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Congenital, familial and genetic disorders – page 50

  • 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
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796 clinical trials

Facioscapulohumeral muscular dystrophy

Not recruiting

Study on the Effects and Safety of RO7204239 for Patients with Facioscapulohumeral Muscular Dystrophy

Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years
  • Substances:Humanised Igg1 Monoclonal Antibody Against Human Latent Myostatin
  • Sponsor:F. Hoffmann-La Roche AG

Porphyria non-acute

Not recruiting

Study of Afamelanotide for Patients with Erythropoietic Protoporphyria (EPP)

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Afamelanotide
  • Sponsor:Clinuvel Europe Limited

Hereditary angioedema with C1 esterase inhibitor deficiency

Not recruiting

Long-term Safety Study of KVD900 for Treating Angioedema Attacks in Adolescents and Adults with Hereditary Angioedema Type I or II

InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Sebetralstat
  • Sponsor:Kalvista Pharmaceuticals Limited

Duchenne muscular dystrophy

Not recruiting

Study on the Safety and Efficacy of Viltolarsen for Boys with Duchenne Muscular Dystrophy

InvestigationalNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years
  • Substances:Viltolarsen
  • Sponsor:Ns Pharma Inc.

Spinal muscular atrophy

Not recruiting

Study on the Effectiveness and Safety of Risdiplam for Children with Spinal Muscular Atrophy After Gene Therapy

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Substances:Risdiplam
  • Sponsor:F. Hoffmann-La Roche AG

Hypertrophic cardiomyopathy

Not recruiting

Study on Aficamten for Adults with Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy

Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Aficamten
  • Sponsor:Cytokinetics Inc.

Factor IX deficiency

Not recruiting

Long-term Safety Study of Etranacogene Dezaparvovec in Adult Men with Hemophilia B

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

Tourette's disorder

Not recruiting

Study on the Effects of Gemlapodect for Adults and Adolescents with Tourette Syndrome

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Gemlapodect
  • Sponsor:Noema Pharma AG

Congenital cystic kidney disease

Not recruiting

Study of Empagliflozin for Patients with Autosomal Dominant Polycystic Kidney Disease

Registered drug
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Empagliflozin
  • Sponsor:Medizinische Hochschule Hannover

Ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency

Not recruiting

Study on the Effects and Safety of INZ-701 for Children with ENPP1 Deficiency

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Recombinant Human Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Fused To The Fc Fragment Of Igg1
  • Sponsor:Inozyme Pharma Inc.

Haemophilia A without inhibitors

Not recruiting

Study on the Effects and Safety of Emicizumab for Infants with Hemophilia A (Ages Birth to 12 Months)

Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Emicizumab
  • Sponsor:F. Hoffmann-La Roche AG

Arteriosclerosis+1

Not recruiting

Study on the Safety and Effects of INZ-701 for Infants with ENPP1 or ABCC6 Deficiency

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years
  • Substances:Recombinant Human Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Fused To The Fc Fragment Of Igg1
  • Sponsor:Inozyme Pharma 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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