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Congenital, familial and genetic disorders – page 57

  • 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

Type IIa hyperlipidaemia

Not recruiting

Study on the Effectiveness of Enlicitide Chloride in Reducing Cardiovascular Events in Patients with Atherosclerotic Cardiovascular Disease

Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Sponsor:Merck Sharp & Dohme LLC

Haemophilia

Not recruiting

Study of Fitusiran for Preventing Bleeding in Boys Aged 1 to 12 with Hemophilia A or B

InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Fitusiran
  • Sponsor:Genzyme Corp.

Mucopolysaccharidosis II

Not recruiting

Study on the Safety of DNL310 for Children with Hunter Syndrome

InvestigationalNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:0–17 years · 18–64 years
  • Substances:Iduronate-2-Sulfatase Fused To A Fc Polypeptide That Binds To The Human Transferrin Receptor
  • Sponsor:Denali Therapeutics Inc.

Mitochondrial DNA depletion

Not recruiting

Study of Doxecitine and Doxribtimine Oral Solution for Treatment of Patients with Thymidine Kinase 2 (TK2) Deficiency Who Have Previously Received Treatment

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Doxecitine
  • Sponsor:UCB Biosciences Inc.

Immune-mediated enterocolitis+1

Not recruiting

Study on MAS825 for Patients with Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations

Registered drug
Countries:CzechiaCzechia
  • Participants:0–17 years · 18–64 years
  • Substances:Ciclosporin
  • Sponsor:Novartis Pharma AG

Facioscapulohumeral muscular dystrophy

Not recruiting

Study on the Safety and Effects of Losmapimod for Patients with Facioscapulohumeral Muscular Dystrophy 1 (FSHD1)

InvestigationalNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years
  • Substances:Losmapimod
  • Sponsor:Fulcrum Therapeutics Inc.

Factor IX deficiency

Not recruiting

Study on Long-Term Safety and Efficacy of Etranacogene Dezaparvovec for Adults with Severe or Moderately Severe Hemophilia B

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Etranacogene Dezaparvovec
  • Sponsor:CSL Behring LLC

Cardiac amyloidosis+1

Not recruiting

Study on the Effectiveness and Safety of Vutrisiran and Patisiran for Patients with Hereditary Transthyretin Amyloidosis

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Patisiran
  • Sponsor:Alnylam Pharmaceuticals Inc.

Factor VIII deficiency

Not recruiting

Study on the Safety and Effectiveness of Giroctocogene Fitelparvovec for Adult Men with Moderate to Severe Hemophilia A

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years
  • Substances:Giroctocogene Fitelparvovec
  • Sponsor:Pfizer Inc.

Cystic fibrosis lung

Not recruiting

Long-Term Safety Study of BI 3720931 for Patients with Cystic Fibrosis from a Previous Trial

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:BI 3720931
  • Sponsor:Boehringer Ingelheim Espana S.A.

Cystic fibrosis lung

Not recruiting

Study on the Safety and Effectiveness of BI 3720931 for Adults with Cystic Fibrosis Who Cannot Use CFTR Modulators

Registered drug
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:BI 3720931
  • Sponsor:Boehringer Ingelheim Espana S.A.

Factor VIII deficiency

Not recruiting

Long-Term Safety Study of Valoctocogene Roxaparvovec for Patients with Severe Hemophilia A

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Valoctocogene Roxaparvovec
  • Sponsor:Biomarin Pharmaceutical 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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