Trial Outcomes & Findings for A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease (NCT NCT03729362)

Last Updated: 2025-09-11

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

125 participants

Primary outcome timeframe

Baseline, Week 52

Results posted on

2025-09-11

Participant Flow

A total of 125 participants were randomized in the study at 62 clinical sites across 24 countries; 123 participants received at least 1 dose of study drug. Two participants randomized to the alglucosidase alfa/placebo group were never dosed because genotyping did not confirm the diagnosis of Pompe disease.

Participant milestones

Participant milestones
Measure	Cipaglucosidase Alfa/Miglustat Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W).	Alglucosidase Alfa/Placebo Alglucosidase alfa co-administered with placebo Q2W.
Overall Study STARTED	85	40
Overall Study Received at Least 1 Dose of Study Drug	85	38
Overall Study COMPLETED	80	37
Overall Study NOT COMPLETED	5	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Cipaglucosidase Alfa/Miglustat Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W).	Alglucosidase Alfa/Placebo Alglucosidase alfa co-administered with placebo Q2W.
Overall Study Withdrawal by Subject	2	0
Overall Study Physician Decision	1	0
Overall Study COVID-19 Pandemic	1	0
Overall Study Discontinued due to COVID-19 related pneumonia	1	0
Overall Study Adverse Event	0	1
Overall Study Genotyping did not confirm diagnosis of Pompe disease	0	2

Baseline Characteristics

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cipaglucosidase Alfa/Miglustat n=85 Participants Cipaglucosidase alfa co-administered with miglustat Q2W.	Alglucosidase Alfa/Placebo n=38 Participants Alglucosidase alfa co-administered with placebo Q2W.	Total n=123 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	74 Participants n=5 Participants	35 Participants n=7 Participants	109 Participants n=5 Participants
Age, Categorical >=65 years	11 Participants n=5 Participants	3 Participants n=7 Participants	14 Participants n=5 Participants
Age, Continuous	47.6 years STANDARD_DEVIATION 13.25 • n=5 Participants	45.1 years STANDARD_DEVIATION 13.30 • n=7 Participants	46.8 years STANDARD_DEVIATION 13.27 • n=5 Participants
Sex: Female, Male Female	49 Participants n=5 Participants	18 Participants n=7 Participants	67 Participants n=5 Participants
Sex: Female, Male Male	36 Participants n=5 Participants	20 Participants n=7 Participants	56 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Asian	5 Participants n=5 Participants	5 Participants n=7 Participants	10 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	74 Participants n=5 Participants	30 Participants n=7 Participants	104 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	5 Participants n=5 Participants	1 Participants n=7 Participants	6 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 52

Population: 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.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=81 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=36 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD)	21.31 meter Standard Error 11.56	7.10 meter Standard Error 7.043	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=84 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in Sitting Forced Vital Capacity (FVC; % Predicted)	-1.04 percentage of predicted FVC Standard Error 0.624	-3.70 percentage of predicted FVC Standard Error 0.953	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=80 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=34 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in the Manual Muscle Test (MMT) Score for the Lower Extremities	1.64 score on a scale Standard Error 0.388	0.68 score on a scale Standard Error 0.603	—

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: Analysis was performed on ITT-LOCF population. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis.

The 6MWD, measured in meters, is the distance walked on the 6MWT.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=85 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 26 in 6MWD	16.45 meter Standard Error 3.360	8.28 meter Standard Error 5.168	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=84 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in the Total Score for the Patient- Reported Outcomes Measurement Information System (PROMIS®) - Physical Function	1.98 score on a scale Standard Error 0.921	0.11 score on a scale Standard Error 1.406	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: Analysis was performed on ITT-LOCF population. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis.

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.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=85 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in the Total Score for the PROMIS® - Fatigue	-1.90 score on a scale Standard Error 0.585	-1.94 score on a scale Standard Error 0.901	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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.

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).

