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Congenital, familial and genetic disorders – page 42

  • 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

Familial hypertriglyceridaemia+1

Not recruiting

Study on the Effects of ISIS 678354 in Patients with Familial Chylomicronemia Syndrome (FCS)

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Isis 678354 Sodium Salt
  • Sponsor:Ionis Pharmaceuticals Inc.

Sickle cell anaemia

Not recruiting

Study on Voxelotor for Reducing Hemolysis in Patients with Sickle Cell Disease

Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Voxelotor
  • Sponsor:Assistance Publique Hopitaux De Paris

Wolfram syndrome

Not recruiting

Study on Tirzepatide for Improving Insulin Production in Patients with Wolfram Syndrome Type 1

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Tirzepatide
  • Sponsor:Ospedale San Raffaele S.r.l.

Stargardt's disease

Not recruiting

Study on the Long-Term Safety of SAR422459 for Patients with Stargardt's Disease

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Lentiviral Vector Containing The Human Abca4 Gene
  • Sponsor:Sanofi-Aventis Recherche & Developpement

Hereditary neuropathic amyloidosis

Not recruiting

Study on NTLA-2001 for Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Ziclumeran
  • Sponsor:Intellia Therapeutics Inc.

DNA mismatch repair protein gene mutation+7

Not recruiting

Study on Atezolizumab for Patients with High-Risk Stage II or Stage III Colorectal Cancer Not Eligible for Oxaliplatin Chemotherapy

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Atezolizumab
  • Sponsor:AIO-Studien gGmbH

K-ras gene mutation+2

Not recruiting

Study Comparing Divarasib, Sotorasib, and Adagrasib for Patients with Advanced or Metastatic KRAS G12C-Positive Non-Small Cell Lung Cancer

Registered drugNo placebo
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Sponsor:F. Hoffmann-La Roche AG

Alport's syndrome

Not recruiting

Study on the Safety of Vonafexor for Patients with Alport Syndrome at Risk of Progression

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years
  • Substances:Vonafexor
  • Sponsor:ENYO Pharma

Pyruvate kinase deficiency anaemia

Not recruiting

Study on Gene Therapy for Pyruvate Kinase Deficiency Using Autologous CD34+ Cells in Adults and Children

Registered drugNo placebo
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Busulfan
  • Sponsor:Rocket Pharmaceuticals Inc.

Microvillous inclusion disease

Not recruiting

Study on the Safety and Effectiveness of Crofelemer for Children with Microvillus Inclusion Disease (MVID)

Investigational
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Crofelemer
  • Sponsor:Napo Pharmaceuticals Inc.

Lymphatic malformation

Not recruiting

Study on the Effects of Topical Sirolimus for Lingual Microcystic Lymphatic Malformations in Children and Adults

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Sirolimus
  • Sponsor:Centre Hospitalier Regional Universitaire De Tours

Hereditary angioedema with C1 esterase inhibitor deficiency

Not recruiting

Study on the Safety and Effects of Sebetralstat for Children Aged 2-11 with Hereditary Angioedema Type I or II

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Sebetralstat
  • Sponsor:Kalvista Pharmaceuticals Limited

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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