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Metabolic Disorders Clinical Trials

A listing of Metabolic Disorders medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (4) clinical trials

Longitudinal Study of Urea Cycle Disorders

Urea cycle disorders are a group of rare genetic diseases that affect how protein is broken down in the body. UCDs are caused by a deficiency in one of six enzymes or two mitochondrial membrane transporters responsible for removing ammonia, a waste product of protein metabolism, from the bloodstream. Normally, ...

Phase

Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)

The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.

Phase

Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases

The prospective clinical phase II protocol MC-FludT.16/NM is to be conducted to verify safety and efficacy of Treosulfan-based conditioning compared to Busulfan-based conditioning in paediatric patients. Based on the given clinical experience with either Treosulfan-based or Busulfan-based conditioning in combination with Fludarabine no increased risk for graft failure is expected ...

Phase

An Extension Trial of Inclisiran Compared to Evolocumab in Participants With Cardiovascular Disease and High Cholesterol

ORION-3, an extension of study MDCO-PCS-15-01 (ORION-1) [NCT02597127], is a Phase II, placebo-controlled, double-blind, randomized trial to evaluate the efficacy, safety, and tolerability of inclisiran (ALN-PCSSC) injection(s). All participants who completed ORION-1 (at least up to and including Day 210) and meet the study criteria will be enrolled into this ...

Phase