Trial · NCT03538899
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 21
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | To demonstrate that ART-SCID patients receiving AProArt-CD34 infusion have superior overall survival (OS) at 24 months post treatment with AProArt-CD34 versus the established outcome of 0% OS for patients who receive no treatment for ART-SCID | 24 months | Patient survival status and (if applicable) cause of death will be recorded to assess overall survival. |
| Secondary | B cell immune reconstitution by 24±2 months compared with historical control cohort of allogeneic transplant recipients | 24 months | B cell immune reconstitution will be defined as cessation of immunoglobulin replacement therapy |
| Secondary | Number of participants with T cell immune reconstitution compared with historical controls who received allogeneic transplants to treat ART-SCID. | 24 months | T cell immune reconstitution will be defined as presence of both of the following: 1) a. CD3\>1000/mm3; 2) CD4\>500/mm3. |
| Secondary | Event Free Survival | 24 months | Events are defined as death, use of a repeat infusion of gene-corrected cells to enhance T/B cell immune reconstitution or requirement for performance of an allogeneic HCT. |
| Other | Adverse event (AE) profile associated with the study intervention from treatment through 15 years after the gene transfer procedure | 15 years | Clinical and laboratory AEs as measured by CTCAE V4 |
| Other | Number of participants with B cell reconstitution beyond 24 months, including B cell numbers and function | 15 years | B cell reconstitution will be defined as presence of at least two of the following: 1) B cell number \>40/mm3; 2) Cessation of immunoglobulin replacement therapy; 3) Protective specific antibody responses. The number of participants who meet the definition for B cell reconstitution will be tabulated. |
| Other | Efficacy of targeting low exposure busulfan in infants and children, calculated by percentage of participants whose treatment meets study parameters using a validated population pharmacokinetic model to calculate individualized busulfan clearance. | 42 days | Achievement of final target cumulative AUC of 20±2 mg\*hr/L in approximately 90% of subjects |
| Other | Repertoire diversity of T and B cells, as assessed by analyzing T cell receptor gene variable beta sequence (TCR-Vb) and immunoglobulin heavy chain (IGH) rearranged receptors and calculating diversity Shannon Index. | 24 months | Measured by deep sequencing of the T cell receptor (TCR) gene variable beta sequence (TCR-Vb) and immunoglobulin heavy chain (IGH) rearranged receptors. The diversity Shannon Index is expected to be above 7.5 by 24±2 months post treatment in 90% of patients. |
| Other | Determine if B cell receptor diversity at 6 months is predictive of B cell reconstitution over 15 years by analyzing B cell receptor gene variable beta sequence and immunoglobulin heavy chain rearranged receptors and calculating diversity Shannon Index. | 15 years | A Shannon Index \>5 is expected to be a biomarker for B cell reconstitution. B cell receptor diversity will be measured throughout study participation. |
| Other | Determine if at 6 months T cell receptor (TCR) diversity and/or absolute CD3, CD4, naïve CD4 counts predict T cell reconstitution at 24 months or need for 2nd treatment throughout the 15 year follow-up period. | 15 years | TCR-Vb diversity and absolute CD3, CD4 and naïve CD4 counts will be measured at 6 months. T cell reconstitution will be measured throughout study participation. |
| Other | Stability of mean vector copy number (VCN) and the location of vector integration sites in sorted T, B, and natural killer (NK) cells, granulocytes and monocytes in peripheral blood over time after infusion of transduced HSC. | 24 months | Mean vector copy number (VCN) and the location of vector integration sites will be measured at 6±1, 12±1 and 24±2 months and then annually through 15 years after infusion of AProArt-CD34. The mean VCN should be \>0.5 copies/cell in T and B cells and \>0.01 in NK, myeloid and granulocytes by 24±2 months post treatment. |
| Other | Characterization of insertion sites in multiple cell lineages as an indicator of diversity, multilineage engraftment, and tabulation of any clonal expansion that might indicate development of a malignancy. | 24 months | Insertion sites will be measured at 6±1, 12±1, 24±2 months, and annually through 15 years after infusion of AProArt-CD34. Cell insertion sites are expected to be diverse with no clone representing \>20% of a lineage at 24 months. |
| Other | Potential benefit of prophylactic sirolimus in reducing the incidence of transient autoimmune hemolytic anemia (AIHA) post infusion of AProArt-CD34, as assessed by number of participants with clinical diagnosis of AIHA. | 24 months | Incidence of autoimmune hemolytic anemia by 24±2 months post infusion following administration of prophylactic sirolimus, as assessed by monitoring of hemoglobin, direct and indirect Coombs, reticulocyte, and lactate dehydrogenase results. Incidence of AIHA requiring treatment is expected to be \<40% of patients on prophylactic sirolimus. |
| Other | Measurement of radiation sensitivity in peripheral blood NK cells for development and validation of a rapid functional assay for radiation-sensitive SCID. | 24 months | Development and validation of an assay for radiation sensitive SCID in peripheral blood NK cells |
| Other | Potency of AProArt-CD34 as indicated by capacity for in vitro differentiation of participant specimens into T cells and assessment of average VCN as a surrogate marker for potency. | 5 years | Demonstration that post-transduction patient CD34+ cells have the capacity for in vitro differentiation into T cells and that average VCN is a surrogate marker for potency. One or more potency assays will be developed and validated for AProArt-transduced CD34+ HSC that give results within 52 days of transduction, and correlation of these with VCN obtained at 6 and 14 days post transduction with AProArt. |
| Other | Hematopoietic recovery in patients with ART-SCID who receive self-inactivating (SIN) lentiviral vector (AProArt)-transduced CD34 cells through a repeat infusion autologous stem cell transplant. | 5 years | Patients will undergo blood tests to measure complete blood count and differential following a repeat infusion of gene corrected cells. |
| Other | Effects of targeted low exposure busulfan conditioning on patient growth/stature | 5 years | Patient height (cm) and weight (kg) will be measured and compared with norms for age |
| Other | Effects of busulfan on patient dental development | 5 years | Dental histories and clinical notes will be reviewed to compare permanent tooth development will be compared with development norms for age from age 5 through age 15 years |
| Other | Patient reported outcome of undergoing treatment with gene-corrected cells as assessed by the PedsQL questionnaires. | 5 years | Age-appropriate Pediatric Quality of Life Inventory (PedsQL) questionnaires will be administered at baseline and years 1, 2, 4, 8, 10, 12, and 15. Four domains (Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning) are reverse-scored on a 5-point Likert scale and transformed to a 0-100 scale, with higher scores indicating better outcomes. |
| Other | Family impact of undergoing treatment with gene-corrected cells. | 15 years | The Pediatric Quality of Life Inventory (PedsQL) Family Impact module will be administered at baseline and years 1, 2, 4, 8, 10, 12, and 15. Responses are indicated on a 5-point Likert scale and transformed to a 0-100 scale, with higher scores indicating better outcomes. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
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