Trial · NCT03759665
N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
NCT03759665, Phase 2, Completed, IntraBio Inc; results first posted 2024-03-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). Trials, record NCT03759665. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03759665
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
- NCT number
- NCT03759665
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 30 participants, actual
- Lead sponsor
- IntraBio Inc
- Conditions
- GM2 Gangliosidosis, Tay-Sachs Disease, Sandhoff Disease
- Interventions
- IB1001
- First posted
- 2018-11-30
- Last updated
- 2024-03-12
- Results first posted
- 2024-03-12
- Limitations and caveats
- This study was ongoing during the COVID-19 pandemic, and the conduct of this study was impacted by the COVID-19 pandemic.
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Clinical Impression of Change in Severity (CI-CS) [Fields et al 2021] | (CI-CS comparing Baseline [Day 1] with IB1001 versus the end of 6-weeks treatment with IB1001 [approximately Day 42]) MINUS (CI-CS comparing the end of 6-weeks treatment with IB1001 [approximately Day 42] versus end of 6-weeks post-treatment washout); | The Clinical Impression of Change in Severity assessment will instruct the blinded rater to consider: compared to the first video, how has the severity of their performance on the 9 Hole Peg Test of the Dominant Hand (9HPT-D) or 8 Meter Walk Test (8MWT) changed (improved or worsened) in 6-weeks as observed in the second video? The Clinical Impression of Change in Severity is evaluated on a 7 point Likert scale (+3=significantly improved to -3= significantly worse). Change values were calculated for each period, i.e. treatment with IB1001 (comparing the end of treatment (Visit 4) with baseline (Visit 2)) and post-treatment washout (comparing the end of washout (Visit 6) with the end of treatment (Visit 4)). Then, the mean change obtained in the post-treatment period was subtracted from the mean change obtained for the treatment with IB1001 period, with a positive value indicating an improvement in the treatment period compared to the post-treatment washout period. |
| Secondary | Key Secondary Endpoint: Individual Components of the CI-CS | Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post 6-week treatment washout. | The Clinical Impression of Change in Severity assessment will instruct the blinded rater to consider: compared to the first video, how has the severity of their performance on the 9 Hole Peg Test of the Dominant Hand (9HPT-D) or 8 Meter Walk Test (8MWT) changed (improved or worsened) in 6-weeks as observed in the second video? The Clinical Impression of Change in Severity is evaluated on a 7 point Likert scale (+3=significantly improved to -3= significantly worse). Change values were calculated for each period, i.e. treatment with IB1001 (comparing the end of treatment (Visit 4) with baseline (Visit 2)) and post-treatment washout (comparing the end of washout (Visit 6) with the end of treatment (Visit 4)). |
| Secondary | Key Secondary Endpoint: Change in Severity Based on Average CI-S | (CI-S comparing baseline period [average for Visit 1 and 2] and end of treatment period [average for Visit 3 and 4]) minus (change in CI-S between end of treatment period [average for Visit 3 and 4] and end of washout period [average for Visit 5 and 6]). | The Change in Severity assessment will instruct the blinded rater to consider the severity of the patient at each visit. The Clinical Impression of Severity (CI-S)-assessment ranged from +3 ="normal not ill at all" to -3="among the most extremely ill patients ". Change values were calculated for each period, i.e. treatment with IB1001 (change between the baseline period (average for Visit 1 and Visit 2) and end of treatment period (average for Visit 3 and Visit 4)) and post-treatment washout (between end of treatment period (average for Visit 3 and Visit 4) and end of washout period (average for Visit 5 and Visit 6). Then, the mean change in the post-treatment period was subtracted from the mean change in the treatment with IB1001 period. |
| Secondary | Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale | Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post 6-week treatment washout | The Clinical Impression of Change in Severity assessment will instruct the blinded rater to consider: compared to the first video, how has the severity of their performance on the 9 Hole Peg Test of the Dominant Hand (9HPT-D) or 8 Meter Walk Test (8MWT) changed (improved or worsened) in 6-weeks as observed in the second video? The Clinical Impression of Change in Severity is evaluated on a 7 point Likert scale (+3=significantly improved to -3= significantly worse). CI-CS scores \<0 were reclassified as worsened (-1), CI-CS scores 0 remained classified as not changed (0), and CI-CS scores \>0 were reclassified as improved (+1). When comparing Visit 4 versus Visit 2 and Visit 6 versus Visit 4, CI-CS scores \<0 were reclassified as worsened (-1), CI-CS scores 0 remained classified as not changed (0), and CI-CS scores \>0 were reclassified as improved (+1). |