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=72 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=30 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in the Total Score for the Gait, Stairs, Gowers' Maneuver, and Chair (GSGC)	-0.567 score on a scale Standard Error 0.280	0.847 score on a scale Standard Error 0.440	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=69 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=33 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in Rasch-Built Pompe-Specific Activity (R-PAct) Total Score	0.04 score on a scale Standard Error 0.387	0.51 score on a scale Standard Error 0.567	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=84 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=36 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
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	0.03 score on a scale Standard Error 1.542	3.61 score on a scale Standard Error 2.400	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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.

SVC is a standard pulmonary function test used to quantify respiratory muscle weakness.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=83 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=35 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in Sitting Slow Vital Capacity (SVC) % Predicted	-2.527 percentage of predicted SVC Standard Error 0.977	-5.368 percentage of predicted SVC Standard Error 1.527	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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.

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).

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=84 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in Maximal Inspiratory Pressure (MIP) % Predicted	1.89 percentage of predicted MIP Standard Error 2.079	-2.31 percentage of predicted MIP Standard Error 3.178	—

SECONDARY outcome

Timeframe: 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).

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=84 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in Maximal Expiratory Pressure (MEP) % Predicted	0.51 percentage of predicted MEP Standard Error 1.996	-1.35 percentage of predicted MEP Standard Error 3.052	—

SECONDARY outcome

Timeframe: 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).

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=84 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in Sniff Nasal Inspiratory Pressure (SNIP) % Predicted	1.40 percentage of predicted SNIP Standard Error 1.918	4.53 percentage of predicted SNIP Standard Error 2.929	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: Analysis was performed on ITT-LOCF population. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis.

The % predicted 6MWD = (actual 6MWD / predicted 6MWD) \* 100. The predicted values were calculated using Enright And Sherrill 1998 Reference Equations.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=85 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in % Predicted 6MWD	4.039 percentage of predicted 6MWD Standard Error 0.716	1.655 percentage of predicted 6MWD Standard Error 1.102	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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.

QMT was measured using the hand-held dynamometer. Larger values (in kg) indicated greater muscle strength.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=85 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in the Quantitative Muscle Test (QMT) Values QMT Value for the Upper Extremities	1.839 kilogram Standard Error 2.098	-0.553 kilogram Standard Error 3.195	—
Change From Baseline to Week 52 in the Quantitative Muscle Test (QMT) Values QMT Value for the Lower Extremities	6.496 kilogram Standard Error 3.185	5.265 kilogram Standard Error 4.854	—
Change From Baseline to Week 52 in the Quantitative Muscle Test (QMT) Values QMT Total Value	8.195 kilogram Standard Error 5.079	5.198 kilogram Standard Error 7.746	—
Change From Baseline to Week 52 in the Quantitative Muscle Test (QMT) Values QMT Value for the Proximal Muscle Group	3.401 kilogram Standard Error 2.920	0.945 kilogram Standard Error 4.477	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=85 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in Other MMT Scores MMT Upper Extremity Score	1.54 score on a scale Standard Error 0.323	0.60 score on a scale Standard Error 0.491	—
Change From Baseline to Week 52 in Other MMT Scores MMT Total Score	3.24 score on a scale Standard Error 0.622	1.02 score on a scale Standard Error 0.966	—
Change From Baseline to Week 52 in Other MMT Scores MMT Proximal Muscle Group Score	1.82 score on a scale Standard Error 0.393	0.70 score on a scale Standard Error 0.599	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Maximum VC is the greater of the two VC values (FVC or SVC).

