Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

2 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-03-31

Study Completion Date

2009-07-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Reduced Intensity Allogeneic PBSCT to Treat Hematologic Malignancies and Hematopoietic Failure States

NCT00997386

Hematologic Neoplasms Multiple Myeloma Anemia, Aplastic +2 more

COMPLETED PHASE2

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

NCT00408447

Sickle Cell Disease Beta Thalassemia

ACTIVE_NOT_RECRUITING PHASE2

Stem Cell Transplant for Hemoglobinopathy

NCT00176852

Sickle Cell Disease Thalassemia Severe Congenital Neutropenia +2 more

COMPLETED PHASE2/PHASE3

Donor Stem Cell Transplant in Treating Patients With Relapsed Hematologic Cancer

NCT00053196

Leukemia Lymphoma Multiple Myeloma +3 more

COMPLETED PHASE2

Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

NCT00301834

Congenital Amegakaryocytic Thrombocytopenia Diamond-blackfan Anemia Leukemia +2 more

COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

* 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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hemoglobinopathies Sickle Cell Disease Thalassemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Busulfex

Given once daily for 4 days

Intervention Type DRUG

Fludarabine

Given intravenously once daily for 4 days

Intervention Type DRUG

Alemtuzumab

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

Intervention Type DRUG

Stem Cell Transfusion

Performed three days after the end of chemotherapy

Intervention Type PROCEDURE

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

CAMPATH

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No