Sirolimus Prophylaxis for aGVHD in TME SCID
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|ClinicalTrials.gov Identifier: NCT02177760|
Recruitment Status : Withdrawn (Preliminary data suggested a better approach available)
First Posted : June 30, 2014
Last Update Posted : December 3, 2015
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.
|Condition or disease||Intervention/treatment||Phase|
|Severe Combined Immunodeficiency Transplacental Maternal Engraftment Stem Cell Transplant||Drug: Sirolimus||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Sirolimus in Prevention of aGVHD in Maternally Engrafted (TME) Severe Combined Immunodeficiency (SCID) Infants Receiving Unconditioned Hematopoietic Stem Cell Transplant (HSCT)|
|Study Start Date :||July 2014|
|Actual Primary Completion Date :||November 2015|
|Actual Study Completion Date :||November 2015|
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.
Other Name: Rapamune
- Incidence of aGVHD [ Time Frame: 105 days ]Incidence of aGVHD (dermatitis, hepatitis, enteritis) as defined by UCSF SOP CL 221.06 by Day +100.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02177760
|United States, California|
|Benioff Children's Hospital at UCSF Medical Center|
|San Francisco, California, United States, 94143|
|Principal Investigator:||Chris Dvorak, MD||University of California, San Francisco|