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=84 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in Maximum Vital Capacity (Maximum VC) % Predicted	-1.286 percentage of predicted maximum VC Standard Error 0.613	-3.695 percentage of predicted maximum VC Standard Error 0.936	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=85 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in PROMIS-Dyspnea and Upper Extremities Total Scores PROMIS-Dyspnea Total Score	-0.41 score on a scale Standard Error 0.426	-1.50 score on a scale Standard Error 0.652	—
Change From Baseline to Week 52 in PROMIS-Dyspnea and Upper Extremities Total Scores PROMIS-Upper Extremities Total Score	0.97 score on a scale Standard Error 0.545	0.87 score on a scale Standard Error 0.833	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=85 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline in the Time to Complete Individual GSGC Component Tests and Timed Up and Go (TUG) Test at Week 52 Time to complete the 10-meter walk	-0.60 seconds Standard Error 0.631	2.06 seconds Standard Error 0.967	—
Change From Baseline in the Time to Complete Individual GSGC Component Tests and Timed Up and Go (TUG) Test at Week 52 Time to complete the 4-stair climb	-6.75 seconds Standard Error 0.851	-3.61 seconds Standard Error 1.308	—
Change From Baseline in the Time to Complete Individual GSGC Component Tests and Timed Up and Go (TUG) Test at Week 52 Time to complete the Gowers' maneuver	-0.36 seconds Standard Error 0.799	-1.95 seconds Standard Error 1.281	—
Change From Baseline in the Time to Complete Individual GSGC Component Tests and Timed Up and Go (TUG) Test at Week 52 Time to arise from a chair	-7.57 seconds Standard Error 0.409	-6.75 seconds Standard Error 0.643	—
Change From Baseline in the Time to Complete Individual GSGC Component Tests and Timed Up and Go (TUG) Test at Week 52 Time to complete the timed up and go test	-0.39 seconds Standard Error 0.768	0.09 seconds Standard Error 1.217	—

SECONDARY outcome

Timeframe: Week 52

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=80 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=36 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Physician's Global Impression of Change (PGIC) Overall Status at Week 52 Improving	31 Participants	10 Participants	—
Physician's Global Impression of Change (PGIC) Overall Status at Week 52 Stable	38 Participants	16 Participants	—
Physician's Global Impression of Change (PGIC) Overall Status at Week 52 Declining	11 Participants	10 Participants	—

SECONDARY outcome

Timeframe: Week 52

Population: Analysis was performed on ITT-OBS population. Number of participants analyzed are based on those who had SGIC data at Week 52. In addition, the outlier subject in the alglucosidase alfa/placebo group was not included in the analysis.

The SGIC is designed to record the participants' impression of their functional status since starting study drug using a 7-point 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.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=81 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=36 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Subject's Global Impression of Change (SGIC) at Week 52 Improving	36 Participants	13 Participants	—
Subject's Global Impression of Change (SGIC) at Week 52 Stable	33 Participants	12 Participants	—
Subject's Global Impression of Change (SGIC) at Week 52 Declining	12 Participants	11 Participants	—

SECONDARY outcome

Timeframe: Week 52

Population: Analysis was performed on ITT-LOCF population. In the cipaglucosidase alfa/miglustat group, 1 subject who had no post-baseline FVC value was counted as not improved and included in the analysis. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis.

A composite subject-level response of the 2 relevant clinical outcomes, 6MWD and FVC (% predicted), was assessed. Prespecified thresholds were used for assessment of improvement consistent with published minimal clinically important difference values for comparable instruments in similar disease.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=85 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Number of Participants Improving on Both 6MWD and % Predicted FVC at Week 52	14 Participants	0 Participants	—

SECONDARY outcome

Timeframe: Baseline up to Week 52

Population: Analysis was performed on safety population which included subjects who had received at least 1 dose of study drug. Number analyzed equals participants evaluable at specified treatment.

Treatment-emergent ADAs were defined as participants who had seroconverted or boosted their preexisting ADA during the study period.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=85 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=38 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Number of Participants With Treatment-Emergent Anti-Drug Antibodies (ADAs) ERT-experienced: Treatment-emergent ADAs	31 participants	5 participants	—
Number of Participants With Treatment-Emergent Anti-Drug Antibodies (ADAs) ERT-naïve: Treatment-emergent ADAs	19 participants	8 participants	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Levels of urinary Hex4, a biomarker of disease substrate, were measured. The assay specifically targets Hex4, the glucose tetrasaccharide (Glc4), which is a biomarker of glycogen storage.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=84 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in Urinary Hexose Tetrasaccharide (Hex4) Level	-1.88 mmol/mol creatinine Standard Deviation 2.380	1.22 mmol/mol creatinine Standard Deviation 4.432	—

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: Analysis was performed on ITT-LOCF population. In the alglucosidase alfa/placebo group, the outlier subject was not included in the analysis.

