NCT00795886

Brief Summary

Objectives: Primary objective: Evaluate toxicity of rapamycin when used for post-bone marrow transplant graft vs. host disease prophylaxis in children with acute lymphoblastic leukemia (ALL). Investigator initiated; four participating institutions; Phase II pilot study

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
63

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Aug 2005

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2005

Completed
3.3 years until next milestone

First Submitted

Initial submission to the registry

November 19, 2008

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 21, 2008

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2010

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2010

Completed
2.2 years until next milestone

Results Posted

Study results publicly available

May 7, 2012

Completed
Last Updated

July 31, 2013

Status Verified

July 1, 2013

Enrollment Period

4.6 years

First QC Date

November 19, 2008

Results QC Date

March 1, 2011

Last Update Submit

July 23, 2013

Conditions

Acute Lymphoblastic Leukemia

Keywords

ALLCancerStem Cell Transplant

Outcome Measures

Primary Outcomes (2)

  • Number of Participants With Transplant-related Mortality

    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.

    24 months after transplant

  • Two Year Overall Survival

    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.

    24 months after transplant

Secondary Outcomes (1)

  • Percentage of Patients Developing Acute Graft vs. Host Disease (GVHD)

    180 days

Study Arms (1)

All participants

EXPERIMENTAL
Drug: RAPAMYCIN

Interventions

RAPAMYCINDRUG

Rapamycin (RAPA, RapamuneR) (sirolimus) is an immunosuppressive agent that was approved by the FDA in 1999. It is a macrocyclic lactone that is structurally similar to Tacrolimus (FK506) and binds to the same intracellular protein as FK506, FKBP1,2,3, but it has an entirely different mechanism of action and a different principal target protein. The target of the RAPA: FKBP complex is the mammalian target of rapamycin (mTOR). Unlike the calcineurin inhibitors cyclosporine (CSA) and - FK506, RAPA exerts its effects by inhibiting growth factor-driven transduction signals in the T-cell response to alloantigen, thus preventing proliferation among T and B lymphocytes3,4.

Also known as: (RAPA, RapamuneR) (sirolimus)
All participants

Eligibility Criteria

AgeUp to 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Pediatric patients' ages (0 - 21 years) with lymphoid malignancies considered for allogeneic bone marrow transplant from HLA-identical sibling donor, single antigen mismatched related or unrelated donor marrow /peripheral blood stem cell (PBSC) or cord blood available for marrow donation.
  • First remission:
  • if remission not achieved by day28
  • high risk cytogenetic features, including t(9;22) or t(4;11) Second or third remission
  • Signed informed consent.

You may not qualify if:

  • \. Organ criteria:
  • Cardiac: ECHO shortening fraction \<27%
  • Renal: Creatinine clearance \<60 ml/min/1.73 m2
  • Hepatic: Bilirubin \>1.5 mg/dl, transaminases \<3x normal
  • Infection: active viral, fungal or bacterial infection including HIV.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Primary Children's Medical Center

Salt Lake City, Utah, 84112, United States

Location

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-LymphomaNeoplasms

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Leukemia, LymphoidLeukemiaNeoplasms by Histologic TypeHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Results Point of Contact

Title
Michael Pulsipher, MD
Organization
Primary Children's Medical Center
Michael.Pulsipher@hsc.utah.edu
801-662-4732

Study Officials

  • Michael Pulsipher, MD

    Primary Children's Hospital

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 19, 2008

First Posted

November 21, 2008

Study Start

August 1, 2005

Primary Completion

March 1, 2010

Study Completion

March 1, 2010

Last Updated

July 31, 2013

Results First Posted

May 7, 2012

Record last verified: 2013-07

Locations

US(1)