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Trial record 7 of 10 for:    Mucolipidoses

Stem Cell Transplantation for Hurler

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
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

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Transplant Patients Patients that received hematopoietic stem cell transplant.

Participant Flow:   Overall Study
    Transplant Patients
STARTED   41 
COMPLETED   41 
NOT COMPLETED   0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Transplant Patients Patients that received hematopoietic stem cell transplant.

Baseline Measures
   Transplant Patients 
Overall Participants Analyzed 
[Units: Participants]
 41 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      41 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 1.8  (1.4) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      23  56.1% 
Male      18  43.9% 
Region of Enrollment 
[Units: Participants]
 
United States   41 


  Outcome Measures

1.  Primary:   Mean Percentage of Donor Cells in Study Population (Chimerism).   [ Time Frame: at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year ]

2.  Secondary:   Number of Patients Surviving on Study   [ Time Frame: at 100 days, 1 year, and 3 years post transplant ]

3.  Secondary:   Number of Patients Who Failed Engraftment.   [ Time Frame: Day 42 Post Transplant ]

4.  Secondary:   Number of Patients With Grade III-IV Acute Graft-versus-host Disease (aGVHD).   [ Time Frame: Day 100 Post Transplant ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data

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.



  More Information

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
e-mail: orcha001@umn.edu



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