Trial · NCT00598481
ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
NCT00598481, Phase 2, Completed, Fondazione Telethon; results first posted 2020-07-31
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID. Trials, record NCT00598481. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00598481
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
- NCT number
- NCT00598481
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 12 participants, actual
- Lead sponsor
- Fondazione Telethon
- Conditions
- Immunologic Deficiency Syndromes
- Interventions
- Gene Therapy, Busulfan
- First posted
- 2008-01-22
- Last updated
- 2024-01-29
- Results first posted
- 2020-07-31
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Survival | baseline to 3 years post gene therapy | From post-treatment to up to 3 years |
| Secondary | Rate of Severe Infections | Before Treatment and 3-months post-treatment up to 3 years | Severe infections were defined as those that required hospitalization or those that prolonged hospitalization. The rate of infection was estimated as number of severe infections over person-years of observation (free from severe infections) before and after treatment administration. The first 3 months after gene therapy were not considered in the post-gene therapy analysis, because all subjects were hospitalized during this period. |
| Secondary | CD3+ Cell Counts | baseline up to 3 years post gene therapy | T-lymphocyte counts (CD3+): mean T-lymphocyte at Baseline and 3 years post gene therapy. Samples were taken from peripheral venous whole blood and tested by cytofluorometry; values are means (10\^6/L). |
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 | Survival | baseline to 3 years post gene therapy | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Rate of Severe Infections | Before Treatment and 3-months post-treatment up to 3 years | Severe Infections per person year | NUMBER | |
| SECONDARY | CD3+ Cell Counts | baseline up to 3 years post gene therapy | Only subjects with available data at each visit were included in the analysis | 10^6 cells/L | GEOMETRIC_MEAN |
Source ClinicalTrials.gov
Results by group7
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Survival | Gene Therapy (Pivotal) | 12 | Participants | ||
| Rate of Severe Infections | Severe Infections (Before Gene Therapy) | 1.1 | Severe Infections per person year | ||
| Rate of Severe Infections | Severe Infections (After Gene Therapy) | 0.429 | Severe Infections per person year | ||
| CD3+ Cell Counts | Gene Therapy ITT Population | Baseline | 112.5 | 95% Confidence Interval 39.4 to 321 | 10^6 cells/L |
| CD3+ Cell Counts | Gene Therapy ITT Population | Year 1 | 348.3 | 95% Confidence Interval 194.5 to 623.8 | 10^6 cells/L |
| CD3+ Cell Counts | Gene Therapy ITT Population | Year 2 | 633 | 95% Confidence Interval 482.9 to 829.8 | 10^6 cells/L |
| CD3+ Cell Counts | Gene Therapy ITT Population | Year 3 | 831.1 | 95% Confidence Interval 596.7 to 1157.6 | 10^6 cells/L |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Baseline | STARTED | Gene Therapy | 12 | |
| Baseline | COMPLETED | Gene Therapy | 12 | |
| Baseline | NOT COMPLETED | Gene Therapy | 0 | |
| Treatment | STARTED | Gene Therapy | 12 | |
| Treatment | COMPLETED | Gene Therapy | 11 | |
| Treatment | NOT COMPLETED | Gene Therapy | 1 |
Source ClinicalTrials.gov
Adverse events159
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 | Autoimmune haemolytic anemia | Blood and lymphatic system disorders | Gene Therapy (Pivotal) | 1 | 12 | |
| other | Neutropenia | Blood and lymphatic system disorders | Gene Therapy (Pivotal) | 4 | 12 | |
| other | Eosinophilia | Blood and lymphatic system disorders | Gene Therapy (Pivotal) | 2 | 12 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Gene Therapy (Pivotal) | 2 | 12 | |
| other | Rhinitis | Infections and infestations | Gene Therapy (Pivotal) | 8 | 12 | |
| other | Device related infection | Infections and infestations | Gene Therapy (Pivotal) | 2 | 12 | |
| other | Upper respiratory tract infection | Infections and infestations | Gene Therapy (Pivotal) | 5 | 12 | |
| other | Gastroenteritis | Infections and infestations | Gene Therapy (Pivotal) | 2 | 12 | |
| other | Oral candidiasis | Infections and infestations | Gene Therapy (Pivotal) | 4 | 12 | |
| other | Bronchitis | Infections and infestations | Gene Therapy (Pivotal) | 3 | 12 |
1 to 10 of 159Source ClinicalTrials.gov
See all 159 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Gene Therapy (Pivotal) | 0 | 12 |
| Total, other adverse events | Gene Therapy (Pivotal) | 12 | 12 |
| Total, serious adverse events | Gene Therapy (Pivotal) | 10 | 12 |
Source ClinicalTrials.gov
Papers8
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.
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/NCT00598481
GET https://conductscience.org/api/trials/v1/NCT00598481/events?page=2