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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 (44) clinical trials

THRIVE is an observational study that will collect information on patients with UCDs. THRIVE will follow enrolled participants for up to 10 years. As an observational study, enrolled patients will not be required to make any additional office visits or take any medicine outside of normal care.

Phase N/A

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

Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale Canada

Familial hypercholesterolemia (FH) is a genetic lipoprotein disorder characterized by elevations in low-density lipoprotein cholesterol (LDL-C) >95th percentile for age and gender. Affected individuals may show clinical manifestations (xanthomas, xanthelasmas and premature arcus corneus) although these manifestations are seen less frequently with early diagnosis and treatment. Untreated, FH will lead ...

Phase N/A

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

This is a single arm dose finding study evaluating the safety and efficacy of a BPX 501 infusion (T cells genetically modified with the inducible Caspase 9 suicide gene) of 3x10E6 to 1X10E7 cells/kg followed by an AP1903 infusion on day 7 after a partially mismatched, related, T cell-depleted hematopoietic ...

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

Biomarker for Hypophosphatasia Disease

Hypophosphatasia (HPP) is a rare genetic disorder characterized the abnormal devel-opment of bones and teeth. These abnormalities occur due to defective mineralization, the process by which bones and teeth take up minerals such as calcium and phosphorus. These minerals are required for proper hardness and strength. Defective mineralization results in ...

Phase N/A

Effect of Liraglutide on Cardiovascular Endpoints in Diabetes Mellitus Type 2 Patients of South Asian Descent

RECRUITMENT AND SCREENING PROCEDURE OF STUDY POPULATION Patients will be recruited from the outpatient clinics of the Leiden University Medical Center, general practitioners, local hospitals and by advertisement. Patients own physicists will be asked to point eligible patients to the opportunity of study participation. If interested, patients will be informed ...

Phase

Biomarker for Patients With Transthyretin-Related Familial Amyloidotic Polyneuropathy

Diseases of diverse etiology can be correlated to the term "polyneuropathy"(PNP). The pathogenesis may be of inflammatory, autoimmune, metabolic, toxic or hereditary nature. Careful clinical and electrodiagnostic assessment, with attention to the pattern of involvement and the types of nerve fibers most affected, narrows the differential diagnosis and helps to ...

Phase N/A

Efficacy of a Probiotic Product on Bone Mineral Density (BMD) in Healthy Women in Early Post-menopausal Phase

The aim of the current study is to evaluate the efficacy of a probiotic product on bone mineral density (BMD) in healthy early post-menopausal women, compared to placebo. The primary endpoint is to assess changes of BMD at lumbar spine after an intervention period of 12 months. Changes in BMD ...

Phase

Study of Treatment and Metabolism in Patients With Urea Cycle Disorders

PROTOCOL OUTLINE: This protocol involves 2 clinical studies. Part A is a metabolic study of glutamine conversion to urea at different levels of protein intake, while on and off medications. Part B is a dose escalation study of a first-generation adenoviral vector with an E1 deletion and an E3 deletion ...

Phase