Pompe DiseaseNCT03865836
Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
A study testing ATB200 and AT2221 in people with Pompe Disease, run by Amicus Therapeutics.
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?
- Any age
- Conditions the study is for
- Pompe Disease
- What is being tested?
- ATB200 and AT2221
- How many people
- Not given
- Where
- No sites listed yet
- 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 Any age
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Subject must have a diagnosis of Pompe disease based on documentation of one of the following:
- deficiency of GAA enzyme
- GAA genotyping
- Patient does not currently qualify for an Amicus sponsored on-going clinical trial or is declining on currently approved ERT (e.g. Myozyme)
- The patient must be willing to receive treatment with ATB200/AT2221 via this program, which includes signing an authorization form for sharing clinical data with Amicus Therapeutics, and its agent Early Access Care LLC.
Who cannot join
- Patient, whether male or female, is planning to conceive a child during the treatment program.
- 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, pose an undue safety risk to the subject or compromise his/her ability to comply with or adversely impact protocol requirements.
- Patient has received any gene therapy at any time.
- 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
No sites are listed yet.
About this study
Official title: Named Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With 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 and will require company, IRB/IEC and single patient IND approval.
- Conditions
- Pompe Disease
- Being tested
- ATB200, AT2221
- Keywords
- expanded access, compassionate use
- 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.
ATB200Biological
AT2221Drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe DiseaseStudies 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
- NCT03865836
- Sponsor's protocol ID
- ATB200-11
- Status last verified
- Oct 2026
- First submitted
- 5 Mar 2019
- 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
- Named Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With Pompe Disease
- ClinicalTrials.gov ID
- NCT03865836
- Acronym / sponsor ID
- ATB200-11
- Conditions
- Pompe Disease
- Keywords
- expanded access, compassionate use
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Amicus TherapeuticsIndustry (pharma, biotech, devices)
- First posted
- 7 Mar 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 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 and will require company, IRB/IEC and single patient IND approval.
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Subject must have a diagnosis of Pompe disease based on documentation of one of the following:
- Patient does not currently qualify for an Amicus sponsored on-going clinical trial or is declining on currently approved ERT (e.g. Myozyme)
- The patient must be willing to receive treatment with ATB200/AT2221 via this program, which includes signing an authorization form for sharing clinical data with Amicus Therapeutics, and its agent Early Access Care LLC.
Key exclusion criteria (5 listed)
- Patient, whether male or female, is planning to conceive a child during the treatment program.
- 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, pose an undue safety risk to the subject or compromise his/her ability to comply with or adversely impact protocol requirements.
- Patient has received any gene therapy at any time.
- 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 |
|---|---|---|
| ATB200 | Biological | – |
| AT2221 | 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.
- Related InfoLink provided by the sponsor.
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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.
Questions about this trial
What is this clinical trial studying?
A study testing ATB200 and AT2221 in people with Pompe Disease, run by Amicus Therapeutics.
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 5 inclusion and 5 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.
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.