Trial · NCT01942590
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 13 of 13
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement | Any point up to week 52 | Worsening muscle involvement, as defined by \>3x increase in CK from baseline that is \>2x the upper limit of normal |
| Primary | Number of Participants With a Change in Aspartate Aminotransferase (AST), Alanine Transaminase (ALT), and Bilirubin Representing Liver Toxicity | Any point up to week 52 | Liver toxicity, as defined by a \>3x increase in AST or ALT from the respective baseline values and/or an increase in direct, indirect or total bilirubin of \>3x the upper limit of normal |
| Secondary | Change in 6 Minute Walk Test | Baseline, week 18 | Assess exercise tolerance in study patients; test administered by physical therapist. Subjects were asked to walk for 6 minutes, unassisted. The distance walked was recorded in meters. |
| Secondary | Change in 6 Minute Walk Test | Baseline, week 52 | Assess exercise tolerance in study patients; test administered by physical therapist. Subjects were asked to walk for 6 minutes, unassisted. The distance walked was recorded in meters. |
| Secondary | Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing | Baseline, Week 18 | Forced vital capacity (FVC) is the total amount of air exhaled during the lung function test. |
| Secondary | Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing | Baseline, Week 52 | Forced vital capacity (FVC) is the total amount of air exhaled during the lung function test. |
| Secondary | Change in Urinary Glc4 Biomarker | Baseline, Week 18 | The Glc4 biomarker is measured in urine and correlates with muscle glycogen content. It is a noninvasive measurement that serves as a biomarker for Pompe disease. |
| Secondary | Change in Urinary Glc4 Biomarker | Baseline, Week 52 | |
| Secondary | GSGC (Gait, Stairs, Gowers, Arising From a Chair.) | Baseline, Week 18, and Week 52 | The GSGC is a criterion referenced assessment designed to measure functional status and change in gross motor function over time and, in particular, to measure clinically relevant change. Consists of 4 components: Gait, Climbing Stairs, Gower's Manuever, Arising From a Chair. Lowest score 4 = normal muscle function, highest score 27 = unable to perform motor function tests. |
| Secondary | Quick Motor Function Test (QMFT) | Baseline, Week 18, and Week 52 | The QMFT is a criterion referenced assessment designed to measure functional status and change in gross motor function over time and, in particular, to measure clinically relevant change. Consists of 16 motor function tests. Lowest score 0 = unable to perform motor function tests, highest score 64 = normal muscle function. |
| Secondary | Late-Life Function and Disability Instrument (LLFDI) | Baseline, Week 18, Week 52 | The Late-Life Function \& Disability Instrument (Late-Life FDI) is an evaluative outcome instrument for community-dwelling older adults. Highest score 240 = normal function and no disability, lowest score 0 = low levels of frequency of participating in life tasks. |
| Secondary | Predicted Maximum Inspiration Pressure (MIP) | Baseline, Week 18, and Week 52 | MIP is a measurement of inspiratory muscle weakness, including weakness of the diaphragm. MIP is decreased in Pompe disease and reflects weakness of respiratory muscles. |
| Secondary | Maximum Expiratory Pressure (MEP) | Baseline, Week 18, and Week 52 | MEP reflects the strength of the abdominal muscles and other expiratory muscles. |
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.
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