Trial · NCT03729362
Registered outcomes33
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 33
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD) | Baseline, Week 52 | The efficacy of cipaglucosidase alfa/miglustat co-administration on ambulatory function was measured by the 6MWT. The 6MWD, measured in meters, is the distance walked on the 6MWT. A greater distance indicated greater endurance. An increase from baseline indicated improvement. |
| Secondary | Change From Baseline to Week 52 in Sitting Forced Vital Capacity (FVC; % Predicted) | Baseline, Week 52 | The efficacy of cipaglucosidase alfa/miglustat co-administration on pulmonary function was measured by sitting FVC (% predicted). FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. |
| Secondary | Change From Baseline to Week 52 in the Manual Muscle Test (MMT) Score for the Lower Extremities | Baseline, Week 52 | The total score for the MMT lower extremity strength included the following 8 body parts: right/left hip flexion, right/left hip abduction, right/left knee flexion and right/left knee extension. The MMT lower extremity score ranged from 0 to 40, with lower scores indicating weaker muscle strength. An increase from baseline indicated increased muscle strength. |
| Secondary | Change From Baseline to Week 26 in 6MWD | Baseline, Week 26 | The 6MWD, measured in meters, is the distance walked on the 6MWT. |
| 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 | Physical Function Short Form 20a (v2.0) consisted of 20 questions. The first 14 questions were each scored on a scale from 1 to 5 as follows: 1 = unable to do; 2 = with much difficulty; 3 = with some difficulty; 4 = with a little difficulty; 5 = without any difficulty; the next 6 questions were each scored on a scale from 1 to 5 as follows: 1 = cannot do; 2 = quite a lot; 3 = somewhat; 4 = very little; 5 = not at all. The total score was calculated by summing up scores (1 to 5) across all items. Total scores range from 20 to 100. A higher score represented a better outcome. |
| Secondary | Change From Baseline to Week 52 in the Total Score for the PROMIS® - Fatigue | Baseline, Week 52 | Fatigue Short Form 8a consisted of 6 questions, each scored on a scale from 1 to 5 as follows: 1 = not at all; 2 = a little bit; 3 = somewhat; 4 = quite a bit; 5 = very much; and 2 questions, each scored on a scale from 1 to 5 as follows: 1 = never; 2 = rarely; 3 = sometimes; 4 = often; 5 = always. The total score was calculated by summing up scores (1 to 5) across all items. A lower score represented lower fatigue symptoms. |
| Secondary | Change From Baseline to Week 52 in the Total Score for the Gait, Stairs, Gowers' Maneuver, and Chair (GSGC) | Baseline, Week 52 | The GSGC consisted of a 10-meter walk for evaluation of gait, a 4-stair climb, Gowers' maneuver, and arising from a chair. Results of the GSGC included the time required to complete the individual tests, individual scores for each of the tests (1 to 7 points for each of gait, 4-stair climb, and Gowers' maneuver, and 1 to 6 points for arising from a chair), and a total score. GSGC total score was the sum of the component scores from the 4 functional tests. The total score ranged from a minimum of 4 points (normal performance) to a maximum of 27 points (worst performance). |
| Secondary | Change From Baseline to Week 52 in Rasch-Built Pompe-Specific Activity (R-PAct) Total Score | Baseline, Week 52 | The R-PAct scale was an 18-item questionnaire to measure limitations in activities and restriction in social participation. Possible responses to questions were as follows: unable to perform, able to perform, but with difficulty, and able to perform without difficulty. The total score was calculated by summing up the observed scores across the 18 items and it ranged from 0 to 36, with higher values representing lower level of disease impact on the muscles. |
| 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 | The EuroQol Visual Analogue Scale (EQ VAS) is a vertical visual analogue scale that records the respondent's own assessment of his or her overall health status at the time of completion. Scores range from 0 (the worst health you can imagine) to 100 (the best health you can imagine). Higher EQ VAS scores represent an improved sense of overall health while lower scores represent a worsening of overall health. |
| Secondary | Change From Baseline to Week 52 in Sitting Slow Vital Capacity (SVC) % Predicted | Baseline, Week 52 | SVC is a standard pulmonary function test used to quantify respiratory muscle weakness. |
