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Congenital, familial and genetic disorders – page 67

  • 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
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803 clinical trials

Cystic fibrosis

Not recruiting

Long-term safety and efficacy study of elexacaftor/tezacaftor/ivacaftor treatment in people with cystic fibrosis who have non-F508del genetic mutations

Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years
  • Substances:Elexacaftor
  • Sponsor:Vertex Pharmaceuticals Inc.

Cystic fibrosis

Not recruiting

Study on Long-term Safety and Efficacy of Elexacaftor, Tezacaftor, and Ivacaftor for Cystic Fibrosis in Patients Aged 12 Months and Older

Registered drugNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years
  • Substances:Elexacaftor
  • Sponsor:Vertex Pharmaceuticals Inc.

Primary hypercholesterolaemia

Not recruiting

Study on Managing High Cholesterol in Cardiovascular Disease Patients Using Inclisiran

Registered drugNo placebo
Countries:HungaryHungary
  • Participants:18–64 years · 65+ years
  • Substances:Inclisiran
  • Sponsor:Semmelweis University

Heritable pulmonary arterial hypertension+2

Not recruiting

Study on Dapagliflozin for Patients with Pulmonary Arterial Hypertension to Improve Exercise Capacity and Heart Function

Registered drug
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Dapagliflozin
  • Sponsor:Rigshospitalet

Ataxia telangiectasia

Not recruiting

Study on the Safety and Effects of EryDex with Dexamethasone Sodium Phosphate for Patients with Ataxia Telangiectasia

InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years · 18–64 years
  • Substances:Dexamethasone Sodium Phosphate
  • Sponsor:Quince Therapeutics S.p.A.

Breast cancer+5

Not recruiting

Study on the Impact of Fluoroestradiol F-18 PET on Treatment for Patients with ER+ HER2- Metastatic Breast Cancer After First-Line Hormone Therapy Relapse

Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Fluoroestradiol F-18
  • Sponsor:GE Healthcare Limited

Adrenoleukodystrophy

Not recruiting

Study on the Effects of Leriglitazone for Adult Men with Cerebral Adrenoleukodystrophy

Investigational
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Leriglitazone
  • Sponsor:Minoryx Therapeutics S.L.

Epilepsy with myoclonic-atonic seizures

Not recruiting

Long-Term Safety Study of Fenfluramine for Patients with Myoclonic Astatic Epilepsy (Doose Syndrome)

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Substances:Fenfluramine Hydrochloride
  • Sponsor:Universitaetsklinikum Schleswig-Holstein AöR

Pseudoxanthoma elasticum

Not recruiting

Study on Preventing Arterial Calcification in Young Patients with Pseudoxanthoma Elasticum Using Etidronate

InvestigationalNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years
  • Substances:Etidronate Disodium
  • Sponsor:Universitair Medisch Centrum Utrecht

Niemann-Pick disease

Not recruiting

Study on the Safety and Effectiveness of Hydroxypropylbetadex and Miglustat for Patients with Niemann-Pick Disease Type C1

Registered drug
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Hydroxypropylbetadex
  • Sponsor:Cyclo Therapeutics Inc.

Hypertrophic cardiomyopathy

Not recruiting

Study of empagliflozin to improve exercise capacity in patients with hypertrophic cardiomyopathy

Registered drug
Countries:PolandPoland
  • Participants:18–64 years · 65+ years
  • Substances:Empagliflozin
  • Sponsor:Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego Panstwowy Instytut Badawczy

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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