Trial · NCT03729362
A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
NCT03729362, Phase 3, Completed, Amicus Therapeutics; results first posted 2023-09-11
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease. Trials, record NCT03729362. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03729362
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
- NCT number
- NCT03729362
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 125 participants, actual
- Lead sponsor
- Amicus Therapeutics
- Conditions
- Pompe Disease (Late-onset)
- Interventions
- Cipaglucosidase Alfa, Miglustat, Alglucosidase Alfa, Placebo
- First posted
- 2018-11-02
- Last updated
- 2025-09-11
- Results first posted
- 2023-09-11
Source ClinicalTrials.gov
Registered outcomes33
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| 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. |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 registered outcomesOutcomes33
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| 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 |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 outcomesResults by group110
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD) | Cipaglucosidase Alfa/Miglustat | 21.31 | Standard Error 11.56 | meter | |
| Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD) | Alglucosidase Alfa/Placebo | 7.10 | Standard Error 7.043 | meter | |
| Change From Baseline to Week 52 in Sitting Forced Vital Capacity (FVC; % Predicted) | Cipaglucosidase Alfa/Miglustat | -1.04 | Standard Error 0.624 | percentage of predicted FVC | |
| Change From Baseline to Week 52 in Sitting Forced Vital Capacity (FVC; % Predicted) | Alglucosidase Alfa/Placebo | -3.70 | Standard Error 0.953 | percentage of predicted FVC | |
| Change From Baseline to Week 52 in the Manual Muscle Test (MMT) Score for the Lower Extremities | Cipaglucosidase Alfa/Miglustat | 1.64 | Standard Error 0.388 | score on a scale | |
| Change From Baseline to Week 52 in the Manual Muscle Test (MMT) Score for the Lower Extremities | Alglucosidase Alfa/Placebo | 0.68 | Standard Error 0.603 | score on a scale | |
| Change From Baseline to Week 26 in 6MWD | Cipaglucosidase Alfa/Miglustat | 16.45 | Standard Error 3.360 | meter | |
| Change From Baseline to Week 26 in 6MWD | Alglucosidase Alfa/Placebo | 8.28 | Standard Error 5.168 | meter | |
| Change From Baseline to Week 52 in the Total Score for the Patient- Reported Outcomes Measurement Information System (PROMIS®) - Physical Function | Cipaglucosidase Alfa/Miglustat | 1.98 | Standard Error 0.921 | score on a scale | |
| Change From Baseline to Week 52 in the Total Score for the Patient- Reported Outcomes Measurement Information System (PROMIS®) - Physical Function | Alglucosidase Alfa/Placebo | 0.11 | Standard Error 1.406 | score on a scale |
1 to 10 of 110Source ClinicalTrials.gov
See all 110 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cipaglucosidase Alfa/Miglustat | 85 | |
| Overall Study | STARTED | Alglucosidase Alfa/Placebo | 40 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Cipaglucosidase Alfa/Miglustat | 85 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Alglucosidase Alfa/Placebo | 38 | |
| Overall Study | COMPLETED | Cipaglucosidase Alfa/Miglustat | 80 | |
| Overall Study | COMPLETED | Alglucosidase Alfa/Placebo | 37 | |
| Overall Study | NOT COMPLETED | Cipaglucosidase Alfa/Miglustat | 5 | |
| Overall Study | NOT COMPLETED | Alglucosidase Alfa/Placebo | 3 |
Source ClinicalTrials.gov
Adverse events108
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Hypertension | Vascular disorders | Cipaglucosidase Alfa/Miglustat | 5 | 85 | |
| other | Hypertension | Vascular disorders | Alglucosidase Alfa/Placebo | 3 | 38 | |
| other | Contusion | Injury, poisoning and procedural complications | Cipaglucosidase Alfa/Miglustat | 5 | 85 | |
| other | Contusion | Injury, poisoning and procedural complications | Alglucosidase Alfa/Placebo | 3 | 38 | |
| other | Fall | Injury, poisoning and procedural complications | Cipaglucosidase Alfa/Miglustat | 25 | 85 | |
| other | Fall | Injury, poisoning and procedural complications | Alglucosidase Alfa/Placebo | 15 | 38 | |
| other | Limb injury | Injury, poisoning and procedural complications | Cipaglucosidase Alfa/Miglustat | 3 | 85 | |
| other | Limb injury | Injury, poisoning and procedural complications | Alglucosidase Alfa/Placebo | 2 | 38 | |
| other | Dyspnoea | Respiratory, thoracic and mediastinal disorders | Cipaglucosidase Alfa/Miglustat | 6 | 85 | |
| other | Dyspnoea | Respiratory, thoracic and mediastinal disorders | Alglucosidase Alfa/Placebo | 1 | 38 |
1 to 10 of 108Source ClinicalTrials.gov
See all 108 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cipaglucosidase Alfa/Miglustat | 0 | 85 |
| Total, all-cause mortality | Alglucosidase Alfa/Placebo | 0 | 38 |
| Total, other adverse events | Cipaglucosidase Alfa/Miglustat | 69 | 85 |
| Total, other adverse events | Alglucosidase Alfa/Placebo | 32 | 38 |
| Total, serious adverse events | Cipaglucosidase Alfa/Miglustat | 8 | 85 |
| Total, serious adverse events | Alglucosidase Alfa/Placebo | 1 | 38 |
Source ClinicalTrials.gov
Papers6
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Cipaglucosidase Alfa/Miglustat vs Alglucosidase Alfa/Placebo: Number of Participants Improving on Both 6MWD and % Predicted FVC at Week 52 | Trial comparisons |
| Cipaglucosidase Alfa/Miglustat vs Alglucosidase Alfa/Placebo: deaths from any cause | Trial comparisons |
| Cipaglucosidase Alfa/Miglustat vs Alglucosidase Alfa/Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
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 trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03729362
GET https://conductscience.org/api/trials/v1/NCT03729362/registered?page=2