Trial · NCT04669535
A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
NCT04669535, Phase 1, Terminated, Terence Flotte; results first posted 2025-05-22
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease. Trials, record NCT04669535. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04669535
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
- NCT number
- NCT04669535
- Status
- Terminated
- Phases
- Phase 1
- Enrollment
- 9 participants, actual
- Lead sponsor
- Terence Flotte
- Conditions
- Tay-Sachs Disease, Sandhoff Disease
- Interventions
- AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2 Middle Dose, AXO-AAV-GM2 High Dose
- First posted
- 2020-12-17
- Last updated
- 2025-09-02
- Results first posted
- 2025-05-22
- Limitations and caveats
While encouraging, the results of this study also demonstrate important limitations of this therapeutic approach. First, the infusion of 1.25 ml of vector material into the parenchyma of the thalamus likely is at or near the maximum feasible dose. With less than complete clinical efficacy and limited ability to increase the dose further, the clinical utility of this approach is far from optimal.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events | At least through 24 weeks post-treatment (infantile-onset participants) to 48 weeks post-treatment (juvenile-onset participants). All AEs are reported through the transition of the study to long-term follow-up. | The measure of total numbers of total treatment emergent adverse events (TEAEs), serious adverse events (SAEs) after treatment with the vector whether related or unrelated to treatment as well as the number of adverse events (AEs) related to AXO-AAV-GM2 vector and neurosurgical procedures. |
| Secondary | Number of Participants With Clinically Significant Abnormal Vital Signs Per Investigator Assessment | 24 weeks (infantile-onset participants) to 48 weeks (juvenile-onset participants) | Any abnormal vital signs that the investigator determined was clinically significant was recorded as an adverse event. |
| Secondary | Number of Participants With Clinically Significant Abnormal Physical Exam Per Investigator Assessment | 24 weeks (infantile-onset participants) to 48 weeks (juvenile-onset participants) | Physical exam findings that the investigator determined to be clinically significant and related to the treatment with AXO-AAV-GM2, surgical procedure or immune suppression. |
| Secondary | Number of Participants With Clinically Significant Abnormal Clinical Safety Laboratory Tests on Blood/Urine/CSF | 24 weeks (infantile-onset participants) to 48 weeks (juvenile-onset participants) | Abnormal laboratory findings in subjects treated with AXO-AAV-GM2 that were felt to be clinically significant and reported as adverse events. |
| Secondary | Serum Cellular and Antibody Immune Response to Vector Capsid/Transgene | 24 weeks (infantile-onset participants) to 48 weeks (juvenile-onset participants) | Subjects with clinically significant serum cellular and antibody immune response to vector capsid/transgene that were reported as adverse events and treated with an increase in steroids. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events | At least through 24 weeks post-treatment (infantile-onset participants) to 48 weeks post-treatment (juvenile-onset participants). All AEs are reported through the transition of the study to long-term follow-up. | All subjects treated with AXO-AAV-GM2 at all doses | number of events | NUMBER |
| SECONDARY | Number of Participants With Clinically Significant Abnormal Vital Signs Per Investigator Assessment | 24 weeks (infantile-onset participants) to 48 weeks (juvenile-onset participants) | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Participants With Clinically Significant Abnormal Physical Exam Per Investigator Assessment | 24 weeks (infantile-onset participants) to 48 weeks (juvenile-onset participants) | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Participants With Clinically Significant Abnormal Clinical Safety Laboratory Tests on Blood/Urine/CSF | 24 weeks (infantile-onset participants) to 48 weeks (juvenile-onset participants) | Subjects treated with AXO-AAV-GM2 | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Serum Cellular and Antibody Immune Response to Vector Capsid/Transgene | 24 weeks (infantile-onset participants) to 48 weeks (juvenile-onset participants) | Subjects with clinically significant serum cellular and antibody immune response to vector capsid/transgene | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group64
