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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 (12) clinical trials

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

The inherited metabolic disorders (IMD) are a heterogeneous group of genetic diseases, most of which involve a single gene mutation resulting in an enzyme defect. In the majority of cases, the enzyme defect leads to the accumulation of substrates that are toxic and/or interfere with normal cellular function. Often times, ...

Phase

Wild-type Reovirus in Combination With Carfilzomib and Dexamethasone in Treating Patients With Relapsed or Refractory Multiple Myeloma

PRIMARY OBJECTIVES: I. Determine safety and tolerability, and define the maximum tolerated dose of reolysin (wild-type reovirus), carfilzomib and dexamethasone in patients with relapsed multiple myeloma. II. Obtain evidence of reovirus entry into myeloma cells via localization of reoviral ribonucleic acid (RNA) in multiple myeloma (MM) cells (in situ hybridization ...

Phase

Umbilical Cord Blood Transplantation From Unrelated Donors

This study is a single-center treatment protocol with four possible preparative regimens, designed to validate the process of umbilical cord blood stem cell transplantation at our institution. Enrolled patients will receive chemotherapy +/-total body radiation as a pre-transplant conditioning regimen. Patients will then receive cord blood stem cells followed by ...

Phase

Efficacy Safety Study and Benefit of Alkali Therapy in Sickle Cell Disease

Primary Objective: To assess the effect of alkali administration on bicarbonate and potassium levels in patients with SCD and depressed serum bicarbonate levels. Secondary Objectives: To assess the effect of alkali administration on improvement in hemolysis and on sequelae of impaired kidney function, ie, LDH, Hgb, reticulocyte count, red cell ...

Phase

Safety and Tolerability of Intravenous LLP2A-Alendronate for Osteopenia Secondary to Glucocorticoids

The study is designed to evaluate safety and tolerability of LLP2A-Ale beginning with a single ascending dose (SAD) cohort followed by multiple ascending dose (MAD) cohorts with matching placebo in each cohort. The population in this study includes patients with low bone density (osteopenia with a T score -1.0) in ...

Phase

Safety Tolerability and PK of PXL770 in Healthy Male Subjects

PXL770 is a direct activator of 5' adenosine monophosphate-activated protein kinase (AMPK) being developed by Poxel S.A. for the treatment of type 2 diabetes mellitus (T2DM). In Part A of this study, we'll test the safety, tolerability and pharmacokinetics (PK) of repeated doses. In Part B, we'll co-administer PXL770 and ...

Phase

Treatment Development of Triheptanoin (G1D)

The trial will use an open-label, standard 3+3 phase i design for determining the MTD of orally-administered C7 in G1D. Triheptanoin: a triglyceride oil containing three odd-carbon chain-length fatty acids (i.e., a triglyceride of 7-carbon heptanoic acid). Triheptanoin will be taken 4 times per day (approximately every 6 hours) by ...

Phase

Dose-escalation Trial of the Safety Pharmacokinetics and Pharmacodynamics of Iron Isomaltoside (Monofer )

IDA is highly prevalent in subjects with cancer and gastrointestinal diseases such as inflammatory bowel diseases, menstruating or pregnant women, and subjects who have undergone bariatric procedure. IDA can have a substantial medical and quality of life (QoL) burden on the subjects, and treatment of these subjects includes controlling the ...

Phase

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

Role of CYP2B6 CYP3A4 and MDR1 in the Metabolic Clearance of Methadone

Methadone maintenance treatment (MMT) has been used to rehabilitate the opiate addict resulting in a higher quality of life for the patient as well as improving social and psychological functioning while reducing the overall cost to society. The maintenance dose of methadone is highly variable between patients, and drug-drug interactions ...

Phase