ATB200 for Acid Maltase Deficiency

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Acid Maltase Deficiency+1 More
ATB200 - Biological
Eligibility
18+
All Sexes
What conditions do you have?
Select

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.

Eligible Conditions
  • Acid Maltase Deficiency
  • Pompe Disease (Late-onset)

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Other trials for Acid Maltase Deficiency

Study Objectives

1 Primary · 17 Secondary · Reporting Duration: baseline, up to approximately 4 years

Year 4
6-Minute Walk Test
Change from baseline Biomarker -CK
Change from baseline Biomarker -uHex4
Change from baseline in muscle strength measured by Manual Muscle Strength testing
Change from baseline in muscle strength measured by Quantitative Muscle Strength testing
Change from baseline in scores of PROMIS - dyspnea questionnaire
Change from baseline in scores of PROMIS - fatigue questionnaire
Change from baseline in scores of PROMIS - physical function questionnaire
Change from baseline in scores of PROMIS - upper extremity questionnaire
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) questionnaires
Immunogenicity: Incidence of neutralizing
Immunogenicity: anti-drug antibodies
Motor Function - Gait, Stairs, Gower, Chair (GSGC) test
Physician's Global Impression of Change
Proportion of participants with Treatment Emergent Adverse Events (TEAE)
Pulmonary Function - Forced vital capacity (FVC)
Subject's Global Impression of Change
The Rasch-built Pompe-specific activity (R-PAct) questionnaires

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Acid Maltase Deficiency

Side Effects for

Miglustat
74%DIARRHOEA
50%FLATULENCE
36%TREMOR
21%HEADACHE
21%PARAESTHESIA
19%FATIGUE
17%DIZZINESS
14%WEIGHT DECREASED
14%CHITOTRIOSIDASE INCREASED
12%HYPOAESTHESIA
12%PLATELET COUNT DECREASED
10%ABDOMINAL DISTENSION
10%MUSCLE SPASMS
10%UPPER RESPIRATORY TRACT INFECTION
10%THROMBOCYTOPENIA
10%NAUSEA
10%HAEMOGLOBIN DECREASED
10%ABDOMINAL PAIN
10%NASOPHARYNGITIS
7%ANAEMIA
7%ANGIOTENSIN CONVERTING ENZYME INCREASED
7%DEPRESSION
7%ABDOMINAL PAIN UPPER
7%BLOOD FOLATE DECREASED
7%BONE PAIN
2%ARTHRALGIA
2%TRANSITIONAL CELL CARCINOMA
2%ABDOMINAL DISCOMFORT
2%CEREBELLAR SYNDROME
2%BACK PAIN
2%CYST
2%BLOOD URINE PRESENT
2%JOINT SWELLING
2%HAEMATOCHEZIA
2%COLON CANCER
2%HYPERREFLEXIA
2%PNEUMONIA
This histogram enumerates side effects from a completed 2010 Phase 3 trial (NCT00319046) in the Miglustat ARM group. Side effects include: DIARRHOEA with 74%, FLATULENCE with 50%, TREMOR with 36%, HEADACHE with 21%, PARAESTHESIA with 21%.

Trial Design

1 Treatment Group

ATB200/AT2221
1 of 1
Experimental Treatment

110 Total Participants · 1 Treatment Group

Primary Treatment: ATB200 · No Placebo Group · Phase 3

ATB200/AT2221Experimental Group · 2 Interventions: AT2221, ATB200 · Intervention Types: Drug, Biological
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Miglustat
FDA approved
Cipaglucosidase alfa
Not yet FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline, up to approximately 4 years

Who is running the clinical trial?

Amicus TherapeuticsLead Sponsor
52 Previous Clinical Trials
2,069 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 1 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 13th, 2021

Last Reviewed: October 7th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.