A Phase 2 interventional study of Sirolimus in Severe Combined Immunodeficiency, Transplacental Maternal Engraftment and Stem Cell Transplant, sponsored by University of California, San Francisco. Withdrawn at 1 site in United States. Open to participants aged 1 Day to 1 Year. Per ClinicalTrials.gov, last updated 2015-12-03.
Sponsored by University of California, San Francisco · Phase 2, Interventional, and Prevention
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.
64 studies on the registry are indexed under Severe Combined Immunodeficiency; 15 are open to participants now.
Browse Severe Combined Immunodeficiency studies →University of California, San Francisco is the lead sponsor of 2,132 studies on the registry; 375 are open to participants now.
Of its 262 completed or terminated interventional studies of FDA-regulated products, 196 (75%) have results posted.
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Exclusion Criteria:
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.
Drug: Sirolimus
Also known as: Rapamune
Incidence of aGVHD
Incidence of aGVHD (dermatitis, hepatitis, enteritis) as defined by UCSF SOP CL 221.06 by Day +100.
Time frame: 105 days
T-regulatory cell enumeration
T-regulatory cell enumeration studies by blood draw prior to prophylaxis and at day +30, day+60, day+100.
Time frame: 105 days
Sirolimus therapeutic drug monitoring
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.
Time frame: 105 days
This study is withdrawn, as verified in Dec 2015. You cannot join it, but the record below documents what was studied.
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Severe Combined Immunodeficiency→
University of California, San Francisco