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

Interventional


Primary Outcome

Incidence of aGVHD

Secondary Outcome

 T-regulatory cell enumeration

Condition

Severe Combined Immunodeficiency

Intervention

Sirolimus

Study Arms / Comparison Groups

 Sirolimus
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.

Publications

* 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

Drug

Estimated Enrollment

0

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
      

Gender

All

Ages

N/A - 1 Year

Accepts Healthy Volunteers

No

Contacts

Chris Dvorak, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02177760

Organization ID

14-13024


Responsible Party

Sponsor

Study Sponsor

University of California, San Francisco


Study Sponsor

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


Verification Date

December 2015