Trial · NCT07113743
Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
NCT07113743, 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. Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease. Trials, record NCT07113743. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07113743
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
- NCT number
- NCT07113743
- Status
- Enrolling by invitation
- Phases
- Phase 1, Phase 2
- Enrollment
- 10 participants, estimated
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- Conditions
- Chronic Granulomatous Disease (CGD)
- Interventions
- Busulfan, Tocilizumab, Eltrombopag, Sirolimus, pCCLChimGp91lentiviral vector containing the human gp91 phox (CYBB) gene
- First posted
- 2025-08-11
- Last updated
- 2026-09-08
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety | Throughout the study | 1\) The primary safety objective of this procedure will be assessed by recording the incidence of adverse events. a) Record clinical adverse events and clinically significant laboratory abnormalities. b) Evaluate overall incidence of adverse events for the study as a whole. c) Monitor the incidence of serious adverse events. |
| Primary | Efficacy | 6 months and 1 year | The primary efficacy objective of this study will be determined by measuring the percentage of subjects who have \>= 10% oxidase positive granulocytes by DHR flow cytometry at month 6 and 12 after transplant. |
| Secondary | Assess immunological reconstitution | Week 4, 5, 6, 7, 8, 10, 12 and months 6, 9, 12, 18 and 24 months. | Restoration of neutrophil oxidase function (\> 10%) as measured by DHR flow cytometry (clinical), and/or cytochrome C (O2 production, research), at 24 months. The % DHR + neutrophils will be measured at week 4, 5, 6, 7, 8, 10, 12 and months 6, 9, 12, 18 and 24 months. |
| Secondary | Assess HSC transduction and engraftment | Week 4, 6, 7, 8, 10, 12 and month 6, 9, 12, 18 and 24) | a) Determine the percentage of transduced CD34+ hematopoietic cells infused. b) Measure the average vector copy number (VCN) in mature blood cells (at week 4, 6, 7, 8, 10, 12 and month 6, 9, 12, 18 and 24) by qPCR or ddPCR. |
| Secondary | Assess health by | 1, 2, 3, 6, 9, 12, 18, and 24 months, compared to baseline | a) The nutritional status (height, weight, serum albumin) at 24 months b) Growth and development of pediatric subjects at 24 months c) Frequency of severe infections or other health issues, including inflammatory complications (requiring IV antibiotics and/or hospitalization for treatment) d) Measurement of inflammatory markers at 1, 2, 3, 6, 9, 12, 18, and 24 months, compared to baseline e) Colitis assessment based on CDAI (Crohn s Disease Activity Index) and fecal calprotectin at 6, 12, and 24 months, compared to baseline f) Quality of life questionnaire (baseline, 6, 12 and 24 months). |
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/NCT07113743
GET https://conductscience.org/api/trials/v1/NCT07113743/registered