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Congenital, familial and genetic disorders – page 27

  • 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

VEXAS syndrome

Not yet recruiting

A Randomized, Double‑Blind, Placebo‑Controlled Study of Momelotinib Dihydrochloride Monohydrate in Patients with VEXAS Syndrome

Investigational
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Momelotinib Dihydrochloride Monohydrate
  • Sponsor:Glaxosmithkline Research & Development Limited

Epidermolysis bullosa

Not yet recruiting

Assessing the efficacy and safety of nemolizumab in patients aged 12 years or older with dystrophic epidermolysis bullosa

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Nemolizumab
  • Sponsor:Centre Hospitalier Universitaire De Nice

Adrenoleukodystrophy

Not yet recruiting

Safety and Early Benefit of Allogeneic Bone Marrow‑Derived Mesenchymal Stromal Cells in Children and Adolescents with X‑Linked Adrenoleukodystrophy

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years
  • Sponsor:Fundacion Para La Investigacion Biomedica Hospital Infantil Universitario Nino Jesus

Hypertrophic cardiomyopathy

Not yet recruiting

Effect of oral mavacamten on left ventricular filling pressure in patients with hypertrophic cardiomyopathy

Registered drugNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Mavacamten
  • Sponsor:Rigshospitalet

Huntington's disease

Not yet recruiting

Phase 3 Study of Pridopidine Hydrochloride for Safety and Efficacy in Adults with Huntington’s Disease

Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Sponsor:Ferrer Internacional S.A.

Breast cancer+1

Not yet recruiting

Long‑term safety study of alpelisib alone or with fulvestrant in adults with breast cancer who benefited from previous alpelisib treatment

Registered drugNo placebo
Countries:BulgariaBulgaria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Alpelisib
  • Sponsor:Novartis Pharma AG

Glycogen storage disease type II

Not yet recruiting

Long‑term study of Pompe disease in all patients receiving alglucosidase alfa drug combination (miglustat, cipaglucosidase alfa, avalglucosidase alfa)

Registered drugNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Alglucosidase Alfa
  • Sponsor:Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

Hereditary haemorrhagic telangiectasia

Not yet recruiting

Safety Study of DIAG723 and Sodium Chloride in Adults with Hereditary Hemorrhagic Telangiectasia

Registered drug
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Sodium Chloride

Phelan-McDermid syndrome

Not yet recruiting

Study of the Safety, Tolerability and Efficacy of Oral Vafidemstat in Adults with Phelan‑McDermid Syndrome (HOPE‑2)

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:18–64 years
  • Substances:Vafidemstat
  • Sponsor:Oryzon Genomics S.A.

Sickle cell disease

Not yet recruiting

A study of etavopivat to reduce vaso‑occlusive crises in children with sickle cell disease

Investigational
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Etavopivat
  • Sponsor:Novo Nordisk A/S

Hypertrophic cardiomyopathy

Not yet recruiting

Study of aficamten and 68Ga‑FAPI‑46 versus placebo in asymptomatic obstructive hypertrophic cardiomyopathy patients

Registered drug
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years
  • Substances:Aficamten
  • Sponsor:Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

Allogenic stem cell transplantation+1

Not yet recruiting

Levofloxacin prophylaxis to reduce bloodstream infections in patients with hematologic malignancies undergoing allogeneic hematopoietic stem cell transplantation

Registered drug
Countries:FinlandFinland
  • Participants:18–64 years
  • Substances:Levofloxacin
  • Sponsor:HUS-Yhtymae

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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