Trial · NCT00688597
Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
NCT00688597, Phase 2, Terminated, Amicus Therapeutics; results first posted 2018-08-17
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease. Trials, record NCT00688597. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00688597
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
- NCT number
- NCT00688597
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 3 participants, actual
- Lead sponsor
- Amicus Therapeutics
- Conditions
- Pompe Disease
- Interventions
- Duvoglustat
- First posted
- 2008-06-03
- Last updated
- 2025-09-11
- Results first posted
- 2018-08-17
- Limitations and caveats
- The serious adverse event of muscle weakness was the main contributor to the study not reaching the target number of participants needed to achieve target power and statistically reliable results.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion Of Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs) | Baseline, Week 11 | The number of participants experiencing severe TEAEs is presented for participants who received duvoglustat treatment in this open-label study. The duration of duvoglustat exposure for Cohort 1 ranged from 2 to 24 days, and their exposure ranged from a total of 7,500 to 32,500 milligrams of duvoglustat. An adverse event (AE) refers to any unfavorable and unintended sign, symptom, syndrome, or illness that develops or worsens during the period of observation in the clinical study. The following guideline was used to grade the intensity of an AE: mild, the AE is easily tolerated and does not interfere with daily activity; moderate, the AE interferes with the daily activity but the participant is still able to function; severe, the AE is incapacitating and requires medical intervention. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module. |
| Secondary | Change In 6-minute Walk Test (6MWT) From Baseline To End Of Study | Baseline, Week 11 | The 6MWT (American Thoracic Society standards) was evaluated in ambulatory participants at screening, baseline, and to the end of the study. It was a standardized test that measured the distance in meters (m) covered over a 6-minute walk. Reference equations used (for 6MWT distance in healthy adults) included: (height in centimeters \[cm\], weight in kilograms \[kg\]) 6MWT distance for men = \[7.57 × height (cm)\] - \[5.02 × age\] - \[1.76 × weight (kg)\] - 309 m; 6MWT distance for women = \[2.11 × height (cm)\] - \[5.78 × age\] - \[2.29 × weight (kg)\] + 667 m |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Proportion Of Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs) | Baseline, Week 11 | Safety Population: All participants who received at least 1 dose of duvoglustat. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change In 6-minute Walk Test (6MWT) From Baseline To End Of Study | Baseline, Week 11 | Safety Population: All participants who received at least 1 dose of duvoglustat. | m | MEAN |
Source ClinicalTrials.gov
Results by group2
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Proportion Of Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs) | Cohort 1 | 1 | Participants | ||
| Change In 6-minute Walk Test (6MWT) From Baseline To End Of Study | Cohort 1 | -19.660 | Standard Deviation 41.1845 | m |
Source ClinicalTrials.gov
Participant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1 | 3 | |
| Overall Study | STARTED | Cohort 2 | 0 | |
| Overall Study | STARTED | Cohort 3 | 0 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Cohort 1 | 3 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Cohort 2 | 0 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Cohort 3 | 0 | |
| Overall Study | COMPLETED | Cohort 1 | 0 | |
| Overall Study | COMPLETED | Cohort 2 | 0 | |
| Overall Study | COMPLETED | Cohort 3 | 0 | |
| Overall Study | NOT COMPLETED | Cohort 1 | 3 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events30
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 | Muscular weakness | General disorders | Cohort 1 | 2 | 3 | |
| other | Muscular weakness | General disorders | Cohort 2 | 0 | 0 | |
| other | Muscular weakness | General disorders | Cohort 3 | 0 | 0 | |
| other | Flatulence | General disorders | Cohort 1 | 1 | 3 | |
| other | Flatulence | General disorders | Cohort 2 | 0 | 0 | |
| other | Flatulence | General disorders | Cohort 3 | 0 | 0 | |
| other | Abdominal pain upper | General disorders | Cohort 1 | 1 | 3 | |
| other | Abdominal pain upper | General disorders | Cohort 2 | 0 | 0 | |
| other | Abdominal pain upper | General disorders | Cohort 3 | 0 | 0 | |
| other | Alanine aminotransferase increased | General disorders | Cohort 1 | 3 | 3 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1 | ||
| Total, all-cause mortality | Cohort 2 | ||
| Total, all-cause mortality | Cohort 3 | ||
| Total, other adverse events | Cohort 1 | 3 | 3 |
| Total, other adverse events | Cohort 2 | 0 | 0 |
| Total, other adverse events | Cohort 3 | 0 | 0 |
| Total, serious adverse events | Cohort 1 | 2 | 3 |
| Total, serious adverse events | Cohort 2 | 0 | 0 |
| Total, serious adverse events | Cohort 3 | 0 | 0 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT00688597
GET https://conductscience.org/api/trials/v1/NCT00688597/events?page=2