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Congenital, familial and genetic disorders – page 37

  • 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

Cystic fibrosis lung

Not recruiting

Safety and effectiveness study of inhaled ARCT-032 (mRNA therapy) in adults with Cystic Fibrosis

InvestigationalNo placebo
Countries:GreeceGreece
  • Participants:18–64 years · 65+ years
  • Substances:MRNA encoding the human CFTR gene
  • Sponsor:Arcturus Therapeutics Europe B.V.

Congenital cystic kidney disease

Not recruiting

Study of ABBV-CLS-628 to evaluate safety and effectiveness in adults with Autosomal Dominant Polycystic Kidney Disease

Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Substances:ABBV-CLS-628
  • Sponsor:Calico Life Sciences LLC

Sickle cell disease

Not recruiting

Study on the Effects of Tebapivat and AG-946 in Patients with Sickle Cell Disease

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:AG-946 phosphate
  • Sponsor:Agios Pharmaceuticals Inc.

Activated PI3 kinase delta syndrome

Not recruiting

Study on the Effects of Leniolisib in Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · Healthy volunteers
  • Substances:Leniolisib Phosphate
  • Sponsor:Pharming Technologies B.V.

Congenital disorder of glycosylation

Not recruiting

Study on the Effects and Safety of GLM101 for Patients with PMM2-CDG

Registered drug
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:M1P dipotassium salt
  • Sponsor:Glycomine Inc.

Breast cancer+2

Not recruiting

Study on Denosumab to Prevent Breast Cancer in Women with BRCA1 Mutation

Registered drug
Countries:AustriaAustria
  • Participants:18–64 years · Healthy volunteers
  • Substances:Denosumab
  • Sponsor:ANZ Breast Cancer Trials Group Limited

Cardiac amyloidosis+2

Not recruiting

Study on How Tafamidis is Absorbed in the Blood of Healthy Adults with Transthyretin Amyloid Cardiomyopathy

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years · Healthy volunteers
  • Substances:Tafamidis
  • Sponsor:Pfizer Inc.

Spinal muscular atrophy

Not recruiting

Study on the Safety and Tolerability of BIIB115 for Children with Spinal Muscular Atrophy Previously Treated with Gene Therapy

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · Healthy volunteers
  • Substances:BIIB115
  • Sponsor:Biogen Idec Research Limited

Hereditary haemorrhagic telangiectasia

Not recruiting

Study on the Effectiveness of Nintedanib for Treating Nosebleeds in Patients with Hereditary Hemorrhagic Telangiectasia

Registered drug
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Nintedanib
  • Sponsor:Hospices Civils De Lyon

MELAS syndrome

Not recruiting

Study on the Safety and Effectiveness of Zagociguat for Treating MELAS in Patients

Investigational
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Zagociguat
  • Sponsor:Tisento Therapeutics Inc.

Severe myoclonic epilepsy of infancy

Not recruiting

Study on the Effects of Bexicaserin for Treating Seizures in Children and Adults with Dravet Syndrome

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Sponsor:Longboard Pharmaceuticals Inc.

Factor VIII deficiency

Not recruiting

Study on Long-Term Effects of Efanesoctocog Alfa in Preventing Joint Bleeds in Patients with Hemophilia A

Registered drugNo placebo
Countries:CroatiaCroatia
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Efanesoctocog Alfa
  • Sponsor:Swedish Orphan Biovitrum AB (publ)

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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