Trial · NCT03538899
Autologous Gene Therapy for Artemis-Deficient SCID
NCT03538899, Phase 1, Phase 2, Recruiting, University of California, San Francisco
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Autologous Gene Therapy for Artemis-Deficient SCID. Trials, record NCT03538899. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03538899
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Autologous Gene Therapy for Artemis-Deficient SCID
- NCT number
- NCT03538899
- Status
- Recruiting
- Phases
- Phase 1, Phase 2
- Enrollment
- 24 participants, estimated
- Lead sponsor
- University of California, San Francisco
- Conditions
- Severe Combined Immunodeficiency
- Interventions
- AProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan
- First posted
- 2018-05-29
- Last updated
- 2026-02-13
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| 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. |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers53
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests2
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as | Source |
|---|---|---|
| Continuous Performance Test | Other outcome | ConductCognition |
| Verbal Learning | Other outcome | ConductCognition |
Data dates
The day each source was copied for this release.
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
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03538899
GET https://conductscience.org/api/trials/v1/NCT03538899/registered?page=2