Overview

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

Status:
Active, not recruiting
Trial end date:
2023-12-01
Target enrollment:
0
Participant gender:
All
Summary
This study is an international, multi-center, study of Pompe disease patients that are currently receiving enzyme-replacement therapy (ERT). The purpose of this study is to find out if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.
Phase:
Phase 1/Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Amicus Therapeutics
Treatments:
Miglustat
Criteria
Key Inclusion Criteria:

- Male and female subjects between 18 and 75years of age, inclusive

- Diagnosis of Pompe disease

Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):

- Has received ERT with alglucosidase alfa for the previous 2-6 years, inclusive

- Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency
of once every other week

- Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )

- Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value

ERT-experienced subjects (non-ambulatory):

- Has received ERT with alglucosidase alfa (Myozyme/Lumizyme) for ≥2 years

- Is wheelchair-bound

ERT-naïve subjects (ambulatory):

- Must be able to walk 200-500 meters on the 6MWT

- Has upright FVC must be 30% to 80% of predicted normal value

- Subject has never received alglucosidase alfa

Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):

- Has received ERT with alglucosidase alfa for >7years, inclusive

- Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency
of once every other week

- Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )

- Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value

Exclusion Criteria:

- Subject has received treatment with prohibited medications within 30 days of Baseline
Visit

- Subject, if female, is pregnant or breastfeeding at screening

- Subject, whether male or female, planning to conceive a child during the study

- Subject has 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

- Subject has a history of allergy or sensitivity to miglustat or other iminosugars

- Subjects with active systemic autoimmune disease such as lupus, scleroderma, or
rheumatoid arthritis. All subjects with autoimmune disease must be discussed with the
Amicus Medical Monitor

- Subjects with active bronchial asthma. All subjects with bronchial asthma must be
discussed with the Amicus Medical Monitor