Pompe Disease (Late-onset)Phase 3NCT03911505
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
A Phase 3 study testing Cipaglucosidase Alfa and Miglustat in people with Pompe Disease (Late-onset), run by Amicus Therapeutics at 17 sites in 6 countries.
Trial summary
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years Read more about this study
- Who is it for?
- Ages 0 to 17 years
- Conditions the study is for
- Pompe Disease (Late-onset)
- What is being tested?
- Cipaglucosidase Alfa and Miglustat
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 17 sitesin 6 countries
- Study dates
- 13 Feb 2020to Sep 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female subjects (ERT-naïve [have never received a dose of rhGAA] or ERT-experienced [have received rhGAA every 2 weeks for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 months before enrollment]) diagnosed with LOPD who are aged 12 to <18 years at screening (Cohort 1 only) or aged 0 months to < 12 years at screening (Cohort 2 only)
- Subject weighs ≤ 115 kg. (Cohort 1 Only)
- Subject must have a diagnosis of LOPD based on documentation as defined in study protocol
- If of reproductive potential and if sexually active, female and male subjects agree to use a highly effective method of contraception throughout the duration of the study and for up to 90 days after their last dose of Cipaglucosidase Alfa/Miglustat
- Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy Adolescents at screening (Cohort 1 only)
- Subject (aged 12 to <18 years; Cohort 1) performs one 6-Minute Walk Test (6MWT) (≥ 75 meters) at screening that is valid, as determined by the clinical evaluator, or subject (aged ≥ 5 to < 12 years; Cohort 2) performs one 6MWT (≥ 40 meters) at screening that is valid, as determined by the clinical evaluator
Who cannot join
- Subject has received any investigational/experimental drug, oral anabolic steroid or derivative, biologic, or device within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before screening
- Subject has received treatment with prohibited medications within 30 days of screening
- Subject has received any gene therapy at any time
- Subject has any intercurrent illness or condition at screening or baseline that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator and/or the medical monitor that the potential subject may have an unacceptable risk by participating in this study
- Subject has a hypersensitivity to any of the excipients in ATB200, approved rhGAA, or AT2221
- Female subject is pregnant or breast-feeding at screening
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- Subject requires the use of ventilation support for > 6 hours per day while awake
- Subject has evidence of moderate to severe hypertrophic cardiomyopathy aligning with classic IOPD
- In the opinion of the investigator, the parent or legally authorized representative is unlikely or unable to comply with the study requirements
- Subject has any prior history of illness or condition known to affect motor function, such as, but not limited to, Guillain-Barre syndrome, cerebral palsy, etc
- Subject who is diagnosed with Pompe disease via newborn screening and is asymptomatic (ie, showing no signs and symptoms of Pompe disease (Cohort 2 Only)
Trial locations and contacts
17 sites in 6 countries.
17 sites in 6 countries
United States9 sites
Woodruff Memorial Research Building
Atlanta, Georgia, 30322
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Duke University Medical Center
Durham, North Carolina, 27710
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia, 22030
University of Florida Clinical Research Center
Gainesville, Florida, 32610
Wolfson Children's Hospital
Jacksonville, Florida, 32207
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
University of Utah, Clinical and Translational Sciences Institute
Salt Lake City, Utah, 84108
St. Louis Children's Hospital
St Louis, Missouri, 63110
Japan4 sites
Gunma University Hospital
Gunma, 371-8511
Tohoku University Hospital
Miyagi, 980-8574
Izumi City General Hospital
Osaka, Izumi-Shi, 594-0073
Tokyo Women's Medical University
Tokyo, 162-8666
Australia1 site
Women's and Children's Hospital
North Adelaide, South Australia, 5006
Germany1 site
SphinCS GmbH Clinical Science for LSD
Hochheim am Main, Hesse, 65239
About this study
Official title: An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years
- Conditions
- Pompe Disease (Late-onset)
- Being tested
- Cipaglucosidase Alfa, Miglustat
- Keywords
- Pompe, rhGAA
- Medical subject headings
- Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cipaglucosidase AlfaBiological
Also called ATB200
Enzyme Replacement Therapy via intravenous infusion
MiglustatDrug
Also called AT2221
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221)
| Study group | Role | What they receive |
|---|---|---|
| Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221) | Experimental | Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule Receives: Biological: Cipaglucosidase Alfa; Drug: Miglustat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (TEAEs) from baseline
Measured: 52 weeks
Secondary outcomes (1)
Assessment of pharmacokinetic parameters
ATB200 and AT2221 concentrations in plasma
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2019
- Study start13 Feb 2020
- Primary completionMar 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe Disease (Late-onset)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amicus Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03911505
- Sponsor's protocol ID
- ATB200-04
- Status last verified
- Sep 2026
- First submitted
- 9 Apr 2019
- Last updated
- 17 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease
- ClinicalTrials.gov ID
- NCT03911505
- Acronym / sponsor ID
- ATB200-04
- Conditions
- Pompe Disease (Late-onset)
- Keywords
- Pompe, rhGAA
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Amicus TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 13 Feb 2020 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 11 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221) | Experimental | Biological: Cipaglucosidase Alfa, Drug: MiglustatParticipants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule |
Who may qualify
- Ages
- 0 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Male or female subjects (ERT-naïve [have never received a dose of rhGAA] or ERT-experienced [have received rhGAA every 2 weeks for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 months before enrollment]) diagnosed with LOPD who are aged 12 to <18 years at screening (Cohort 1 only) or aged 0 months to < 12 years at screening (Cohort 2 only)
- Subject weighs ≤ 115 kg. (Cohort 1 Only)
- Subject must have a diagnosis of LOPD based on documentation as defined in study protocol
- If of reproductive potential and if sexually active, female and male subjects agree to use a highly effective method of contraception throughout the duration of the study and for up to 90 days after their last dose of Cipaglucosidase Alfa/Miglustat
- Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy Adolescents at screening (Cohort 1 only)
Key exclusion criteria (11 listed)
- Subject has received any investigational/experimental drug, oral anabolic steroid or derivative, biologic, or device within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before screening
- Subject has received treatment with prohibited medications within 30 days of screening
- Subject has received any gene therapy at any time
- Subject has any intercurrent illness or condition at screening or baseline that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator and/or the medical monitor that the potential subject may have an unacceptable risk by participating in this study
- Subject has a hypersensitivity to any of the excipients in ATB200, approved rhGAA, or AT2221
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cipaglucosidase AlfaAlso: ATB200Enzyme Replacement Therapy via intravenous infusion | Biological | Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221) |
| MiglustatAlso: AT2221Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221) | Drug | Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221) |
Primary outcome
Incidence of treatment-emergent adverse events (TEAEs) from baseline
Time frame: 52 weeks
1 secondary outcome
Assessment of pharmacokinetic parameters
Time frame: 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
17 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Cipaglucosidase Alfa and Miglustat in people with Pompe Disease (Late-onset), run by Amicus Therapeutics at 17 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, Japan, Australia, Canada, Germany and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (TEAEs) from baseline (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2026.