Change from baseline to Week 52 in serum CK level. CK levels were measured as part of the serum chemistry panel.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=85 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=37 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Change From Baseline to Week 52 in Serum Creatine Kinase (CK) Level	-130.5 U/L Standard Deviation 231.18	60.2 U/L Standard Deviation 159.49	—

SECONDARY outcome

Timeframe: Days 1 and 364 (Week 52)

Population: Number analyzed equals participants evaluable at specified time points.

On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis in ERT-experienced participants at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=56 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=26 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Population Pharmacokinetics (PK): Maximum Observed Concentration (Cmax) of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Experienced Participants Using Plasma Total GAA Protein Level by Signature Peptide Assay and Plasma Miglustat Concentration Day 1	280 μg/mL Geometric Coefficient of Variation 18.5	289 μg/mL Geometric Coefficient of Variation 13.2	—
Population Pharmacokinetics (PK): Maximum Observed Concentration (Cmax) of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Experienced Participants Using Plasma Total GAA Protein Level by Signature Peptide Assay and Plasma Miglustat Concentration Day 364	293 μg/mL Geometric Coefficient of Variation 19.9	283 μg/mL Geometric Coefficient of Variation 17.6	—

SECONDARY outcome

Timeframe: Days 1 and 364 (Week 52)

Population: Number analyzed equals participants evaluable at specified timepoints.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=56 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=26 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Population PK: Area Under the Concentration-Time Curve (AUC) of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Experienced Participants Using Plasma Total GAA Protein Level by Signature Peptide Assay and Plasma Miglustat Concentration Day 1	1395 μg·h/mL Geometric Coefficient of Variation 21.5	1700 μg·h/mL Geometric Coefficient of Variation 17.6	—
Population PK: Area Under the Concentration-Time Curve (AUC) of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Experienced Participants Using Plasma Total GAA Protein Level by Signature Peptide Assay and Plasma Miglustat Concentration Day 364	1476 μg·h/mL Geometric Coefficient of Variation 21.8	1688 μg·h/mL Geometric Coefficient of Variation 23.9	—

SECONDARY outcome

Timeframe: Days 1 and 364 (Week 52)

Population: Number analyzed equals participants evaluable at specified timepoints.

On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis in ERT-naïve participants at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=18 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=7 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Population PK: Cmax of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Naïve Participants Day 1	273 μg/mL Geometric Coefficient of Variation 18.1	342 μg/mL Geometric Coefficient of Variation 31.0	—
Population PK: Cmax of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Naïve Participants Day 364	290 μg/mL Geometric Coefficient of Variation 17.4	359 μg/mL Geometric Coefficient of Variation 28.1	—

SECONDARY outcome

Timeframe: Days 1 and 364 (Week 52)

Population: Number analyzed equals participants evaluable at specified timepoints.

On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis in ERT-naïve participants at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=18 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=7 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Population PK: AUC of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Naïve Subjects Day 1	1343 μg·h/mL Geometric Coefficient of Variation 25.7	1859 μg·h/mL Geometric Coefficient of Variation 22.4	—
Population PK: AUC of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Naïve Subjects Day 364	1457 μg·h/mL Geometric Coefficient of Variation 19.2	1964 μg·h/mL Geometric Coefficient of Variation 26.8	—

SECONDARY outcome

Timeframe: Day 1

A noncompartmental analysis was performed on ERT-naïve subjects, who underwent serial PK sampling during the study. On Day 1, serial blood samples were collected for ERT-naïve participants just prior to initiation of cipaglucosidase alfa/alglucosidase alfa infusion (time 0) and at 1, 2, 3, 3.5, 4, 4.5, 6, 8, 10, and 24 hours after the start of cipaglucosidase alfa/alglucosidase alfa infusion for plasma total human acid α-glucosidase (GAA) protein signature peptide T09 and plasma miglustat determinations.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=12 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=4 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat n=12 Participants Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Noncompartmental Analysis: Cmax of Plasma Total GAA Protein by Signature Peptide T09 in ERT-Naïve Subjects	260 μg/mL Geometric Coefficient of Variation 18.4	364 μg/mL Geometric Coefficient of Variation 66.7	2768 μg/mL Geometric Coefficient of Variation 30.8

