Trial Outcomes & Findings for Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies (NCT NCT00153985)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

2 participants

Primary outcome timeframe

3 years

Results posted on

2013-07-30

Participant Flow

Activated for enrollment 3/4/2004. Closed to enrollment 4/25/2008. Participating institutions included: Dana-Farber Cancer Institute, Boston, Massachusetts, Feist-Weiller Cancer Center, LSU Health Sciences Center, Shreveport, Louisiana and Winship Cancer Institute, Emory University, Atlanta, Georgia

All enrolled patients received a stem cell transplant.

Participant milestones

Participant milestones
Measure	Transplant for Severe Hemoglobinopathies Patients with severe hemoglobinopathies (eg. sickle cell disease, thalassemia major) with related donors who are identical at 6 HLA loci: (HLA-A, HLA-B, HLA-DRB1). The preparative regimen consisted of Busulfex, fludarabine and alemtuzumab.
Overall Study STARTED	2
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Transplant for Severe Hemoglobinopathies n=2 Participants Patients with severe hemoglobinopathies (eg. sickle cell disease, thalassemia major) with related donors who are identical at 6 HLA loci: (HLA-A, HLA-B, HLA-DRB1). The preparative regimen consisted of Busulfex, fludarabine and alemtuzumab.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	25 years STANDARD_DEVIATION 2 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants

PRIMARY outcome

Timeframe: 3 years

Population: All patients enrolled.

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 measures

Outcome measures
Measure	Transplant for Severe Hemoglobinopathies n=2 Participants Patients with severe hemoglobinopathies (eg. sickle cell disease, thalassemia major) with related donors who are identical at 6 HLA loci: (HLA-A, HLA-B, HLA-DRB1). The preparative regimen consisted of Busulfex, fludarabine and alemtuzumab.
Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy.	2 participants

SECONDARY outcome

Timeframe: 3 years

Population: All patients enrolled.

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

Outcome measures

Outcome measures
Measure	Transplant for Severe Hemoglobinopathies n=2 Participants Patients with severe hemoglobinopathies (eg. sickle cell disease, thalassemia major) with related donors who are identical at 6 HLA loci: (HLA-A, HLA-B, HLA-DRB1). The preparative regimen consisted of Busulfex, fludarabine and alemtuzumab.
Solid Organ Toxicity Related to the Conditioning Regimen.	2 participants

SECONDARY outcome

Timeframe: 3 years

Population: All patients enrolled.

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

Outcome measures

Outcome measures
Measure	Transplant for Severe Hemoglobinopathies n=2 Participants Patients with severe hemoglobinopathies (eg. sickle cell disease, thalassemia major) with related donors who are identical at 6 HLA loci: (HLA-A, HLA-B, HLA-DRB1). The preparative regimen consisted of Busulfex, fludarabine and alemtuzumab.
The Incidence of Grade II-IV Acute Graft vs. Host Disease.	2 participants

Adverse Events

Transplant for Severe Hemoglobinopathies

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Transplant for Severe Hemoglobinopathies n=2 participants at risk Patients with severe hemoglobinopathies (eg. sickle cell disease, thalassemia major) with related donors who are identical at 6 HLA loci: (HLA-A, HLA-B, HLA-DRB1). The preparative regimen consisted of Busulfex, fludarabine and alemtuzumab.
Immune system disorders Graft versus host disease	50.0% 1/2 • Number of events 1 • Through study completion in 2009 (or patient death)

Other adverse events

Other adverse events
Measure	Transplant for Severe Hemoglobinopathies n=2 participants at risk Patients with severe hemoglobinopathies (eg. sickle cell disease, thalassemia major) with related donors who are identical at 6 HLA loci: (HLA-A, HLA-B, HLA-DRB1). The preparative regimen consisted of Busulfex, fludarabine and alemtuzumab.
Infections and infestations Infection	50.0% 1/2 • Number of events 1 • Through study completion in 2009 (or patient death)

Additional Information

Catherine Wu, MD

Dana-Farber Cancer Institute

Phone: 617-632-5943

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place