Phase 2
Completed N=41
Stem Cell Transplantation for Hurler
Mucopolysaccharidosis I · Mucopolysaccharidosis VI · Mannosidosis · Mucolipidosis Type II (I-cell Disease)
Source: ClinicalTrials.gov NCT00176917 ↗
Enrolled (actual)
41
Serious AEs
0.0%
Results posted
Sep 2009
Primary outcomePrimary: Mean Percentage of Donor Cells in Study Population (Chimerism). — 85.8; 73.2; 84.6; 81.1 Percentage
Summary
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for Hurler syndrome, Maroteaux Lamy syndrome, Mannosidosis, or I-cell disease.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Percentage of Donor Cells in Study Population (Chimerism). |
85.8; 73.2; 84.6; 81.1; 81.6; 91.5 | — |
| SECONDARY Number of Patients Surviving on Study |
37; 28; 27 | — |
| SECONDARY Number of Patients Who Failed Engraftment. |
1 | — |
| SECONDARY Number of Patients With Grade III-IV Acute Graft-versus-host Disease (aGVHD). |
2 | — |
Eligibility Criteria
Inclusion Criteria
- Patients with Mucopolysaccharidosis, type I (e.g., Hurler syndrome), Maroteaux-Lamy syndrome (MPS VI), Alpha Mannosidosis, or mucolipidosis type II (I-cell disease) who have an HLA-identical or mismatched (at 1 antigen) related marrow, PBSC, or cord blood donor.
- Patients with Mucopolysaccharidosis, type I, Maroteaux-Lamy syndrome (MPS VI), Alpha Mannosidosis, or mucolipidosis type II (I-cell disease) who have an HLA-identical or HLA-1 antigen mismatched unrelated marrow, PBSC, or HLA-0-2 antigen mismatched umbilical cord blood donor.
- Patients with MPS type I, Maroteaux Lamy Syndrome (MPS VI), or mucolipidosis type II (I-cell disease) will have a mental developmental index within two standard deviations of the normal mean, as best as can be determined using Bayley scales of infant development or other standardized testing, recognizing that these may be affected by speech and/or hearing impairment.
- Adequate organ function:
- Cardiac: ejection fraction >40%; no decompensated congestive heart failure or uncontrolled arrhythmia
- Renal: serum creatinine 50 kg may be at risk for having cell doses below the goal of ≥ 10 x 106 CD 34 cells/kg and therefore will not be eligible to receive unrelated PBSCs.
Data sourced from ClinicalTrials.gov (NCT00176917). 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.