| Secondary | Key Secondary Endpoint: CI-CS Score for the Non-Primary Anchor Test | CI-CS of the non-primary anchor test was evaluated, comparing the CI-CS of Visit 4 (end of treatment) versus Visit 2 (baseline) and of Visit 6 (end of washout) versus Visit 4 (end of treatment) as done for the primary anchor test. | The Clinical Impression of Change in Severity assessment will instruct the blinded rater to consider: compared to the first video, how has the severity of their performance on the 9 Hole Peg Test of the Dominant Hand (9HPT-D) or 8 Meter Walk Test (8MWT) changed (improved or worsened) in 6-weeks as observed in the second video? The Clinical Impression of Change in Severity is evaluated on a 7 point Likert scale (+3=significantly improved to -3= significantly worse). Change values were calculated for each period, i.e. treatment with IB1001 (comparing the end of treatment (Visit 4) with baseline (Visit 2)) and post-treatment washout (comparing the end of washout (Visit 6) with the end of treatment (Visit 4)). |
| Secondary | Spinocerebellar Ataxia Functional Index (SCAFI) [Schmitz-Hübsch et al, 2008] | Change values were calculated for each period, i.e. treatment with IB1001 (comparing the end of treatment (Visit 4) with baseline (Visit 2)) and post-treatment washout (comparing the end of washout (Visit 6) with the end of treatment (Visit 4)). | Spinocerebellar Ataxia Functional Index (SCAFI) is composed of 8 Meter Walk Test, 9-Hole Peg Test of Dominant and Non-Dominant Hand (9HPT-D/9HPT-ND) (the 3 tests are timed assessments; each is done twice and values are averaged; the 8MWT and 9HPT-D and 9HPT-ND values are converted from times to rates, and the results expressed as a composite Z-score of each test relative to baseline) and the PATA rate (counted number how often a patient can repeat the syllables "PATA" within 10 seconds), a measure of speech performance. The scores of these 3 were transformed to Z-scores (=individual's average of both trials to perform the respective task -mean of study population at baseline) / SD of study population at baseline). A Z-score of 0 equates to the population mean at baseline. For all 3, higher Z-scores (above mean) mean better performance. The SCAFI total score was calculated as the arithmetic mean of the non-missing Z-scores for the 3. A higher total score means better performance. |
| Secondary | Scale for Assessment and Rating of Ataxia (SARA) Score [Schmitz-Hübsch Etal, 2006; Subramony, 2007] | Baseline to end of treatment with IB1001 (Parent Study Day 42); End of treatment with IB1001 to the end of post treatment washout | The Scale for Assessment and Rating of Ataxia has 8 items that are related to gait, stance, sitting, speech, finger-chase test, nose-finger test, fast alternating movements, and heel-shin test. The range is 0-40 points, with a lower score representing neurological improvement and a higher score representing neurological worsening. Change values were calculated for each period, i.e. treatment with IB1001 (comparing the end of treatment (Visit 4) with baseline (Visit 2)) and post-treatment washout (comparing the end of washout (Visit 6) with the end of treatment (Visit 4)). |
| Secondary | EuroQuol- 5 Dimension (EQ-5D) Quality of Life Scale: Visual Analogue Scale (VAS) | Baseline to end of treatment with IB1001 (Parent Study 6-week treatment): observed VAS values at visit 4; End of treatment with IB1001 to the end of post treatment washout: observed VAS values at visit 6. | For posting, health-related quality of life based on the EQ-5D visual analogue scale (VAS) was presented as a secondary endpoint. EQ-5D VAS is a 0-100 scale where patients are asked to indicate their overall health, with a score of 0 indicating worst health and a score of 100indicating best health. |
| Secondary | Modified Disability Rating Scale (mDRS) [Iturriaga et al. 2006] | Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post treatment washout. | Overall neurological status based on six domains (ambulation, manipulation,language, swallowing, seizures and ocular movements). The Modified Disability Rating Scale (mDRS) ranges from 0-24, where 0 is the best neurological status and 24 is the worst. Change values were calculated for each period, i.e. treatment with IB1001 (comparing the end of treatment (Visit 4) with baseline (Visit 2)) and post-treatment washout (comparing the end of washout (Visit 6) with the end of treatment (Visit 4)) |
| Secondary | Investigator's Clinical Global Impressions of Change (CGI-c) | Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post treatment washout. | The Clinical Global Impression of Change assessed by the investigator is evaluated on a 7 point Likert scale ranging from 1='very much improved' to 7='very much worse' Change values were calculated for each period, i.e. treatment with IB1001 (comparing the end of treatment (Visit 4) with baseline (Visit 2)) and post-treatment washout (comparing the end of washout (Visit 6) with the end of treatment (Visit 4)). |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Clinical Impression of Change in Severity (CI-CS) [Fields et al 2021] | (CI-CS comparing Baseline [Day 1] with IB1001 versus the end of 6-weeks treatment with IB1001 [approximately Day 42]) MINUS (CI-CS comparing the end of 6-weeks treatment with IB1001 [approximately Day 42] versus end of 6-weeks post-treatment washout); | Modified Intention to Treat (mITT) Analysis Set is defined as all patients who receive at least one dose of the study drug and with one video recording during the baseline period (Visit 1 or 2, or both) and treatment period (Visit 3 or Visit 4, or both). | score on a scale | MEAN |
