Trial Outcomes & Findings for Rapamycin for Immunosuppression and B Cell Modulation Post Stem Cell Transplant for Acute Lymphoblastic Leukemia (ALL) (NCT NCT00795886)

Results Overview

Death not associated with relapse. We determined that if transplant related mortality(TRM) 25% at day 100 or if Grade III-IV (severe, life threatening, or disabling) acute GVHD was \>30% a stopping rule would be triggered.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

63 participants

Primary outcome timeframe

24 months after transplant

Results posted on

2013-07-31

Participant Flow

Patients were recruited between Aug 2003 and Aug 2008 from 4 pediatric centers in the United States. (Childrens Hospital of Philadelphia; Methodist Childrens Hospital of South Texas (San Antonio); Primary Childrens Medical Center (Salt Lake City); and Childrens Hospital of Pittsburgh.

Participant milestones

Participant milestones
Measure	Rapamycin This includes all study participants.
Overall Study STARTED	63
Overall Study COMPLETED	53
Overall Study NOT COMPLETED	10

Reasons for withdrawal

Reasons for withdrawal
Measure	Rapamycin This includes all study participants.
Overall Study Death	10

Baseline Characteristics

Rapamycin for Immunosuppression and B Cell Modulation Post Stem Cell Transplant for Acute Lymphoblastic Leukemia (ALL)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Rapamycin n=63 Participants This includes all study participants.
Age, Categorical <=18 years	61 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	9.9 years STANDARD_DEVIATION 5.3 • n=5 Participants
Sex: Female, Male Female	25 Participants n=5 Participants
Sex: Female, Male Male	38 Participants n=5 Participants
Region of Enrollment United States	63 participants n=5 Participants

PRIMARY outcome

Timeframe: 24 months after transplant

Population: All participants enrolled in the trial.

Death not associated with relapse. We determined that if transplant related mortality(TRM) 25% at day 100 or if Grade III-IV (severe, life threatening, or disabling) acute GVHD was \>30% a stopping rule would be triggered.

Outcome measures

Outcome measures
Measure	Rate of Transplant Related Mortality n=63 Participants All participants enrolled in the trial
Number of Participants With Transplant-related Mortality	8 participants

PRIMARY outcome

Timeframe: 24 months after transplant

Population: All patients enrolled in study.

The probability that a given patient will be alive two years after transplantation. The Kaplan-Meier product limit method was used to compute the probability of overall survival to 2 years. Greenwood's formula was used to compute the standard error.

Outcome measures

Outcome measures
Measure	Rate of Transplant Related Mortality n=63 Participants All participants enrolled in the trial
Two Year Overall Survival	0.73 probability Standard Error 0.058

SECONDARY outcome

Timeframe: 180 days

Cumulative incidence of Grade 2-4 acute GVHD at 180 days.

Outcome measures

Outcome measures
Measure	Rate of Transplant Related Mortality n=63 Participants All participants enrolled in the trial
Percentage of Patients Developing Acute Graft vs. Host Disease (GVHD)	38 percentage of participants Interval 26.0 to 50.0

Adverse Events

Rapamycin

Serious events: 8 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Rapamycin n=63 participants at risk This includes all study participants.
Blood and lymphatic system disorders Transplant related mortality	12.7% 8/63 • Number of events 8 • Over the 3 years of the trial and two years after completing.

Other adverse events

Other adverse events
Measure	Rapamycin n=63 participants at risk This includes all study participants.
Hepatobiliary disorders Venoocclusive Disease	4.8% 3/63 • Number of events 3 • Over the 3 years of the trial and two years after completing.

Additional Information

Michael Pulsipher, MD

Primary Children's Medical Center

Phone: 801-662-4732

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place