Phase 2 N=2 Treatment

Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

Hemoglobinopathies · Sickle Cell Disease · Thalassemia

Bottom Line

View on ClinicalTrials.gov: NCT00153985 ↗

Enrolled (actual)

Serious AEs

50.0%

Results posted

Mar 2013

Primary outcome: Primary: Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy. — 2 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Busulfex (Drug); Fludarabine (Drug); Alemtuzumab (Drug); Stem Cell Transfusion (Procedure)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Dana-Farber Cancer Institute
Primary completion: Mar 2008

Outcome Measures

Outcome	Result	p-value
PRIMARY Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy.	2	—
SECONDARY Solid Organ Toxicity Related to the Conditioning Regimen.	2	—
SECONDARY The Incidence of Grade II-IV Acute Graft vs. Host Disease.	2	—

Summary

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.

Eligibility Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00153985). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.