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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

Study 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.Check my eligibility

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.See study design

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, up to approximately 4 years

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Proportion of participants with Treatment Emergent Adverse Events (TEAE)

Secondary outcome measures

6-Minute Walk Test

Change from baseline Biomarker -CK

Change from baseline Biomarker -uHex4

+14 more

Side effects data

From 2010 Phase 3 trial • 42 Patients • NCT00319046

74%

DIARRHOEA

50%

FLATULENCE

36%

TREMOR

21%

HEADACHE

21%

PARAESTHESIA

19%

FATIGUE

17%

DIZZINESS

14%

WEIGHT DECREASED

14%

CHITOTRIOSIDASE INCREASED

12%

PLATELET COUNT DECREASED

12%

HYPOAESTHESIA

10%

HAEMOGLOBIN DECREASED

10%

UPPER RESPIRATORY TRACT INFECTION

10%

NASOPHARYNGITIS

10%

MUSCLE SPASMS

10%

ABDOMINAL DISTENSION

10%

ABDOMINAL PAIN

10%

THROMBOCYTOPENIA

10%

NAUSEA

DEPRESSION

BLOOD FOLATE DECREASED

ANGIOTENSIN CONVERTING ENZYME INCREASED

ANAEMIA

ABDOMINAL PAIN UPPER

BONE PAIN

HAEMATOCHEZIA

BLOOD URINE PRESENT

TRANSITIONAL CELL CARCINOMA

BACK PAIN

CEREBELLAR SYNDROME

ABDOMINAL DISCOMFORT

ARTHRALGIA

CYST

JOINT SWELLING

COLON CANCER

HYPERREFLEXIA

PNEUMONIA

100%

80%

60%

40%

20%

Study treatment Arm

Miglustat

Trial Design

1Treatment groups

Experimental Treatment

Group I: ATB200/AT2221Experimental Treatment2 Interventions

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

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Amicus TherapeuticsLead Sponsor

54 Previous Clinical Trials

2,614 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this clinical trial available in more than one hospital in the city?

"There are 20 different clinical sites running this trial, but some notable locations include the IU Health Neuroscience Center in Indianapolis, Indiana, the Heritage Medical Research Clinic in Calgary, Alberta, and the McMaster University Medical Centre in Hamilton, Ontario."

Answered by AI

What similar research has been done with ATB200?

"ATB200 was first studied in 2016 at Duke University Medical Center, and there have been 12 completed clinical trials since then. Currently, there are 6 ongoing trials, many of which are based in Indianapolis, Indiana."

Answered by AI

Could you tell me the recruitment numbers for this trial?

"This research is no longer recruiting willing participants. The trial was originally advertised on December 18th, 2019 and has not been updated since June 9th, 2022. For those still looking for other studies, there are 18 clinical trials actively recruiting patients with acid maltase deficiency and 6 more for ATB200."

Answered by AI

Do you have any openings for new participants in this clinical trial?

"This study is no longer looking for new test subjects. The trial was first posted on December 18th, 2019 and was last updated on June 9th, 2022. Currently, there are 18 other clinical trials searching for patients with acid maltase deficiency and 6 trials for ATB200 that are actively looking for new participants."

Answered by AI