Trial Outcomes & Findings for Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease (NCT NCT00688597)
NCT ID: NCT00688597
Last Updated: 2025-09-11
Results Overview
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.
TERMINATED
PHASE2
3 participants
Baseline, Week 11
2025-09-11
Participant Flow
Participant milestones
| Measure |
Cohort 1
Regimen 1: Low-dose duvoglustat (2.5 grams \[g\]) once a day (QD) for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 2
Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 3
Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks.
|
|---|---|---|---|
|
Overall Study
STARTED
|
3
|
0
|
0
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
3
|
0
|
0
|
|
Overall Study
COMPLETED
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
3
|
0
|
0
|
Reasons for withdrawal
| Measure |
Cohort 1
Regimen 1: Low-dose duvoglustat (2.5 grams \[g\]) once a day (QD) for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 2
Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 3
Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks.
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
2
|
0
|
0
|
|
Overall Study
Physician Decision
|
1
|
0
|
0
|
Baseline Characteristics
Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
Baseline characteristics by cohort
| Measure |
Cohort 1
n=3 Participants
Regimen 1: Low-dose duvoglustat (2.5 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 2
Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 3
Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks.
|
Total
n=3 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
49.07 years
STANDARD_DEVIATION 11.02 • n=5 Participants
|
—
|
—
|
49.07 years
STANDARD_DEVIATION 11.02 • n=4 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
—
|
—
|
2 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
—
|
—
|
1 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 11Population: Safety Population: All participants who received at least 1 dose of duvoglustat.
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.
Outcome measures
| Measure |
Cohort 1
n=3 Participants
Regimen 1: Low-dose duvoglustat (2.5 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 2
Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 3
Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks.
|
|---|---|---|---|
|
Proportion Of Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs)
|
1 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline, Week 11Population: Safety Population: All participants who received at least 1 dose of duvoglustat.
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
Outcome measures
| Measure |
Cohort 1
n=3 Participants
Regimen 1: Low-dose duvoglustat (2.5 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 2
Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 3
Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks.
|
|---|---|---|---|
|
Change In 6-minute Walk Test (6MWT) From Baseline To End Of Study
|
-19.660 m
Standard Deviation 41.1845
|
—
|
—
|
Adverse Events
Cohort 1
Cohort 2
Cohort 3
Serious adverse events
| Measure |
Cohort 1
n=3 participants at risk
Regimen 1: Low-dose duvoglustat (2.5 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 2
Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 3
Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks.
|
|---|---|---|---|
|
General disorders
Muscular weakness
|
66.7%
2/3 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
Other adverse events
| Measure |
Cohort 1
n=3 participants at risk
Regimen 1: Low-dose duvoglustat (2.5 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 2
Regimen 1: High-dose duvoglustat (5.0 g) QD for 3 days, followed by no drug for 4 days, for 11 weeks.
|
Cohort 3
Regimen 2: High-dose duvoglustat (5.0 g) QD for 7 days, followed by no drug for 7 days, for 11 weeks.
|
|---|---|---|---|
|
General disorders
Muscular weakness
|
66.7%
2/3 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
|
General disorders
Flatulence
|
33.3%
1/3 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
|
General disorders
Abdominal pain upper
|
33.3%
1/3 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
|
General disorders
Alanine aminotransferase increased
|
100.0%
3/3 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
|
General disorders
Aspartate aminotransferase increased
|
100.0%
3/3 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
|
General disorders
Blood creatine phosphokinase increased
|
100.0%
3/3 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
|
General disorders
Fall
|
66.7%
2/3 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
|
General disorders
Dysphagia
|
33.3%
1/3 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
|
General disorders
Joint injury
|
33.3%
1/3 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
—
0/0 • Day 1 (after dosing) through end of follow up.
Due to the discontinuation of the study, AEs were not encoded.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The investigator can only publish the results from this trial provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review. If requested, the investigator will remove information deemed confidential or proprietary by the sponsor and will withhold publication for an additional period of time to allow the sponsor to take appropriate measures to establish and preserve its proprietary rights.
- Publication restrictions are in place
Restriction type: OTHER