Clinical trial NCT02177760: 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)
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Severe Combined Immunodeficiency
Transplacental Maternal Engraftment
Stem Cell Transplant
Keywords: SCID
severe combined immunodeficiency
maternal engraftment
aGVHD prophylaxis
sirolimus
Lead sponsor: University of California, San Francisco
Sponsor class: OTHER
First posted: 2014-06-30
Enrollment: 0
Sex: ALL
Minimum age (years): 1
Maximum age (years): 1
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

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.


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

Primary outcomes:
measure: Incidence of aGVHD
timeFrame: 105 days
Secondary outcomes:
measure: T-regulatory cell enumeration
timeFrame: 105 days
measure: Sirolimus therapeutic drug monitoring
timeFrame: 105 days
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: Benioff Children's Hospital at UCSF Medical Center
state: California
Study officials:
affiliation: University of California, San Francisco
name: Chris Dvorak, MD
role: PRINCIPAL_INVESTIGATOR
References:
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