Trial · NCT02612129
Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C
NCT02612129, Phase 2, Phase 3, Completed, ZevraDenmark; results first posted 2023-06-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C. Trials, record NCT02612129. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02612129
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C
- NCT number
- NCT02612129
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 50 participants, actual
- Lead sponsor
- ZevraDenmark
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- arimoclomol, Placebo
- First posted
- 2015-11-23
- Last updated
- 2024-11-29
- Results first posted
- 2023-06-09
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in the Niemann-Pick Disease Type C (NPC) Disease Severity Assessed Based on the 5-domain NPCCSS Total Scores | Baseline to Month 12 | NPC disease severity was assessed based on the 5-domain NPC Clinical Severity Scale (NPCCSS). The 5-domain NPCCSS focuses on domains identified by participants, caregivers, and NPC experts as the most clinically relevant when assessing disease progression in NPC: Ambulation, fine motor skills, swallow, cognition, and speech. The scale is derived from the original 17-domain NPCCSS. Each domain is rated on a scale of 0-5 based on clinical assessments, observations, and interviews with participants/caregiver. The total score is a sum of the score of each of the 5 domains and ranges from 0-25, with a higher score indicating more severe clinical impairment. |
| Secondary | Percentage of Responders in Clinical Global Impression Scale of Improvement (CGI-I) - Defined as Percentage of Participants Where the CGI-I Score Remains Stable or Shows Improvement (This Outcome Measure Was Considered Co-primary by the FDA) | Month 12 | The CGI-I is a 7-point scale that rates total improvement of participant's condition. The clinician rates the participants from 1=Very much improved, 2=Much improved, 3=Minimally improved, 4=No change, 5=Minimally worse, 6=Much worse, or 7=Very much worse. Scores thus range from 1-7 with lower scores indicating greater improvement. Responders were the participants with a score of 1 or 2 at Month 12. |
| Secondary | Percentage of Responders in 5-domain NPCCSS - Defined as Participants Where the 5-domain NPCCSS Score Remains Stable or Improves as Compared to Baseline | Baseline to Month 12 | NPC disease severity was assessed based on the 5-domain NPC Clinical Severity Scale (NPCCSS). The 5-domain NPCCSS focuses on domains identified by participants, caregivers, and NPC experts as the most clinically relevant when assessing disease progression in NPC: Ambulation, fine motor skills, swallow, cognition, and speech. The scale is derived from the original 17-domain NPCCSS. Each domain is rated on a scale of 0-5 based on clinical assessments, observations, and interviews with participants/caregiver. The total score is a sum of the score of each of the 5 domains and ranges from 0-25, with a higher score indicating more severe clinical impairment. Stable was defined as a participant's total score for the 5 domains being the same at month 12 as at baseline. Improvement was defined as a participant's total score at month 12 being lower than at baseline. |
| Secondary | Time to Worsening | Baseline to Month 12 | Time to worsening was defined as the time until the participant reached the predefined minimal clinically important difference (MCID) of 2 points compared to baseline on the 5-domain NPC Clinical Severity Scale (NPCCSS). The 5-domain NPCCSS focuses on domains identified by participants, caregivers, and NPC experts as the most clinically relevant when assessing disease progression in NPC: Ambulation, fine motor skills, swallow, cognition, and speech. The scale is derived from the original 17-domain NPCCSS. Each domain is rated on a scale of 0-5 based on clinical assessments, observations, and interviews with participants/caregiver. The total score is a sum of the score of each of the 5 domains and ranges from 0-25, with a higher score indicating more severe clinical impairment. The values reported per group are the 25th percentile Kaplan-Meier estimates and 95% confidence interval. |
| Secondary | Percentage of Participants With Worsening | Months 6 and 12 | Worsening was defined as participants that have reached the predefined MCID of 2 points on their 5-domain NPC Clinical Severity Scale (NPCCSS). The 5-domain NPCCSS focuses on domains identified by participants, caregivers, and NPC experts as the most clinically relevant when assessing disease progression in NPC: Ambulation, fine motor skills, swallow, cognition, and speech. The scale is derived from the original 17-domain NPCCSS. Each domain is rated on a scale of 0-5 based on clinical assessments, observations, and interviews with participants/caregiver. The total score is a sum of the score of each of the 5 domains and ranges from 0-25, with a higher score indicating more severe clinical impairment. |
| Secondary | Change From Baseline in 17-domain NPCCSS Apart From Hearing Domains (i.e. Hearing and Auditory Brainstem Response) | Baseline to 6 and 12 months | The NPC Clinical Severity Scale (NPCCSS) is a disease-specific, clinician-reported outcome measure developed to characterize and quantify NPC disease progression. The 17-domain NPCCSS includes clinical signs and symptoms in nine major and eight minor domains, which are rated on scales of 0-5 (for the major domains) or 0-2 (for the minor domains). The total score is the sum of the score of each of the 17 domains and ranges from 0 to 61, with a high score indicating a more severe clinical impairment. |
| Secondary | Change From Baseline in 5-domain NPCCSS Score | Baseline to 6 months | The 5-domain NPC Clinical Severity Scale (NPCCSS) focuses on domains identified by participants, caregivers, and NPC experts as the most clinically relevant when assessing disease progression in NPC: Ambulation, fine motor skills, swallow, cognition, and speech. The scale is derived from the original 17-domain NPCCSS. Each domain is rated on a scale of 0-5 based on clinical assessments, observations, and interviews with participants/caregiver. The total score is a sum of the score of each of the 5 domains and ranges from 0-25, with a higher score indicating more severe clinical impairment. |
| Secondary | Changes From Baseline in Each Individual Domain of the NPCCSS | Baseline to 6 and 12 months | The NPC Clinical Severity Scale (NPCCSS) is a disease-specific, clinician-reported outcome measure developed to characterize and quantify disease progression. The 17-domain NPCCSS includes clinical signs and symptoms in nine major (ambulation, cognition, eye movement, fine motor, hearing, memory, seizures, speech, swallowing,) and eight minor (auditory brainstem response, behavior, gelastic cataplexy, hyperreflexia, incontinence, narcolepsy, psychiatric, respiratory problems) domains, which are rated on scales of 0-5 (for the major domains) or 0-2 (for the minor domains). A higher score indicates a more severe clinical impairment. |
| Secondary | Change From Baseline in the NPC Clinical Database (NPC-CDB) Score (Modified "Stampfer Score") | Baseline to 6 and 12 months | The NPC Clinical Database (NPC-cdb) score aims to reflect the current clinical status of the participant. The NPC-cdb score represents both historical symptoms and a current status. The test consists of ten areas: visceral signs, development, motor function, ocular-motor abnormalities, seizures/cataplexy/narcolepsy, cognitive abilities and memory, behavioral and psychiatric abnormalities, speech, hearing, and abilities in daily life. The current status score is a severity-weighted sum of 72 symptoms considered as disease-relevant at the time of assessment. Each symptom contributes with a score between 1 and 5, the maximum score is 125. An increase in score reflects a reduction in the participant's abilities. |
| Secondary | Percentage of Participants With Change From Baseline in Quality of Life (EQ-5D-Y) | Baseline to 6 and 12 months | The EQ-5D-Y descriptive system includes 5 descriptive items: Mobility, self-care, doing usual activities, having pain or discomfort, and feeling anxiety or depressed. Each dimension has 3 levels: No problems, some problems, and a lot of problems. The change in the 5 individual items of the EQ-5D-Y per participant was explored by using the pareto principle at 6 and 12 months to show the number (%) of participants who felt: * Better (better on at least one dimension and no worse in any other dimension), * Worse (worse in at least one dimension, and no better in any other dimension) |
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 | Change From Baseline in the Niemann-Pick Disease Type C (NPC) Disease Severity Assessed Based on the 5-domain NPCCSS Total Scores | Baseline to Month 12 | Full Analysis Set (FAS) included those participants who were randomized and who had received at least one dose of randomized treatment medication. Overall Number analyzed is the number of participants evaluated at a specified timepoint. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Responders in Clinical Global Impression Scale of Improvement (CGI-I) - Defined as Percentage of Participants Where the CGI-I Score Remains Stable or Shows Improvement (This Outcome Measure Was Considered Co-primary by the FDA) | Month 12 | FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. | percentage of responders | NUMBER |
| SECONDARY | Percentage of Responders in 5-domain NPCCSS - Defined as Participants Where the 5-domain NPCCSS Score Remains Stable or Improves as Compared to Baseline | Baseline to Month 12 | FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. | percentage of responders | NUMBER |
| SECONDARY | Time to Worsening | Baseline to Month 12 | FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. | months | NUMBER |
| SECONDARY | Percentage of Participants With Worsening | Months 6 and 12 | FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in 17-domain NPCCSS Apart From Hearing Domains (i.e. Hearing and Auditory Brainstem Response) | Baseline to 6 and 12 months | FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. Here, Number analyzed signifies number of participants evaluated at specified timepoint. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in 5-domain NPCCSS Score | Baseline to 6 months | FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Changes From Baseline in Each Individual Domain of the NPCCSS | Baseline to 6 and 12 months | FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. Here, Number analyzed signifies number of participants evaluated at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in the NPC Clinical Database (NPC-CDB) Score (Modified Stampfer Score) | Baseline to 6 and 12 months | FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Participants With Change From Baseline in Quality of Life (EQ-5D-Y) | Baseline to 6 and 12 months | FAS included those participants who were randomized and who had received at least one dose of randomized treatment medication. Here, Overall Number of Participants Analyzed is the number of participants with data for the outcome measure and Number analyzed signifies number of participants evaluated at specified timepoint. | percentage of participants | NUMBER |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group200
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in the Niemann-Pick Disease Type C (NPC) Disease Severity Assessed Based on the 5-domain NPCCSS Total Scores | Arimoclomol (12-month Double-blind Phase) | 0.76 | 95% Confidence Interval -0.05 to 1.56 | score on a scale | |
| Change From Baseline in the Niemann-Pick Disease Type C (NPC) Disease Severity Assessed Based on the 5-domain NPCCSS Total Scores | Placebo (12-month Double-blind Phase) | 2.15 | 95% Confidence Interval 1.05 to 3.25 | score on a scale | |
Percentage of Responders in Clinical Global Impression Scale of Improvement (CGI-I) - Defined as Percentage of Participants Where the CGI-I Score Remains Stable or Shows Improvement (This Outcome Measure Was Considered Co-primary by the FDA) | Arimoclomol (12-month Double-blind Phase) | 58.8 | percentage of responders | ||
Percentage of Responders in Clinical Global Impression Scale of Improvement (CGI-I) - Defined as Percentage of Participants Where the CGI-I Score Remains Stable or Shows Improvement (This Outcome Measure Was Considered Co-primary by the FDA) | Placebo (12-month Double-blind Phase) | 56.3 | percentage of responders | ||
| Percentage of Responders in 5-domain NPCCSS - Defined as Participants Where the 5-domain NPCCSS Score Remains Stable or Improves as Compared to Baseline | Arimoclomol (12-month Double-blind Phase) | 50.0 | percentage of responders | ||
| Percentage of Responders in 5-domain NPCCSS - Defined as Participants Where the 5-domain NPCCSS Score Remains Stable or Improves as Compared to Baseline | Placebo (12-month Double-blind Phase) | 37.5 | percentage of responders | ||
| Time to Worsening | Arimoclomol (12-month Double-blind Phase) | 5.2 | 95% Confidence Interval 2.9 to 12.0 | months | |
| Time to Worsening | Placebo (12-month Double-blind Phase) | 5.5 | 95% Confidence Interval 1.0 to 6.5 | months | |
| Percentage of Participants With Worsening | Arimoclomol (12-month Double-blind Phase) | Worsening at Month 6 | 35.3 | percentage of participants | |
| Percentage of Participants With Worsening | Placebo (12-month Double-blind Phase) | Worsening at Month 6 | 50.0 | percentage of participants |
1 to 10 of 200Source ClinicalTrials.gov
See all 200 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Single Dose Treatment Period | STARTED | Arimoclomol Single PK Dose | 28 | |
| Single Dose Treatment Period | STARTED | Arimoclomol (12-month Double-blind Phase) | 0 | |
| Single Dose Treatment Period | STARTED | Placebo (12-month Double-blind Phase) | 0 | |
| Single Dose Treatment Period | COMPLETED | Arimoclomol Single PK Dose | 27 | |
| Single Dose Treatment Period | COMPLETED | Arimoclomol (12-month Double-blind Phase) | 0 | |
| Single Dose Treatment Period | COMPLETED | Placebo (12-month Double-blind Phase) | 0 | |
| Single Dose Treatment Period | NOT COMPLETED | Arimoclomol Single PK Dose | 1 | |
| Single Dose Treatment Period | NOT COMPLETED | Arimoclomol (12-month Double-blind Phase) | 0 | |
| Single Dose Treatment Period | NOT COMPLETED | Placebo (12-month Double-blind Phase) | 0 | |
| Continuous Treatment Period | STARTED | Arimoclomol Single PK Dose | 0 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events438
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 | Abdominal Pain Upper | Gastrointestinal disorders | Arimoclomol Single PK Dose | 1 | 28 | |
| other | Abdominal Pain Upper | Gastrointestinal disorders | Arimoclomol (12-month Double-blind Phase) | 1 | 34 | |
| other | Abdominal Pain Upper | Gastrointestinal disorders | Placebo (12-month Double-blind Phase) | 0 | 16 | |
| other | Pyrexia | General disorders | Arimoclomol Single PK Dose | 1 | 28 | |
| other | Pyrexia | General disorders | Arimoclomol (12-month Double-blind Phase) | 6 | 34 | |
| other | Pyrexia | General disorders | Placebo (12-month Double-blind Phase) | 3 | 16 | |
| other | Drug Hypersensitivity | Immune system disorders | Arimoclomol Single PK Dose | 1 | 28 | |
| other | Drug Hypersensitivity | Immune system disorders | Arimoclomol (12-month Double-blind Phase) | 1 | 34 | |
| other | Drug Hypersensitivity | Immune system disorders | Placebo (12-month Double-blind Phase) | 0 | 16 | |
| other | Upper Respiratory Tract Infection | Infections and infestations | Arimoclomol Single PK Dose | 1 | 28 |
1 to 10 of 438Source ClinicalTrials.gov
See all 438 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Arimoclomol Single PK Dose | 0 | 28 |
| Total, all-cause mortality | Arimoclomol (12-month Double-blind Phase) | 1 | 34 |
| Total, all-cause mortality | Placebo (12-month Double-blind Phase) | 0 | 16 |
| Total, other adverse events | Arimoclomol Single PK Dose | 4 | 28 |
| Total, other adverse events | Arimoclomol (12-month Double-blind Phase) | 30 | 34 |
| Total, other adverse events | Placebo (12-month Double-blind Phase) | 13 | 16 |
| Total, serious adverse events | Arimoclomol Single PK Dose | 2 | 28 |
| Total, serious adverse events | Arimoclomol (12-month Double-blind Phase) | 5 | 34 |
| Total, serious adverse events | Placebo (12-month Double-blind Phase) | 6 | 16 |
Source ClinicalTrials.gov
Papers2
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/NCT02612129
GET https://conductscience.org/api/trials/v1/NCT02612129/results?page=2