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Congenital, familial and genetic disorders – page 37

  • 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

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)

MELAS syndrome

Not recruiting

Long-term safety study of zagociguat tablets in patients with MELAS syndrome (Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) who completed previous trial

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

Hereditary angioedema

Not recruiting

Study on the Effect and Safety of BW-20805 for Adults with Hereditary Angioedema

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:BW-20805
  • Sponsor:Shanghai Argo Biopharmaceutical Co. Ltd.

VEXAS syndrome

Not recruiting

Study of pacritinib effectiveness and safety compared to placebo in patients with VEXAS syndrome

Investigational
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Pacritinib
  • Sponsor:Sobi Inc.

Osteogenesis imperfecta

Not recruiting

Study on Treating Osteogenesis Imperfecta in Patients Using Teriparatide and Zoledronic Acid

Registered drugNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Calcium Carbonate
  • Sponsor:NHS Lothian

Fabry's disease

Not recruiting

Long-Term Safety Study of ST-920 Gene Therapy for Patients with Fabry Disease

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Isaralgagene Civaparvovec
  • Sponsor:PTC Therapeutics Inc.

Cortical dysplasia+1

Not recruiting

Study on the Safety and Effectiveness of Sirolimus for Treating Drug-Resistant Epilepsy in Children with Rare Brain Disorders Linked to mTOR Pathway Activation

Registered drugNo placebo
Countries:PolandPoland
  • Participants:0–17 years
  • Substances:Sirolimus
  • Sponsor:Instytut Pomnik Centrum Zdrowia Dziecka

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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