PROPEL Study - A Study Comparing ATB200/AT2221 With Alglucosidase/Placebo in Adult Subjects With LOPD

• Register: ClinicalTrials.gov
• Last refreshed on: 18 October 2021
• Main ID: NCT03729362
• Date of registration: 10/10/2018
• Prospective Registration: Yes
• Primary sponsor: Amicus Therapeutics
• Public title: PROPEL Study - A Study Comparing ATB200/AT2221 With Alglucosidase/Placebo in Adult Subjects With LOPD
• 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
• Date of first enrolment: December 4, 2018
• Target sample size: 123
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03729362
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Austria, Belgium, Bosnia and Herzegovina, Bulgaria, Canada, Denmark, France, Germany, Greece, Hungary, Italy, Japan, Korea, Republic of, Netherlands, New Zealand, Poland, Slovenia, Spain, Sweden, Taiwan, United Kingdom, United States

Key inclusion & exclusion criteria

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

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Pompe Disease (Late-onset)

Intervention(s)

Drug: AT2221

Biological: alglucosidase alfa

Biological: ATB200

Primary Outcome(s)

6-Minute Walk Test [Time Frame: 12 months]

Secondary Outcome(s)

Physician Overall Clinical Impression [Time Frame: 12 months]

Pulmonary Function - Slow Vital Capacity (SVC) [Time Frame: 12 months]

Biomarkers/Pharmacodynamics of muscle injury and disease substrate [Time Frame: 12 months]

EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) questionnaires [Time Frame: 12 months]

Immunogenicity [Time Frame: 12 months]

Motor Function - Timed Up and Go (TUG) [Time Frame: 12 months]

popPK: CLT [Time Frame: 12 months]

Pulmonary Function - Maximum Expiratory Pressure [Time Frame: 12 months]

popPK: Tmax [Time Frame: 12 months]

Manual Muscle Strength [Time Frame: 12 months]

Pulmonary Function - Sniff Nasal Inspiratory Pressure (SNIP) [Time Frame: 12 months]

popPK: Cmax [Time Frame: 12 months]

Subject's Global Impression of Change questionnaires [Time Frame: 12 months]

Motor Function - Gait, Stairs, Gower, Chair (GSGC) test [Time Frame: 12 months]

popPK: AUC0-inf [Time Frame: 12 months]

popPK: t1/2 [Time Frame: 12 months]

PROMIS instruments questionnaires [Time Frame: 12 months]

Pulmonary Function - Forced vital capacity (FVC) [Time Frame: 12 months]

Quantitative Muscle Strength [Time Frame: 12 months]

Number of participants with TEAEs and SARs [Time Frame: 12 months]

Pulmonary Function - Maximum Inspiratory Pressure (MIP) [Time Frame: 12 months]

The Rasch-built Pompe-specific activity (R-PAct) questionnaires [Time Frame: 12 months]

Secondary ID(s)

ATB200-03

Source(s) of Monetary Support

Please refer to primary and secondary sponsors