Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and… (NCT05432310) | Clinical Trial Compass
RecruitingPhase 1/2
Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
United States20 participantsStarted 2023-01-04
Plain-language summary
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
Who can participate
Age range1 Month
SexALL
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Inclusion criteria
✓. Provision of written informed consent prior to any study related procedures. In this study consent must be provided by the parents/legal guardians and, where applicable according to local laws, a signed assent from the child,
✓. Subjects ≥30 days of age,
✓. With a diagnosis of ADA-SCID based on:
✓. Lymphopenia (absolute lymphocyte count (ALC) \<400 cells/mL) OR absence or low number of T cells (absolute CD3+ count \< 300 cells/mL), or
✓. Severely decreased T lymphocyte blastogenic responses to phytohemagglutinin (either \<10% of lower limit of normal controls for the diagnostic laboratory, or \<10% of the response of the normal control of the day, or stimulation index \<10), or
✓. Identification of SCID by neonatal screening revealing low T Cell Receptor Excision Circles (TREC) levels.
✓. Ineligible for matched family allogeneic bone marrow (BM) transplantation, defined as the absence of a medically eligible HLA-identical sibling or family donor, with normal immune function, who could serve as an allogeneic bone marrow donor.
✓. Females of child-bearing age will be required to provide a negative pregnancy test 30 days prior to Visit 2.
Exclusion criteria
✕. Ineligible for autologous HSCT as per clinical site criteria
✕. Other conditions which in the opinion of the Principal Investigator and/or Co Investigators, contraindicate the mobilization of peripheral blood or the leukapheresis process, the administration of busulfan and the infusion of transduced cells, or which indicate an inability of the subject or subject's parent/legal guardian to comply with the protocol