Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders (NCT01372228) | Clinical Trial Compass
TerminatedPhase 1/2
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
Stopped: company did not pursue this indication
United States3 participantsStarted 2011-04
Plain-language summary
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
Who can participate
SexALL
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Inclusion criteria
✓. Patients must have a confirmed diagnosis of inherited metabolic disorder / inborn error of metabolism. Diagnosis should be confirmed by appropriate test(s) (enzyme and/or mutation analysis) before study entry. Patients must not be eligible for myeloablative chemotherapy as a preparative regimen for transplant due to age, co-morbidities or organ dysfunction.
✓. Patients must have adequate function of other organ systems as measured by:
✓. Patient must have a related donor (identical or mismatched for 1, 2 or 3 Human Leukocyte Antigen (HLA)-A, -B or -DR loci).
✓. Patient, and parent, or legal guardian must have given written informed consent according to FDA guidelines.
✓. Patients must have a minimum life expectancy of at least 6 months.
✓. Female patients of childbearing potential cannot be pregnant or lactating/breast-feeding and must be either surgically sterile, postmenopausal (no menses for the previous 12 months), or must be practicing an effective method of birth control as determined by the investigator (e.g., oral contraceptives, double barrier methods, hormonal injectable or implanted contraceptives, tubal ligation, or partner with vasectomy).
✓. There is no upper or lower age limit for this study.
Exclusion criteria
✕. Patients with uncontrolled seizures, apnea, evidence of recurrent or uncontrolled aspiration, or need for chronic mechanical ventilation.
✕. Patients with allogeneic stem cell transplant with cytoreductive therapy in the past 6 months.
What they're measuring
1
Production of missing enzyme at levels greater than or equal to 10% of normal
✕. Subjects must not have had previous radiation therapy that would preclude total body irradiation (TBI) (as determined by radiation therapist)
✕. Uncontrolled infection or severe concomitant diseases, which in the judgment of the Principal Investigator, could not tolerate reduced intensity transplantation.
✕. Subjects with a positive human immunodeficiency virus (HIV) antibody test result
✕. Subjects who are pregnant, as indicated by a positive serum human chorionic gonadotropin (HCG) test
✕. Subjects whose only donor is pregnant at the time of intended transplant
✕. Subjects of childbearing potential who are not practicing adequate contraception as defined by the investigator at the site