Trial · NCT03729362
Outcomes33
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
1 to 20 of 33
Download this page as JSON| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD) | Baseline, Week 52 | Analysis was performed on Intent-to-treat (ITT)-observed (OBS) population. Number of participants analyzed are based on those who had 6MWD result at Week 52. In addition, 1 outlier subject in the alglucosidase alfa/placebo group was not included in the analysis. | meter | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in Sitting Forced Vital Capacity (FVC; % Predicted) | Baseline, Week 52 | Analysis was performed on ITT-Last Observation Carried Forward (LOCF) population. In the cipaglucosidase alfa/miglustat group, 1 subject had no post-baseline values and was not included in the analysis. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis. | percentage of predicted FVC | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in the Manual Muscle Test (MMT) Score for the Lower Extremities | Baseline, Week 52 | Analysis was performed on the ITT-LOCF population. In the cipaglucosidase alfa/miglustat group, 1 subject had neither baseline nor post-baseline values and 4 subjects had no post-baseline values; these subjects were not included in the analysis. In the alglucosidase alfa/placebo group, 3 subjects had neither baseline nor post-baseline values, and were not included in the analysis. In addition, the outlier subject was not included in the analysis. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 26 in 6MWD | Baseline, Week 26 | Analysis was performed on ITT-LOCF population. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis. | meter | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in the Total Score for the Patient- Reported Outcomes Measurement Information System (PROMIS®) - Physical Function | Baseline, Week 52 | Analysis was performed on ITT-LOCF population. In the cipaglucosidase alfa/miglustat group, 1 subject had neither baseline nor post-baseline values, and was not included in the analysis. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in the Total Score for the PROMIS® - Fatigue | Baseline, Week 52 | Analysis was performed on ITT-LOCF population. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in the Total Score for the Gait, Stairs, Gowers' Maneuver, and Chair (GSGC) | Baseline, Week 52 | Analysis was performed on ITT-LOCF population. In the cipaglucosidase alfa/miglustat group, 11 subjects had neither baseline nor post-baseline values, and 2 had no post-baseline values; these subjects were not included in the analysis. In the alglucosidase alfa/placebo group, 5 subjects had neither baseline nor post-baseline values, and 2 had no post-baseline values; these subjects were not included in the analysis. In addition, the outlier subject was not included in the analysis. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in Rasch-Built Pompe-Specific Activity (R-PAct) Total Score | Baseline, Week 52 | Analysis was performed on ITT-LOCF population. In the cipaglucosidase alfa/miglustat group, 16 subjects had neither baseline nor post-baseline values, and were not included in the analysis. In the alglucosidase alfa/placebo group, 4 subjects had neither baseline nor post-baseline values, and were not included in the analysis. In addition, the outlier subject was not included in the analysis. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in European Quality of Life-5 Dimensions 5 Response Levels (EQ-5D-5L) Based on the EuroQol Visual Analogue Scale (EQ VAS) Quantitative Score | Baseline, Week 52 | Analysis was performed on ITT-LOCF population. In the cipaglucosidase alfa/miglustat group, 1 subject had neither baseline nor post-baseline values, and was not included in the analysis. In the alglucosidase alfa/placebo group, 1 subject had neither baseline nor post-baseline values, and was not included in the analysis. In addition, the outlier subject was not included in the analysis. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in Sitting Slow Vital Capacity (SVC) % Predicted | Baseline, Week 52 | Analysis was performed on ITT-LOCF population. In the cipaglucosidase alfa/miglustat group, 2 subjects had no post-baseline values and were not included in the analysis. In the alglucosidase alfa/placebo group, 1 subject had no baseline value, 1 subject had no post-baseline values, and they were not included in the analysis. In addition, the outlier subject was not included in the analysis. | percentage of predicted SVC | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in Maximal Inspiratory Pressure (MIP) % Predicted | Baseline, Week 52 | Analysis was performed on ITT-LOCF population. In the cipaglucosidase alfa/miglustat group, 1 subject had no post-baseline value and was not included in the analysis. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis. | percentage of predicted MIP | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in Maximal Expiratory Pressure (MEP) % Predicted | Baseline, Week 52 | Analysis was performed on ITT-LOCF population. In the cipaglucosidase alfa/miglustat group, 1 subject had no post-baseline value and was not included in the analysis. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis. | percentage of predicted MEP | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in Sniff Nasal Inspiratory Pressure (SNIP) % Predicted | Baseline, Week 52 | Analysis was performed on ITT-LOCF population. In the cipaglucosidase alfa/miglustat group, 1 subject had no post-baseline value and was not included in the analysis. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis. | percentage of predicted SNIP | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in % Predicted 6MWD | Baseline, Week 52 | Analysis was performed on ITT-LOCF population. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis. | percentage of predicted 6MWD | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in the Quantitative Muscle Test (QMT) Values | Baseline, Week 52 | Analyses were performed on ITT-LOCF population. Number of participants analyzed for each of the QMT parameters displayed are based upon the number of participants who had both baseline and post-baseline values for each respective QMT parameter. In addition, the outlier subject in the alglucosidase alfa/placebo group was not included in the analyses. | kilogram | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in Other MMT Scores | Baseline, Week 52 | Analyses were performed on ITT-LOCF population. Number of participants analyzed for each of the MMT parameters displayed are based upon the number of participants who had both baseline and post-baseline values for each respective MMT parameter. In addition, the outlier subject in the alglucosidase alfa/placebo group was not included in the analyses. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in Maximum Vital Capacity (Maximum VC) % Predicted | Baseline, Week 52 | Analysis was performed on ITT-LOCF population. In the cipaglucosidase alfa/miglustat group, 1 subject had no post-baseline value and was not included in the analysis. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis. | percentage of predicted maximum VC | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 52 in PROMIS-Dyspnea and Upper Extremities Total Scores | Baseline, Week 52 | Analyses were performed on ITT-LOCF population. Number of participants analyzed for each of the PROMIS total scores displayed are based upon the number of participants who had both baseline and post-baseline values for each respective PROMIS total score (PROMIS-Dyspnea and Upper Extremities Total Scores). In addition, the outlier subject in the alglucosidase alfa/placebo group was not included in the analyses. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the Time to Complete Individual GSGC Component Tests and Timed Up and Go (TUG) Test at Week 52 | Baseline, Week 52 | Analyses were performed on ITT-LOCF population. Number of participants analyzed for each of the GSGC components and the TUG test displayed are based upon the number of participants who had both baseline and post-baseline values for each respective GSGC component and the TUG test. In addition, the outlier subject in the alglucosidase alfa/placebo group was not included in the analyses. | seconds | LEAST_SQUARES_MEAN |
| SECONDARY | Physician's Global Impression of Change (PGIC) Overall Status at Week 52 | Week 52 | Analysis was performed on ITT-OBS population. Number of participants analyzed are based on those who had PGIC data at Week 52. In addition, the outlier subject in the alglucosidase alfa/placebo group was not included in the analysis. | Participants | NUMBER |
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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