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Completed

NCT Number: NCT00153985

Allogeneic Stem Cell Transplantation Following 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.

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Key information

Conditions

Hemoglobinopathies Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Graft vs Host Disease Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobin SC Disease Immune System Diseases Sickle Cell Disease Thalassemia

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Winship Cancer Institute-Emory University, Atlanta, Georgia, United States

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About this study

  • 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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

busulfex

Drug

Given once daily for 4 days

Fludarabine

Drug

Given intravenously once daily for 4 days

Alemtuzumab

Drug

One day before fludarabine and busulfex are started, alemtuzumab will be given once daily for 5 days.

Other names: CAMPATH

Stem Cell Transfusion

Procedure

Performed three days after the end of chemotherapy

Primary outcomes

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

    Time frame: 3 years

    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.

Secondary outcomes

  1. Solid Organ Toxicity Related to the Conditioning Regimen.

    Time frame: 3 years

    Outcome was measured by the assessment of organ toxicity related to Busulfex, fludarabine and alemtuzumab.

  2. The Incidence of Grade II-IV Acute Graft vs. Host Disease.

    Time frame: 3 years

    Outcome was measured by incidence and severity of acute and chronic GVHD following donor stem cell infusion.

Sponsors and collaborators

Lead sponsor

Dana-Farber Cancer Institute

Other

Collaborators

  • Beth Israel Deaconess Medical Center
  • Brigham and Women's Hospital
  • Emory University
  • Feist-Weiller Cancer Center at Louisiana State University Health Sciences
  • Massachusetts General Hospital
  • Ohio State University

Registry information

Official study title

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

Important dates

Study start
2004
Primary completion
2008
Study completion
2009
First posted
Sep 12, 2005
Registry last updated
Jul 30, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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