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

NCT01626092 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2017-12-05

Study results available
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Summary

This study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) in patients with high-risk lysosomal and peroxisomal disorders using a novel conditioning regimen for hematopoietic cell transplantation (HCT). After a reduced-intensity conditioning regimen using volumetric-modulated arc therapy (VMAT)-delivered low-dose total body irradiation (TBI) with highly conformal marrow boosting, patients will be transplanted using either a related or unrelated allograft. The cell source may be marrow, peripheral blood or cord blood based on donor availability.

Conditions

  • Lysosomal Storage Disease
  • Peroxisomal Disorder

Interventions

DRUG

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.

DRUG

Clofarabine

A daily dose of 40 mg/m2 will be administered IV over 2 hours on days -9, -8, -7, -6, and -5.

DRUG

Melphalan

A single dose of 140 mg/m2 will be given IV on day -4 over 15 minutes.

RADIATION

Total Body Irradiation with Marrow Boosting

1. Dose to total body 200 cGy in single dose 2. Dose to bone marrow of the following bones 800 cGy in 5 divided daily fractions (160 cGy x 5 doses ). Treated with up to 3 abutting VMAT photon arcs from top of head to maximum inferior border.

BIOLOGICAL

Hematopoietic stem cell transplantation

Patients will be transplanted using either a related or unrelated allograft. The cell source may be marrow, peripheral blood or cord blood based on donor availability.

DRUG

Cyclosporine A

Patients will receive CsA therapy beginning on day -3. Dosing of CsA will be 2.5 mg/kg/dose IV; if the recipient body weight is \<40 kg, dosing will be 3 times daily, and if \> 40 kg, twice daily. An attempt will be made to maintain a trough cyclosporine level of 200 mg/L to 400 mg/L.

DRUG

Mycophenolate mofetil

Patients will receive mycophenolate mofetil (MMF) therapy beginning on day -3. Dosing of MMF will be 1 gram three times daily (total daily dose 3 grams/day) if the recipient is \>50 kg, or 15 mg/kg/dose three times daily if the recipient is ≤50 kg. The same dosage is used orally or intravenously.

Sponsors & Collaborators

  • Masonic Cancer Center, University of Minnesota

    lead OTHER

Principal Investigators

  • Weston Miller, MD · Masonic Cancer Center, University of Minnesota

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-07-11
Primary Completion
2013-11-30
Completion
2013-11-30

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01626092 on ClinicalTrials.gov