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Congenital, familial and genetic disorders – page 29

  • 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
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796 clinical trials

Hereditary spastic paraplegia

Not yet recruiting

Phase IIb Open‑Label Study of Intrathecal MELPIDA (AAV9/AP4M1) in Patients with Hereditary Spastic Paraplegia Type 50 (SPG50)

InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years
  • Sponsor:Region Hovedstaden

Osteogenesis imperfecta

Not yet recruiting

Phase 2 Study of AGA2115 in Adults with Osteogenesis Imperfecta (COL1A1/COL1A2 Variants) Evaluating Bone Density

Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Sponsor:Angitia Inc. Ltd.

Metachromatic leukodystrophy

Not yet recruiting

Long‑Term Monitoring of Malignancy Risk in Early‑Onset Metachromatic Leukodystrophy Patients Treated with atidarsagene autotemcel

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Atidarsagene Autotemcel
  • Sponsor:Orchard Therapeutics (Europe) Limited

Muscular dystrophy

Not yet recruiting

Long-Term Safety and Efficacy of Oral Ribitol in Participants with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I/R9)

InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years
  • Substances:Ribitol
  • Sponsor:ML Bio Solutions Inc.

Arteriovenous malformation

Not yet recruiting

Long‑Term Effects of Sirolimus on Volume and Color of Port‑Wine Stain in Patients with Vascular Malformation Undergoing Surgery and Laser Therapy

Registered drugNo placebo
Countries:NorwayNorway
  • Participants:0–17 years · 18–64 years
  • Substances:Sirolimus
  • Sponsor:Oslo University Hospital HF

Alpha-1 antitrypsin deficiency

Not yet recruiting

Safety and Efficacy of TSRA-196 (rnaivt9315 and rnacs24757) in Adults with Lung or Liver Disease due to Alpha‑1 Antitrypsin Deficiency

InvestigationalNo placebo
Countries:IrelandIreland
  • Participants:18–64 years · 65+ years

Heart disease congenital

Not yet recruiting

Safety and pharmacodynamics of cangrelor tetrasodium in paediatric patients with congenital heart disease undergoing percutaneous vascular procedures

Registered drugNo placebo
Countries:AustriaAustria
  • Participants:0–17 years
  • Substances:Cangrelor Tetrasodium
  • Sponsor:Chiesi Farmaceutici S.p.A.

Chronic granulomatous disease

Not yet recruiting

Safety and efficacy of autologous CD34+ cells transduced with a vector encoding human NCF1 in children and adults with p47‑deficient chronic granulomatous disease

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years

Cystic fibrosis

Not yet recruiting

Safety and Efficacy of VX-828, Deutivacaftor, and Tezacaftor in Adults with Cystic Fibrosis

Registered drug
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Sponsor:Vertex Pharmaceuticals Inc.

Sickle cell disease

Not yet recruiting

Methoxyflurane versus oral oxycodone hydrochloride and morphine sulfate for pain relief in sickle cell disease vaso‑occlusive crises

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

Duchenne muscular dystrophy

Not yet recruiting

Phase 3 Randomized Double‑Blind Placebo‑Controlled Study of Intravenous sgt-003 in Ambulatory Male Patients with Duchenne Muscular Dystrophy

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:SGT-003
  • Sponsor:Solid Biosciences Inc.

Type IIa hyperlipidaemia

Not yet recruiting

Atorvastatin, Placebo, and No Treatment in Adults with Familial Hypercholesterolaemia and Statin-Associated Muscle Symptoms

Registered drug
Countries:NorwayNorway
  • Participants:18–64 years · 65+ years
  • Substances:Atorvastatin
  • Sponsor:Oslo University Hospital HF

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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