Trial · NCT02534844
VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
NCT02534844, Phase 2, Phase 3, Completed, Mandos LLC; results first posted 2023-02-22
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease. Trials, record NCT02534844. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02534844
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
- NCT number
- NCT02534844
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 56 participants, actual
- Lead sponsor
- Mandos LLC
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- Parts A/B: Adrabetadex, Parts A/B: Sham Control
- First posted
- 2015-08-28
- Last updated
- 2023-02-22
- Other registries' IDs
- eudract:2015-002548-15
- Results first posted
- 2023-02-22
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Parts A/B: Change From Baseline to Week 52 in 4-Item Composite Score of Niemann Pick Type C Severity Scale (NPC-SS) Score | Baseline, Week 52 | The NPC-SS composite score is the sum of the ambulation, cognition, fine motor, and swallowing domains of the NPC-SS. Each of the four NPC-SS components (ambulation, cognition, fine motor, and swallowing) are rated on a scale from 0 (better) to 5 (worse). The total score was the sum of individual components scores which ranges from 0 (best) to 20 (worst), with higher scores indicating more severe clinical impairment. |
| Primary | Parts A/B: Number of Participants Classified With Each Score on the Clinician Global Impression of Change (CGIC) at Week 52 | Week 52 | The Clinician CGIC is a 7-point Likert scale. The scale requires assessment of change from a baseline level of disease activity, with anchors ranging from markedly improved, moderately improved, and minimally improved to no change and corresponding worsening (minimally, moderately, markedly). The Investigator rates his/her impression of the change in each participant's condition at week 52 on a scale from marked improvement (1) to marked worsening (7). |
| Secondary | Parts A/B: Change From Baseline to Week 52 in NPC-SS Total Score (Excluding Hearing and Auditory Brainstem Response [ABR]) | Baseline, Week 52 | The NPC-SS total score is based on 9 domains including ambulation, fine motor skills, cognition, swallowing, memory, speech, eye movement, hearing (sensorineural) and seizures. The hearing domain and auditory brainstem response modifiers are removed from the total NPC-SS total score for this measure. A Likert-like scale is used to assign the remaining 8 major domain scores of 0 to 5 (better to worse). The total score was the sum of individual component scores which ranges from 0 (best) to 40 (worst), with higher scores indicating more severe clinical impairment. |
| Secondary | Parts A/ B: Number of Participants Classified as CGIC Responders at Week 52 | Week 52 | The Clinician CGIC is a 7-point Likert scale. The scale requires assessment of change from a baseline level of disease activity, with anchors ranging from markedly improved, moderately improved, and minimally improved to no change and corresponding worsening (minimally, moderately, markedly). The Investigator rates his/her impression of the change in each participant's condition at week 52 on a scale from marked improvement (1) to marked worsening (7). CGIC Responders are defined as participants who received the caregiver's rating of no change, minimally improved, moderately improved, or markedly improved from baseline to Week 52. |
| Secondary | Parts A/B: Number of Participants Classified as Responders on NPC-SS Total Score (Excluding Hearing and ABR) at Week 52 | Week 52 | The NPC-SS total score is based on 9 domains including ambulation, fine motor skills, cognition, swallowing, memory, speech, eye movement, hearing (sensorineural) and seizures. The hearing domain and ABR modifiers are removed from the total NPC-SS total score for this measure. A Likert-like scale is used to assign the remaining 8 major domain scores of 0 to 5 (better to worse). The total score was the sum of individual components scores which ranges from 0 (best) to 40 (worst), with higher scores indicating more severe clinical impairment. Responders on NPC-SS Total Score are defined as participants with no change or improvement on NPC-SS total score from baseline to Week 52. |
| Secondary | Parts A/B: EQ-5D-3L Questionnaire Visual Analog Scale (VAS) Score (for Health Status) at Baseline and at Week 52 | Baseline, Week 52 | The EQ-5D-3L assessment is a self-reported, simple, descriptive system measuring 5 dimensions including mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. A vertical VAS allows the participants to indicate their health state that day, and ranges from 0 (worst imaginable) to 100 (best imaginable), with higher scores indicating better health state. |
| Secondary | Parts A/B: Number of Participants Treated for at Least 6 Months Who Qualified for the Rescue Option | Baseline up to Week 26 | Participants who manifested significant disease progression according to predefined clinical criteria after treatment of 26 weeks or more had the option to rescue. Number of participants who qualified for the rescue option following a minimum of 26 weeks of treatment were analyzed. |
