Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Therapeutic area

Congenital, familial and genetic disorders – page 65

  • 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
Quick filters

796 clinical trials

Thalassaemia alpha

Not recruiting
Registered drug
Countries:GreeceGreece
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Luspatercept
  • Sponsor:Celgene Corp.

Familial haemophagocytic lymphohistiocytosis+1

Not recruiting
InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Substances:Tadekinig Alfa
  • Sponsor:AB2 Bio S.A.

Leukocyte adhesion deficiency+1

Not recruiting
InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Autologous Cd34+ Haematopoietic Stem Cells Genetically Modified With Lentiviral Vector Encoding The Cd18 Gene
  • Sponsor:Rocket Pharmaceuticals Inc.

Netherton's syndrome

Not recruiting
Registered drug
Countries:FranceFrance
  • Participants:18–64 years
  • Substances:Ds-2325A
  • Sponsor:Daiichi Sankyo Inc.

Sickle cell anaemia with crisis+1

Not recruiting
Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Sponsor:F. Hoffmann-La Roche AG

Sickle cell anaemia with crisis+1

Not recruiting
Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Crovalimab
  • Sponsor:F. Hoffmann-La Roche AG

Corneal dystrophy

Not recruiting
Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Ripasudil
  • Sponsor:Kowa Research Institute Inc.

Factor VIII deficiency+3

Not recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Sponsor:Apcintex Limited

Facioscapulohumeral muscular dystrophy

Not recruiting
Investigational
Countries:ItalyItaly
  • Participants:18–64 years · 65+ years
  • Substances:Humanised Igg1 Monoclonal Antibody Against Tfr1 Conjugated To Double Stranded Sirna Oligonucleotide Against Dux4 Mrna Via A Non-Cleavable Linker
  • Sponsor:Avidity Biosciences Inc.

Early onset familial Alzheimer's disease

Not recruiting
Investigational
Countries:ItalyItaly
  • Participants:18–64 years · 65+ years
  • Substances:Florquinitau (18F)
  • Sponsor:AbbVie Deutschland GmbH & Co. KG

Patent ductus arteriosus

Not recruiting
Registered drug
Countries:IrelandIreland
  • Participants:0–17 years
  • Substances:Ibuprofen
  • Sponsor:Royal College Of Surgeons In Ireland

Tourette's disorder

Not recruiting
InvestigationalNo placebo
Countries:BulgariaBulgaria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Ecopipam Hydrochloride
  • Sponsor:Emalex Biosciences 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
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.