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Congenital, familial and genetic disorders – page 10

  • 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

Gastrooesophageal cancer+2

Recruiting

Study of Simvastatin with Nivolumab and Oxaliplatin for Patients with Advanced Stomach or Esophageal Cancer with ARID1A Mutation

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:18–64 years · 65+ years
  • Substances:Capecitabine
  • Sponsor:IRCCS Istituto Nazionale Tumori Fondazione Pascale

Myotonic dystrophy

Recruiting

Study on Long-Term Safety and Efficacy of VX-670 for Adults with Myotonic Dystrophy Type I

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Substances:VX-670
  • Sponsor:Vertex Pharmaceuticals Inc.

Sickle cell disease

Recruiting

Study on Rilzabrutinib for Patients Aged 10-65 with Sickle Cell Disease

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Rilzabrutinib
  • Sponsor:Sanofi-Aventis Recherche & Developpement

Sickle cell disease

Recruiting

Study on the Safety and Effects of BMS-986470 for Patients with Sickle Cell Disease

Investigational
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:BMS-986470
  • Sponsor:Bristol-Myers Squibb Services Unlimited Company

Lymphatic malformation+3

Recruiting

Study of RLY-2608 for Adults and Children with PIK3CA-Related Overgrowth and Malformations

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:RLY-2608
  • Sponsor:Relay Therapeutics Inc.

Primary hyperoxaluria

Recruiting

Study on the Safety and Effects of ABO-101 with mRNA-002 and gRNA-001 for Patients with Primary Hyperoxaluria Type 1

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:GRNA-001
  • Sponsor:Arbor Biotechnologies Inc.

Duchenne muscular dystrophy+1

Recruiting

Study of ENTR-601-45 Safety and Effectiveness in Patients with Duchenne Muscular Dystrophy Who Can Skip Exon 45

Registered drug
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Sodium Chloride
  • Sponsor:Entrada Therapeutics Inc.

Trisomy 21+1

Recruiting

Study of levetiracetam to prevent seizures in adults who have both Down syndrome and Alzheimer's disease

Registered drug
Countries:SpainSpain
  • Participants:18–64 years · 65+ years
  • Substances:Levetiracetam
  • Sponsor:Fundacio Institut De Recerca De L Hospital De La Santa Creu I Sant Pau

Von Willebrand's disease

Recruiting

Study on the Safety and Effectiveness of Emicizumab for Patients with TYPE 3 Von Willebrand Disease

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Efmoroctocog Alfa
  • Sponsor:F. Hoffmann-La Roche AG

Duchenne muscular dystrophy+1

Recruiting

Study of ENTR-601-44 in Children with Duchenne Muscular Dystrophy Who Are Eligible for Exon 44 Skipping Treatment

Registered drug
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:ENTR-601-44
  • Sponsor:Entrada Therapeutics Inc.

Alagille syndrome+1

Recruiting

Study on Long-Term Safety and Effects of Maralixibat for Patients with Alagille Syndrome

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Maralixibat
  • Sponsor:Mirum Pharmaceuticals Inc.

Heritable pulmonary arterial hypertension+1

Recruiting

Study on Sotatercept for Improving Heart Function in Patients with Pulmonary Arterial Hypertension

Registered drugNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years
  • Substances:Sotatercept
  • Sponsor:Amsterdam UMC Stichting

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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