Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

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

Status

Terminated

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02179359

Enrollment

Registered

2014-07-01

Start date

2014-09-02

Completion date

2024-11-21

Last updated

2025-03-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Keywords

Stem Cell Transplant

Brief summary

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

Interventions

DRUGReduced Toxicity Ablative Regimen

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

DRUGReduced Intensity Preparative Regimen

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

DRUGMyeloablative Preparative Regimen

* Alemtuzumab * Cyclophosphamide * Busulfan * Stem Cell Infusion Day 0

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 55 Years

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

Design outcomes

Primary

Measure	Time frame
incidence of graft failure	42 days

Secondary

Measure	Time frame	Description
overall survival	6 months, 1 and 2 years	—
disease free survival	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)

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026