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Congenital, familial and genetic disorders – page 46

  • 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

Hereditary non-polyposis colorectal cancer syndrome+1

Not recruiting

Study on the Effect of Low-Dose Aspirin in Preventing New or Recurrent Colorectal Polyps in Patients with Lynch Syndrome

Registered drug
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Acetylsalicylic Acid
  • Sponsor:Assistance Publique Hopitaux De Paris

Osteochondrodysplasia

Not recruiting

Study on the Effects of TransCon CNP in Children Aged 2-11 with Achondroplasia

Investigational
Countries:DenmarkDenmark
  • Participants:0–17 years
  • Substances:C-Type Natriuretic Peptide Conjugated To A Multi-Arm Polyethylene Glycol Carrier Molecule Through A Cleavable Linker
  • Sponsor:Ascendis Pharma Growth Disorders A/S

Familial hypertriglyceridaemia

Not recruiting

Study on the Effects of ARO-APOC3 for Adults with Familial Chylomicronemia Syndrome

Investigational
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:Synthetic Double-Stranded Sirna Oligonucleotide Directed Against Apolipoprotein C-Iii Mrna And Covalently Linked To A Ligand Containing Three N-Acetylgalactosamine Residues
  • Sponsor:Arrowhead Pharmaceuticals Inc.

Ornithine transcarbamoylase deficiency

Not recruiting

Study of Avalotcagene Ontaparvovec for Patients Aged 12 and Older with Late-onset Ornithine Transcarbamylase Deficiency

Registered drug
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Prednisolone
  • Sponsor:Ultragenyx Pharmaceutical Inc.

EGFR gene mutation+1

Not recruiting

Study on the Effectiveness and Safety of BAY 2927088 Compared to Standard Treatment in Patients with Advanced Non-Small Cell Lung Cancer with HER2 Mutations

Registered drugNo placebo
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:BAY 2927088
  • Sponsor:Bayer AG

Arteriosclerosis+2

Not recruiting

Long-term Safety Study of INZ-701 for Patients with ENPP1 and ABCC6 Deficiencies, Including Pseudoxanthoma Elasticum and Generalized Arterial Calcification of Infancy

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Recombinant Human Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Fused To The Fc Fragment Of Igg1
  • Sponsor:Inozyme Pharma Inc.

Duchenne muscular dystrophy

Not recruiting

Study on DMD06-MAB Injection for Duchenne Muscular Dystrophy in Non-Ambulant Patients

InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:DMD06-MAB
  • Sponsor:Ospedale San Raffaele S.r.l.

Metachromatic leukodystrophy

Not recruiting

Study on Gene Therapy with Atidarsagene Autotemcel for Patients with Metachromatic Leukodystrophy

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Atidarsagene Autotemcel
  • Sponsor:Orchard Therapeutics (Europe) Limited

Spinal muscular atrophy

Not recruiting

Study of NMD670 tablets to improve muscle strength in adults with TYPE 3 spinal muscular atrophy who can walk

Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:NMD670
  • Sponsor:NMD Pharma A/S

Neonatal alloimmune thrombocytopenia

Not recruiting

Study on the Safety and Effects of RLYB212 in Pregnant Women at Risk for Fetal and Neonatal Alloimmune Thrombocytopenia

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Substances:Anti-(integrin BETA-3) human monoclonal antibody
  • Sponsor:Rallybio Ipa LLC

Activated PI3 kinase delta syndrome

Not recruiting

Study of Leniolisib for Children Aged 4 to 11 with Activated PI3K Delta Syndrome

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Leniolisib Phosphate
  • Sponsor:Pharming Technologies B.V.

Choroidal dystrophy+1

Not recruiting

Long-term Safety and Efficacy Study of Gene Therapy for Choroideremia and X-Linked Retinitis Pigmentosa Using AAV2-REP1 and AAV8-RPGR in Previously Treated Patients

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years
  • Substances:Timrepigene Emparvovec
  • Sponsor:Biogen Idec Research Limited

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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