NCT02435901

HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT) IN PATIENTS WITH HIGH RISK HEMOGLOBINOPATHIES LIKE SICKLE CELL DISEASE AND β-THALESSEMIA-MAJOR USING REDUCED INTENSITY CONDITIONING REGIMEN

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 29 (actual)
• Sponsor: Northwell Health · Academic / Other
• Sex: All
• Age: 1 Year – 21 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the use of reduced intensity conditioning regimen in patients with high risk hemoglobinopathy Sickle Cell and B-Thalassemia Major in combination with standard immunosuppressive medications, followed by a routine stem cell transplant in order to assess whether or not it is as effective as myeloablative high dose chemotherapy and transplant.

Detailed description

Standard myeloablative regimens are toxic to non-hematopoietic tissue and are associated with treatment related mortality and morbidity (TRM). Preparative regimens that are not myeloablative are associated with a greatly decreased incidence of TRM. In addition to providing a less toxic regimen, the reduced intensity chemotherapy preparative regimen also remains immunosuppressive enough to allow donor engraftment. Recent report of non-myeloablative regimens which resulted in engraftment of allogeneic stem cell in hematological malignancies raises the possibility that this conditioning regimen might be useful in achieving engraftment in non hematological disorder. In an effort to achieve stable engraftment with any suitable donor stem cell source and to minimize toxicity the investigators have developed a new reduced intensity conditioning regimen for high risk hemoglobinopathies with the main aim of significantly suppressing the recipient's immune system and facilitate engraftment. Non-myeloablative or reduced-intensity immunosuppressive preparative regimens have achieved a stable, mixed chimerism engraftment and successful allogeneic bone marrow transplants.

Conditions

• Sickle Cell Disease
• Beta Thalassemia-Major

Interventions

Type	Name	Description
DRUG	alemtuzumab (Campath IH)	Alemtuzumab (Campath IH) is given daily over first 4 days, Day -20 to Day -17
DRUG	Fludarabine	Fludarabine 35/m2 is given daily over 4 days on Day -7 to Day -4.
DRUG	Melphalan	Melphalan 70mg/m2 is given daily over 2 days on Day -3 to Day -2.
DRUG	Cyclosporine	Immunosuppressant to prevent graft vs host disease is given on Day -1 prior to stem cell infusion
DRUG	Mycophenolate mofetil	Immunosuppressant to prevent graft vs host disease is given on Day -1.
DRUG	Tacrolimus	Immunosuppressant to prevent graft vs host disease is given Day -1 prior to stem cell infusion
BIOLOGICAL	Hematopoietic Stem Cell Transplantation	Human Leukocyte Antigen (HLA) matched or mismatched; related or unrelated hematopoietic stem cells to be transplanted on Day 0.

Timeline

• Start date: 2008-12-01
• Primary completion: 2019-03-01
• Completion: 2019-12-01
• First posted: 2015-05-06
• Last updated: 2021-08-24
• Results posted: 2020-11-05

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study