| SECONDARY | Key Secondary Endpoint: Individual Components of the CI-CS | Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post 6-week treatment washout. | Modified Intention to Treat (mITT) Analysis Set is defined as all patients who receive at least one dose of the study drug and with one video recording during the baseline period (Visit 1 or 2, or both) and treatment period (Visit 3 or Visit 4, or both). The overall mITT comprised 29 participants. Of these, 29 participants had values for the CI-CS analysis in the Treatment with IB1001 period and 27 participants in the Post-Treatment washout period. | score on a scale | MEAN |
| SECONDARY | Key Secondary Endpoint: Change in Severity Based on Average CI-S | (CI-S comparing baseline period [average for Visit 1 and 2] and end of treatment period [average for Visit 3 and 4]) minus (change in CI-S between end of treatment period [average for Visit 3 and 4] and end of washout period [average for Visit 5 and 6]). | Modified Intention to Treat (mITT) Analysis Set is defined as all patients who receive at least one dose of the study drug and with one video recording during the baseline period (Visit 1 or 2, or both) and treatment period (Visit 3 or Visit 4, or both). | score on a scale | MEAN |
| SECONDARY | Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale | Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post 6-week treatment washout | The Modified Intention to Treat (mITT) analysis set was defined as all patients who receive at least one dose of the study drug and with one video recording during the baseline period (Visit 1 or 2, or both) and treatment period (Visit 3 or Visit 4, or both). The overall mITT comprised 29 participants. Of these, 29 participants had values for the CI-CS analysis in the Treatment with IB1001 period and 27 participants in the Post-Treatment washout period. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Key Secondary Endpoint: CI-CS Score for the Non-Primary Anchor Test | CI-CS of the non-primary anchor test was evaluated, comparing the CI-CS of Visit 4 (end of treatment) versus Visit 2 (baseline) and of Visit 6 (end of washout) versus Visit 4 (end of treatment) as done for the primary anchor test. | Modified Intention to Treat (mITT) Analysis Set is defined as all patients who receive at least one dose of the study drug and with one video recording during the baseline period (Visit 1 or 2, or both) and treatment period (Visit 3 or Visit 4, or both). The overall mITT comprised 29 participants. Of these, 22 participants had values for the CI-CS Score for the Non-Primary Anchor Test analysis in the Treatment with IB1001 period and 20 participants in the Post-Treatment washout period | score on a scale | MEAN |
| SECONDARY | Spinocerebellar Ataxia Functional Index (SCAFI) [Schmitz-Hübsch et al, 2008] | Change values were calculated for each period, i.e. treatment with IB1001 (comparing the end of treatment (Visit 4) with baseline (Visit 2)) and post-treatment washout (comparing the end of washout (Visit 6) with the end of treatment (Visit 4)). | Modified Intention to Treat (mITT) Analysis Set is defined as all patients who receive at least one dose of the study drug and with one video recording during the baseline period (Visit 1 or 2, or both) and treatment period (Visit 3 or Visit 4, or both). The overall mITT comprised 29 participants. Of these, 26 participants had values for the SCAFI Total score analysis in the Treatment with IB1001 period and 25 participants in the Post-Treatment washout period | Z-scores | MEAN |
| SECONDARY | Scale for Assessment and Rating of Ataxia (SARA) Score [Schmitz-Hübsch Etal, 2006; Subramony, 2007] | Baseline to end of treatment with IB1001 (Parent Study Day 42); End of treatment with IB1001 to the end of post treatment washout | The Modified Intention to Treat (mITT) analysis set was defined as all patients who receive at least one dose of the study drug and with one video recording during the baseline period (Visit 1 or 2, or both) and treatment period (Visit 3 or Visit 4, or both). The overall mITT comprised 29 participants. Of these, 28 participants had values for the SARA analysis in the Treatment with IB1001 period and 27 participants in the Post-Treatment washout period. | score on a scale | MEAN |
| SECONDARY | EuroQuol- 5 Dimension (EQ-5D) Quality of Life Scale: Visual Analogue Scale (VAS) | Baseline to end of treatment with IB1001 (Parent Study 6-week treatment): observed VAS values at visit 4; End of treatment with IB1001 to the end of post treatment washout: observed VAS values at visit 6. | The Modified Intention to Treat (mITT) analysis set was defined as all patients who receive at least one dose of the study drug and with one video recording during the baseline period (Visit 1 or 2, or both) and treatment period (Visit 3 or Visit 4, or both). The overall mITT comprised 29 participants. Of these, 28 participants had values for the EQ-5D VAS analysis in the Treatment with IB1001 period and 27 participants in the Post-Treatment washout period. | score on a scale | MEAN |