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events | AXO-AAV-GM2 Starting Dose | Total number of TEAEs | 21 | number of events | |
| Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events | AXO-AAV-GM2 Low Dose | Total number of TEAEs | 43 | number of events | |
| Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events | AXO-AAV-GM2 Middle Dose | Total number of TEAEs | 51 | number of events | |
| Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events | AXO-AAV-GM2 High Dose | Total number of TEAEs | 18 | number of events | |
| Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events | AXO-AAV-GM2 Starting Dose | Total Number of Treatment Emergent SAEs | 1 | number of events | |
| Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events | AXO-AAV-GM2 Low Dose | Total Number of Treatment Emergent SAEs | 9 | number of events | |
| Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events | AXO-AAV-GM2 Middle Dose | Total Number of Treatment Emergent SAEs | 8 | number of events | |
| Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events | AXO-AAV-GM2 High Dose | Total Number of Treatment Emergent SAEs | 1 | number of events | |
| Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events | AXO-AAV-GM2 Starting Dose | AEs Related to Vector | 0 | number of events | |
| Incidence, Severity, Seriousness and Relatedness to Treatment of Treatment Emergent Adverse Events | AXO-AAV-GM2 Low Dose | AEs Related to Vector | 4 | number of events |
1 to 10 of 64Source ClinicalTrials.gov
See all 64 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | AXO-AAV-GM2 Starting Dose | 1 | |
| Overall Study | STARTED | AXO-AAV-GM2 Low Dose | 3 | |
| Overall Study | STARTED | AXO-AAV-Middle Dose | 3 | |
| Overall Study | STARTED | AXO-AAV-GM2 High Dose | 2 | |
| Overall Study | COMPLETED | AXO-AAV-GM2 Starting Dose | 0 | |
| Overall Study | COMPLETED | AXO-AAV-GM2 Low Dose | 2 | |
| Overall Study | COMPLETED | AXO-AAV-Middle Dose | 3 | |
| Overall Study | COMPLETED | AXO-AAV-GM2 High Dose | 2 | |
| Overall Study | NOT COMPLETED | AXO-AAV-GM2 Starting Dose | 1 | |
| Overall Study | NOT COMPLETED | AXO-AAV-GM2 Low Dose | 1 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events368
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 | Anemia | Blood and lymphatic system disorders | AXO-AAV-GM2 Starting Dose | 1 | 1 | 1 |
| other | Anemia | Blood and lymphatic system disorders | AXO-AAV-GM2 Low Dose | 1 | 3 | 1 |
| other | Anemia | Blood and lymphatic system disorders | AXO-AAV-GM2 Middle Dose | 1 | 3 | 1 |
| other | Anemia | Blood and lymphatic system disorders | AXO-AAV-GM2 High Dose | 1 | 2 | 1 |
| other | Neutrophil Count Decrease | Blood and lymphatic system disorders | AXO-AAV-GM2 Starting Dose | 1 | 1 | 2 |
| other | Neutrophil Count Decrease | Blood and lymphatic system disorders | AXO-AAV-GM2 Low Dose | 0 | 3 | 0 |
| other | Neutrophil Count Decrease | Blood and lymphatic system disorders | AXO-AAV-GM2 Middle Dose | 0 | 3 | 0 |
| other | Neutrophil Count Decrease | Blood and lymphatic system disorders | AXO-AAV-GM2 High Dose | 0 | 2 | 0 |
| other | Atrial Fibrillation | Cardiac disorders | AXO-AAV-GM2 Starting Dose | 0 | 1 | 0 |
| other | Atrial Fibrillation | Cardiac disorders | AXO-AAV-GM2 Low Dose | 0 | 3 | 0 |
1 to 10 of 368Source ClinicalTrials.gov
See all 368 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | AXO-AAV-GM2 Starting Dose | 1 | 1 |
| Total, all-cause mortality | AXO-AAV-GM2 Low Dose | 1 | 3 |
| Total, all-cause mortality | AXO-AAV-GM2 Middle Dose | 0 | 3 |
| Total, all-cause mortality | AXO-AAV-GM2 High Dose | 0 | 2 |
| Total, other adverse events | AXO-AAV-GM2 Starting Dose | 1 | 1 |
| Total, other adverse events | AXO-AAV-GM2 Low Dose | 3 | 3 |
| Total, other adverse events | AXO-AAV-GM2 Middle Dose | 3 | 3 |
| Total, other adverse events | AXO-AAV-GM2 High Dose | 2 | 2 |
| Total, serious adverse events | AXO-AAV-GM2 Starting Dose | 1 | 1 |
| Total, serious adverse events | AXO-AAV-GM2 Low Dose | 3 | 3 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers2
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/NCT04669535
GET https://conductscience.org/api/trials/v1/NCT04669535/results?page=2