Recruiting

Hematopoietic Stem Cell Transplant

Sponsor:

Masonic Cancer Center, University of Minnesota

Code:

NCT01666080

Conditions

Hematologic Disorders

Hemoglobinopathies

Immunodeficiencies

Eligibility Criteria

Sex: All

Age: 0 - 55

Healthy Volunteers: Not accepted

Interventions

Busulfan

Fludarabine

Total body irradiation

Stem cell transplant

Keppra

Study Details

Brief summary:

This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) in patients with non-malignant or malignant diseases. This regimen, consisting of busulfan, fludarabine, and low dose total body irradiation (TBI), is designed to promote engraftment in patients who failed to achieve an acceptable level of donor-derived engraftment following a previous allogeneic HCT.

Conditions

Hematologic Disorders

Hemoglobinopathies

Immunodeficiencies

Study ID

NCT01666080

Start date

Aug, 2012

Status verified date

Jul, 2026

Completion date

Jun, 2028

Anticipated

Primary completion date

Jun, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 55

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Diagnosis of any disease for which a second or greater hematopoietic stem cell transplant is needed due to insufficient donor chimerism following hematopoietic recovery after previous HSCT. Determination of "insufficiency of donor chimerism" will be made by the treating transplant physician. Occasionally donor derived engraftment may be present, but sustained aplasia or failed recovery of sufficient hematopoiesis requires administration of a second graft. This intervention may be used for both situations.
  • Donor Availability: Patients considered for transplantation must have a sufficient graft as based on current criteria of the University of Minnesota Blood and Marrow Transplantation Program

  • Transplantation using sufficiently matched related donors (such as matched siblings) or unrelated donors will be considered. Both granulocyte-colony stimulating factor (GCSF) stimulated peripheral blood grafts and bone marrow grafts will be considered, although bone marrow will be the priority.
  • Cord blood grafts, both related and unrelated, are also eligible. As this protocol will use a reduced intensity regimen, this protocol will use the current recommendations of the University of Minnesota for choosing cord blood grafts. If a single cord blood unit cell dose is insufficient, double cord transplantation should be considered if sufficiently matched cord blood units are available. The priority of choosing cord blood donors is based on the current institutional recommendations.
  • Exclusion of Metabolic Disorder or other Inherited Disorder Carrier Status from related donor and unrelated cord blood grafts as appropriate for primary disease.

At the discretion of the treating transplant physician, an allograft from the previous donor may be used, if available.

  • Age, Performance Status, Consent

  • Age: 0 to 55 years
  • Consent: voluntary written consent (adult or parental/guardian)

Exclusion Criteria:

  • Previous irradiation that precludes the safe administration of an additional dose of 200 cGy of total body irradiation (TBI). Radiation Oncology will evaluate all patients who have had previous radiation therapy or TBI for approval to receive an additional 200 cGy of TBI
  • Pregnant or breastfeeding
  • Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted
  • HIV positive
  • While it would be advantageous to begin therapy on this second transplant regimen > 6 months following a prior myeloablative regimen or >2 months after a reduced intensity regimen, it is recognized that there are circumstances where this may not be practical.

Study Design

Enrollment

30 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

other: Reduced Intensity Conditioning

Includes patients receiving a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using reduced intensity conditioning (RIC). Patients will receive busulfan, fludarabine, total body irradiation and stem cell transplant. Keppra will be given for seizure prophylaxis.

Interventions

Busulfan

0.4 mg/kg (0.5 mg/kg if <4 years of age) intravenously (IV) every 6 hours on Days -8 and -7.

Fludarabine

40 mg/m\^2 intravenously (IV) over 1 hour on days -6 through -2.

Total body irradiation

200 cGy on Day -1

Stem cell transplant

stem cell infusion on day 0

Keppra

Keppra will be given for seizure prophylaxis during busulfan administration as per the standard institutional protocol.

Primary outcome measure

  • Time to Engraftment [ Time Frame: Day 42 ]

Central Contacts and Locations

Central contacts

Troy Lund, M.D., Ph.D.

612-625-2508mill4991@umn.edu

Paul Orchard, M.D.

612-626-2313orcha001@umn.edu

Locations

Masonic Cancer Center, University of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55455

Contacts

Weston P. Miller, M.D.

612-626-2778mill4991@umn.eud

Principal Investigator:

Weston P. Miller, M.D.

More Information

Sponsor

Masonic Cancer Center, University of Minnesota

Last update posted

Jul 6, 2026

Last verified

Jul, 2026

Keywords

  • second stem cell transplant
  • donor hematopoietic engraftment
  • hematopoietic stem cell transplantation
  • inherited metabolic disorder

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Masonic Cancer Center, University of Minnesota on 2026-07-06.