Trial Outcomes & Findings for Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes (NCT NCT00004143)

Results Overview

Number of patients with neutrophil engraftment: Absolute Neutrophil Count (ANC) \> 500/μL and hemoglobin level remaining above 10 g/dL without transfusion support, with tests showing at least 2.5% donor cells present. Primary graft failure is defined as absence of establishment of adequate donor hematopoiesis by day 42 with bone marrow cellularity \< 5%, peripheral White Blood Count (WBC) \< 500/μL, peripheral ANC \< 100/μL, and/or platelets \< 10,000/μL by day 120 with absence of megakaryocytes in the bone marrow (in the absence of disease relapse).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

2 participants

Primary outcome timeframe

1 year post transplant

Results posted on

2014-12-05

Participant Flow

The protocol was reopened in 2003 for non-malignant conditions only. Enrollment closed in 2008 due to lack of enrollment.

Participant milestones

Participant milestones
Measure	All Patients
Overall Study STARTED	2
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Patients n=2 Participants
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
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year post transplant

Number of patients with neutrophil engraftment: Absolute Neutrophil Count (ANC) \> 500/μL and hemoglobin level remaining above 10 g/dL without transfusion support, with tests showing at least 2.5% donor cells present. Primary graft failure is defined as absence of establishment of adequate donor hematopoiesis by day 42 with bone marrow cellularity \< 5%, peripheral White Blood Count (WBC) \< 500/μL, peripheral ANC \< 100/μL, and/or platelets \< 10,000/μL by day 120 with absence of megakaryocytes in the bone marrow (in the absence of disease relapse).

Outcome measures

Outcome measures
Measure	All Patients n=2 Participants
Number of Patients With Neutrophil Engraftment	2 participants

PRIMARY outcome

Timeframe: 1 year post transplant

Number of patients with platelet engraftment - Platelets \> 20,000/μL and hemoglobin level remaining above 10 g/dL without transfusion support, with tests showing at least 2.5% donor cells present. Primary graft failure is defined as absence of establishment of adequate donor hematopoiesis by day 42 with bone marrow cellularity \< 5%, peripheral White Blood Count (WBC) \< 500/μL, peripheral ANC \< 100/μL, and/or platelets \< 10,000/μL by day 120 with absence of megakaryocytes in the bone marrow (in the absence of disease relapse).

Outcome measures

Outcome measures
Measure	All Patients n=2 Participants
Number of Patients With Platelet Engraftment	2 participants

PRIMARY outcome

Timeframe: 60 days post transplant

Number of patients with Grade 3-4 acute Graft Versus Host Disease (GVHD). GVHD will be monitored at least two times per week through day 45, then weekly through day 60 and graded by 2 persons at each institution, to ensure internal consistency in grading.

Outcome measures

Outcome measures
Measure	All Patients n=2 Participants
Number of Patients With Grade 3-4 Acute Graft Versus Host Disease (GVHD)	2 participants

PRIMARY outcome

Timeframe: 45 days post transplant

An unexpected adverse event is one that differs in the nature, severity, or frequency from (a) the research procedures that are described in the protocol-related documents, (such as the IRB-approved research protocol and informed consent document) as expected, and/or (b) the characteristics of the subject population being studied.

Outcome measures

Outcome measures
Measure	All Patients n=2 Participants
Number of Participants With Grade 3-4 Unexpected Adverse Events	0 participants

PRIMARY outcome

Timeframe: 100 days

Number of patients who died due to transplant-related complications

Outcome measures

Outcome measures
Measure	All Patients n=2 Participants
Number of Participants With Transplant-related Mortality	0 participants

SECONDARY outcome

Timeframe: 2 years

Number of patients alive 2 years after transplant

Outcome measures

Outcome measures
Measure	All Patients n=2 Participants
Overall Survival	2 participants

Adverse Events

All Patients

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	All Patients n=2 participants at risk
Cardiac disorders Hypertension	50.0% 1/2 • Number of events 1 • 45 days post transplant
Nervous system disorders Headache	50.0% 1/2 • Number of events 1 • 45 days post transplant
Cardiac disorders Cardiac ischemia / infarction	50.0% 1/2 • Number of events 1 • 45 days post transplant
Infections and infestations Parainfluenza	50.0% 1/2 • Number of events 1 • 45 days post transplant

Additional Information

Mitchell Horwitz, MD

Duke University Medical Center

Phone: 919-668-1045

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place