First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and Whe… (NCT02675465) | Clinical Trial Compass
CompletedPhase 1/2
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
United States, Australia, Germany29 participantsStarted 2016-04
Plain-language summary
This is an international, multi-center, open-label study designed to evaluate if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Adults with Diagnosis of Pompe disease
Cohort 1: Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):
* Male and female subjects between 18 and 65 years of age, inclusive
* Received ERT with alglucosidase alfa (Myozyme/Lumizyme) for the previous 2-6 years, inclusive
* Was receiving alglucosidase alfa at a frequency of once every other week
* Must have been able to walk 200-500 meters on the 6-Minute Walk Test (6MWT)
* Had upright Forced Vial Capacity (FVC) 30-80% of predicted normal value
Cohort 2: ERT-experienced subjects (non-ambulatory):
* Male and female subjects between 18 and 65 years of age, inclusive
* Had been receiving ERT with alglucosidase alfa for ≥2 years at a regular or set frequency
* Was wheelchair-bound
Cohort 3: ERT-naïve subjects (ambulatory):
* Male and female subjects between 18 and 65 years of age, inclusive
* Must have been able to walk 200-500 meters on the 6MWT
* Had upright FVC 30-80% of predicted normal value
Cohort 4: ERT-experienced subject (ambulatory):
* Male and female subjects between 18 and 75 years of age, inclusive
* Had been receiving ERT with alglucosidase alfa for ≥7 years, inclusive
* Was receiving alglucosidase alfa at a frequency of once every other week
* Must have been able to walk 75-600 meters on the 6MWT
* Had upright FVC 30-85% of predicted normal value
Exclusion Criteria:
* Received treatment with prohibited medications within 30 days of Baseline Visit
* Subject, if female, was pregnant or breastfeeding…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug
Timeframe: Stage 3 (2 year treatment) and Stage 4 (Extension) combined, (mean = 71 months on treatment)
2
Plasma Human Acid α-glucosidase (GAA) Activity Levels as Measured by Maximum Observed Plasma Concentration (Cmax).
Timeframe: 18 Weeks
3
Plasma GAA Activity Levels as Measured by Time to Reach the Maximum Observed Plasma Concentration (Tmax).
Timeframe: 18 Weeks
4
Plasma GAA Activity Levels as Measured by Area Under the Plasma Drug Concentration-time Curve (AUC).