SECONDARY outcome

Timeframe: Day 1

A noncompartmental analysis was performed on ERT-naïve subjects, who underwent serial PK sampling during the study. On Day 1, serial blood samples were collected for ERT-naïve participants just prior to initiation of cipaglucosidase alfa/alglucosidase alfa infusion (time 0) and at 1, 2, 3, 3.5, 4, 4.5, 6, 8, 10, and 24 hours after the start of cipaglucosidase alfa/alglucosidase alfa infusion for plasma total GAA protein signature peptide T09 and plasma miglustat determinations.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=12 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=4 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat n=12 Participants Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Noncompartmental Analysis: AUC From Time 0 (Predose) to the Time of Last Quantifiable Concentration of Plasma Total GAA Protein by Signature Peptide T09 in ERT-Naïve Subjects	1264 μg·h/mL Geometric Coefficient of Variation 28.9	1656 μg·h/mL Geometric Coefficient of Variation 28.9	20588 μg·h/mL Geometric Coefficient of Variation 36.8

SECONDARY outcome

Timeframe: Days 1 and 364 (Week 52)

Population: Number analyzed based upon number of subjects with evaluable data for population PK analysis at specified timepoints.

On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=56 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=18 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Comparison of Cmax of Cipaglucosidase Alfa in ERT-Experienced and ERT-Naïve Populations Day 1	280 μg/mL Geometric Coefficient of Variation 18.5	273 μg/mL Geometric Coefficient of Variation 18.1	—
Comparison of Cmax of Cipaglucosidase Alfa in ERT-Experienced and ERT-Naïve Populations Day 364	296 μg/mL Geometric Coefficient of Variation 19.9	290 μg/mL Geometric Coefficient of Variation 17.4	—

SECONDARY outcome

Timeframe: Days 1 and 364 (Week 52)

Population: Number analyzed based upon number of subjects with evaluable data for population PK analysis at specified timepoints.

On Days 1 and 364 (Week 52), sparse blood samples were collected for PK analysis at 0, 1, 4, 6, 12, and 24 hours post-dose. Collection of the 12-hour sample was optional.

Outcome measures

Outcome measures
Measure	Cipaglucosidase Alfa/Miglustat n=56 Participants Cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W)	Alglucosidase Alfa/Placebo n=18 Participants Alglucosidase alfa co-administered with placebo every 2 weeks (Q2W)	Miglustat Participants who were exposed to at least 1 dose of miglustat and had at least 1 PK assessment.
Comparison of AUC of Cipaglucosidase Alfa in ERT- Experienced and ERT-Naïve Populations Day 1	1395 μg·h/mL Geometric Coefficient of Variation 21.5	1343 μg·h/mL Geometric Coefficient of Variation 25.7	—
Comparison of AUC of Cipaglucosidase Alfa in ERT- Experienced and ERT-Naïve Populations Day 364	1476 μg·h/mL Geometric Coefficient of Variation 21.8	1457 μg·h/mL Geometric Coefficient of Variation 19.2	—

Adverse Events

Cipaglucosidase Alfa/Miglustat

Serious events: 8 serious events

Other events: 69 other events

Deaths: 0 deaths

Alglucosidase Alfa/Placebo

Serious events: 1 serious events

Other events: 32 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cipaglucosidase Alfa/Miglustat n=85 participants at risk All participants who received at least 1 dose of study drug (cipaglucosidase alfa/miglustat).	Alglucosidase Alfa/Placebo n=38 participants at risk All participants who received at least 1 dose of study drug (alglucosidase alfa/placebo).
Vascular disorders Aortic aneurysm	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	0.00% 0/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Injury, poisoning and procedural complications Contusion	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	0.00% 0/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Injury, poisoning and procedural complications Ilium fracture	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	0.00% 0/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Injury, poisoning and procedural complications Skin laceration	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	0.00% 0/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Surgical and medical procedures Removal of internal fixation	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	0.00% 0/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Cardiac disorders Bradycardia	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	0.00% 0/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Immune system disorders Anaphylactoid reaction	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	0.00% 0/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Nervous system disorders Cerebrovascular accident	0.00% 0/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	2.6% 1/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Gastrointestinal disorders Abdominal pain	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	0.00% 0/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Gastrointestinal disorders Enteritis	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	0.00% 0/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Gastrointestinal disorders Vomiting	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	0.00% 0/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Infections and infestations Viral myositis	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	0.00% 0/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).

