Clinical Trial: Sirolimus Prophylaxis for aGVHD in TME SCID

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

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.


Detailed Summary:
Sponsor: University of California, San Francisco

Current Primary Outcome: Incidence of aGVHD [ Time Frame: 105 days ]

Incidence of aGVHD (dermatitis, hepatitis, enteritis) as defined by UCSF SOP CL 221.06 by Day +100.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • T-regulatory cell enumeration [ Time Frame: 105 days ]
    T-regulatory cell enumeration studies by blood draw prior to prophylaxis and at day +30, day+60, day+100.
  • Sirolimus therapeutic drug monitoring [ Time Frame: 105 days ]
    Sirolimus therapeutic drug monitoring will be done via blood draws(after initial 4th dose; thereafter every 4th dose with adjustment; once stable it will be measured weekly). We will determine the average dose in milligrams per kilograms that would be needed to reach therapeutic trough levels of 5-8 ng/mL.


Original Secondary Outcome: Same as current

Information By: University of California, San Francisco

Dates:
Date Received: June 19, 2014
Date Started: July 2014
Date Completion:
Last Updated: December 1, 2015
Last Verified: December 2015