Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

NCT01372228•Talaris Therapeutics Inc.

Summary

The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.

Detailed Description

The objective for the study is to establish chimerism following reduced intensity conditioning with no grade III/IV GVHD. The primary endpoint we will follow is production of the missing enzyme at ≥ 10% of the normal level at day 180 post-transplant in \> 90% of patients.

Eligibility

Age

All ages

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. 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.
•Inborn errors of metabolism / Inherited Metabolic Disorders (IMD) eligible for this study include the following:
•* Hurler Syndrome (MPS I)
•* Hurler-Scheie Syndrome with early neurologic involvement and/or sensitization to ERT
•* Hunter Syndrome (MPS II)
•* Sanfilippo Syndrome (MPS III)
•* Krabbe Disease (Globoid Leukodystrophy)
•* Metachromatic Leukodystrophy (MLD)
•* Adrenoleukodystrophy (ALD and AMN)
•* Sandhoff Disease
+14 more criteria

Exclusion Criteria

•1. Patients with uncontrolled seizures, apnea, evidence of recurrent or uncontrolled aspiration, or need for chronic mechanical ventilation.
•2. Patients with allogeneic stem cell transplant with cytoreductive therapy in the past 6 months.
•3. Subjects must not have had previous radiation therapy that would preclude total body irradiation (TBI) (as determined by radiation therapist)
•4. Uncontrolled infection or severe concomitant diseases, which in the judgment of the Principal Investigator, could not tolerate reduced intensity transplantation.
•5. Subjects with a positive human immunodeficiency virus (HIV) antibody test result
•6. Subjects who are pregnant, as indicated by a positive serum human chorionic gonadotropin (HCG) test
•7. Subjects whose only donor is pregnant at the time of intended transplant
•8. Subjects of childbearing potential who are not practicing adequate contraception as defined by the investigator at the site
•9. Jehovah's witnesses being unwilling to be transfused
•10. Patients that have any comorbid condition which, in the view of the Principal Investigators, renders the patient at too high a risk from treatment complications and regimen related morbidity/mortality.
+1 more criteria

Locations (1)

Duke University Medical Center

Durham, North Carolina, United States

Key Dates

Start Date

April 1, 2011

Primary Completion Date

April 1, 2016

Completion Date

April 1, 2016

Last Updated

April 12, 2023

Enrollment

ACTUAL participants

Conditions

Hurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)Sanfilippo Syndrome (MPS III)Krabbe Disease (Globoid Leukodystrophy)Metachromatic Leukodystrophy (MLD)Adrenoleukodystrophy (ALD and AMN)Sandhoff DiseaseTay Sachs DiseasePelizaeus Merzbacher (PMD)Niemann-Pick DiseaseAlpha-mannosidosis

Interventions

hematopoietic stem cell infusion

BIOLOGICAL

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

NCT00146770

Mucopolysaccharidosis IHurler's Syndrome+2 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: April 12, 2023Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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