Clinical Trial: HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

Brief 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 Summary:

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.


Sponsor: Northwell Health

Current Primary Outcome: Assessment of Stem Cell Engraftment [ Time Frame: 1 year ]

Sustained stem cell engraftment of donor cells will be evaluated by chimerism (FISH fluorescence in situ hybridization OR VNTR (Variable Number of Tandem Repeats), based on recipient/donor gender, at 30 days, 100 days, 6 months and 1 year following the use of reduced intensity conditioning.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Assessment of treatment related mortality and morbidity [ Time Frame: 2 years ]
    Patients will be evaluated for incidence and severity of graft versus host disease, infection, and cardiopulmonary complications.
  • Event Free Survival [ Time Frame: 2 years ]
  • Immunological Reconstitution [ Time Frame: 2 years ]
    Measure the rate of T and B cell immune reconstitution by laboratory studies at 8 weeks, 6 months, one year and two years post transplant.


Original Secondary Outcome: Same as current

Information By: Northwell Health

Dates:
Date Received: April 24, 2015
Date Started: December 2008
Date Completion: January 2019
Last Updated: May 12, 2017
Last Verified: May 2017