Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Short… (NCT01852071) | Clinical Trial Compass
CompletedPhase 1/2
Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
United States46 participantsStarted 2013-08-02
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 the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA 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 – 17 Years
SexALL
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Inclusion criteria
✓Children ≥ 1.0 months of age with a diagnosis of ADA-deficient SCID based on A. Decreased ADA enzymatic activity in erythrocytes, leukocytes, skin fibroblasts, or in cultured fetal cells to levels consistent with ADA-deficient SCID as determined by reference laboratory or confirmed ADA gene mutation(s) known to cause disease , AND
✓. Family history of first order relative with ADA deficiency and clinical and laboratory evidence of severe immunologic deficiency, OR
✓. Evidence of severe immunologic deficiency in subject prior to institution of immune restorative therapy, based on
✓. lymphopenia (absolute lymphocyte count \<400 cells/mcL) OR absence or low number of T cells (absolute CD3+ count \<300 cells/mcL) OR
✓. severely decreased T lymphocyte blastogenic responses to phytohemagglutinin (either \<10% of lower limit of normal controls for the diagnostic laboratory, \<10% of the response of the normal control of the day, or stimulation index \<10)
Exclusion criteria
✕. Age ≤ 1.0 months Appropriate organ function as outlined below must be observed within 60 days of entering this trial.
✕. Hematologic
✕. Anemia (hemoglobin \< 10.5 g/dl at \< 2 years of age, or \< 11.5 g/dl at \> 2 years of age).
. International Normalised Ratio (INR) or Prothrombin Time (PT) \> 2 times the upper limits of normal or Partial Thromboplastin Time (PTT) \> 2.33 times the upper limit of normal (patients with a correctable deficiency controlled on medication will not be excluded).
✕. Cytogenetic abnormalities on peripheral blood or bone marrow or amniotic fluid (if available).