Trial · NCT00794508
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
NCT00794508, Phase 2, Completed, Donald B. Kohn, M.D.; results first posted 2016-05-30
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID. Trials, record NCT00794508. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00794508
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
- NCT number
- NCT00794508
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 10 participants, actual
- Lead sponsor
- Donald B. Kohn, M.D.
- Conditions
- Severe Combined Immunodeficiency
- Interventions
- ADA gene transfer
- First posted
- 2008-11-20
- Last updated
- 2021-04-23
- Results first posted
- 2016-05-30
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Adverse Events | 2 years | Examine the safety of the procedure: harvesting bone marrow, isolating CD34+ hematopoietic stem/progenitor cells, performing ex vivo gene transduction with the MND-ADA gamma-retroviral vector, giving 90 mg/m2 busulfan to "make space" in the bone marrow to aid engraftment, and re-infusing the autologous gene-modified cells. |
| Secondary | Number of Participants With Greater Than 1% of Gene-Modified Cells in the Peripheral Blood | 2 years | As measured by quantitative polymerase chain reaction in peripheral blood cells separated into mononuclear and granulocyte fractions. |
| Secondary | Number of Participants Reaching the Normal Range of ADA Enzyme Activity | 2 years | As measured by ADA enzyme activity in peripheral blood mononuclear cells |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Adverse Events | 2 years | participants | NUMBER | |
| SECONDARY | Number of Participants With Greater Than 1% of Gene-Modified Cells in the Peripheral Blood | 2 years | participants | NUMBER | |
| SECONDARY | Number of Participants Reaching the Normal Range of ADA Enzyme Activity | 2 years | participants | NUMBER |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Adverse Events | Gamma-retroviral Mediated ADA Gene Transfer | Expected Grade 3 or 4 AE related to procedure | 10 | participants | |
| Number of Participants With Adverse Events | Gamma-retroviral Mediated ADA Gene Transfer | Unexpected Grade 3 or 4 AE related to procedure | 9 | participants | |
| Number of Participants With Adverse Events | Gamma-retroviral Mediated ADA Gene Transfer | Grade 3 or 4 AE related to study product | 0 | participants | |
| Number of Participants With Adverse Events | Gamma-retroviral Mediated ADA Gene Transfer | Alive | 10 | participants | |
| Number of Participants With Adverse Events | Gamma-retroviral Mediated ADA Gene Transfer | Deaths | 0 | participants | |
| Number of Participants With Greater Than 1% of Gene-Modified Cells in the Peripheral Blood | Gamma-retroviral Mediated ADA Gene Transfer | For peripheral blood mononuclear cells | 10 | participants | |
| Number of Participants With Greater Than 1% of Gene-Modified Cells in the Peripheral Blood | Gamma-retroviral Mediated ADA Gene Transfer | for peripheral blood granulocytes | 4 | participants | |
| Number of Participants Reaching the Normal Range of ADA Enzyme Activity | Gamma-retroviral Mediated ADA Gene Transfer | 9 | participants |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Gamma-retroviral-mediated ADA Gene Transfer | 10 | |
| Overall Study | COMPLETED | Gamma-retroviral-mediated ADA Gene Transfer | 10 | |
| Overall Study | NOT COMPLETED | Gamma-retroviral-mediated ADA Gene Transfer | 0 |
Source ClinicalTrials.gov
Adverse events2
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | neutropenia | Blood and lymphatic system disorders | Retroviral-mediated ADA Gene Transfer | 9 | 10 | |
| serious | Infection requiring hospitalization | Immune system disorders | Retroviral-mediated ADA Gene Transfer | 5 | 10 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Retroviral-mediated ADA Gene Transfer | ||
| Total, other adverse events | Retroviral-mediated ADA Gene Transfer | 9 | 10 |
| Total, serious adverse events | Retroviral-mediated ADA Gene Transfer | 5 | 10 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants1
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| R01FD003005 | MND-ADA Transduced CD34 Cells for ADA-SCID | KOHN, DONALD B | UNIVERSITY OF CALIFORNIA LOS ANGELES | FOOD AND DRUG ADMINISTRATION | 2005 to 2011 |
Source NIH RePORTER
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/NCT00794508
GET https://conductscience.org/api/trials/v1/NCT00794508/registered