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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

  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
Intent-to-Treat 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.

Participant Flow:   Overall Study
    Intent-to-Treat
STARTED   18 
COMPLETED   18 
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
Intent-to-Treat 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.

Baseline Measures
   Intent-to-Treat 
Overall Participants Analyzed 
[Units: Participants]
 18 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      18 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 4.7  (4.3) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      7  38.9% 
Male      11  61.1% 
Region of Enrollment 
[Units: Participants]
 
United States   18 


  Outcome Measures

1.  Primary:   Number of Patients Achieving Engraftment   [ Time Frame: Day 100 ]

2.  Secondary:   Overall Survival   [ Time Frame: Day 100, 1 Year, 3 Years ]


  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
Other secondary objectives outlined in study protocol were not analyzed; number of patients were too few to be relevant.


  More Information

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
e-mail: orcha001@umn.edu


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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