Trial · NCT04138277
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
NCT04138277, Phase 3, Completed, Amicus Therapeutics; results first posted 2026-01-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD). Trials, record NCT04138277. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04138277
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
- NCT number
- NCT04138277
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 119 participants, actual
- Lead sponsor
- Amicus Therapeutics
- Conditions
- Pompe Disease (Late-onset)
- Interventions
- AT2221, ATB200
- First posted
- 2019-10-24
- Last updated
- 2026-03-05
- Results first posted
- 2026-01-12
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| 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." |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| 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) | Open-label extension (OLE) Safety Population defined as all subjects who took at least one dose of ATB200/AT2221 combination treatment in Study ATB200-07 | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in 6-Minute Walk Distance (6MWD) | baseline, Week 208 | Motor function was evaluated using the Full Analysis Set (consisting of subjects in the study who had both a valid baseline and at least 1 post-baseline assessment for at least 1 of the main efficacy endpoints). Baseline and Month 208 assessment available for 39 subjects in the Treatment Continued Group and 18 subjects in the Treatment Switched Group. | meters | MEAN |
| SECONDARY | Change From Baseline in Sitting % Predicted Forced Vital Capacity (FVC) | baseline, Week 208 | Pulmonary function was evaluated using the Full Analysis Set (consisting of subjects in the study who had both a valid baseline and at least 1 post-baseline assessment for at least 1 of the main efficacy endpoints). Baseline and Month 208 assessment available for 41 subjects in the Treatment Continued Group and 15 subjects in the Treatment Switched Group. | percent predicted FVC | MEAN |
| SECONDARY | Change From Baseline in Manual Muscle Testing (MMT) Lower Extremity Score | baseline, Week 208 | Muscle strength was evaluated using the Full Analysis Set (consisting of subjects in the study who had both a valid baseline and at least 1 post-baseline assessment for at least 1 of the main efficacy endpoints). Baseline and Month 208 assessment available for 35 subjects in the Treatment Continued Group and 15 subjects in the Treatment Switched Group. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in the Total Score for Patient-reported Outcomes Measurement Information System (PROMIS®) - Physical Function | baseline, Week 208 | PROMIS-Physical Function was evaluated using the Full Analysis Set (consisting of subjects in the study who had both a valid baseline and at least 1 post-baseline assessment for at least 1 of the main efficacy endpoints). Baseline and Month 208 assessment available for 42 subjects in the Treatment Continued Group and 19 subjects in the Treatment Switched Group. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in the Total Score for PROMIS® - Fatigue | baseline, Week 208 | PROMIS-Fatigue was evaluated using the Full Analysis Set (consisting of subjects in the study who had both a valid baseline and at least 1 post-baseline assessment for at least 1 of the main efficacy endpoints). Baseline and Month 208 assessment available for 42 subjects in the Treatment Continued Group and 19 subjects in the Treatment Switched Group. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Gait, Stairs, Gower, Chair (GSGC) Test | baseline, Week 208 | GSGC was evaluated using the Full Analysis Set (consisting of subjects in the study who had both a valid baseline and at least 1 post-baseline assessment for at least 1 of the main efficacy endpoints). Baseline and Month 208 assessment available for 33 subjects in the Treatment Continued Group and 14 subjects in the Treatment Switched Group. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) Responses | baseline, Week 208 | EQ-5D-5L was evaluated using the Full Analysis Set (consisting of subjects in the study who had both a valid baseline and at least 1 post-baseline assessment for at least 1 of the main efficacy endpoints). Baseline and Month 208 assessment available for 42 subjects in the Treatment Continued Group and 19 subjects in the Treatment Switched Group. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Overall Physical Well-being (Subject's Global Impression of Change [SGIC], Question 1) | baseline, Week 208 | The SGIC was evaluated using the Full Analysis Set (consisting of subjects in the study who had both a valid baseline and at least 1 post-baseline assessment for at least 1 of the main efficacy endpoints). Baseline and Month 208 assessment available for 42 subjects in the Treatment Continued Group and 19 subjects in the Treatment Switched Group. