 3, and Huntington's Disease

InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years
  • Substances:Vo659
  • Sponsor:Vico Therapeutics B.V.

Sickle cell disease+1

Recruiting

Long-term safety study of oral etavopivat tablets in children, adolescents and adults with sickle cell disease or thalassaemia who completed previous etavopivat treatment

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Etavopivat
  • Sponsor:Novo Nordisk A/S

Adrenogenital syndrome

Recruiting

Study on the Effectiveness of Dexamethasone in Preventing Prenatal Virilization in Female Patients with Congenital Adrenal Hyperplasia

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years
  • Substances:Dexamethasone
  • Sponsor:Philipps-Universitaet Marburg

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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