ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
- Conditions
- Pompe Disease (Late-onset)
- Interventions
- Registration Number
- NCT03911505
- Lead Sponsor
- Amicus Therapeutics
- Brief Summary
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to \< 18 years
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 22
- Male or female subjects (ERT-naïve [have never received a dose of rhGAA] or ERT-experienced [have received rhGAA every 2 weeks for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 months before enrollment]) diagnosed with LOPD who are aged 12 to <18 years at screening (Cohort 1 only) or aged 0 months to < 12 years at screening (Cohort 2 only)
- Subject weighs ≤ 115 kg. (Cohort 1 Only)
- Subject must have a diagnosis of LOPD based on documentation as defined in study protocol
- If of reproductive potential and if sexually active, female and male subjects agree to use a highly effective method of contraception throughout the duration of the study and for up to 90 days after their last dose of Cipaglucosidase Alfa/Miglustat
- Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy Adolescents at screening (Cohort 1 only)
- Subject (aged 12 to <18 years; Cohort 1) performs one 6-Minute Walk Test (6MWT) (≥ 75 meters) at screening that is valid, as determined by the clinical evaluator, or subject (aged ≥ 5 to < 12 years; Cohort 2) performs one 6MWT (≥ 40 meters) at screening that is valid, as determined by the clinical evaluator
- Subject has received any investigational/experimental drug, oral anabolic steroid or derivative, biologic, or device within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before screening
- Subject has received treatment with prohibited medications within 30 days of screening
- Subject has received any gene therapy at any time
- Subject has any intercurrent illness or condition at screening or baseline that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator and/or the medical monitor that the potential subject may have an unacceptable risk by participating in this study
- Subject has a hypersensitivity to any of the excipients in ATB200, approved rhGAA, or AT2221
- Female subject is pregnant or breast-feeding at screening
- Subject requires the use of ventilation support for > 6 hours per day while awake
- Subject has evidence of moderate to severe hypertrophic cardiomyopathy aligning with classic IOPD
- In the opinion of the investigator, the parent or legally authorized representative is unlikely or unable to comply with the study requirements
- Subject has any prior history of illness or condition known to affect motor function, such as, but not limited to, Guillain-Barre syndrome, cerebral palsy, etc
- Subject who is diagnosed with Pompe disease via newborn screening and is asymptomatic (ie, showing no signs and symptoms of Pompe disease (Cohort 2 Only)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221) Cipaglucosidase Alfa Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221) Miglustat Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule
- Primary Outcome Measures
Name Time Method Incidence of treatment-emergent adverse events (TEAEs) from baseline 52 weeks
- Secondary Outcome Measures
Name Time Method Assessment of pharmacokinetic parameters 52 weeks ATB200 and AT2221 concentrations in plasma
Trial Locations
- Locations (23)
Woodruff Memorial Research Building
🇺🇸Atlanta, Georgia, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
National Taiwan University Hospital
🇨🇳Taipei, Taiwan
UCSF Benioff Children's Hospital
🇺🇸Oakland, California, United States
University of Florida Clinical Research Center
🇺🇸Gainesville, Florida, United States
Wolfson Children's Hospital
🇺🇸Jacksonville, Florida, United States
University of Calgary
🇨🇦Calgary, Alberta, Canada
San Gerardo Hospital
🇮🇹Monza, Italy
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Infusion Associates
🇺🇸Grand Rapids, Michigan, United States
St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
🇺🇸Fairfax, Virginia, United States
SphinCS GmbH Clinical Science for LSD
🇩🇪Hochheim, Hessen, Germany
Izumi City General Hospital
🇯🇵Osaka, Izumi-Shi, Japan
Gunma University Hospital
🇯🇵Gunma, Japan
Tohoku University Hospital
🇯🇵Miyagi, Japan
Tokyo Women's Medical University
🇯🇵Tokyo, Japan
Women's and Children's Hospital
🇦🇺North Adelaide, South Australia, Australia
Neuromuscular Research Center
🇺🇸Phoenix, Arizona, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
University of Utah, Clinical and Translational Sciences Institute
🇺🇸Salt Lake City, Utah, United States