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Physician's Global Impression of Change (PGIC) Overall Status | baseline, Week 208 | The PGIC was evaluated using the Full Analysis Set (consisting of subjects in the study who had both a valid baseline and at least 1 post-baseline assessment for at least 1 of the main efficacy endpoints). Baseline and Month 208 assessment available for 40 subjects in the Treatment Continued Group and 19 subjects in the Treatment Switched Group. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group126
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug | Treatment Continued Group | Subjects with TEAEs | 80 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug | Treatment Switched Group | Subjects with TEAEs | 37 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug | Treatment Continued Group | Subjects with TESAEs | 17 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug | Treatment Switched Group | Subjects with TESAEs | 10 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug | Treatment Continued Group | Subjects with TEAEs leading to discontinuation | 6 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug | Treatment Switched Group | Subjects with TEAEs leading to discontinuation | 2 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug | Treatment Continued Group | Subjects with TEAE leading to death | 1 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug | Treatment Switched Group | Subjects with TEAE leading to death | 0 | Participants | |
| Change From Baseline in 6-Minute Walk Distance (6MWD) | Treatment Continued Group | -33.3 | Standard Deviation 37.60 | meters | |
| Change From Baseline in 6-Minute Walk Distance (6MWD) | Treatment Switched Group | -8.6 | Standard Deviation 62.77 | meters |
1 to 10 of 126Source ClinicalTrials.gov
See all 126 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Pretreatment | STARTED | Treatment Continued Group | 82 | |
| Pretreatment | STARTED | Treatment Switched Group | 37 | |
| Pretreatment | COMPLETED | Treatment Continued Group | 81 | |
| Pretreatment | COMPLETED | Treatment Switched Group | 37 | |
| Pretreatment | NOT COMPLETED | Treatment Continued Group | 1 | |
| Pretreatment | NOT COMPLETED | Treatment Switched Group | 0 | |
| Treatment | STARTED | Treatment Continued Group | 81 | |
| Treatment | STARTED | Treatment Switched Group | 37 | |
| Treatment | COMPLETED | Treatment Continued Group | 58 | |
| Treatment | COMPLETED | Treatment Switched Group | 30 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events220
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 | Anemia | Blood and lymphatic system disorders | Treatment Continued Group | 2 | 81 | |
| other | Anemia | Blood and lymphatic system disorders | Treatment Switched Group | 2 | 37 | |
| other | Ear pain | Ear and labyrinth disorders | Treatment Continued Group | 6 | 81 | |
| other | Ear pain | Ear and labyrinth disorders | Treatment Switched Group | 0 | 37 | |
| other | Diarrhea | Gastrointestinal disorders | Treatment Continued Group | 21 | 81 | |
| other | Diarrhea | Gastrointestinal disorders | Treatment Switched Group | 7 | 37 | |
| other | Nausea | Gastrointestinal disorders | Treatment Continued Group | 15 | 81 | |
| other | Nausea | Gastrointestinal disorders | Treatment Switched Group | 5 | 37 | |
| other | Abdominal pain | Gastrointestinal disorders | Treatment Continued Group | 7 | 81 | |
| other | Abdominal pain | Gastrointestinal disorders | Treatment Switched Group | 4 | 37 |
1 to 10 of 220Source ClinicalTrials.gov
See all 220 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Treatment Continued Group | 1 | 81 |
| Total, all-cause mortality | Treatment Switched Group | 0 | 37 |
| Total, other adverse events | Treatment Continued Group | 80 | 81 |
| Total, other adverse events | Treatment Switched Group | 37 | 37 |
| Total, serious adverse events | Treatment Continued Group | 17 | 81 |
| Total, serious adverse events | Treatment Switched Group | 10 | 37 |
Source ClinicalTrials.gov
Papers3
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Treatment Continued Group vs Treatment Switched Group: deaths from any cause | Trial comparisons |
| Treatment Continued Group vs Treatment Switched Group: 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/NCT04138277
GET https://conductscience.org/api/trials/v1/NCT04138277/results?page=2