Other adverse events

Additional Information

Patient advocacy

Amicus Therapeutics, Inc.

Phone: 001 6096622000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60

Measure	Cipaglucosidase Alfa/Miglustat n=85 participants at risk All participants who received at least 1 dose of study drug (cipaglucosidase alfa/miglustat).	Alglucosidase Alfa/Placebo n=38 participants at risk All participants who received at least 1 dose of study drug (alglucosidase alfa/placebo).
Vascular disorders Hypertension	5.9% 5/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	7.9% 3/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Injury, poisoning and procedural complications Contusion	5.9% 5/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	7.9% 3/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Injury, poisoning and procedural complications Fall	29.4% 25/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	39.5% 15/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Injury, poisoning and procedural complications Limb injury	3.5% 3/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Respiratory, thoracic and mediastinal disorders Dyspnoea	7.1% 6/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	2.6% 1/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Respiratory, thoracic and mediastinal disorders Nasal congestion	5.9% 5/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	11.8% 10/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Nervous system disorders Balance disorder	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Nervous system disorders Dizziness	9.4% 8/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	7.9% 3/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Nervous system disorders Headache	23.5% 20/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	23.7% 9/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
General disorders Asthenia	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	10.5% 4/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
General disorders Fatigue	9.4% 8/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	13.2% 5/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
General disorders Infusion site bruising	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
General disorders Infusion site erythema	0.00% 0/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
General disorders Pain	7.1% 6/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	2.6% 1/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
General disorders Pyrexia	7.1% 6/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	2.6% 1/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Psychiatric disorders Panic attack	2.4% 2/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Gastrointestinal disorders Abdominal distension	5.9% 5/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Gastrointestinal disorders Abdominal pain	2.4% 2/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	7.9% 3/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Gastrointestinal disorders Abdominal pain upper	4.7% 4/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	7.9% 3/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Gastrointestinal disorders Constipation	2.4% 2/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	7.9% 3/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Gastrointestinal disorders Diarrhoea	12.9% 11/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	10.5% 4/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Gastrointestinal disorders Dyspepsia	2.4% 2/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Gastrointestinal disorders Flatulence	3.5% 3/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Gastrointestinal disorders Nausea	11.8% 10/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	21.1% 8/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Skin and subcutaneous tissue disorders Pruritus	2.4% 2/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	7.9% 3/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Skin and subcutaneous tissue disorders Rash	2.4% 2/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	7.9% 3/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Skin and subcutaneous tissue disorders Skin lesion	0.00% 0/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Musculoskeletal and connective tissue disorders Arthralgia	15.3% 13/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	13.2% 5/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Musculoskeletal and connective tissue disorders Back pain	10.6% 9/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	18.4% 7/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Musculoskeletal and connective tissue disorders Groin pain	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Musculoskeletal and connective tissue disorders Muscle spasms	9.4% 8/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	2.6% 1/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Musculoskeletal and connective tissue disorders Muscular weakness	3.5% 3/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	13.2% 5/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Musculoskeletal and connective tissue disorders Musculoskeletal pain	11.8% 10/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Musculoskeletal and connective tissue disorders Myalgia	16.5% 14/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	13.2% 5/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Musculoskeletal and connective tissue disorders Pain in extremity	12.9% 11/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Infections and infestations Nasopharyngitis	22.4% 19/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	7.9% 3/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Infections and infestations Pharyngitis	1.2% 1/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Infections and infestations Rhinitis	7.1% 6/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Infections and infestations Sinusitis	4.7% 4/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	7.9% 3/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Infections and infestations Upper respiratory tract infection	3.5% 3/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	15.8% 6/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).
Infections and infestations Urinary tract infection	14.1% 12/85 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).	5.3% 2/38 • Day 1 (after dosing) through Week 52 and follow-up (30 days after last dose).