Trial · NCT02675465
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
NCT02675465, Phase 1, Phase 2, Completed, Amicus Therapeutics; results first posted 2025-10-23
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221. Trials, record NCT02675465. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02675465
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
- NCT number
- NCT02675465
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 29 participants, actual
- Lead sponsor
- Amicus Therapeutics
- Conditions
- Pompe Disease
- Interventions
- ATB200, AT2221
- First posted
- 2016-02-05
- Last updated
- 2025-10-23
- Results first posted
- 2025-10-23
Source ClinicalTrials.gov
Registered outcomes10
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 Adverse Events (AEs) Leading to Discontinuation of Study Drug | Stage 3 (2 year treatment) and Stage 4 (Extension) combined, (mean = 71 months on treatment) | Number of subjects with TEAE, TESAE, and AE leading to discontinuation during the 2 year treatment period and extension (Stage 3 and 4 combined) |
| Primary | Plasma Human Acid α-glucosidase (GAA) Activity Levels as Measured by Maximum Observed Plasma Concentration (Cmax). | 18 Weeks | Plasma GAA levels (Cmax) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat |
| Primary | Plasma GAA Activity Levels as Measured by Time to Reach the Maximum Observed Plasma Concentration (Tmax). | 18 Weeks | Plasma GAA levels (Tmax) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat |
| Primary | Plasma GAA Activity Levels as Measured by Area Under the Plasma Drug Concentration-time Curve (AUC). | 18 Weeks | Plasma GAA levels (AUC) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat |
| Secondary | Change From Baseline in 6-minute Walk Distance (6MWD) | Baseline, Month 60 | Motor function was measured in ambulatory subjects using 6MWD (meters). |
| Secondary | Change From Baseline in Pulmonary Function Tests | Baseline, Month 60 | Pulmonary function was measured by sitting and supine % predicted forced vital capacity (FVC) |
| Secondary | Change From Baseline in Muscle Strength Tests | Baseline, Month 60 | Muscle strength was measured by total manual muscle test (MMT) score. Total MMT score ranges from 0 to 80 based on all 16 muscle groups, which are right/left shoulder abduction, right/left shoulder adduction, right/left elbow flexion, right/left elbow extension, right/left hip flexion, right/left hip abduction, right/left knee flexion, and right/left knee extension. Higher scores indicate less disease impact on muscle functions. |
| Secondary | Change From Baseline in Fatigue Severity Score (FSS) | Baseline, Month 60 | The Fatigue Severity Score (FSS) consists of 9 questions, each scored on a scale from 1 ("completely disagree") to 7 ("completely agree"). The total score ranges from 9 to 63, with higher values representing higher level of fatigue due to the disease condition. |
| Secondary | Change From Baseline in Overall Physical Wellbeing (Subject's Global Impression of Change [SGIC], Question1) | Baseline, Month 60 | The Subject's Global Impression of Change overall physical wellbeing (question 1) is scored on a 7-point rating scale. Improved = response of 5 or higher, No change = response of 4, and Declined = response of 3 or lower. |
| Secondary | Change From Baseline in Overall Physical Wellbeing (Physician's Global Impression of Change [PGIC]) | Baseline, Month 60 | The Physician's Global Impression of Change overall physical wellbeing is scored on a 7-point rating scale. Improved = response of 5 or higher, No change = response of 4, and Declined = response of 3 or lower. |
Source ClinicalTrials.gov
Outcomes10
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 Adverse Events (AEs) Leading to Discontinuation of Study Drug | Stage 3 (2 year treatment) and Stage 4 (Extension) combined, (mean = 71 months on treatment) | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Plasma Human Acid α-glucosidase (GAA) Activity Levels as Measured by Maximum Observed Plasma Concentration (Cmax). | 18 Weeks | Pharmacokinetic (PK) analysis only performed in Cohort 1 and Cohort 3. One subject each in Cohort 1 and Cohort 3 did not have PK sample available following first dose. | nmol/mL/hr | GEOMETRIC_MEAN |
