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Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

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ClinicalTrials.gov Identifier: NCT00668564
Recruitment Status : Terminated (Replaced by another study)
First Posted : April 29, 2008
Results First Posted : July 13, 2011
Last Update Posted : December 28, 2017
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Hurler's Syndrome
Maroteaux-Lamy Syndrome
Sly Syndrome
Alpha Mannosidosis
Fucosidosis
Aspartylglucosaminuria
Sphingolipidoses
Krabbe Disease
Wolman's Disease
Niemann-Pick Disease Type B
Niemann-Pick Disease, Type C
Interventions Procedure: Stem Cell Transplantation
Drug: Cyclophosphamide
Drug: Campath-1H
Drug: Busulfan
Enrollment 18
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Intent-to-Treat
Hide Arm/Group Description All patients treated with study regimen; Bone Marrow Transplant - cord blood transplant; Cyclophosphamide (50 mg/kg intravenous [IV] days 1-4 prior to transplant); Busulfan (if < or = 12 kg: 1.1 mg/kg or if > 12 kg: 0.8 mg/kg IV every 6 hours on days 6-9 before transplant) and Campath-1H (once per day 0.3 mg/kg IV on days 10-12 before transplant.
Period Title: Overall Study
Started 18
Completed 18
Not Completed 0
Arm/Group Title Intent-to-Treat
Hide Arm/Group Description All patients treated with study regimen; Bone Marrow Transplant - cord blood transplant; Cyclophosphamide (50 mg/kg intravenous [IV] days 1-4 prior to transplant); Busulfan (if < or = 12 kg: 1.1 mg/kg or if > 12 kg: 0.8 mg/kg IV every 6 hours on days 6-9 before transplant) and Campath-1H (once per day 0.3 mg/kg IV on days 10-12 before transplant.
Overall Number of Baseline Participants 18
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
<=18 years
18
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 18 participants
4.7  (4.3)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
Female
7
  38.9%
Male
11
  61.1%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 18 participants
18
1.Primary Outcome
Title Number of Patients Achieving Engraftment
Hide Description Rate of successful engraftment - patients who achieved and sustained donor engraftment; donor chimerism by day 100 of at least 90% after undergoing hematopoietic stem cell transplantation.
Time Frame Day 100
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intent-to-Treat
Hide Arm/Group Description:
All patients treated with study regimen; Bone Marrow Transplant - cord blood transplant; Cyclophosphamide (50 mg/kg intravenous [IV] days 1-4 prior to transplant); Busulfan (if < or = 12 kg: 1.1 mg/kg or if > 12 kg: 0.8 mg/kg IV every 6 hours on days 6-9 before transplant) and Campath-1H (once per day 0.3 mg/kg IV on days 10-12 before transplant.
Overall Number of Participants Analyzed 18
Measure Type: Number
Unit of Measure: Participants
14
2.Secondary Outcome
Title Overall Survival
Hide Description Number of patients alive at timepoints.
Time Frame Day 100, 1 Year, 3 Years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Year 3 survival endpoint was not done due to study being terminated prematurely.
Arm/Group Title Intent-to-Treat
Hide Arm/Group Description:
All patients treated with study regimen; Bone Marrow Transplant - cord blood transplant; Cyclophosphamide (50 mg/kg intravenous [IV] days 1-4 prior to transplant); Busulfan (if < or = 12 kg: 1.1 mg/kg or if > 12 kg: 0.8 mg/kg IV every 6 hours on days 6-9 before transplant) and Campath-1H (once per day 0.3 mg/kg IV on days 10-12 before transplant.
Overall Number of Participants Analyzed 18
Measure Type: Number
Unit of Measure: Participants
Day 100 14
1 Year 12
Time Frame Day 1 through Day 100 post-transplant.
Adverse Event Reporting Description Only Serious Adverse Events were collected for this study.
 
Arm/Group Title Intent-to-Treat
Hide Arm/Group Description All patients treated with study regimen; Bone Marrow Transplant - cord blood transplant; Cyclophosphamide (50 mg/kg intravenous [IV] days 1-4 prior to transplant); Busulfan (if < or = 12 kg: 1.1 mg/kg or if > 12 kg: 0.8 mg/kg IV every 6 hours on days 6-9 before transplant) and Campath-1H (once per day 0.3 mg/kg IV on days 10-12 before transplant.
All-Cause Mortality
Intent-to-Treat
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Intent-to-Treat
Affected / at Risk (%) # Events
Total   8/18 (44.44%)    
Blood and lymphatic system disorders   
Graft failure  1  2/18 (11.11%)  2
General disorders   
Death  1  6/18 (33.33%)  6
Auto recovery  1  2/18 (11.11%)  2
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Intent-to-Treat
Affected / at Risk (%) # Events
Total   0/18 (0.00%)    
Other secondary objectives outlined in study protocol were not analyzed; number of patients were too few to be relevant.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Paul Orchard, M.D.
Organization: Masonic Cancer Center, University of Minnesota
Phone: 612-626-2313
Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00668564     History of Changes
Other Study ID Numbers: MT2008-02
0801M25202 ( Other Identifier: IRB, University of Minnesota )
First Submitted: April 25, 2008
First Posted: April 29, 2008
Results First Submitted: June 14, 2011
Results First Posted: July 13, 2011
Last Update Posted: December 28, 2017