Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

February 5, 2024 updated by: Masonic Cancer Center, University of Minnesota

MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders

This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

25

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • University of Minnesota Medical Center, Fairview
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 53 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated
  • Acceptable stem cell source identified
  • Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score)
  • Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
  • Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase <5 times the upper limit of institutional normal
  • Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%

Exclusion Criteria:

  • active, uncontrolled infection
  • pregnant or breastfeeding
  • HIV positive

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Reduced Toxicity Ablative Regimen
For use in patients with a matched sibling donor or unrelated UCB donor and DBA patients who are <12 years and/or have mild/moderate iron exposure.
Drug: Reduced Toxicity Ablative Regimen
  • Anti-thymocyte Globulin (ATG)
  • Fludarabine
  • Busulfan
  • Stem Cell Infusion Day 0
Experimental: Reduced Intensity Preparative Regimen
For use in patients with unrelated donor bone marrow and for DBA patients who are >12 years and/or have significant iron exposure.
Drug: Reduced Intensity Preparative Regimen
  • Alemtuzumab
  • Cyclophosphamide
  • Fludarabine
  • Total Body Irradiation (TBI)
  • Stem Cell Infusion Day 0
Experimental: Myeloablative Preparative Regimen
For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia.
Drug: Myeloablative Preparative Regimen
  • Alemtuzumab
  • Cyclophosphamide
  • Busulfan
  • Stem Cell Infusion Day 0

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
incidence of graft failure
Time Frame: 42 days
42 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
overall survival
Time Frame: 6 months, 1 and 2 years
6 months, 1 and 2 years
disease free survival
Time Frame: 6 months, 1 and 2 years
patient no longer needing red blood cell transfusion and/or a hemoglobin S level at that of the donor ( sickle cell disease only)
6 months, 1 and 2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Masonic Cancer Center, University of Minnesota

Investigators

  • Principal Investigator: Ashish Gupta, MBBS, MPH, Masonic Cancer Center, University of Minnesota

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 2, 2014

Primary Completion (Estimated)

January 1, 2025

Study Completion (Estimated)

August 1, 2025

Study Registration Dates

First Submitted

June 27, 2014

First Submitted That Met QC Criteria

June 27, 2014

First Posted (Estimated)

July 1, 2014

Study Record Updates

Last Update Posted (Estimated)

February 6, 2024

Last Update Submitted That Met QC Criteria

February 5, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2014OC034
  • MT2014-10C (Other Identifier: Masonic Cancer Center, University of Minnesota)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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