| SECONDARY | Modified Disability Rating Scale (mDRS) [Iturriaga et al. 2006] | Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post treatment washout. | The Modified Intention to Treat (mITT) analysis set was defined as all patients who receive at least one dose of the study drug and with one video recording during the baseline period (Visit 1 or 2, or both) and treatment period (Visit 3 or Visit 4, or both). The overall mITT comprised 29 participants. Of these, 28 participants had values for the mDRS analysis in the Treatment with IB1001 period and 27 participants in the Post-Treatment washout period | score on a scale | MEAN |
| SECONDARY | Investigator's Clinical Global Impressions of Change (CGI-c) | Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post treatment washout. | The Modified Intention to Treat (mITT) analysis set was defined as all patients who receive at least one dose of the study drug and with one video recording during the baseline period (Visit 1 or 2, or both) and treatment period (Visit 3 or Visit 4, or both). The overall mITT comprised 29 participants. Of these, 28 participants had values for theInvestigator's CGI-C analysis in the Treatment with IB1001 period and 27 participants in the Post-Treatment washout period. | score on a scale | MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group26
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Clinical Impression of Change in Severity (CI-CS) [Fields et al 2021] | Parent Study | 0.71 | Standard Deviation 2.09 | score on a scale | |
| Key Secondary Endpoint: Individual Components of the CI-CS | Parent Study | Treatment with IB1001 | 0.34 | Standard Deviation 1.59 | score on a scale |
| Key Secondary Endpoint: Individual Components of the CI-CS | Parent Study | Post-TreatmentWashout | -0.36 | Standard Deviation 1.33 | score on a scale |
| Key Secondary Endpoint: Change in Severity Based on Average CI-S | Parent Study | 0.09 | Standard Deviation 0.71 | score on a scale | |
| Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale | Parent Study | Treatment with IB1001 / -1 (Worsened) | 12 | Participants | |
| Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale | Parent Study | Treatment with IB1001 / 0 (No observable change) | 1 | Participants | |
| Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale | Parent Study | Treatment with IB1001 / +1 (Improved) | 16 | Participants | |
| Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale | Parent Study | Post-treatment washout / -1 (Worsened) | 16 | Participants | |
| Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale | Parent Study | Post-treatment washout / 0 (No observable change) | 3 | Participants | |
| Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale | Parent Study | Post-treatment washout / +1 (Improved) | 8 | Participants |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment With IB1001 | STARTED | Parent Study | 30 | |
| Treatment With IB1001 | COMPLETED | Parent Study | 27 | |
| Treatment With IB1001 | NOT COMPLETED | Parent Study | 3 | |
| Post-Treatment Washout | STARTED | Parent Study | 27 | |
| Post-Treatment Washout | COMPLETED | Parent Study | 27 | |
| Post-Treatment Washout | NOT COMPLETED | Parent Study | 0 |
Source ClinicalTrials.gov
Adverse events26
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 | Fall | Injury, poisoning and procedural complications | Treatment With IB1001 | 7 | 30 | 10 |
| other | Fall | Injury, poisoning and procedural complications | Post-Treatment Washout | 6 | 30 | 7 |
| other | Contusion | Injury, poisoning and procedural complications | Treatment With IB1001 | 0 | 30 | 0 |
| other | Contusion | Injury, poisoning and procedural complications | Post-Treatment Washout | 3 | 30 | 4 |
| other | Tremor | Nervous system disorders | Treatment With IB1001 | 4 | 30 | 4 |
| other | Tremor | Nervous system disorders | Post-Treatment Washout | 1 | 30 | 1 |
| other | Balance disorder | Nervous system disorders | Treatment With IB1001 | 2 | 30 | 2 |
| other | Balance disorder | Nervous system disorders | Post-Treatment Washout | 1 | 30 | 1 |
| other | Seizure | Nervous system disorders | Treatment With IB1001 | 0 | 30 | 0 |
| other | Seizure | Nervous system disorders | Post-Treatment Washout | 2 | 30 | 2 |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Treatment With IB1001 | 0 | 30 |
| Total, all-cause mortality | Post-Treatment Washout | 0 | 30 |
| Total, other adverse events | Treatment With IB1001 | 17 | 30 |
| Total, other adverse events | Post-Treatment Washout | 15 | 30 |
| Total, serious adverse events | Treatment With IB1001 | 2 | 30 |
| Total, serious adverse events | Post-Treatment Washout | 0 | 30 |
Source ClinicalTrials.gov
Papers4
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Treatment With IB1001 vs Post-Treatment Washout: deaths from any cause | Trial comparisons |
| Treatment With IB1001 vs Post-Treatment Washout: serious adverse events | Trial comparisons |
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.
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