CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 41 enrolled
Drug / intervention
Busulfan, Cyclophosphamide, ATG +1 moredrug
Likely dose
Busulfan IV twice daily for 4 days, cyclophosphamide IV once daily for 4 days, anti-thymocyte globulin IV twice daily for 3 days (prior to transplantation)AI-extracted
Key inclusion· 7
  • ✓Confirmed diagnosis of Mucopolysaccharidosis type I (Hurler syndrome), MPS VI (Maroteaux-Lamy syndrome), Alpha Mannosidosis, or mucolipidosis type II (I-cell disease)
  • ✓HLA-identical or mismatched (at 1 antigen) related donor (bone marrow, PBSC, or cord blood)
  • ✓HLA-identical or HLA-1 antigen mismatched unrelated bone marrow or PBSC donor, or HLA-0-2 antigen mismatched umbilical cord blood donor
  • ✓For MPS I, MPS VI, or mucolipidosis II: mental developmental index within two standard deviations of normal mean on Bayley scales or equivalent standardized testing
Key exclusion· 5
  • ✕Presence of major organ dysfunction
  • ✕Pregnancy
  • ✕Evidence of HIV infection or known HIV positive serology
  • ✕Patient or parent psychologically incapable of undergoing BMT with associated strict isolation or documented history of medical non-compliance

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00176917
NCT00176917Phase 2Completed
CompareCT.gov

Hematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis)

Masonic Cancer Center, University of Minnesota·interventional·Posted Sep 15, 2005·Updated Dec 28, 2017

In Brief

A Phase 2 clinical trial evaluating Stem Cell Transplant and Busulfan, Cyclophosphamide, ATG for Mucopolysaccharidosis I and 3 related conditions. Completed, enrolled 41 participants across 1 site.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)
CountriesUnited States
Collaborators--

Timeline

Phase 2CompletedFinished
19992000200120022003200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedSep 15, 2005
Enrollment StartMay 1, 1999
Primary CompletionMay 1, 2008
Study CompletionMay 1, 2010
TodayJul 2, 2026
Enrollment to primary: 9 yearsPosted 20.8 years ago

Interventions

Stem Cell Transplantprocedure

The purpose of hematopoietic cell transplantation is to introduce hematopoietic cells from a normal donor that contains the enzyme able to get rid of the substances that have accumulated in the body of patients with storage diseases. Hematopoietic cells can come from bone marrow, peripheral blood (i.e., the blood circulating in our body's blood vessels) or umbilical cord blood (i.e. blood taken from the umbilical cord after a baby is born and umbilical cord is cut).

Busulfan, Cyclophosphamide, ATGdrug

Prior to transplantation, subjects will receive BUSULFAN intravenously (IV) via the Hickman line twice daily for 4 days, CYCLOPHOSPHAMIDE intravenously via the Hickman line once a day for 4 days, and ANTI-THYMOCYTE GLOBULIN IV via the Hickman line twice daily for three days before the transplant. These three drugs are being given to help the new marrow "take" and grow. METHYLPREDNISOLONE will be given as a pre-medication for the ATG.