Trial · NCT06851767
Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
NCT06851767, Phase 1, Phase 2, Enrolling by invitation, National Institute of Allergy and Infectious Diseases (NIAID)
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy. Trials, record NCT06851767. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT06851767
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
- NCT number
- NCT06851767
- Status
- Enrolling by invitation
- Phases
- Phase 1, Phase 2
- Enrollment
- 18 participants, estimated
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- Conditions
- X-linked Severe Combined Immunodeficiency, X-SCID, XSCID
- Interventions
- Plerixafor, Filgrastim, Palifermin, Busulfan, Base-edited hematopoietic stem and progenitor cells
- First posted
- 2025-02-28
- Last updated
- 2026-10-13
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Quantify frequency and severity of adverse events (AEs) related to study agent from infusion to 12 months after infusion. | 12 months | Evaluate the safety of treatment with BE-HSPC IL2RG in participants with X-SCID. |
| Secondary | Evaluate percentage of participants with >= 5% mutation-repaired alleles in PBMCs. | 24 months | Measure efficacy of treatment by assessing efficiency of base editor at repair of mutations. |
| Secondary | Evaluate editing efficiency in peripheral blood cells (such as T, B, and natural killer [NK] cells). | 24 months | Measure efficacy of treatment by assessing molecular evidence for mutation repair. |
| Secondary | Evaluate Immune reconstitution: a. T, B, and NK cell number improvement from baseline. b. Emergence of naive T cells and CD31+ recent thymic emigrants.c. B-cell function: immunoglobulin (Ig) production. d. Specific responses to vacci... | 24 months | Measure efficacy of treatment by assessing immune reconstitution as indicated by 1) increase in immune cell numbers; 2) restoration of thymic function with production of na(SqrRoot) ve T cells and CD31+ T cells; 3) Evaluate restoration of B-cell function. |
| Secondary | Evaluate clinical efficacy by improvement from baseline problems such as recurrent infection, chronic norovirus, protein-losing enteropathy, gastrointestinal complaints, growth failure, malnutrition, or immune dysregulation. | 24 months | Measure efficacy of treatment by assessing comparison of clinical status before and after treatment as indicator of response to improved immune function. |
| Secondary | Evaluate frequency and severity of all study agent-related AEs and serious adverse events (SAEs) from time of study product infusion. | 24 months | Measure efficacy of treatment by assessing intervention-related AE rate. |
Source ClinicalTrials.gov
Outcomes0
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.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
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/NCT06851767
GET https://conductscience.org/api/trials/v1/NCT06851767/registered