Clinical trial NCT03911505: ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
Official title: An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Pompe Disease (Late-onset)
Keywords: Pompe
rhGAA
Lead sponsor: Amicus Therapeutics
Sponsor class: INDUSTRY
First posted: 2019-04-11
Enrollment: 22
Sex: ALL
Minimum age (years): 0
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: Inclusion Criteria:

1. 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)
2. Subject weighs ≤ 115 kg. (Cohort 1 Only)
3. Subject must have a diagnosis of LOPD based on documentation as defined in study protocol
4. 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
5. Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy Adolescents at screening (Cohort 1 only)
6. 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

Exclusion Criteria:

1. 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
2. Subject has received treatment with prohibited medications within 30 days of screening
3. Subject has received any gene therapy at any time
4. 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
5. Subject has a hypersensitivity to any of the excipients in ATB200, approved rhGAA, or AT2221
6. Female subject is pregnant or breast-feeding at screening
7. Subject requires the use of ventilation support for \> 6 hours per day while awake
8. Subject has evidence of moderate to severe hypertrophic cardiomyopathy aligning with classic IOPD
9. In the opinion of the investigator, the parent or legally authorized representative is unlikely or unable to comply with the study requirements
10. 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
11. 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)

Primary outcomes:
measure: Incidence of treatment-emergent adverse events (TEAEs) from baseline
timeFrame: 52 weeks
Secondary outcomes:
measure: Assessment of pharmacokinetic parameters
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Neuromuscular Research Center
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital
state: California
city: Gainesville
country: United States
facility: University of Florida Clinical Research Center
state: Florida
city: Jacksonville
country: United States
facility: Wolfson Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Woodruff Memorial Research Building
state: Georgia
city: Grand Rapids
country: United States
facility: Infusion Associates
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Saint Louis
country: United States
facility: St. Louis Children's Hospital
state: Missouri
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Pittsburgh
country: United States
facility: UPMC Children's Hospital of Pittsburgh
state: Pennsylvania
city: Salt Lake City
country: United States
facility: University of Utah, Clinical and Translational Sciences Institute
state: Utah
city: Fairfax
country: United States
facility: Lysosomal and Rare Disorders Research and Treatment Center, Inc.
state: Virginia
city: North Adelaide
country: Australia
facility: Women's and Children's Hospital
state: South Australia
city: Calgary
country: Canada
facility: University of Calgary
state: Alberta
city: Hochheim
country: Germany
facility: SphinCS GmbH Clinical Science for LSD
state: Hessen
city: Monza
country: Italy
facility: San Gerardo Hospital
city: Osaka
country: Japan
facility: Izumi City General Hospital
state: Izumi-Shi
city: Gunma
country: Japan
facility: Gunma University Hospital
city: Miyagi
country: Japan
facility: Tohoku University Hospital
city: Tokyo
country: Japan
facility: Tokyo Women's Medical University
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
Study officials:
References: