Trial · NCT04671472
Efficacy Confirmation Study of NPC-09
NCT04671472, Phase 3, Completed, Nobelpharma
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Efficacy Confirmation Study of NPC-09. Trials, record NCT04671472. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04671472
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy Confirmation Study of NPC-09
- NCT number
- NCT04671472
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 14 participants, actual
- Lead sponsor
- Nobelpharma
- Conditions
- GNE Myopathy, Distal Myopathy With Rimmed Vacuoles (DMRV), Hereditary Inclusion Body Myopathy (hIBM), Nonaka Disease
- Interventions
- NPC-09, NPC-09 placebo
- First posted
- 2020-12-17
- Last updated
- 2022-05-16
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change from Baseline in the upper extremity composite score (the sum of the average of the right and left muscle strength measured by HHD (Hand held dynamometer) (kg) for grip, shoulder abductors, elbow flexors and elbow extensors) | Week 48 | Muscle strength based on the maximum voluntary isometric contraction (MVIC) is measured. |
| Secondary | The effective rate in comprehensive judgement by the investigator | Week 48 | The investigator judges based on below 1 \~ 4 items. 1. The manual muscle testing (0 - 5) or grip strength (kg) 2. the upper extremity composite score (kg) 3. the change in the upper extremity composite score (kg) compared with the transition of non-active drug administered GNE myopathy patients 4. the other secondary endpoints |
| Secondary | Change from Baseline in GNE Myopathy Functional Activity Scale (GNEM-FAS) upper extremity domain score | Week 48 | Upper extremity functional activity is assessed using the upper extremity domain sore of the GNEM-FAS instrument. It consists of 8 items (score 0-4 for each item), total domain score is 0 to 32 with higher scores representing greater activity. |
| Secondary | Changes from Baseline in Individual muscle strength: Grip, shoulder abductors, elbow flexors, and elbow extensors comprising the upper extremity composite score (kg) | Week 48 | Muscle strength based on the maximum voluntary isometric contraction (MVIC) is measured. |
| Secondary | Change from baseline in knee extensors muscle strength (kg) | Week 48 | Muscle strength based on the maximum voluntary isometric contraction (MVIC) is measured. |
| Secondary | Change from Baseline in GNE Myopathy Functional Activity Scale (GNEM-FAS) mobility domain score | Week 48 | Mobility functional activity is assessed using the mobility domain sore of the GNEM-FAS instrument. It consists of 10 items (score 0-4 for each item), total domain score is 0 to 40 with higher scores representing greater activity. |
| Secondary | Change from Baseline in GNE Myopathy Functional Activity Scale (GNEM-FAS) self-care domain score | Week 48 | Self-care functional activity is assessed using the self-care domain sore of the GNEM-FAS instrument. It consists of 7 items (score 0-4 for each item), total domain score is 0 to 28 with higher scores representing greater activity. |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37568154 | 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 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/NCT04671472
GET https://conductscience.org/api/trials/v1/NCT04671472/registered