Phase 2
Completed N=46
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Mucopolysaccharidosis · Hurler Syndrome · Hunter Syndrome · Maroteaux-Lamy Syndrome
Source: ClinicalTrials.gov NCT01043640 ↗
Enrolled (actual)
46
Serious AEs
17.4%
Results posted
May 2017
Primary outcomePrimary: Number of Patients With Donor Derived Engraftment — 42 Participants
Summary
Rationale: Chemotherapy administration before a donor stem cell transplant is necessary to stop the patient's immune system from rejecting the donor's stem cells. When healthy stem cells from a donor are infused into the patient, the donor white blood cells can provide the missing enzyme that causes the metabolic disease. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving a monoclonal antibody, alemtuzumab, before transplant and cyclosporine and mycophenolate mofetil before and after transplant may stop this from happening. This may be an effective treatment for inherited metabolic disorders.
Purpose: The design of this study is to achieve donor cell engraftment in patients with standard-risk inherited metabolic diseases with limited peri-transplant morbidity and mortality. This will be achieved through the administration of the chemotherapy regimen described. The intention is to follow transplanted patient for years after transplant monitoring them for complications of their disease and assisting families with a multifaceted interdisciplinary approach.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients With Donor Derived Engraftment |
42 | — |
| SECONDARY Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD) |
32 | — |
| SECONDARY Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD) |
2 | — |
| SECONDARY Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD) |
5 | — |
| SECONDARY Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD) |
2 | — |
| SECONDARY Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD) |
5 | — |
| SECONDARY Number of Patients Who Died Peri-Transplant |
2 | — |
| SECONDARY Donor Cell Chimerism Following Transplant |
99 | — |
Eligibility Criteria
Inclusion Criteria
- Must have diagnosis of one of the following: mucopolysaccharidosis disorder, glycoprotein metabolic disorder, sphingolipidoses or inherited leukodystrophy, peroxisomal disorder or other inherited diseases of metabolism
- Must have an acceptable graft source as defined by University of Minnesota criteria
- Adequate organ function
Exclusion Criteria
- Pregnant - menstruating females must have a negative serum pregnancy test within 14 days of treatment start
- Evidence of human immunodeficiency virus (HIV) infection or known HIV positive serology
Data sourced from ClinicalTrials.gov (NCT01043640). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.