Sirolimus Prophylaxis for aGVHD in TME SCID

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Brief Title

Sirolimus Prophylaxis for aGVHD in TME SCID

Official Title

Sirolimus in Prevention of aGVHD in Maternally Engrafted (TME) Severe Combined Immunodeficiency (SCID) Infants Receiving Unconditioned Hematopoietic Stem Cell Transplant (HSCT)

Brief Summary

      Study Design: SCID infants receiving an unconditioned haploidentical transplant will be
      started on Sirolimus (0.05 mg/kg/day) day -5 for Acute Graft-Versus-Host Disease (aGVHD)
      prophylaxis. Sirolimus levels will be monitored with goal sirolimus trough level of 5-8
      ng/mL. Patients will be monitored for signs of aGVHD as defined by UCSF SOP CL 221.06 through
      day +100. Sirolimus will be tapered once T-regulatory cell to CD4 effector cell ratio is > or
      = 9%.

      Setting: Inpatient BMT Unit Benioff Children's Hospital at UCSF Medical Center

      Study Subjects: 15 infants with diagnosis of maternally engrafted T cells SCID by CA Newborn
      screen receiving unconditioned haploidentical HSCT

      Main Outcome Measures: Incidence of aGVHD (dermatitis, hepatitis, enteritis) as defined by
      SOP CL 221.06 by Day +100.

      Hypothesis 1. Patients placed on sirolimus prophylaxis will have lower incidence of aGVHD
      compared to historical controls.

      Hypothesis 2. Lower doses of sirolimus milligram per kilogram will be required to maintain
      goal troughs of 5-8 ng/mL.

Study Phase

Phase 2

Study Type


Primary Outcome

Incidence of aGVHD

Secondary Outcome

 T-regulatory cell enumeration


Severe Combined Immunodeficiency



Study Arms / Comparison Groups

Description:  Sirolimus (0.05 mg/kg/day) day -5 for aGVHD prophylaxis through day +100 or until T-regulatory cells >9% of CD4 effector cells; whichever comes first.


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

July 2014

Completion Date

November 2015

Primary Completion Date

November 2015

Eligibility Criteria

        Inclusion Criteria:

          -  Infants diagnosed with SCID on CA newborn screen

          -  Evidence of Maternal Engraftment

          -  Unconditioned haploidentical hematopoeitic stem cell transplant

        Exclusion Criteria:

          -  Evidence of acute graft vs. host disease




N/A - 1 Year

Accepts Healthy Volunteers



Chris Dvorak, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Responsible Party


Study Sponsor

University of California, San Francisco

Study Sponsor

Chris Dvorak, MD, Principal Investigator, University of California, San Francisco

Verification Date

December 2015