| Secondary | Change From Baseline to Week 52 in Maximal Inspiratory Pressure (MIP) % Predicted | Baseline, Week 52 | The percent predicted values of MIP were calculated as: % predicted = (actual result / predicted result)\* 100, where the predicted results were obtained using the reference equations from Uldry and Fitting (1995). |
| Secondary | Change From Baseline to Week 52 in Maximal Expiratory Pressure (MEP) % Predicted | Baseline, Week 52 | The percent predicted values of MEP were calculated as: % predicted = (actual result / predicted result) \* 100, where the predicted results were obtained using the reference equations from Uldry and Fitting (1995). |
| Secondary | Change From Baseline to Week 52 in Sniff Nasal Inspiratory Pressure (SNIP) % Predicted | Baseline, Week 52 | The percent predicted values of SNIP were calculated as: % predicted = (actual result / predicted result) \* 100, where the predicted results were obtained using the reference equations from Evans and Whitelaw (2009). |
| Secondary | Change From Baseline to Week 52 in % Predicted 6MWD | Baseline, Week 52 | The % predicted 6MWD = (actual 6MWD / predicted 6MWD) \* 100. The predicted values were calculated using Enright And Sherrill 1998 Reference Equations. |
| Secondary | Change From Baseline to Week 52 in the Quantitative Muscle Test (QMT) Values | Baseline, Week 52 | QMT was measured using the hand-held dynamometer. Larger values (in kg) indicated greater muscle strength. |
| Secondary | Change From Baseline to Week 52 in Other MMT Scores | Baseline, Week 52 | Each manual muscle test was evaluated on a scoring scale from 0 to 5, as follows: 0 = no muscle movement; 1 = visible muscle movement, but no movement at the joint; 2 = movement at the joint, but not against gravity; 3 = movement against gravity, but not against added resistance; 4 = movement against resistance, but less than normal; 5 = normal strength. Upper extremity score was the sum of scores for right/left shoulder abduction, right/left shoulder adduction, right/left elbow extension, and right/left elbow flexion, with the total score ranging from 0 to 40. Proximal muscle group score, the sum of scores for right/left hip flexion, right/left hip abduction, right/left shoulder abduction, and right/left shoulder adduction, with the total score ranging from 0 to 40. MMT total score was the sum of the lower and upper extremity scores and ranged from 0 to 80. Lower scores indicated lower overall muscle strength. An increase from baseline indicated improvement in muscle strength. |
| Secondary | Change From Baseline to Week 52 in Maximum Vital Capacity (Maximum VC) % Predicted | Baseline, Week 52 | Maximum VC is the greater of the two VC values (FVC or SVC). |
| Secondary | Change From Baseline to Week 52 in PROMIS-Dyspnea and Upper Extremities Total Scores | Baseline, Week 52 | The Upper Extremities Short Form 7a consisted of 7 items each scored on a decreasing scale from 1 to 5 as follows: 1 = unable to do; 2 = with much difficulty; 3 = with some difficulty; 4 = with a little difficulty; 5 = without any difficulty. Dyspnea Severity Short Form 10a consisted of 10 items each scored on a scale from 0 to 3 as follows: 0 = no shortness of breath; 1 = mildly short of breath; 2 = moderately short of breath; 3 = severely short of breath. A total score was generated for each instrument by adding up each item. Total scores for upper extremities range from 7 to 35. Total scores for dyspnea range from 0 to 30. A higher score for upper extremities represented improvement in symptoms. A lower score for dyspnea severity represented improvement in symptoms. |
| 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 | Motor function test assessed the time to complete individual GSGC component tests (10-meter walk, 4- stair climb, Gowers' maneuver, and arise from a chair) and the TUG test. The TUG test assessed the time a subject needed to rise from a chair, walk 3 meters, turn around, walk back to the chair, and sit down. |
| Secondary | Physician's Global Impression of Change (PGIC) Overall Status at Week 52 | Week 52 | Physician's Global Impression of Change is based on a single item that is scored on a 7-point rating scale ranging from 1 "very much worse" to 7 "very much improved". A tertiary response variable (improving, declining, stable) was defined as follows: "Improving", which consisted of improved, moderately improved, and very much improved; "Declining", which consisted of worse, moderately worse, and very much worse; and "Stable", which equaled to no change. |
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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