Trial · NCT04138277
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 | Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug | Entire extension study (mean = 40.5 months on treatment) | Number of subjects with TEAE, TESAE, and TEAE leading to discontinuation during this long-term extension study |
| Secondary | Change From Baseline in 6-Minute Walk Distance (6MWD) | baseline, Week 208 | Motor function was measured using the 6-minute walk distance (meters) |
| Secondary | Change From Baseline in Sitting % Predicted Forced Vital Capacity (FVC) | baseline, Week 208 | Pulmonary function was measured by sitting % predicted forced vital capacity (FVC) |
| Secondary | Change From Baseline in Manual Muscle Testing (MMT) Lower Extremity Score | baseline, Week 208 | Strength was measured by manual muscle testing (MMT) using the Medical Research Council grading scale (0 to 5 points, with 5 indicating normal function). Change from baseline values \>0 represent improvement. |
| Secondary | Change From Baseline in the Total Score for Patient-reported Outcomes Measurement Information System (PROMIS®) - Physical Function | baseline, Week 208 | 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 in the Total Score for PROMIS® - Fatigue | baseline, Week 208 | 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 resulting in a total score range from 6 (minimum) to 30 (maximum). A lower score represented lower fatigue symptoms. |
| Secondary | Change From Baseline in Gait, Stairs, Gower, Chair (GSGC) Test | baseline, Week 208 | 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 in EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) Responses | baseline, Week 208 | The EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) questionnaire comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: Level 1=no problems, Level 2=slight problems, Level 3=moderate problems, Level 4=severe problems, and Level 5=extreme problems. In this categorical assessment, subjects were asked to indicate their health state by ticking the box next to the most appropriate statement in each of the 5 dimensions. Outcomes for change from baseline at Week 208 include 'no change', 'worsening' relative to baseline category, or 'improvement' relative to baseline category. Changes (worsening or improvement) are shown by magnitude of change (how many Levels) in each categorical assessment. |
| Secondary | Change From Baseline in Overall Physical Well-being (Subject's Global Impression of Change [SGIC], Question 1) | baseline, Week 208 | The Subject's Global Impression of Change overall physical well-being (question 1) is scored on a 7-point rating scale ranging from "very much worse" to "very much improved." |
| Secondary | Change From Baseline in Physician's Global Impression of Change (PGIC) Overall Status | baseline, Week 208 | The Physician's Global Impression of Change (PGIC) is designed to record the physician's assessment of the subject's status, taking into account the subject's signs and symptoms and other neuromuscular symptoms, and signs relative to their status at the Baseline Visit. The PGIC 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." |
| Secondary | Percent Change From Baseline in Creatine Kinase (U/L) | baseline, Week 208 | Percent change from baseline to Week 208 in the levels of biomarker creatine kinase (CK) |
| Secondary | Percent Change From Baseline in Urine Hex4 (mmol/Mol Creatinine) | baseline, Week 208 | Percent change from baseline to Week 208 in the levels of biomarker urine Hex4. |
| Secondary | Proportion of Subjects With Positive Anti-drug Antibodies at Baseline and Week 208 | baseline, Week 208 | Immunogenicity was assessed by the number (%) of subjects with positive specific anti-cipaglucosidase antibodies at baseline and at Week 208 |
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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