ATB200/AT2221 for Pompe Disease

No longer recruiting at 63 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Amicus Therapeutics

Disqualifiers: Gene therapy, Pregnant, Breastfeeding, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment for individuals with late-onset Pompe disease, a rare condition affecting muscle strength. The treatment combines two drugs, ATB200 (an enzyme replacement therapy) and AT2221 (a pharmacological chaperone), to determine if they can improve muscle function and quality of life. The trial is open to those who have completed a previous study on this treatment. Participants should not plan to receive gene therapy or join another study for Pompe disease. As a Phase 3 trial, this is the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that ATB200 and AT2221 have been tested together for safety in people with Pompe disease. One study found that these treatments were generally well-tolerated over a long period. Participants received ATB200 intravenously and took AT2221 orally.

Some side effects occurred, but most were mild to moderate, with common issues including headache, nausea, and tiredness. Serious side effects were rare. Another study demonstrated that these treatments improved symptoms in mice, offering hope for human treatment.

These findings suggest that ATB200 and AT2221 can be safely used together, although some side effects may occur. As this trial is in a later phase, there is increased confidence in the treatment's safety for people. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about ATB200/AT2221 for Pompe disease because it offers a potentially game-changing approach to treatment. Unlike existing therapies that primarily rely on enzyme replacement, ATB200, combined with AT2221 (Miglustat), works to stabilize and enhance the enzyme's effectiveness. This combination aims to improve muscle function and reduce the storage of glycogen in cells more efficiently. By targeting both the enzyme stability and its uptake into cells, this treatment could offer patients improved outcomes compared to current standard therapies.

What evidence suggests that this trial's treatments could be effective for late-onset Pompe disease?

Research has shown that the combination of ATB200 and AT2221, which participants in this trial will receive, may help treat late-onset Pompe disease (LOPD). In one study, this treatment stabilized symptoms and improved some daily activities over 18 months. Studies in mice also demonstrated improvements in the disease's core problems. Another study confirmed these benefits, indicating that this combination can effectively address the disease. Overall, the evidence suggests that ATB200 and AT2221 could be a promising treatment option for people with LOPD. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for adults with late-onset Pompe disease who finished Study ATB200-03. Participants can't join if they're pregnant, breastfeeding, planning to have a child during the study, or if they plan to get gene therapy or join another Pompe disease study.

Inclusion Criteria

Subject must have completed Study ATB200-03.

Exclusion Criteria

Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.

Subject, if female, is pregnant or breastfeeding.

Subject, whether male or female, is planning to conceive a child during the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label Extension

Participants receive ATB200 co-administered with AT2221 capsule (Miglustat) until regulatory approval or marketing authorization

Up to approximately 4 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• AT2221
• ATB200

Trial Overview The trial is testing the long-term safety and effectiveness of two drugs, AT2221 and ATB200, in treating adult patients with late-onset Pompe disease. It's an international study where everyone gets the same treatment.

How Is the Trial Designed?

Group I: ATB200/AT2221 Experimental Treatment 2 Interventions

Participants received ATB200 co-administered with AT2221 capsule (Miglustat)

Who Is Running the Clinical Trial?

Amicus Therapeutics

Lead Sponsor

Trials

55

Recruited

2,700+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04138277

NCT04138277 | A Study to Assess the Long-term Safety ... Change from baseline in scores of EQ-5D-5L questionnaire to assess the efficacy of ATB200/AT2221 co-administration. baseline, up to approximately 4 years.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT03729362

NCT03729362 | A Study Comparing ATB200/AT2221 With ... 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 ...

3. ir.amicusrx.com ir.amicusrx.com/static-files/5d3c0beb-87c9-4482-b396-7b232205e855

Results From ATB200-02: First-in-Human Study of ... ATB200 Co-Administered With AT2221 for. Pompe Disease (18-Month Results) ... ̶ Data from a phase 1/2 trial of ATB200/AT2221 for the treatment of patients with.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11055775/

104-week efficacy and safety of cipaglucosidase alfa plus ... In pre-clinical studies, cipa + mig improved multiple defects along the Pompe disease pathogenic cascade in GAA knockout mice, including reduced ...

5. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2023/761204Orig1s000IntegratedR.pdf

761204Orig1s000 - accessdata.fda.gov ATB200/AT2221: Stabilization of ATB200 and Alglucosidase Alfa by Miglustat (AT2221). (Study #RRB200-010). The study objective was to show ...

6. clinicaltrial.be clinicaltrial.be/en/details/4565?per_page=20&only_recruiting=0&only_eligible=0&only_active=0

A Study to Assess the Long-term Safety and Efficacy of AT... A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects ...