Trial Outcomes & Findings for T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders (NCT NCT00176826)

NCT ID: NCT00176826

Last Updated: 2018-01-23

Results Overview

• Recruitment status: TERMINATED
• Study phase: PHASE2/PHASE3
• Target enrollment: 22 participants
• Primary outcome timeframe: Day 100 Post Transplant
• Results posted on: 2018-01-23

Participant Flow

Subjects were recruited from the clinic or hospital where they were being seen for their disease. The study was discussed with them at the time that treatment options were being presented.

Patients had to have a suitable donor identified prior to the subject starting the conditioning regimen.

Participant milestones

Measure	Intent-To-Treat Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.
Overall Study STARTED	22
Overall Study COMPLETED	22
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders

Baseline characteristics by cohort

Measure	Intent-To-Treat n=22 Participants Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.
Age, Categorical <=18 years	22 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	1.7 years STANDARD_DEVIATION 0.9 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	16 Participants n=5 Participants
Region of Enrollment United States	22 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 100 Post Transplant

Outcome measures

Measure	Intent-To-Treat n=22 Participants Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.
Time to Transplant Engraftment	19.8 days Standard Deviation 5.2

SECONDARY outcome

Timeframe: Day 100 Post Transplant

Outcome measures

Measure	Intent-To-Treat n=22 Participants Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.
Number of Patients With Treatment Related Mortality.	6 participants

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Measure	Intent-To-Treat n=22 Participants Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.
Number of Patients Surviving (Disease-free)	14 participants

SECONDARY outcome

Timeframe: Day 100 Post Transplant

Outcome measures

Measure	Intent-To-Treat n=22 Participants Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.
Number of Patients With Grade II-IV Graft-Versus-Host Disease (GVHD)	4 participants

SECONDARY outcome

Timeframe: Day 100 Post transplant

Outcome measures

Measure	Intent-To-Treat n=22 Participants Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.
Number of Patients With Graft Failure	2 participants

SECONDARY outcome

Timeframe: Day 100 Post Transplant

Outcome measures

Measure	Intent-To-Treat n=22 Participants Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.
Number of Patients With III-IV Graft-Versus-Host Disease (GVHD)	1 participants

SECONDARY outcome

Timeframe: 3 years

Outcome measures

Measure	Intent-To-Treat n=22 Participants Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.
Number of Patients Surviving (Disease-free)	10 participants

Adverse Events

Intent-To-Treat

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Intent-To-Treat n=22 participants at risk Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Secondary Malignancy	4.5% 1/22 • Number of events 1 • From the time consent was signed to the end of follow-up, which was 3 years.
General disorders Death	4.5% 1/22 • Number of events 1 • From the time consent was signed to the end of follow-up, which was 3 years.

Other adverse events

Adverse event data not reported

Additional Information

Dr. Angela Smith

University of Minnesota

Phone: 612-626-2778

Email: smith719@umn.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place