| Secondary | Parts A/B: Change From Baseline to Week 52 in Each of the 9 Clinical Domains of the NPC-SS | Baseline, Week 52 | The NPC-SS total score is based on 9 domains including ambulation, fine motor skills, cognition, swallowing, memory, speech, eye movement, hearing (sensorineural) and seizures. A Likert-like scale is used to assign to each domain score of 0 to 5 (better to worse) with higher scores indicating more severe clinical impairment. |
| Secondary | Parts A/B: Change From Baseline to Week 52 in the Total NPC-SS (With Hearing Domain and ABR Modifier Included) | Baseline, Week 52 | The NPC-SS total score is based on 9 domains including ambulation, fine motor skills, cognition, swallowing, memory, speech, eye movement, hearing (sensorineural) and seizures. A Likert-like scale is used to assign to each domain score of 0 to 5 (better to worse). The total score was the sum of individual components scores which ranges from 0 (best) to 45 (worst), with higher scores indicating more severe clinical impairment. |
| Secondary | Parts A/B: Time to One Point Increase (Worsening) in NPC-SS Composite Score | Baseline up to Week 52 | The NPC-SS composite score is the sum of the ambulation, cognition, fine motor, and swallowing domains of the NPC-SS. Each of the four NPC-SS components (ambulation, cognition, fine motor, and swallowing) are rated on a scale from 0 (better) to 5 (worse). The total score was the sum of individual components scores which ranges from 0 (best) to 20 (worst), with higher scores indicating more severe clinical impairment. The product-limit survival analysis method is used to estimate the time to one point increase (worsening) in NPC-SS composite score. Time to worsening in NPC-SS Composite Score defined as the interval from study drug administration to a one point increase in the NPC-SS composite score. If a subject discontinued from the study prior to Week 52, then the subject was censored at time of discontinuation. If a subject completed the Week 52 visit, then the subject was censored at the time of last study visit. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Parts A/B: Change From Baseline to Week 52 in 4-Item Composite Score of Niemann Pick Type C Severity Scale (NPC-SS) Score | Baseline, Week 52 | Modified intent-to-treat (mITT) population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, number analyzed = participants evaluable at specified time-point. | score on a scale | MEAN |
| PRIMARY | Parts A/B: Number of Participants Classified With Each Score on the Clinician Global Impression of Change (CGIC) at Week 52 | Week 52 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Parts A/B: Change From Baseline to Week 52 in NPC-SS Total Score (Excluding Hearing and Auditory Brainstem Response [ABR]) | Baseline, Week 52 | mITT population included all randomized participants who received at least one treatment. As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, overall number of participants analyzed = participants evaluable for this outcome measure. Number analyzed = participants evaluable at specified time-point. | score on a scale | MEAN |
| SECONDARY | Parts A/ B: Number of Participants Classified as CGIC Responders at Week 52 | Week 52 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Parts A/B: Number of Participants Classified as Responders on NPC-SS Total Score (Excluding Hearing and ABR) at Week 52 | Week 52 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Parts A/B: EQ-5D-3L Questionnaire Visual Analog Scale (VAS) Score (for Health Status) at Baseline and at Week 52 | Baseline, Week 52 | mITT population included all randomized participants who received at least one treatment. As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, overall number of participants analyzed = participants evaluable for this outcome measure. Number analyzed = participants evaluable at specified time-point. | score on a scale | MEAN |
| SECONDARY | Parts A/B: Number of Participants Treated for at Least 6 Months Who Qualified for the Rescue Option | Baseline up to Week 26 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Parts A/B: Change From Baseline to Week 52 in Each of the 9 Clinical Domains of the NPC-SS | Baseline, Week 52 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, Number analyzed = participants evaluable at specified time-point. | score on a scale | MEAN |
| SECONDARY | Parts A/B: Change From Baseline to Week 52 in the Total NPC-SS (With Hearing Domain and ABR Modifier Included) | Baseline, Week 52 | mITT population included all randomized participants who received at least one treatment). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. Here, overall number of participants analyzed = participants evaluable for this outcome measure, and number analyzed = participants evaluable at specified time-point. | score on a scale | MEAN |
| SECONDARY | Parts A/B: Time to One Point Increase (Worsening) in NPC-SS Composite Score | Baseline up to Week 52 | mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study. | Days | MEDIAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group88
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Parts A/B: Change From Baseline to Week 52 in 4-Item Composite Score of Niemann Pick Type C Severity Scale (NPC-SS) Score | Sham Control | Baseline | 8.1 | Standard Deviation 4.27 | score on a scale |
