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Enzyme Replacement Therapy

ATB200/AT2221 for Pompe Disease

Phase 3
Waitlist Available
Research Sponsored by Amicus Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, up to approximately 4 years
Awards & highlights
Pivotal Trial
No Placebo-Only Group

Summary

This trial is for people with late-onset Pompe disease who have completed a previous study. It is an extension of that study to continue to evaluate the safety and efficacy of the study drug.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While specific side effects are not listed here, participants will be monitored for any adverse reactions due to long-term use of the drugs AT2221 and ATB200 as part of their ongoing treatment for Pompe disease.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, up to approximately 4 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, up to approximately 4 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
6-Minute Walk Test
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) questionnaires
Motor Function - Gait, Stairs, Gower, Chair (GSGC) test
+4 more

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: ATB200/AT2221Experimental Treatment2 Interventions
Participants received ATB200 co-administered with AT2221 capsule (Miglustat)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AT2221
2016
Completed Phase 2
~30
ATB200
2016
Completed Phase 2
~30

Find a Location

Who is running the clinical trial?

Amicus TherapeuticsLead Sponsor
54 Previous Clinical Trials
2,617 Total Patients Enrolled

Media Library

AT2221 (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04138277 — Phase 3
Pompe Disease Research Study Groups: ATB200/AT2221
Pompe Disease Clinical Trial 2023: AT2221 Highlights & Side Effects. Trial Name: NCT04138277 — Phase 3
AT2221 (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04138277 — Phase 3
~0 spots leftby Dec 2024
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