Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03729362
Registration number
NCT03729362
Ethics application status
Date submitted
10/10/2018
Date registered
2/11/2018
Date last updated
11/09/2023
Titles & IDs
Public title
A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
Query!
Scientific title
A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo
Query!
Secondary ID [1]
0
0
ATB200-03
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
PROPEL
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Pompe Disease (Late-onset)
0
0
Query!
Condition category
Condition code
Metabolic and Endocrine
0
0
0
0
Query!
Metabolic disorders
Query!
Metabolic and Endocrine
0
0
0
0
Query!
Other metabolic disorders
Query!
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Other interventions - Cipaglucosidase Alfa
Treatment: Drugs - Miglustat
Other interventions - Alglucosidase Alfa
Treatment: Drugs - Placebo
Experimental: Cipaglucosidase Alfa/Miglustat - Participants received cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W).
Active Comparator: Alglucosidase Alfa/Placebo - Participants received alglucosidase alfa co-administered with placebo Q2W.
Other interventions: Cipaglucosidase Alfa
Participants received an intravenous (IV) infusion dose over a 4-hour duration every 2 weeks (Q2W).
Treatment: Drugs: Miglustat
Participants received weight-based doses 1 hour prior to cipaglucosidase alfa infusion Q2W.
Other interventions: Alglucosidase Alfa
Participants received an IV infusion dose over a 4-hour duration Q2W.
Treatment: Drugs: Placebo
Miglustat matching placebo was administered orally 1 hour prior to alglucosidase alfa infusion Q2W.
Query!
Intervention code [1]
0
0
Other interventions
Query!
Intervention code [2]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD)
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Baseline, Week 52
Query!
Secondary outcome [1]
0
0
Change From Baseline to Week 52 in Sitting Forced Vital Capacity (FVC; % Predicted)
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Baseline, Week 52
Query!
Secondary outcome [2]
0
0
Change From Baseline to Week 52 in the Manual Muscle Test (MMT) Score for the Lower Extremities
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
Baseline, Week 52
Query!
Secondary outcome [3]
0
0
Change From Baseline to Week 26 in 6MWD
Query!
Assessment method [3]
0
0
The 6MWD, measured in meters, is the distance walked on the 6MWT.
Query!
Timepoint [3]
0
0
Baseline, Week 26
Query!
Secondary outcome [4]
0
0
Change From Baseline to Week 52 in the Total Score for the Patient- Reported Outcomes Measurement Information System (PROMIS®) - Physical Function
Query!
Assessment method [4]
0
0
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.
Query!
Timepoint [4]
0
0
Baseline, Week 52
Query!
Secondary outcome [5]
0
0
Change From Baseline to Week 52 in the Total Score for the PROMIS® - Fatigue
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
Baseline, Week 52
Query!
Secondary outcome [6]
0
0
Change From Baseline to Week 52 in the Total Score for the Gait, Stairs, Gowers' Maneuver, and Chair (GSGC)
Query!
Assessment method [6]
0
0
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).
Query!
Timepoint [6]
0
0
Baseline, Week 52
Query!
Secondary outcome [7]
0
0
Change From Baseline to Week 52 in Rasch-Built Pompe-Specific Activity (R-PAct) Total Score
Query!
Assessment method [7]
0
0
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.
Query!
Timepoint [7]
0
0
Baseline, Week 52
Query!
Secondary outcome [8]
0
0
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
Query!
Assessment method [8]
0
0
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.
Query!
Timepoint [8]
0
0
Baseline, Week 52
Query!
Secondary outcome [9]
0
0
Change From Baseline to Week 52 in Sitting Slow Vital Capacity (SVC) % Predicted
Query!
Assessment method [9]
0
0
SVC is a standard pulmonary function test used to quantify respiratory muscle weakness.
Query!
Timepoint [9]
0
0
Baseline, Week 52
Query!
Secondary outcome [10]
0
0
Change From Baseline to Week 52 in Maximal Inspiratory Pressure (MIP) % Predicted
Query!
Assessment method [10]
0
0
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).
Query!
Timepoint [10]
0
0
Baseline, Week 52
Query!
Secondary outcome [11]
0
0
Change From Baseline to Week 52 in Maximal Expiratory Pressure (MEP) % Predicted
Query!
Assessment method [11]
0
0
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).
Query!
Timepoint [11]
0
0
Baseline, Week 52
Query!
Secondary outcome [12]
0
0
Change From Baseline to Week 52 in Sniff Nasal Inspiratory Pressure (SNIP) % Predicted
Query!
Assessment method [12]
0
0
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).
Query!
Timepoint [12]
0
0
Baseline, Week 52
Query!
Secondary outcome [13]
0
0
Change From Baseline to Week 52 in % Predicted 6MWD
Query!
Assessment method [13]
0
0
The % predicted 6MWD = (actual 6MWD / predicted 6MWD) * 100. The predicted values were calculated using Enright And Sherrill 1998 Reference Equations.
Query!
Timepoint [13]
0
0
Baseline, Week 52
Query!
Secondary outcome [14]
0
0
Change From Baseline to Week 52 in the Quantitative Muscle Test (QMT) Values
Query!
Assessment method [14]
0
0
QMT was measured using the hand-held dynamometer. Larger values (in kg) indicated greater muscle strength.
Query!
Timepoint [14]
0
0
Baseline, Week 52
Query!
Secondary outcome [15]
0
0
Change From Baseline to Week 52 in Other MMT Scores
Query!
Assessment method [15]
0
0
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.
Query!
Timepoint [15]
0
0
Baseline, Week 52
Query!
Secondary outcome [16]
0
0
Change From Baseline to Week 52 in Maximum Vital Capacity (Maximum VC) % Predicted
Query!
Assessment method [16]
0
0
Maximum VC is the greater of the two VC values (FVC or SVC).
Query!
Timepoint [16]
0
0
Baseline, Week 52
Query!
Secondary outcome [17]
0
0
Change From Baseline to Week 52 in PROMIS-Dyspnea and Upper Extremities Total Scores
Query!
Assessment method [17]
0
0
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.
Query!
Timepoint [17]
0
0
Baseline, Week 52
Query!
Secondary outcome [18]
0
0
Change From Baseline in the Time to Complete Individual GSGC Component Tests and Timed Up and Go (TUG) Test at Week 52
Query!
Assessment method [18]
0
0
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.
Query!
Timepoint [18]
0
0
Baseline, Week 52
Query!
Secondary outcome [19]
0
0
Physician's Global Impression of Change (PGIC) Overall Status at Week 52
Query!
Assessment method [19]
0
0
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.
Query!
Timepoint [19]
0
0
Week 52
Query!
Secondary outcome [20]
0
0
Subject's Global Impression of Change (SGIC) at Week 52
Query!
Assessment method [20]
0
0
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.
Query!
Timepoint [20]
0
0
Week 52
Query!
Secondary outcome [21]
0
0
Number of Participants Improving on Both 6MWD and % Predicted FVC at Week 52
Query!
Assessment method [21]
0
0
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.
Query!
Timepoint [21]
0
0
Week 52
Query!
Secondary outcome [22]
0
0
Number of Participants With Treatment-Emergent Anti-Drug Antibodies (ADAs)
Query!
Assessment method [22]
0
0
Treatment-emergent ADAs were defined as participants who had seroconverted or boosted their preexisting ADA during the study period.
Query!
Timepoint [22]
0
0
Baseline up to Week 52
Query!
Secondary outcome [23]
0
0
Change From Baseline to Week 52 in Urinary Hexose Tetrasaccharide (Hex4) Level
Query!
Assessment method [23]
0
0
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.
Query!
Timepoint [23]
0
0
Baseline, Week 52
Query!
Secondary outcome [24]
0
0
Change From Baseline to Week 52 in Serum Creatine Kinase (CK) Level
Query!
Assessment method [24]
0
0
Change from baseline to Week 52 in serum CK level. CK levels were measured as part of the serum chemistry panel.
Query!
Timepoint [24]
0
0
Baseline, Week 52
Query!
Secondary outcome [25]
0
0
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
Query!
Assessment method [25]
0
0
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.
Query!
Timepoint [25]
0
0
Days 1 and 364 (Week 52)
Query!
Secondary outcome [26]
0
0
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
Query!
Assessment method [26]
0
0
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.
Query!
Timepoint [26]
0
0
Days 1 and 364 (Week 52)
Query!
Secondary outcome [27]
0
0
Population PK: Cmax of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Naïve Participants
Query!
Assessment method [27]
0
0
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.
Query!
Timepoint [27]
0
0
Days 1 and 364 (Week 52)
Query!
Secondary outcome [28]
0
0
Population PK: AUC of Cipaglucosidase Alfa and Alglucosidase Alfa in ERT-Naïve Subjects
Query!
Assessment method [28]
0
0
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.
Query!
Timepoint [28]
0
0
Days 1 and 364 (Week 52)
Query!
Secondary outcome [29]
0
0
Noncompartmental Analysis: Cmax of Plasma Total GAA Protein by Signature Peptide T09 in ERT-Naïve Subjects
Query!
Assessment method [29]
0
0
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 a-glucosidase (GAA) protein signature peptide T09 and plasma miglustat determinations.
Query!
Timepoint [29]
0
0
Day 1
Query!
