First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
a study on Pompe Disease
Summary
- Eligibility
- for people ages 18-75 (full criteria)
- Location
- at UC Irvine
- Dates
- study startedcompletion around
Description
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.
Official Title
An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease
Details
This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the effect of a highly targeted rhGAA (ATB200) co-administered with a chaperone (AT2221).
The study aims to evaluate safety, tolerability, pharmacodynamics (PD), and immunogenicity of ATB200 co-administered with AT2221. The study will be conducted in 3 stages.
In Stage 1, safety, tolerability, and PK will be evaluated following sequential single ascending doses of intravenously infused ATB200.
In Stage 2, safety, tolerability, and PK will be evaluated following single- and multiple-ascending dose combinations of ATB200 and AT2221.
In Stage 3, long term safety and efficacy will be assessed following 24 month treatment of ATB200 co-administered with AT2221 (Miglustat)
In Stage 4, treatment period will begin at the end of Stage 3 and will continue as open label extension until commercialization, study discontinuation or subject withdrawal, with functional assessments every 6 months
No Muscle biopsies will be performed in this study.
Keywords
Pompe Disease, Pompe, rhGAA, Glycogen Storage Disease Type II, Miglustat, ATB200, ATB200 + AT2221
Eligibility
You can join if…
Open to people ages 18-75
- 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
You CAN'T join if...
- 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
Locations
- University of California Irvine
Orange California 92868 United States - Neuromuscular Research Centre
Phoenix Arizona 85028 United States
Details
- Status
- in progress, not accepting new patients
- Start Date
- Completion Date
- (estimated)
- Sponsor
- Amicus Therapeutics
- ID
- NCT02675465
- Phase
- Phase 1/2 Pompe Disease Research Study
- Study Type
- Interventional
- Participants
- Expecting 32 study participants
- Last Updated