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Trial record 7 of 11 for:    Mucolipidoses

Stem Cell Transplantation for Hurler

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ClinicalTrials.gov Identifier: NCT00176917
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : September 4, 2009
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 Mucopolysaccharidosis I
Mucopolysaccharidosis VI
Mannosidosis
Mucolipidosis Type II (I-cell Disease)
Interventions Procedure: Stem Cell Transplant
Drug: Busulfan, Cyclophosphamide, ATG
Enrollment 41

Recruitment Details  
Pre-assignment Details  
Arm/Group Title Transplant Patients
Hide Arm/Group Description Patients that received hematopoietic stem cell transplant.
Period Title: Overall Study
Started 41
Completed 41
Not Completed 0
Arm/Group Title Transplant Patients
Hide Arm/Group Description Patients that received hematopoietic stem cell transplant.
Overall Number of Baseline Participants 41
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 41 participants
<=18 years
41
 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 41 participants
1.8  (1.4)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 41 participants
Female
23
  56.1%
Male
18
  43.9%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 41 participants
41
1.Primary Outcome
Title Mean Percentage of Donor Cells in Study Population (Chimerism).
Hide Description Donor-derived engraftment determined by restriction fragment length polymorphism (RFLP).
Time Frame at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Day 21 (24 patients included), Day 42 (15 pts), Day 60 (29 pts), Day 100 (25 pts), 6 Months (18 pts), 1 Year (16 pts).
Arm/Group Title Transplant Patients
Hide Arm/Group Description:
Patients that received hematopoietic stem cell transplant.
Overall Number of Participants Analyzed 41
Mean (Standard Deviation)
Unit of Measure: Percentage
21 Days Post Transplant 85.8  (28.4)
42 Days Post Transplant 73.2  (31.6)
60 Days Post Transplant 84.6  (27.9)
100 Days Post Transplant 81.1  (26.8)
6 Months Post Transplant 81.6  (29.6)
1 Year Post Transplant 91.5  (18.8)
2.Secondary Outcome
Title Number of Patients Surviving on Study
Hide Description Number of patients surviving (alive) at specified timepoints.
Time Frame at 100 days, 1 year, and 3 years post transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Day 100 and 1 Year timepoints include all 41 patients. Year 3 includes 36 patients (5 pts not yet at followup timepoint.)
Arm/Group Title Transplant Patients
Hide Arm/Group Description:
Patients that received hematopoietic stem cell transplant.
Overall Number of Participants Analyzed 41
Measure Type: Number
Unit of Measure: Participants
Day 100 Post Transplant 37
1 Year Post Transplant 28
3 Years Post Transplant 27
3.Secondary Outcome
Title Number of Patients Who Failed Engraftment.
Hide Description Toxicity (undesireable effect) of hematologic donor cell engraftment is determined by failure to engraft at Day 42.
Time Frame Day 42 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
1 patient of 41 failed engraftment - per protocol.
Arm/Group Title Transplant Patients
Hide Arm/Group Description:
Patients that received hematopoietic stem cell transplant.
Overall Number of Participants Analyzed 41
Measure Type: Number
Unit of Measure: Participants
1
4.Secondary Outcome
Title Number of Patients With Grade III-IV Acute Graft-versus-host Disease (aGVHD).
Hide Description Toxicity (undesireable effect) of this stem cell transplant preparative regimen due to acute graft-versus-host disease.
Time Frame Day 100 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:
Patients that received hematopoietic stem cell transplant.
Overall Number of Participants Analyzed 41
Measure Type: Number
Unit of Measure: Participants
2
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Transplant Patients
Hide Arm/Group Description Patients that received hematopoietic stem cell transplant.
All-Cause Mortality
Transplant Patients
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Transplant Patients
Affected / at Risk (%)
Total   0/41 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Transplant Patients
Affected / at Risk (%)
Total   0/41 (0.00%) 

Based on variability in age, diagnosis and condition of these patients, the data on enzyme levels and neuropsych testing is extremely difficult to report.

See Secondary Outcome Measures #3 and #4; no other adverse events were collected.

Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Paul J. 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: NCT00176917     History of Changes
Obsolete Identifiers: NCT00005899
Other Study ID Numbers: UMN-MT1999-07
0104M93821 ( Other Identifier: IRB, University of Minnesota )
First Submitted: September 12, 2005
First Posted: September 15, 2005
Results First Submitted: July 28, 2009
Results First Posted: September 4, 2009
Last Update Posted: December 28, 2017