Secondary outcome [30]
0
0
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
Query!
Assessment method [30]
0
0
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.
Query!
Timepoint [30]
0
0
Day 1
Query!
Secondary outcome [31]
0
0
Comparison of Cmax of Cipaglucosidase Alfa in ERT-Experienced and ERT-Naïve Populations
Query!
Assessment method [31]
0
0
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.
Query!
Timepoint [31]
0
0
Days 1 and 364 (Week 52)
Query!
Secondary outcome [32]
0
0
Comparison of AUC of Cipaglucosidase Alfa in ERT- Experienced and ERT-Naïve Populations
Query!
Assessment method [32]
0
0
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.
Query!
Timepoint [32]
0
0
Days 1 and 364 (Week 52)
Query!
Eligibility
Key inclusion criteria
1. Subject must provide signed informed consent prior to any study-related procedures
being performed.
2. Male and female subjects are = 18 years old and weigh = 40 kg at screening.
3. Female subjects of childbearing potential and male subjects must agree to use
medically accepted methods of contraception during the study and for 90 days after the
last dose of study drug.
4. Subject must have a diagnosis of LOPD based on documentation of one of the following:
1. deficiency of GAA enzyme
2. GAA genotyping
5. Subject is classified as one of the following with respect to ERT status:
1. ERT-experienced, defined as currently receiving standard of care ERT
(alglucosidase alfa) at the recommended dose and regimen (ie, 20 mg/kg dose every
2 weeks) for = 24 months
2. ERT-naïve, defined as never having received investigational or commercially
available ERT
6. Subject has a sitting FVC = 30% of the predicted value for healthy adults (National
Health and Nutrition Examination Survey III) at screening.
7. Subject performs two 6MWTs at screening that are valid, as determined by the clinical
evaluator, and that meet all of the following criteria:
1. both screening values of 6MWD are = 75 meters
2. both screening values of 6MWD are = 90% of the predicted value for healthy adults
3. the lower value of 6MWD is within 20% of the higher value of 6MWD
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
Exclusion Criteria
1. Subject has received any investigational therapy or pharmacological treatment for
Pompe disease, other than alglucosidase alfa, within 30 days or 5 half-lives of the
therapy or treatment, whichever is longer, before Day 1 or is anticipated to do so
during the study.
2. Subject has received gene therapy for Pompe disease
3. Subject is taking any of the following prohibited medications within 30 days before
Day 1:
- miglitol (eg, Glyset)
- miglustat (eg, Zavesca)
- acarbose (eg, Precose or Glucobay)
- voglibose (eg, Volix, Vocarb, or Volibo)
Note: None of these medications have a half-life that, when multiplied by 5, is longer
than 30 days.
4. Subject requires the use of invasive or noninvasive ventilation support for > 6 hours
per day while awake.
5. Subject has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa,
or AT2221.
6. Subject has a medical condition or any other extenuating circumstance that may, in the
opinion of the investigator or medical monitor, pose an undue safety risk to the
subject or may compromise his/her ability to comply with or adversely impact protocol
requirements. This includes clinical depression (as diagnosed by a psychiatrist or
other mental health professional) with uncontrolled or poorly controlled symptoms.
7. Subject, if female, is pregnant or breastfeeding at screening.
8. Subject, whether male or female, is planning to conceive a child during the study.
9. Subject does not have documentation of diagnosis of Pompe disease and refuses to
undergo genetic testing.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
4/12/2018
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
15/01/2021
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
125
Query!
Recruitment in Australia
Recruitment state(s)
QLD,SA,VIC
Query!
Recruitment hospital [1]
0
0
Royal Brisbane & Women's Hospital - Herston
Query!
Recruitment hospital [2]
0
0
Royal Adelaide Hospital - Adelaide
Query!
Recruitment hospital [3]
0
0
Monash Medical Centre - Melbourne
Query!
Recruitment hospital [4]
0
0
Westmead Hospital - Westmead
Query!
Recruitment postcode(s) [1]
0
0
4029 - Herston
Query!
Recruitment postcode(s) [2]
0
0
5000 - Adelaide
Query!
Recruitment postcode(s) [3]
0
0
3168 - Melbourne
Query!
Recruitment postcode(s) [4]
0
0
2145 - Westmead
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Arizona
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Arkansas
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
California
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Florida
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Georgia
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Indiana
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Kansas
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Minnesota
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Missouri
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Montana
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
New Jersey
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
New York
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
North Carolina
Query!
Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
Ohio
Query!
Country [15]
0
0
United States of America
Query!
State/province [15]
0
0
Oregon
Query!
Country [16]
0
0
United States of America
Query!
State/province [16]
0
0
Pennsylvania
Query!