| Parts A/B: Change From Baseline to Week 52 in 4-Item Composite Score of Niemann Pick Type C Severity Scale (NPC-SS) Score | Adrabetadex | Baseline | 8.8 | Standard Deviation 3.12 | score on a scale |
| Parts A/B: Change From Baseline to Week 52 in 4-Item Composite Score of Niemann Pick Type C Severity Scale (NPC-SS) Score | Sham Control | Change from Baseline at Week 52 | -0.1 | Standard Deviation 1.55 | score on a scale |
| Parts A/B: Change From Baseline to Week 52 in 4-Item Composite Score of Niemann Pick Type C Severity Scale (NPC-SS) Score | Adrabetadex | Change from Baseline at Week 52 | 0.0 | Standard Deviation 2.45 | score on a scale |
| Parts A/B: Number of Participants Classified With Each Score on the Clinician Global Impression of Change (CGIC) at Week 52 | Sham Control | Markedly improved (1) | 0 | Participants | |
| Parts A/B: Number of Participants Classified With Each Score on the Clinician Global Impression of Change (CGIC) at Week 52 | Adrabetadex | Markedly improved (1) | 0 | Participants | |
| Parts A/B: Number of Participants Classified With Each Score on the Clinician Global Impression of Change (CGIC) at Week 52 | Sham Control | Moderately improved (2) | 1 | Participants | |
| Parts A/B: Number of Participants Classified With Each Score on the Clinician Global Impression of Change (CGIC) at Week 52 | Adrabetadex | Moderately improved (2) | 5 | Participants | |
| Parts A/B: Number of Participants Classified With Each Score on the Clinician Global Impression of Change (CGIC) at Week 52 | Sham Control | Minimally improved (3) | 4 | Participants | |
| Parts A/B: Number of Participants Classified With Each Score on the Clinician Global Impression of Change (CGIC) at Week 52 | Adrabetadex | Minimally improved (3) | 9 | Participants |
1 to 10 of 88Source ClinicalTrials.gov
See all 88 results by groupParticipant flow48
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part A | STARTED | Part A: Adrabetadex 900 mg | 3 | |
| Part A | STARTED | Part A: Adrabetadex 1200 mg | 3 | |
| Part A | STARTED | Part A: Adrabetadex 1800 mg | 3 | |
| Part A | STARTED | Part A: Sham Control | 3 | |
| Part A | STARTED | Part B: Adrabetadex 900 mg | 0 | |
| Part A | STARTED | Part B: Sham Control | 0 | |
| Part A | Received at Least One Dose of Study Drug | Part A: Adrabetadex 900 mg | 3 | |
| Part A | Received at Least One Dose of Study Drug | Part A: Adrabetadex 1200 mg | 3 | |
| Part A | Received at Least One Dose of Study Drug | Part A: Adrabetadex 1800 mg | 3 | |
| Part A | Received at Least One Dose of Study Drug | Part A: Sham Control | 3 |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 participant flowAdverse events1,110
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 | Anaemia | Blood and lymphatic system disorders | Part A: Adrabetadex 900 mg | 0 | 3 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | Part A: Adrabetadex 1200 mg | 0 | 3 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | Part A: Adrabetadex 1800 mg | 0 | 3 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | Part A: Sham Control | 0 | 3 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | Part B: Adrabetadex 900 mg | 2 | 29 | 2 |
| other | Anaemia | Blood and lymphatic system disorders | Part B: Sham Control | 1 | 15 | 1 |
| other | Deafness | Ear and labyrinth disorders | Part A: Adrabetadex 900 mg | 0 | 3 | 0 |
| other | Deafness | Ear and labyrinth disorders | Part A: Adrabetadex 1200 mg | 0 | 3 | 0 |
| other | Deafness | Ear and labyrinth disorders | Part A: Adrabetadex 1800 mg | 1 | 3 | 1 |
| other | Deafness | Ear and labyrinth disorders | Part A: Sham Control | 0 | 3 | 0 |
1 to 10 of 1,110Source ClinicalTrials.gov
See all 1,110 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part A: Adrabetadex 900 mg | 0 | 3 |
| Total, all-cause mortality | Part A: Adrabetadex 1200 mg | 0 | 3 |
| Total, all-cause mortality | Part A: Adrabetadex 1800 mg | 1 | 3 |
| Total, all-cause mortality | Part A: Sham Control | 0 | 3 |
| Total, all-cause mortality | Part B: Adrabetadex 900 mg | 0 | 29 |
| Total, all-cause mortality | Part B: Sham Control | 1 | 15 |
| Total, other adverse events | Part A: Adrabetadex 900 mg | 3 | 3 |
| Total, other adverse events | Part A: Adrabetadex 1200 mg | 3 | 3 |
| Total, other adverse events | Part A: Adrabetadex 1800 mg | 3 | 3 |
| Total, other adverse events | Part A: Sham Control | 3 | 3 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40525490 | DERIVED |
Source PubMed
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 comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Adrabetadex vs Sham Control: Parts A/B: Number of Participants Treated for at Least 6 Months Who Qualified for the Rescue Option | Trial comparisons |
| Adrabetadex vs Sham Control: Parts A/ B: Number of Participants Classified as CGIC Responders at Week 52 | Trial comparisons |
| Adrabetadex vs Sham Control: Parts A/B: Number of Participants Classified as Responders on NPC-SS Total Score (Excluding Hearing and ABR) at Week 52 | 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.
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