| PRIMARY | Plasma GAA Activity Levels as Measured by Time to Reach the Maximum Observed Plasma Concentration (Tmax). | 18 Weeks | PK analysis only performed in Cohort 1 and Cohort 3. One subject each in Cohort 1 and Cohort 3 did not have PK sample available following first dose. | h | MEDIAN |
| PRIMARY | Plasma GAA Activity Levels as Measured by Area Under the Plasma Drug Concentration-time Curve (AUC). | 18 Weeks | PK analysis only performed in Cohort 1 and Cohort 3. One subject each in Cohort 1 and Cohort 3 did not have PK sample available following first dose. | nmol*hr/mL | GEOMETRIC_MEAN |
| SECONDARY | Change From Baseline in 6-minute Walk Distance (6MWD) | Baseline, Month 60 | Motor function was evaluated using the Efficacy Population (consisting of subjects who took at least 1 dose of study drug (20 mg/kg cipaglucosidase alfa + 260 mg miglustat) and had both a baseline and at least 1 post-baseline assessment for any efficacy endpoint. 6-minute walk distance (6MWD) was only performed in ambulatory subjects (Cohorts 1, 3, and 4). Baseline and Month 60 assessment available for 9 subjects in Cohort 1, 6 subjects in Cohort 3, and 4 subjects in Cohort 4. | meters | MEAN |
| SECONDARY | Change From Baseline in Pulmonary Function Tests | Baseline, Month 60 | Number of participants analyzed per cohort represents the number of subjects assessed at the specific visit for the parameter. Eight subjects in Cohort 1, 1 subject in Cohort 2, and 5 subjects in Cohort 4 had FVC values reported at Month 60. | percent predicted FVC | MEAN |
| SECONDARY | Change From Baseline in Muscle Strength Tests | Baseline, Month 60 | Total MMT score was evaluated using the Efficacy Population consisting of all enrolled subjects who took at least 1 dose of study drug (20 mg/kg cipaglucosidase alfa + 260 mg miglustat co-administration) in Stage 3 and had both a baseline and at least 1 post-baseline assessment for any efficacy endpoint. Number of participants analyzed per cohort represents the number of subjects assessed at the specific visit for the parameter. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Fatigue Severity Score (FSS) | Baseline, Month 60 | The Efficacy Population consists of all enrolled subjects who took at least 1 dose of study drug (20 mg/kg cipaglucosidase alfa + 260 mg miglustat co-administration) in Stage 3 and had both a baseline and at least 1 post-baseline assessment for any efficacy endpoint. Number of participants analyzed for FSS are those in each cohort who completed the assessment at baseline and Month 60. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Overall Physical Wellbeing (Subject's Global Impression of Change [SGIC], Question1) | Baseline, Month 60 | Number of participants analyzed per cohort represents the number of subjects assessed at the specific visit for the parameter. Eight subjects in Cohort 1, 2 subjects in Cohort 2, and 5 subjects in Cohort 4 had SGIC values reported at Month 60. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Overall Physical Wellbeing (Physician's Global Impression of Change [PGIC]) | Baseline, Month 60 | Number of participants analyzed per cohort represents the number of subjects assessed at the specific visit for the parameter. Eight subjects in Cohort 1, 3 subjects in Cohort 2, and 5 subjects in Cohort 4 had PGIC values reported at Month 60. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group67
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 Adverse Events (AEs) Leading to Discontinuation of Study Drug | Cohort 1 | Number of subjects with TEAEs | 11 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug | Cohort 2 | Number of subjects with TEAEs | 6 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug | Cohort 3 | Number of subjects with TEAEs | 6 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug | Cohort 4 | Number of subjects with TEAEs | 6 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug | Cohort 1 | Number of subjects with TESAEs | 7 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug | Cohort 2 | Number of subjects with TESAEs | 4 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug | Cohort 3 | Number of subjects with TESAEs | 4 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug | Cohort 4 | Number of subjects with TESAEs | 6 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug | Cohort 1 | Number of subjects with AEs leading to discontinuation | 1 | Participants | |
| Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug | Cohort 2 | Number of subjects with AEs leading to discontinuation | 2 | Participants |
1 to 10 of 67Source ClinicalTrials.gov
See all 67 results by groupParticipant flow72
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Stage 1 SAD (Cohort 1 Only) | STARTED | Cohort 1 (ERT Experienced; Ambulatory): SAD 5 to 20 mg/kg ATB200 | 11 | |
| Stage 1 SAD (Cohort 1 Only) | STARTED | Cohort 1 (ERT Experienced; Ambulatory) MAD 20 mg/kg ATB200 + 130 to 260 mg AT2221 | 0 | |
| Stage 1 SAD (Cohort 1 Only) | STARTED | Cohort 1 (ERT Experienced; Ambulatory): | 0 | |
| Stage 1 SAD (Cohort 1 Only) | STARTED | Cohort 2 (ERT Experienced; Non-ambulatory): 20 mg/kg ATB200 + 260 mg AT2221 | 0 | |
| Stage 1 SAD (Cohort 1 Only) | STARTED | Cohort 3 (ERT Naive; Ambulatory): 20 mg/kg ATB200 + 260 mg AT2221 | 0 | |
| Stage 1 SAD (Cohort 1 Only) | STARTED | Cohort 4 (ERT Experienced; Ambulatory): 20 mg/kg ATB200 + 260 mg AT2221 | 0 | |
| Stage 1 SAD (Cohort 1 Only) | COMPLETED | Cohort 1 (ERT Experienced; Ambulatory): SAD 5 to 20 mg/kg ATB200 | 11 | |
| Stage 1 SAD (Cohort 1 Only) | COMPLETED | Cohort 1 (ERT Experienced; Ambulatory) MAD 20 mg/kg ATB200 + 130 to 260 mg AT2221 | 0 | |
| Stage 1 SAD (Cohort 1 Only) | COMPLETED | Cohort 1 (ERT Experienced; Ambulatory): | 0 | |
| Stage 1 SAD (Cohort 1 Only) | COMPLETED | Cohort 2 (ERT Experienced; Non-ambulatory): 20 mg/kg ATB200 + 260 mg AT2221 | 0 |
1 to 10 of 72Source ClinicalTrials.gov
See all 72 participant flowAdverse events2,696
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 | Fall | Injury, poisoning and procedural complications | Cohort 1: ATB200 5 mg | 1 | 11 | 1 |
| other | Fall | Injury, poisoning and procedural complications | Cohort 1: ATB 200 10 mg | 1 | 11 | 1 |
| other | Fall | Injury, poisoning and procedural complications | Cohort 1: ATB200 20 mg | 0 | 11 | 0 |
| other | Fall | Injury, poisoning and procedural complications | Cohort 1: ATB200 20 mg + 130/260 mg Miglustat | 2 | 11 | 2 |
| other | Fall | Injury, poisoning and procedural complications | Cohort 1 ATB200 20 mg/kg + Miglustat 260 mg | 6 | 11 | 36 |
| other | Fall | Injury, poisoning and procedural complications | Cohort 2 ATB200 20 mg/kg + Miglustat 260 mg | 2 | 6 | 4 |
| other | Fall | Injury, poisoning and procedural complications | Cohort 3 ATB200 20 mg/kg + Miglustat 260 mg | 6 | 6 | 43 |
| other | Fall | Injury, poisoning and procedural complications | Cohort 4 ATB200 20 mg/kg + Miglustat 260 mg | 4 | 6 | 27 |
| other | Nasopharyngitis | Infections and infestations | Cohort 1: ATB200 5 mg | 0 | 11 | 0 |
| other | Nasopharyngitis | Infections and infestations | Cohort 1: ATB 200 10 mg | 1 | 11 | 1 |
1 to 10 of 2,696Source ClinicalTrials.gov
See all 2,696 adverse eventsAdverse event totals24
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: ATB200 5 mg | 0 | 11 |
| Total, all-cause mortality | Cohort 1: ATB 200 10 mg | 0 | 11 |
| Total, all-cause mortality | Cohort 1: ATB200 20 mg | 0 | 11 |
| Total, all-cause mortality | Cohort 1: ATB200 20 mg + 130/260 mg Miglustat | 0 | 11 |
| Total, all-cause mortality | Cohort 1 ATB200 20 mg/kg + Miglustat 260 mg | 0 | 11 |
| Total, all-cause mortality | Cohort 2 ATB200 20 mg/kg + Miglustat 260 mg | 1 | 6 |
| Total, all-cause mortality | Cohort 3 ATB200 20 mg/kg + Miglustat 260 mg | 0 | 6 |
| Total, all-cause mortality | Cohort 4 ATB200 20 mg/kg + Miglustat 260 mg | 0 | 6 |
| Total, other adverse events | Cohort 1: ATB200 5 mg | 7 | 11 |
| Total, other adverse events | Cohort 1: ATB 200 10 mg | 5 | 11 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 adverse event totalsPapers3
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 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/NCT02675465
GET https://conductscience.org/api/trials/v1/NCT02675465/results?page=2