Non-Malignant Marrow Disorder Treatment using Reduced Intensity Conditioning – Phase II

Study of Reduced-Intensity Conditioning In Patients With Non-Malignant Disorders Undergoing Umbilical Cord, Bone Marrow, or Peripheral Blood Stem Cell Transplantation

Protocol Description

The purpose of this study is to determine whether patients who receive a hematopoietic stem cell transplant (HSCT) as part of therapy for a non-malignant disorder of the bone marrow can benefit from reduced-intensity conditioning (RIC) rather than the standard myeloablative conditioning. As part of standard preparation for conditions treated with HSCT, chemotherapy is provided in advance to reduce the chance that the patient’s body will reject the donor stem cells. While the highest tolerable chemotherapy is administered for malignant (cancer) cases, researchers believe that for non-malignant cases, RIC will reduce harmful side effects and lead to improved quality of life following treatment.

Study Level: Study Level: Phase II

Eligibility Criteria

Subject to certain exclusion criteria, the study is accepting participants of both genders between the ages of 2 months to 35 years , who have a non-malignant disease that can be treated with a HSCT.
Males: 2 months to 35 years old
Females: 2 months to 35 years old

Requirements

Prior to HSCT, participants will be placed into one of three study groups based on their conditions. Patients with or without immune deficiency and with a normal or near normal lymphocyte count will receive standard myeloablative conditioning. Patients with moderate lymphopenia or without lymphopenia but a recent history of CMV, adenovirus, RSV, or Parainfluenza 3 infections will received a half dose of the chemotherapy provided in the first group. The third group will be comprised of patients with severe lymphopenia and will be provided a test dose of the chemotherapy combination.
Visits: Requires hospitalization for the transplant and chemotherapy that precedes transplant
Duration: Patients will be followed at 3-month intervals in the first year, and thereafter annually.

Status: Enrolling by Invitation

Primary Investigator

Paul Szabolcs, MD

Contact Information

For information about the study or enrollment, please contact:
Paul Szabolcs, MD
412-692-5427