Trial · NCT03759639
N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
NCT03759639, Phase 2, Completed, IntraBio Inc; results first posted 2023-11-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC). Trials, record NCT03759639. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03759639
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
- NCT number
- NCT03759639
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 33 participants, actual
- Lead sponsor
- IntraBio Inc
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- IB1001
- First posted
- 2018-11-30
- Last updated
- 2023-11-28
- Results first posted
- 2023-11-28
- Limitations and caveats
- This study was ongoing during the coronavirus disease 2019 (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 the CI-CS comparing the end of 6-weeks treatment with IB1001 (Approximately Day 42) versus the 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). |
| 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). |
| Secondary | Key Secondary Endpoint: Change in Severity Based on Average CI-S | CI-CS comparing baseline period and end of treatment period minus the change in CI-S between end of treatment period and end of washout period. | 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). |
| 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) or8 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). |
| 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 versus Visit 2 and of Visit 6 versus Visit 4 as done for the primary anchor test. | The Clinical Impression of Change in Severity is evaluated on a 7 point Likert scale (+3=significantly improved to -3= significantly worse). |
| Secondary | Spinocerebellar Ataxia Functional Index (SCAFI) [Schmitz-Hübsch et al, 2008] | 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 | 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 et al, 2006; Subramony, 2007] | 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 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. |
| Secondary | EuroQuol- 5 Dimension (EQ-5D) Quality of Life Scale: Visual Analogue Scale (VAS) | 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 | 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 100 indicating 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 6-week treatment washout | Overall neurological status based on six domains (ambulation, manipulation, language, swallowing, seizures and ocular movements). The Modified Disability Rating Scale ranges from 0-24, where 0 is the best neurological status and 24 is the worst. |
| 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 6-week 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' |
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 the CI-CS comparing the end of 6-weeks treatment with IB1001 (Approximately Day 42) versus the end of 6-weeks post-treatment washout | Modified Intention to Treat (mITT) Analysis Set - 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 - Defined as all patients who receive at east 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 32 participants. | score on a scale | MEAN |
| SECONDARY | Key Secondary Endpoint: Change in Severity Based on Average CI-S | CI-CS comparing baseline period and end of treatment period minus the change in CI-S between end of treatment period and end of washout period. | Modified Intention to Treat (mITT) Analysis Set - 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 | Modified Intention to Treat (mITT) Analysis Set - 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 32 participants. Of these, 32 participants had values for the CI-CS Score Reclassified on a 3-Point Scale analysis in the Treatment with IB1001 period and 28 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 versus Visit 2 and of Visit 6 versus Visit 4 as done for the primary anchor test. | Modified Intention to Treat (mITT) Analysis Set - 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 32 participants. Of these, 30 participants had values for the SCAFI Total score analysis in the Treatment with IB1001 period and 26 participants in the Post-Treatment washout period | score on a scale | MEAN |
| SECONDARY | Spinocerebellar Ataxia Functional Index (SCAFI) [Schmitz-Hübsch et al, 2008] | 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 - 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 32 participants. Of these, 30 participants had values for the SCAFI Total score analysis in the Treatment with IB1001 period and 26 participants in the Post-Treatment washout period | Z-scores | MEAN |
| SECONDARY | Scale for Assessment and Rating of Ataxia (SARA) Score [Schmitz-Hübsch et al, 2006; Subramony, 2007] | 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 32 participants. Of these, 31 participants had values for the SARA analysis in the Treatment with IB1001 period and 28 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-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 32 participants. Of these, 26 participants had values for the EQ-5D analysis in the Treatment with IB1001 period and 26 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 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 32 participants. Of these, 31 participants had values for the mDRS analysis in the Treatment with IB1001 period and 28 participants in the Post-Treatment washout period. | units 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 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 32 participants. Of these, 31 participants had values for the Investigator's CGI-C analysis in the Treatment with IB1001 period and 28 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 | 0.86 | Standard Deviation 2.52 | score on a scale | |
| Key Secondary Endpoint: Individual Components of the CI-CS | Parent Study | Treatment with IB1001 | 0.48 | Standard Deviation 1.34 | score on a scale |
| Key Secondary Endpoint: Individual Components of the CI-CS | Parent Study | Post-Treatment Washout | -0.38 | Standard Deviation 1.45 | score on a scale |
| Key Secondary Endpoint: Change in Severity Based on Average CI-S | Parent Study | 0.08 | Standard Deviation 1.12 | score on a scale | |
| Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale | Parent Study: | Treatment with IB1001 / -1 (Worsened) | 9 | Participants | |
| Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale | Parent Study: | Treatment with IB1001 / 0 (No observable Change) | 3 | Participants | |
| Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale | Parent Study: | Treatment with IB1001 / +1 (Improved) | 20 | 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) | 2 | Participants | |
| Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale | Parent Study: | Post-treatment washout / +1 (Improved) | 10 | 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 | 33 | |
| Treatment With IB1001 | COMPLETED | Parent Study | 31 | |
| Treatment With IB1001 | NOT COMPLETED | Parent Study | 2 | |
| Post-Treatment Washout | STARTED | Parent Study | 31 | |
| Post-Treatment Washout | COMPLETED | Parent Study | 31 | |
| Post-Treatment Washout | NOT COMPLETED | Parent Study | 0 |
Source ClinicalTrials.gov
Adverse events24
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 | Rhinitis | Infections and infestations | Treatment With IB1001 | 2 | 33 | 2 |
| other | Rhinitis | Infections and infestations | Post-Treatment Washout | 1 | 31 | 1 |
| other | Seizure | Nervous system disorders | Treatment With IB1001 | 3 | 33 | 3 |
| other | Seizure | Nervous system disorders | Post-Treatment Washout | 1 | 31 | 1 |
| other | Diarrhoea | Gastrointestinal disorders | Treatment With IB1001 | 3 | 33 | 4 |
| other | Diarrhoea | Gastrointestinal disorders | Post-Treatment Washout | 1 | 31 | 1 |
| other | Epistaxis | Respiratory, thoracic and mediastinal disorders | Treatment With IB1001 | 2 | 33 | 2 |
| other | Epistaxis | Respiratory, thoracic and mediastinal disorders | Post-Treatment Washout | 0 | 31 | 0 |
| other | Rash pruritic | Skin and subcutaneous tissue disorders | Treatment With IB1001 | 2 | 33 | 2 |
| other | Rash pruritic | Skin and subcutaneous tissue disorders | Post-Treatment Washout | 0 | 31 | 0 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 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 | 33 |
| Total, all-cause mortality | Post-Treatment Washout | 0 | 31 |
| Total, other adverse events | Treatment With IB1001 | 11 | 33 |
| Total, other adverse events | Post-Treatment Washout | 5 | 31 |
| Total, serious adverse events | Treatment With IB1001 | 4 | 33 |
| Total, serious adverse events | Post-Treatment Washout | 0 | 31 |
Source ClinicalTrials.gov
Papers3
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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