Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

This study has been completed.
Sponsor:
Collaborators:
Beth Israel Deaconess Medical Center
Massachusetts General Hospital
Brigham and Women's Hospital
Emory University
Feist-Weiller Cancer Center at Louisiana State University Health Sciences
Ohio State University
Information provided by (Responsible Party):
Catherine Wu, MD, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier:
NCT00153985
First received: September 8, 2005
Last updated: July 24, 2013
Last verified: July 2013

September 8, 2005
July 24, 2013
March 2004
March 2008   (final data collection date for primary outcome measure)
Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy. [ Time Frame: 3 years ] [ Designated as safety issue: No ]
Outcome was measured by ANC >500 for three consecutive days prior to day 30 after PBSC infusion, >25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods prior to day 45 after PBSC infusion and >25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods after day 180 after PBSC infusion.
To determine if the preparative regimen of busulfex, fludarabine and alemtuzumab (CAMPATH) will generate stable engraftment with donor stem cells in patients with severe hemoglobinopathy.
Complete list of historical versions of study NCT00153985 on ClinicalTrials.gov Archive Site
  • Solid Organ Toxicity Related to the Conditioning Regimen. [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]
    Outcome was measured by the assessment of organ toxicity related to Busulfex, fludarabine and alemtuzumab.
  • The Incidence of Grade II-IV Acute Graft vs. Host Disease. [ Time Frame: 3 years ] [ Designated as safety issue: No ]
    Outcome was measured by incidence and severity of acute and chronic GVHD following donor stem cell infusion.
  • To assess the safety of busulfex, fludarabine and alemtuzumab
  • to describe the incidence and severity of acute or chronic graft vs. host disease.
Not Provided
Not Provided
 
Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies
Multi-Center Study Using Allogeneic Stem Cell Transplantation Following Reduced Intensity Chemotherapy in Patients With Hemoglobinopathies

The purpose of this study is to determine if treatment with reduced-dose busulfex, fludarabine and alemtuzumab (CAMPATH) followed by sten cell infusion will allow for donor stem cells to grow in patients with hemoglobinopathies bone marrow and restore circulating blood counts. In addition the incidence and severity of side effects and of graft vs. host disease (GVHD) will be monitored.

  • In order to undergo transplant procedure, patients will be admitted to the hospital for approximately 10-14 days.
  • To prepare patient's bone marrow to accept donor stem cells, they will receive fludarabine and busulfex. Fludarabine will be given intravenously once daily for 4 days. Busulfex will be given once daily for the same 4 days.
  • One day before patients receive busulfex and fludarabine, they will also be given alemtuzumab intravenously once daily for 5 days.
  • Three days after the end of chemotherapy, patients will receive the infusion of donor stem cells.
  • If patients have thalassemia, they will receive subcutaneous injections of filgrastim starting on day one after the donor stem cell transfusion and will continue receiving filgrastim every day until it appears that the donor stem cells have been accepted. If the patient has sickle cell disease, filgrastim will not be given,
  • Additional drugs will be given to help prevent infection (i.e. antibiotics).
  • After stem cell infusion patients will be examined and have blood tests weekly for 1 month. Bone marrow biopsies, and blood work will also be performed 1 month, 3 months, 6 months and 1 year after stem cell infusion.
  • Patients will be on the study for about 12 months. After study is completed progress will be monitored on an annual basis.
Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Hemoglobinopathies
  • Sickle Cell Disease
  • Thalassemia
  • Drug: Busulfex
    Given once daily for 4 days
  • Drug: Fludarabine
    Given intravenously once daily for 4 days
  • Drug: Alemtuzumab
    One day before fludarabine and busulfex are started, alemtuzumab will be given once daily for 5 days.
    Other Name: CAMPATH
  • Procedure: Stem Cell Transfusion
    Performed three days after the end of chemotherapy
Not Provided
Armistead PM, Mohseni M, Gerwin R, Walsh EC, Iravani M, Chahardouli B, Rostami S, Zhang W, Neuberg D, Rioux J, Ghavamzadeh A, Ritz J, Antin JH, Wu CJ. Erythroid-lineage-specific engraftment in patients with severe hemoglobinopathy following allogeneic hematopoietic stem cell transplantation. Exp Hematol. 2008 Sep;36(9):1205-15. doi: 10.1016/j.exphem.2008.04.004. Epub 2008 Jun 11.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
2
July 2009
March 2008   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Patients with sickle cell disease should have one or more of the following: acute chest syndrome requiring hospitalization; nonhemorrhagic stroke or central nervous system event lasting longer than 24 hours; recurrent caso-occlusive pain or recurrent priapism; sickle neuropathy; bilateral proliferative retinopathy and major visual impairment of at least one eye; osteonecrosis of multiple joints; transfusion dependence; vaso-occlusive.
  • Patients with thalassemia should have one or more of the following: transfusion dependence; iron overload; presence of 2 or more alloantibodies against red cell antigens.

Exclusion Criteria:

  • Pregnancy
  • Acute hepatitis
  • Cardiac ejection fraction < 30%
  • Severe renal impairment
  • Severe residual functional neurologic impairment
  • Evidence of HIV infection
Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00153985
03-338
Yes
Catherine Wu, MD, Dana-Farber Cancer Institute
Dana-Farber Cancer Institute
  • Beth Israel Deaconess Medical Center
  • Massachusetts General Hospital
  • Brigham and Women's Hospital
  • Emory University
  • Feist-Weiller Cancer Center at Louisiana State University Health Sciences
  • Ohio State University
Principal Investigator: Catherine J. Wu, MD Dana-Farber Cancer Institute
Dana-Farber Cancer Institute
July 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP