Trial · NCT04231266
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 12 of 12
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Muscle strength of ankle dorsiflexion, knee flexion, knee extension, shoulder abduction, elbow flexion and grip measured by fixed-frame Quantitative Muscle Assessment (QMA) | Minimum 2 years, until 24 months from randomization of last subject | The primary endpoint is the change in muscle strength decline under treatment compared to placebo. The primary analysis is based on the disease progression ratio (γ) comparing the rate of progression from baseline until last visit, under placebo to that under treatment. |
| Secondary | Inclusion Body Myositis Functional Rating Scale (IBMFRS) | Minimum 2 years, until 24 months from randomization of last subject | Change in patient-reported function as measured by the Inclusion Body Myositis Functional Rating Scale (IBMFRS). |
| Other | Adverse Events | Minimum 2 years, until 24 months from randomization of last subject | Safety and tolerability will be evaluated by comparing the frequency of adverse events (AEs) across groups, collected using information from in-person assessments, clinical laboratory tests, vital signs, electronic diary reports, and physical examinations. |
| Other | Correlation muscle strength measured Exploratory GNEM Functional Questions (ExGNEM) | Minimum 2 years, until 24 months from randomization of last subject | Evaluate the effect of ManNAc on patient-reported physical functioning assessed by ExGNEM, a six-question rating scale that assesses lower body function. |
| Other | Adult Myopathy Assessment Tool | Minimum 2 years, until 24 months from randomization of last subject | Evaluate the effect of ManNAc on physical function as measured by the Adult Myopathy Assessment Tool (AMAT), a 13-item standardized test that assesses physical performance, to be performed at baseline and every 6 months thereafter until completion of the study. |
| Other | Six-Minute Walk Test | Minimum 2 years, until 24 months from randomization of last subject | Evaluate the effect of ManNAc on physical function as measured by the Six-Minute Walk Test (whenever possible) to be performed at baseline and every 6 months thereafter until completion of the study. |
| Other | Timed Up and Go Test | Minimum 2 years, until 24 months from randomization of last subject | Evaluate the effect of ManNAc on physical function as measured by the Timed Up and Go, performance test to evaluate functional mobility, to be performed at baseline and every 6 months thereafter until completion of the study. |
| Other | Functional Reach Test | Minimum 2 years, until 24 months from randomization of last subject | Evaluate the effect of ManNAc on physical function as measured by the Functional Reach, a measure of stability, to be performed at baseline and every 6 months thereafter until completion of the study. |
| Other | Jebsen Hand Function Test | Baseline and every 12 months thereafter | Evaluate the effect of ManNAc on physical function as measured by the Jebsen Hand Function Test, a performance measure which assesses unilateral hand function, to be performed at baseline and every 6 months thereafter until completion of the study. |
| Other | Inclusion Body Myositis Functional Rating Scale | Minimum 2 years, until 24 months from randomization of last subject | Evaluate effect of ManNAc on activities of daily living (ADLs) compared to placebo as measured by the Inclusion Body Myositis Functional Rating Scale (IBMFRS), a patient-reported outcome completed at baseline, and every 6 months thereafter until completion of the study. |
| Other | Human Activity Profile | Minimum 2 years, until 24 months from randomization of last subject | Evaluate effect of ManNAc on activities of daily living (ADLs) compared to placebo as measured by the Human Activity Profile, a patient-reported outcome completed at baseline, and every 6 months thereafter until completion of the study. |
| Other | Activities-specific Balance Confidence (ABC) scale | Minimum 2 years, until 24 months from randomization of last subject | Evaluate effect of ManNAc on activities of daily living (ADLs) compared to placebo as measured by the Activities-specific Balance Confidence (ABC) scale, a patient-reported outcome completed at baseline, and every 6 months thereafter until completion of the study. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
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 list from software.
Twenty rows a page, as JSON. No key.
GET https://conductscience.org/api/trials/v1/NCT04231266/registered