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
This is further along than 85% of similar trials
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
This is further along than 85% of similar trials
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
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 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.
Popular Clinical Trials
Top Clinical Trials in Cities
Relevant Clinical Trials
Unbiased Results
We believe in providing patients with all the options.Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.Back to top
Conditions
Cancer Related
Treatments