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Congenital, familial and genetic disorders – page 34

  • 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
Quick filters

803 clinical trials

Factor VIII deficiency+1

Not yet recruiting

Study on Joint Health in Hemophilia A and B Patients Using Efmoroctocog Alfa or Eftrenonacog Alfa

Registered drugNo placebo
Countries:BulgariaBulgaria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Efmoroctocog Alfa
  • Sponsor:Swedish Orphan Biovitrum AB (publ)

Krabbe's disease

Not yet recruiting

Study on Gene Therapy with AAVrh10 for Infants with Krabbe Disease Undergoing Stem Cell Transplantation

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years
  • Substances:Adeno-Associated Virus Serotype Rh10 Containing The Human Galc Gene
  • Sponsor:Forge Biologics Inc.

Acute myeloid leukaemia+1

Not yet recruiting

Study of Cytarabine and Daunorubicin for Treating Myeloid Leukemia in Children with Down Syndrome

Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Cytarabine
  • Sponsor:GPOH gGmbH

Arrhythmogenic right ventricular dysplasia

Not yet recruiting

Study on Spironolactone for Patients with Arrhythmogenic Right Ventricular Dysplasia (ARVD)

Registered drug
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Spironolactone
  • Sponsor:Hospices Civils De Lyon

Chronic kidney disease+1

Not recruiting

A Study Testing How Well BAY 3401016 Works and How Safe It Is in Adults Aged 18 to 45 Years with Alport Syndrome

Investigational
Countries:CzechiaCzechia
  • Participants:18–64 years
  • Sponsor:Bayer AG

Hereditary angioedema with C1 esterase inhibitor deficiency+1

Not recruiting

Study of navenibart to prevent attacks in adults and adolescents with Hereditary Angioedema

Investigational
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Navenibart
  • Sponsor:Astria Therapeutics Inc.

Familial hypertriglyceridaemia+1

Not recruiting

Study of ISIS 678354 for Patients with Severe High Triglycerides

Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:ISIS 678354 sodium salt
  • Sponsor:Ionis Pharmaceuticals Inc.

Primary immunodeficiency syndrome

Not recruiting

Study on the Effects of TAK-881 and Hyaluronidase in Adults with Primary Immunodeficiency Diseases

Registered drugNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Human Normal Immunoglobulin
  • Sponsor:Takeda Development Center Americas Inc.

Primary immunodeficiency syndrome

Not recruiting

Study on Long-term Safety of TAK-881 (Human Normal Immunoglobulin, Hyaluronidase) for Patients with Primary Immunodeficiency Diseases

InvestigationalNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Human Normal Immunoglobulin
  • Sponsor:Baxalta Innovations GmbH

Familial hypertriglyceridaemia+1

Not recruiting

Study of Olezarsen (ISIS 678354) in Patients with Severe Hypertriglyceridemia: Testing Effects on Triglyceride Levels

Investigational
Countries:BulgariaBulgaria
  • Participants:18–64 years · 65+ years
  • Substances:ISIS 678354 sodium salt
  • Sponsor:Ionis Pharmaceuticals Inc.

Activated PI3 kinase delta syndrome

Not recruiting

Drug interaction study of leniolisib with a drug combination in healthy volunteers for Activated PI3K Delta Syndrome

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · Healthy volunteers
  • Substances:Caffeine
  • Sponsor:Pharming Technologies B.V.

Hereditary ataxia

Not recruiting

Safety and tolerability of intrathecal S233107 in participants with spinocerebellar ataxia type 3: a Phase 1b/2a first‑in‑human randomized study

Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years
  • Sponsor:Institut De Recherches Internationales Servier IRIS

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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