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Sirolimus Prophylaxis for aGVHD in TME SCID

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02177760
Recruitment Status : Withdrawn (Preliminary data suggested a better approach available)
First Posted : June 30, 2014
Last Update Posted : December 3, 2015
Information provided by (Responsible Party):
University of California, San Francisco

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.

Condition or disease Intervention/treatment Phase
Severe Combined Immunodeficiency Transplacental Maternal Engraftment Stem Cell Transplant Drug: Sirolimus Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
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

Arm Intervention/treatment
Experimental: Sirolimus
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
Other Name: Rapamune

Primary Outcome Measures :
  1. Incidence of aGVHD [ Time Frame: 105 days ]
    Incidence of aGVHD (dermatitis, hepatitis, enteritis) as defined by UCSF SOP CL 221.06 by Day +100.

Secondary Outcome Measures :
  1. 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.

  2. 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.

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Ages Eligible for Study:   up to 1 Year   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

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 identifier (NCT number): NCT02177760

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United States, California
Benioff Children's Hospital at UCSF Medical Center
San Francisco, California, United States, 94143
Sponsors and Collaborators
University of California, San Francisco
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Principal Investigator: Chris Dvorak, MD University of California, San Francisco

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Responsible Party: University of California, San Francisco Identifier: NCT02177760    
Other Study ID Numbers: 14-13024
First Posted: June 30, 2014    Key Record Dates
Last Update Posted: December 3, 2015
Last Verified: December 2015
Keywords provided by University of California, San Francisco:
severe combined immunodeficiency
maternal engraftment
aGVHD prophylaxis
Additional relevant MeSH terms:
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Severe Combined Immunodeficiency
Immunologic Deficiency Syndromes
Immune System Diseases
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs