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Congenital, familial and genetic disorders – page 38

  • 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

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

Hereditary angioedema

Not recruiting

Study on NTLA-2002 for Treating Hereditary Angioedema in Patients: Evaluating the Effects of Ziclumeran and Lonvoguran

Registered drug
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Lonvoguran
  • Sponsor:Intellia Therapeutics Inc.

Hereditary angioedema

Not recruiting

Study of Deucrictibant Extended-Release Tablets for Prevention of Hereditary Angioedema Attacks in Adults and Adolescents

Registered drug
Countries:BulgariaBulgaria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Deucrictibant
  • Sponsor:Pharvaris Netherlands B.V.

Hodgkin's disease refractory+1

Not recruiting

Study of Ruxolitinib with Brentuximab or Pembrolizumab for Patients with Relapsed or Refractory Classical Hodgkin Lymphoma

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:18–64 years · 65+ years
  • Substances:Brentuximab Vedotin
  • Sponsor:Universita Degli Studi Di Perugia

Arginase deficiency

Not recruiting

Title: Safety and effectiveness study of pegzilarginase weekly injections in children under 2 years old with Arginase 1 Deficiency

Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Pegzilarginase
  • Sponsor:Immedica Pharma AB

Prader-Willi syndrome

Not recruiting

Long-Term Study on Oxytocin for Children with Prader-Willi Syndrome Up to Age 4

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Oxytocin
  • Sponsor:Centre Hospitalier Universitaire De Toulouse

Mucopolysaccharidosis II

Not recruiting

Study on the Effects and Safety of JR-141 and Idursulfase for Patients with Hunter Syndrome

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Idursulfase
  • Sponsor:Jcr Pharmaceuticals Co. Ltd.

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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