Pompe Disease Infantile-OnsetNCT04327973
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
A study testing ATB200 and AT2221 in people with Pompe Disease Infantile-Onset, run by Amicus Therapeutics at 4 sites in 3 countries.
Trial summary
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221. Read more about this study
- Who is it for?
- Ages 0 to 17 years
- Conditions the study is for
- Pompe Disease Infantile-Onset
- What is being tested?
- ATB200 and AT2221
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 4 sitesin 3 countries
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
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
- Child
Who may be able to join
- Patient is male or female and 0 to < 18 years old.
- Patient has a diagnosis of Pompe disease, based on documentation of GAA genotyping demonstrating variants associated with IOPD.
- Patient does not currently qualify for or is unable to enroll (eg, due to location, etc) in any commercial drug-sponsored ongoing clinical trial.
- Patient is currently being treated with an approved ERT and is declining (eg, worsening in motor function, respiratory function/parameters, cardiac function, etc) based on treating physician's clinical judgement.
- The patient's legal guardian or representative has given informed consent (and assent when age appropriate for regional requirements) to treatment prior to administering ATB200/AT2221 in a manner consistent with all national requirements.
- If of reproductive potential and sexually active, female subjects agree to use a highly effective method of contraception throughout the duration of the treatment and for up to 90 days after their last dose of ATB200/AT2221.
Who cannot join
- Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221
- Patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator and/or Amicus, pose an undue safety risk to the patient.
- Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb,or Volibo)
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
United States2 sites
- Available
UF Health Shands Hospital
Gainesville, Florida, 32610
- Available
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
About this study
Official title: Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe Disease
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
Read the detailed description
This program is being offered on a patient by patient basis.
- Conditions
- Pompe Disease Infantile-Onset
- Being tested
- ATB200, AT2221
- Keywords
- expanded access, compassionate use
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATB200Biological
Participants received ATB200 co-administered with AT2221 capsule (Miglustat)
AT2221Drug
Participants received ATB200 co-administered with AT2221 capsule (Miglustat)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe Disease Infantile-OnsetStudies 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Amicus Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04327973
- Sponsor's protocol ID
- ATB200-15
- Status last verified
- Oct 2026
- First submitted
- 27 Mar 2020
- Last updated
- 27 Oct 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe Disease
- ClinicalTrials.gov ID
- NCT04327973
- Acronym / sponsor ID
- ATB200-15
- Conditions
- Pompe Disease Infantile-Onset
- Keywords
- expanded access, compassionate use
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Amicus TherapeuticsIndustry (pharma, biotech, devices)
- First posted
- 31 Mar 2020 (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 an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
Detailed description
This program is being offered on a patient by patient basis.
Study design
Who may qualify
- Ages
- 0 to 17 years
- Sex
- All
- Age groups
- Child
Key inclusion criteria (6 listed)
- Patient is male or female and 0 to < 18 years old.
- Patient has a diagnosis of Pompe disease, based on documentation of GAA genotyping demonstrating variants associated with IOPD.
- Patient does not currently qualify for or is unable to enroll (eg, due to location, etc) in any commercial drug-sponsored ongoing clinical trial.
- Patient is currently being treated with an approved ERT and is declining (eg, worsening in motor function, respiratory function/parameters, cardiac function, etc) based on treating physician's clinical judgement.
- The patient's legal guardian or representative has given informed consent (and assent when age appropriate for regional requirements) to treatment prior to administering ATB200/AT2221 in a manner consistent with all national requirements.
Key exclusion criteria (3 listed)
- Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221
- Patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator and/or Amicus, pose an undue safety risk to the patient.
- Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb,or Volibo)
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 |
|---|---|---|
| ATB200Participants received ATB200 co-administered with AT2221 capsule (Miglustat) | Biological | – |
| AT2221Participants received ATB200 co-administered with AT2221 capsule (Miglustat) | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2026.
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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.
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ATB200 and AT2221 in people with Pompe Disease Infantile-Onset, run by Amicus Therapeutics at 4 sites in 3 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 3 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: status not confirmed, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Italy, Taiwan. 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact For Site (866-9AMICUS, patientadvocacy@amicusrx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
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 27 Oct 2026.