Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02179359
• Other study ID #: 2014OC034
• Secondary ID: MT2014-10C
• Status: Recruiting
• Phase: N/A
• Start date: September 2, 2014
• Est. completion date: August 2025

Study information

• Verified date: February 2024
• Source: Masonic Cancer Center, University of Minnesota
• Contact: Lisa Burke
• Phone: 612-273-8482
• Email: lburke3[@]Fairview.org
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 25
• Est. completion date: August 2025
• Est. primary completion date: January 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 55 Years

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

Drug:
• Reduced Toxicity Ablative Regimen
Anti-thymocyte Globulin (ATG) Fludarabine Busulfan Stem Cell Infusion Day 0
• Reduced Intensity Preparative Regimen
Alemtuzumab Cyclophosphamide Fludarabine Total Body Irradiation (TBI) Stem Cell Infusion Day 0
• Myeloablative Preparative Regimen
Alemtuzumab Cyclophosphamide Busulfan Stem Cell Infusion Day 0

Locations

Country	Name	City	State
United States	University of Minnesota Medical Center, Fairview	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Masonic Cancer Center, University of Minnesota

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	incidence of graft failure		42 days
Secondary	overall survival		6 months, 1 and 2 years
Secondary	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)	6 months, 1 and 2 years