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Congenital, familial and genetic disorders – page 9

  • 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

Factor IX deficiency

Recruiting
Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Etranacogene Dezaparvovec
  • Sponsor:CSL Behring LLC

Cystic fibrosis

Recruiting
Registered drugNo placebo
Countries:ItalyItaly
  • Participants:18–64 years
  • Substances:Tamoxifen
  • Sponsor:Azienda Ospedaliera Universitaria Integrata Verona

Turner's syndrome

Recruiting
Registered drugNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years · Healthy volunteers
  • Substances:Estradiol
  • Sponsor:Region Midtjylland

Trisomy 21+1

Recruiting
InvestigationalNo placebo
Countries:SpainSpain
  • Participants:18–64 years · 65+ years · Healthy volunteers
  • Substances:Izaflortaucipir (18F)
  • Sponsor:Fundacio Institut De Recerca De L'Hospital De La Santa Creu I Sant Pau

Huntington's disease

Recruiting
InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · Healthy volunteers
  • Substances:2-[2-[3-[4-(2-(18F)FLUORANYLETHOXY)PHENYL]-7-METHYL-4-OXOQUINAZOLIN-2-YL]ETHYL]-4-PROPAN-2-YLOXYISOINDOLE-1,3-DIONE
  • Sponsor:Assistance Publique Hopitaux De Paris

Congenital cystic kidney disease+1

Recruiting
InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years · Healthy volunteers
  • Substances:Pyruvic Acid
  • Sponsor:Aarhus University

Urethral valves

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Amoxicillin
  • Sponsor:Centre Hospitalier Universitaire De Bordeaux

Gastrooesophageal cancer+2

Recruiting
Registered drugNo placebo
Countries:ItalyItaly
  • Participants:18–64 years · 65+ years
  • Substances:Capecitabine
  • Sponsor:IRCCS Istituto Nazionale Tumori Fondazione Pascale

Myotonic dystrophy

Recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Substances:Vx-670
  • Sponsor:Vertex Pharmaceuticals Inc.

Sickle cell disease

Recruiting
Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Rilzabrutinib
  • Sponsor:Sanofi-Aventis Recherche & Developpement

Sickle cell disease

Recruiting
Investigational
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:BMS-986470
  • Sponsor:Bristol-Myers Squibb Services Unlimited Company

Lymphatic malformation+3

Recruiting
InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:RLY-2608
  • Sponsor:Relay Therapeutics 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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