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Multi-Center Study Using Allogeneic Stem Cell Transplantation Following Reduced Intensity Chemotherapy in Patients With Hemoglobinopathies


Phase 2
18 Years
N/A
Not Enrolling
Both
Hemoglobinopathies, Sickle Cell Disease, Thalassemia

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Trial Information

Multi-Center Study Using Allogeneic Stem Cell Transplantation Following Reduced Intensity Chemotherapy in Patients With Hemoglobinopathies


- 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.


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

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy.

Outcome Description:

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.

Outcome Time Frame:

3 years

Safety Issue:

No

Principal Investigator

Catherine J. Wu, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Dana-Farber Cancer Institute

Authority:

United States: Institutional Review Board

Study ID:

03-338

NCT ID:

NCT00153985

Start Date:

March 2004

Completion Date:

July 2009

Related Keywords:

  • Hemoglobinopathies
  • Sickle Cell Disease
  • Thalassemia
  • Hemoglobinopathies
  • Sickle cell anemia
  • sickle cell-hemoglobin C disease
  • sickle cell-B-thalassemia
  • transfusion-dependant thalassemia
  • allogeneic transplant
  • nonmyeloablative transplant
  • Stem cell transfusion
  • graft vs. host disease
  • Anemia, Sickle Cell
  • Hemoglobinopathies
  • Thalassemia

Name

Location

Beth Israel Deaconess Medical CenterBoston, Massachusetts  02215
Dana-Farber Cancer InstituteBoston, Massachusetts  02115
Massachusetts General HospitalBoston, Massachusetts  02114-2617
Winship Cancer Institute-Emory UniversityAtlanta, Georgia  30322
Feist-Weiller Cancer Center-LSUShreveport, Louisiana  71130
Ohio State University College of MedicineColumbus, Ohio  43210