Phase 2 N=11 Treatment

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Pompe Disease

Bottom Line

View on ClinicalTrials.gov: NCT02675465 ↗

Enrolled (actual)

Serious AEs

28.8%

Results posted

Oct 2025

Primary outcome: Primary: Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug — 11; 6; 6; 6 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: ATB200 (Drug); AT2221 (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Amicus Therapeutics
Primary completion: Aug 2024

Outcome Measures

Outcome	Result	p-value
PRIMARY Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug	11; 6; 6; 6; 7; 4	—
PRIMARY Plasma Human Acid α-glucosidase (GAA) Activity Levels as Measured by Maximum Observed Plasma Concentration (Cmax).	108836; 132400; 119624; 105842	—
PRIMARY Plasma GAA Activity Levels as Measured by Time to Reach the Maximum Observed Plasma Concentration (Tmax).	3.49; 3.97; 3.57; 3.66	—
PRIMARY Plasma GAA Activity Levels as Measured by Area Under the Plasma Drug Concentration-time Curve (AUC).	608180; 762484; 670754; 638984	—
SECONDARY Change From Baseline in 6-minute Walk Distance (6MWD)	9.2; -34.9; 27.7	—
SECONDARY Change From Baseline in Pulmonary Function Tests	-2.8; -8.0; 5.0; 3.8; 0.8; -1.0	—
SECONDARY Change From Baseline in Muscle Strength Tests	2.0; 0.0; 1.0; 3.4	—
SECONDARY Change From Baseline in Fatigue Severity Score (FSS)	0.9; -4.3; -0.5; -14.0	—
SECONDARY Change From Baseline in Overall Physical Wellbeing (Subject's Global Impression of Change [SGIC], Question1)	3; 0; 3; 4; 2; 2	—
SECONDARY Change From Baseline in Overall Physical Wellbeing (Physician's Global Impression of Change [PGIC])	2; 1; 2; 2; 3; 2	—

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.

Eligibility Criteria

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 at screening
Subject, whether male or female, planned to conceive a child during the study
Had a medical or any other extenuating condition or circumstance that may, in opinion of investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with protocol requirements
Had a history of allergy or sensitivity to alglucosidase alfa, miglustat or other iminosugars (Cohorts 1, 2, and 4)
Required invasive ventilatory support, or used noninvasive ventilatory support ≥ 6 hours a day while awake (Cohorts 1, 3, and 4)
Had active systemic autoimmune disease such as lupus, scleroderma, or rheumatoid arthritis; subjects with autoimmune disease must have been discussed with the Amicus Medical Monitor
Had active bronchial asthma; subjects with bronchial asthma must have been discussed with the Amicus Medical Monitor

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02675465). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.