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Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:
NCT01043640
First received: January 5, 2010
Last updated: March 31, 2017
Last verified: March 2017
Results First Received: March 31, 2017  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: No masking;   Primary Purpose: Treatment
Conditions: Mucopolysaccharidosis
Hurler Syndrome
Hunter Syndrome
Maroteaux-Lamy Syndrome
Sly Syndrome
Alpha Mannosidosis
Fucosidosis
Aspartylglucosaminuria
Adrenoleukodystrophy (ALD)
Krabbe Disease
Metachromatic Leukodystrophy (MLD)
Sphingolipidoses
Peroxisomal Disorders
Interventions: Drug: Campath-1H
Drug: Cyclophosphamide
Drug: Busulfan
Procedure: Allogeneic stem cell transplantation
Drug: Cyclosporine A
Drug: Mycophenolate Mofetil

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Transplant Patients

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.


Participant Flow:   Overall Study
    Transplant Patients
STARTED   46 
COMPLETED   46 
NOT COMPLETED   0 



  Baseline Characteristics
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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

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.


Baseline Measures
   Transplant Patients 
Overall Participants Analyzed 
[Units: Participants]
 46 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      46 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      11  23.9% 
Male      35  76.1% 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Number of Patients With Donor Derived Engraftment   [ Time Frame: Day 100 Post Transplant ]

2.  Secondary:   Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD)   [ Time Frame: Day 100 Post Transplant ]

3.  Secondary:   Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD)   [ Time Frame: Day 100 Post Transplant ]

4.  Secondary:   Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD)   [ Time Frame: Day 100 Post Transplant ]

5.  Secondary:   Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD)   [ Time Frame: Day 100 Post Transplant ]

6.  Secondary:   Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD)   [ Time Frame: Day 100 Post Transplant ]

7.  Secondary:   Number of Patients Who Died Peri-Transplant   [ Time Frame: By Day 100 Post Transplant ]

8.  Secondary:   Donor Cell Chimerism Following Transplant   [ Time Frame: Day 28 ]

9.  Secondary:   Donor Cell Chimerism Following Transplant   [ Time Frame: Day 42 ]

10.  Secondary:   Donor Cell Chimerism Following Transplant   [ Time Frame: Day 100 ]

11.  Secondary:   Donor Cell Chimerism Following Transplant   [ Time Frame: 6 months ]

12.  Secondary:   Donor Cell Chimerism Following Transplant   [ Time Frame: One year ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr. Paul Orchard
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: NCT01043640     History of Changes
Other Study ID Numbers: 2009LS088
MT2009-19 ( Other Identifier: Blood and Marrow Transplantation Program )
Study First Received: January 5, 2010
Results First Received: March 31, 2017
Last Updated: March 31, 2017