Country [17]
0
0
United States of America
Query!
State/province [17]
0
0
Texas
Query!
Country [18]
0
0
United States of America
Query!
State/province [18]
0
0
Utah
Query!
Country [19]
0
0
United States of America
Query!
State/province [19]
0
0
Virginia
Query!
Country [20]
0
0
Argentina
Query!
State/province [20]
0
0
Buenos Aires
Query!
Country [21]
0
0
Austria
Query!
State/province [21]
0
0
Innsbruck
Query!
Country [22]
0
0
Belgium
Query!
State/province [22]
0
0
Leuven
Query!
Country [23]
0
0
Bosnia and Herzegovina
Query!
State/province [23]
0
0
Banja Luka
Query!
Country [24]
0
0
Bulgaria
Query!
State/province [24]
0
0
Sofia
Query!
Country [25]
0
0
Canada
Query!
State/province [25]
0
0
Alberta
Query!
Country [26]
0
0
Canada
Query!
State/province [26]
0
0
Ontario
Query!
Country [27]
0
0
Denmark
Query!
State/province [27]
0
0
Aarhus N
Query!
Country [28]
0
0
Denmark
Query!
State/province [28]
0
0
Copenhagen
Query!
Country [29]
0
0
France
Query!
State/province [29]
0
0
Bron
Query!
Country [30]
0
0
France
Query!
State/province [30]
0
0
Garches
Query!
Country [31]
0
0
France
Query!
State/province [31]
0
0
Lille
Query!
Country [32]
0
0
France
Query!
State/province [32]
0
0
Marseille
Query!
Country [33]
0
0
France
Query!
State/province [33]
0
0
Nice
Query!
Country [34]
0
0
Germany
Query!
State/province [34]
0
0
Bavaria
Query!
Country [35]
0
0
Germany
Query!
State/province [35]
0
0
NRW
Query!
Country [36]
0
0
Germany
Query!
State/province [36]
0
0
Sachsen-Anhalt
Query!
Country [37]
0
0
Greece
Query!
State/province [37]
0
0
Attica
Query!
Country [38]
0
0
Hungary
Query!
State/province [38]
0
0
Budapest
Query!
Country [39]
0
0
Hungary
Query!
State/province [39]
0
0
Pécs
Query!
Country [40]
0
0
Hungary
Query!
State/province [40]
0
0
Szeged
Query!
Country [41]
0
0
Italy
Query!
State/province [41]
0
0
NAP
Query!
Country [42]
0
0
Japan
Query!
State/province [42]
0
0
Hokkaido
Query!
Country [43]
0
0
Japan
Query!
State/province [43]
0
0
Fukuoka
Query!
Country [44]
0
0
Japan
Query!
State/province [44]
0
0
Kagoshima
Query!
Country [45]
0
0
Japan
Query!
State/province [45]
0
0
Osaka
Query!
Country [46]
0
0
Japan
Query!
State/province [46]
0
0
Tokyo
Query!
Country [47]
0
0
Korea, Republic of
Query!
State/province [47]
0
0
Gyeongsangnam-do
Query!
Country [48]
0
0
Korea, Republic of
Query!
State/province [48]
0
0
Seoul
Query!
Country [49]
0
0
Netherlands
Query!
State/province [49]
0
0
Rotterdam
Query!
Country [50]
0
0
New Zealand
Query!
State/province [50]
0
0
Auckland
Query!
Country [51]
0
0
Poland
Query!
State/province [51]
0
0
Malogoskie
Query!
Country [52]
0
0
Poland
Query!
State/province [52]
0
0
Rzeszów
Query!
Country [53]
0
0
Slovenia
Query!
State/province [53]
0
0
Ljubljana
Query!
Country [54]
0
0
Spain
Query!
State/province [54]
0
0
Barcelona
Query!
Country [55]
0
0
Sweden
Query!
State/province [55]
0
0
Gothenburg
Query!
Country [56]
0
0
Taiwan
Query!
State/province [56]
0
0
Taipei
Query!
Country [57]
0
0
United Kingdom
Query!
State/province [57]
0
0
Birmingham
Query!
Country [58]
0
0
United Kingdom
Query!
State/province [58]
0
0
Cambridge
Query!
Country [59]
0
0
United Kingdom
Query!
State/province [59]
0
0
London
Query!
Country [60]
0
0
United Kingdom
Query!
State/province [60]
0
0
Salford
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Amicus Therapeutics
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
This is a phase 3 double-blind randomized study to study the efficacy and safety of
intravenous ATB200 Co-administered with oral AT2221 in adult subjects with Late Onset Pompe
Disease compared with Alglucosidase Alfa/placebo.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT03729362
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03729362
Download to PDF