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Congenital, familial and genetic disorders – page 8

  • 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
Recruitment status
Quick filters

796 clinical trials

Osteopetrosis

Recruiting
Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Plerixafor
  • Sponsor:Fondazione Telethon Ets

Congenital pseudarthrosis

Recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Autologous Adipose-Derived Stem Cells
  • Sponsor:Novadip Biosciences

Myotonic dystrophy

Recruiting
InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years · 18–64 years
  • Substances:Humanised Igg1 Monoclonal Antibody Against Tfr1 Conjugated To Double Stranded Sirna Oligonucleotide Against Dmpk Via A Non-Cleavable Linker
  • Sponsor:Avidity Biosciences Inc.

Li-Fraumeni syndrome

Recruiting
Registered drugNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Metformin
  • Sponsor:Medizinische Hochschule Hannover

Cystic fibrosis

Recruiting
Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Elexacaftor
  • Sponsor:IRCCS Istituto Giannina Gaslini

K-ras gene mutation+1

Recruiting
Registered drugNo placebo
Countries:FinlandFinland
  • Participants:18–64 years · 65+ years
  • Substances:Bevacizumab
  • Sponsor:Merck Sharp & Dohme LLC

Vascular malformation

Recruiting
Registered drugNo placebo
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Enoxaparin Sodium
  • Sponsor:Fundacio Sant Joan De Deu

Stargardt's disease

Recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Substances:ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE 8 ENCODING THE ABCA4 PROTEIN, C-REGION
  • Sponsor:Splicebio S.L.

Hypochondroplasia

Recruiting
Registered drug
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Infigratinib
  • Sponsor:Qed Therapeutics Inc.

Phenylketonuria

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Sepiapterin
  • Sponsor:PTC Therapeutics Inc.

Sickle cell anaemia

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Iohexol
  • Sponsor:Centre Hospitalier Regional Universitaire De Tours

Hypertrophic cardiomyopathy

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Rivaroxaban
  • Sponsor:Centre Hospitalier Universitaire De Rennes

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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