JPRN-jRCT2031210014
Recruiting
Phase 3
An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of ATB200/AT2221 in Pediatric Subjects Aged 0 to < 18 Years with Pompe Disease - ZIP Study
ConditionsPompe disease
Overview
- Phase
- Phase 3
- Intervention
- Not specified
- Conditions
- Pompe disease
- Sponsor
- Wright Jacquelyn
- Enrollment
- 22
- Status
- Recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\. Male or female subjects (ERT\-naive \[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 \>\= 25 kg and \<\= 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 ATB200/AT2221
- •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
Outcomes
Primary Outcomes
Not specified
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