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Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Primary Purpose

Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia

Status

Recruiting

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

Reduced Toxicity Ablative Regimen

Reduced Intensity Preparative Regimen

Myeloablative Preparative Regimen

About this trial

This is an interventional treatment trial for Sickle Cell Disease focused on measuring Stem Cell Transplant

Eligibility Criteria

undefined - 55 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

University of Minnesota Medical Center, FairviewRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

Reduced Toxicity Ablative Regimen

Reduced Intensity Preparative Regimen

Myeloablative Preparative Regimen

Arm Description

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.

For use in patients with unrelated donor bone marrow and for DBA patients who are >12 years and/or have significant iron exposure.

For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia.

Outcomes

Primary Outcome Measures

incidence of graft failure

Secondary Outcome Measures

overall survival

disease free survival

patient no longer needing red blood cell transfusion and/or a hemoglobin S level at that of the donor ( sickle cell disease only)

Full Information

NCT ID

NCT02179359

First Posted

June 27, 2014

Last Updated

April 14, 2023

Sponsor

Masonic Cancer Center, University of Minnesota

1. Study Identification

Unique Protocol Identification Number

NCT02179359

Brief Title

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Recruiting

Study Start Date

September 2, 2014 (Actual)

Primary Completion Date

January 2024 (Anticipated)

Study Completion Date

August 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Masonic Cancer Center, University of Minnesota

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders

Keywords

Stem Cell Transplant

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

25 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Reduced Toxicity Ablative Regimen

Arm Type

Experimental

Arm Description

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.

Arm Title

Reduced Intensity Preparative Regimen

Arm Type

Experimental

Arm Description

For use in patients with unrelated donor bone marrow and for DBA patients who are >12 years and/or have significant iron exposure.

Arm Title

Myeloablative Preparative Regimen

Arm Type

Experimental

Arm Description

For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia.

Intervention Type

Drug

Intervention Name(s)

Reduced Toxicity Ablative Regimen

Intervention Description

Anti-thymocyte Globulin (ATG) Fludarabine Busulfan Stem Cell Infusion Day 0

Intervention Type

Drug

Intervention Name(s)

Reduced Intensity Preparative Regimen

Intervention Description

Alemtuzumab Cyclophosphamide Fludarabine Total Body Irradiation (TBI) Stem Cell Infusion Day 0

Intervention Type

Drug

Intervention Name(s)

Myeloablative Preparative Regimen

Intervention Description

Alemtuzumab Cyclophosphamide Busulfan Stem Cell Infusion Day 0

Primary Outcome Measure Information:

Title

incidence of graft failure

Time Frame

42 days

Secondary Outcome Measure Information:

Title

overall survival

Time Frame

6 months, 1 and 2 years

Title

disease free survival

Description

patient no longer needing red blood cell transfusion and/or a hemoglobin S level at that of the donor ( sickle cell disease only)

Time Frame

6 months, 1 and 2 years

10. Eligibility

Sex

All

Maximum Age & Unit of Time

55 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Lisa Burke

Phone

612-273-8482

lburke3@Fairview.org

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Ashish Gupta, MBBS, MPH

Organizational Affiliation

Masonic Cancer Center, University of Minnesota

Official's Role

Principal Investigator

Facility Information:

Facility Name

University of Minnesota Medical Center, Fairview

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55455

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Lisa Burke

Phone

612-273-8482

lburke3@Fairview.org

12. IPD Sharing Statement

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Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

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