Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe… (NCT03315078) | Clinical Trial Compass
UnknownPhase 1/2
Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency
United States13 participantsStarted 2012-04
Plain-language summary
The purpose of this study is to evaluate the safety and effectiveness of lentiviral gene transfer treatment at restoring immune function to participants with X-linked severe combined immunodeficiency (XSCID) who are 2 to 40 years of age, and have significant impairment of immunity.
Who can participate
Age range2 Years – 40 Years
SexALL
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Inclusion Criteria:
* A proven mutation in the common gamma chain gene as defined by direct sequencing of patient DNA
* Human leukocyte antigen (HLA) typing of the patient will have been performed before enrollment
* No available HLA matched sibling donor as determined before enrollment.
* Must be between 2 and 40 years of age and weigh 10 kg
* If previously transplanted, must be 18 months post-hematopoietic stem cell transplant (HSCT)
* Expected survival of at least 120 days.
* Documented to be negative for HIV infection by genome polymerase chain reaction (PCR)
* The patient must be judged by the primary evaluating physician to have a suitable family and social situation consistent with ability to comply with protocol procedures and the long-term follow-up requirements.
* Medical lab data (historical) of severe B cell dysfunction (low or absent immunoglobulin G \[IgG\] levels, failed immune response to vaccines); OR demonstrated requirement for intravenous gamma globulin (IVIG) (significant drop over 3 to 6 weeks between peak and trough IgG levels).
* Must be willing to have blood and tissue samples stored. IN ADDITION, patients must satisfy the following Laboratory Criteria AND Clinical Criteria:
* Laboratory Criteria: (at least 1 must be present)
* CD4+ lymphocytes: absolute number less than or equal to 50% of the lower limit of normal (LLN)
* CD4+CD45RA+ lymphocytes: absolute number less than or equal to 50% of the LLN OR T-cell receptor excision circles (TRECs) le…
What they're measuring
1
Level of autologous transduced T-lymphocytes with functional γc
Timeframe: Measured through Year 2
2
Incidence of serious side effects due to lentiviral gene transfer
Timeframe: Measured through Year 15
3
Distribution of integration sites of the lentiviral vector in reconstituted peripheral blood cells
Timeframe: Measured through Year 15
Trial details
NCT IDNCT03315078
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)