Sirolimus Prophylaxis for aGVHD in TME SCID

University of California San Francisco (UCSF)

Status and phase: Withdrawn; Phase 2

Conditions: Stem Cell Transplant; Transplacental Maternal Engraftment; Severe Combined Immunodeficiency

Treatments: Drug: Sirolimus

Study type: Interventional

Funder types: Other

Identifiers

NCT02177760

14-13024

Details and patient eligibility

About

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.

Sex: All

Ages: 1 day to 1 year old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

Sirolimus

Experimental group

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.

Treatment:

Drug: Sirolimus