Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

2 participants

Study Classification

INTERVENTIONAL

Study Start Date

2001-09-30

Study Completion Date

2003-11-30

Brief Summary

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The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high dose chemotherapy with transplantation.

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Detailed Description

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Hemoglobinopathies, such as sickle cell disease and thalassemia major, are genetic diseases associated with significant morbidity and premature death. Allogeneic bone marrow transplantation (BMT) is the only potential cure for severe hemoglobinopathies. Typical regimens have used high doses of chemotherapy or chemo-radiotherapy to ablate recipient hematopoiesis and to prevent graft rejection. The widespread use of this treatment has been limited by toxicity, risk of end-organ damage, and donor availability. This study will use a nonmyeloablative regimen of fludarabine and busulfan to attempt to generate consistent engraftment with donor hematopoietic stem cells in patients with severe hemoglobinopathy.

G-CSF mobilization of the donor's peripheral blood white blood cells will precede donor apheresis. A nonmyeloablative conditioning regimen of fludarabine and busulfan will be administered to patients prior to allogeneic peripheral blood stem cell infusions. FK506 and prednisone will be administered for graft versus host disease (GVHD) prophylaxis. Patients will be evaluated for engraftment, donor: host hematopoietic chimerism, toxicity, and hemoglobinopathy.

Conditions

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Hemoglobinopathies Anemia, Sickle Cell Hemoglobin SC Disease Thalassemia Thalassemia Major

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Allogeneic stem cell transplantation

Participants will receive a nonmyeloablative conditioning regimen of fludarabine and busulfan prior to allogeneic peripheral blood stem cell (CD34+) infusions. FK506 and prednisone will be administered for graft versus host disease (GVHD) prophylaxis.

Group Type EXPERIMENTAL

Busulfan

Intervention Type DRUG

0.8 mg/kg/d administered as a single intravenous infusion over 3 hours for 4 days. All infusions are anticipated to be given in the outpatient clinic.

Fludarabine

Intervention Type DRUG

30 mg/m\^2/d administered as a bolus infusion over 30 minutes for 4 days. All infusions are anticipated to be given in the outpatient clinic.

FK506

Intervention Type DRUG

0.15 mg/kg taken orally daily for 12 to 14 weeks

Prednisone

Intervention Type DRUG

0.5 mg/kg taken orally four times daily on Day 7 and increase to 1 mg/kg taken orally four times daily on Day 14. Participants will continue regimen until Day 30 before a 20-25% taper per week.

Interventions

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Busulfan

0.8 mg/kg/d administered as a single intravenous infusion over 3 hours for 4 days. All infusions are anticipated to be given in the outpatient clinic.

Intervention Type DRUG

Fludarabine

30 mg/m\^2/d administered as a bolus infusion over 30 minutes for 4 days. All infusions are anticipated to be given in the outpatient clinic.

Intervention Type DRUG

FK506

0.15 mg/kg taken orally daily for 12 to 14 weeks

Intervention Type DRUG

Prednisone

0.5 mg/kg taken orally four times daily on Day 7 and increase to 1 mg/kg taken orally four times daily on Day 14. Participants will continue regimen until Day 30 before a 20-25% taper per week.

Intervention Type DRUG

Other Intervention Names

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Busulfex Fludara Oforta Prograf Tacromilus

Eligibility Criteria

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Inclusion Criteria

* All patients must:

* Have related donors who are identical at 6 human leukocyte antigens (HLA) loci (A, B and DR) by molecular typing
* Have a performance status from 0-2
* Give written informed consent
* Patients with sickle cell disease should have 1 or more of the following:

* Acute chest syndrome requiring recurrent hospitalization or exchange transfusion
* Nonhemorrhagic stroke or central nervous system event lasting longer than 24 hours
* Recurrent vaso-occlusive pain (2 episodes or more per year) or recurrent priapism
* Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate 30-50 percent of normal predicted value)
* Bilateral proliferative retinopathy and major visual impairment in at least 1 eye
* Osteonecrosis of multiple joints
* Patients with thalassemia should have 1 or more of the following:

* Transfusion dependence, defined as a transfusion requirement of greater than or equal to 6 units of packed red blood cells over the past 12 months
* Iron overload, defined as serum ferritin greater than 500 mcg/L in the absence of infection or biopsy-proven iron overload
* Presence of 2 or more alloantibodies against red cell antigens

Exclusion Criteria

* Pregnancy
* Acute hepatitis (transaminases greater than 3 times the normal value)
* Cardiac ejection fraction less than 30 percent
* Severe renal impairment (glomerular filtration rate less than 30 percent of predicted normal value)
* Severe residual functional neurologic impairment (other than hemiplegia alone)
* Seropositivity for the human immunodeficiency virus (HIV)

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No