Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

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. Identifier: NCT01626092
Recruitment Status : Completed
First Posted : June 22, 2012
Results First Posted : October 11, 2016
Last Update Posted : December 5, 2017
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: Lysosomal Storage Disease
Peroxisomal Disorder
Interventions: Drug: Campath-1H
Drug: Clofarabine
Drug: Melphalan
Radiation: Total Body Irradiation with Marrow Boosting
Biological: Hematopoietic stem cell transplantation
Drug: Cyclosporine A
Drug: Mycophenolate mofetil

  Participant Flow

  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.
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Reporting Groups
Intent-To-Treat Patients

Patients with high-risk lysosomal and peroxisomal disorders treated with preparative regimen (Campath-1H 0.3 mg/kg intravenous (IV) on days -12 through -8, clofarabine 40 mg/m^2 IV on days -9 through -5, melphalan 140 mg/m^2 IV on day -4 and Total Body Irradiation with Marrow Boosting [ first dose of 200 cGy single dose; 5 doses of 160cGy for marrow boosting - 1000cGy cumulative exposure] by Volumetric-Modulated Arc Therapy [VMAT] on days -3 through -1). Hematopoietic stem cell transplantation will be infused on Day 0. Post-transplant immunosuppression to follow: Mycophenolate mofetil (MMF) begin day -3 and continue to day +30 or 7 days after engraftment, whichever is later; Cyclosporine A (CsA) begin day -3 and then taper at day +100 if related donor, day +180 for unrelated donor.

Campath-1H: A daily dose of 0.3 mg/kg IV over 2 hours will be administered on days - 12, -11, -10, -9, and -8

Clofarabine: A daily dose of 40 mg/m2 will be administered IV over 2 hours on days -9

Baseline Measures
   Intent-To-Treat Patients 
Overall Participants Analyzed 
[Units: Participants]
[Units: Participants]
Count of Participants
<=18 years      3 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
Female      0   0.0% 
Male      3 100.0% 
Region of Enrollment 
[Units: Participants]
United States   3 

  Outcome Measures

1.  Primary:   Donor (Allogeneic) Hematopoietic Engraftment   [ Time Frame: Day 100 Following Hematopoietic Cell Transplant (HCT) ]

2.  Secondary:   Transplant-Related Mortality   [ Time Frame: Day 100 following HCT ]

3.  Secondary:   Neurologic Outcomes   [ Time Frame: Changes from Baseline, Days 30, 60, 100, Year 1, Year 2, Year 3 Following HCT ]

  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
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  More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact:  
Name/Title: Weston P Miller, MD
Organization: Masonic Cancer Center, University of Minnesota
phone: 612-626-2778

Responsible Party: Masonic Cancer Center, University of Minnesota Identifier: NCT01626092     History of Changes
Other Study ID Numbers: 2011LS147
MT2011-24 ( Other Identifier: Blood and Marrow Transplantation Program )
First Submitted: June 20, 2012
First Posted: June 22, 2012
Results First Submitted: August 17, 2016
Results First Posted: October 11, 2016
Last Update Posted: December 5, 2017