For Healthcare Providers
CAMPSIITE™ is a multicenter, open-label, single-arm Phase I/II/III study enrolling boys with neuronopathic MPS II, aged 4 months up to 5 years of age, to evaluate the efficacy, safety, and pharmacodynamics (PD) of RGX-121 gene therapy.
Summary of Key Points
- Emerging natural history data highlights the urgency of treating neuronopathic MPS II early.
- Interim data from the Phase I/II portion of CAMPSIITE demonstrate that RGX-121 has been well-tolerated as of Aug. 1, 2022. Interim data from the dose level selected for the pivotal expansion (Cohort 3) showed biomarkers approaching normal levels of GAGs.1
- Available data from Cohorts 1 and 2 showed CNS activity up to 2 years after RGX-121 administration as demonstrated by improvements in neurodevelopmental function and caregiver-reported outcomes.2
- CAMPSIITE is currently enrolling pediatric neuronopathic MPS II patients from 4 months to 5 years of age at the pivotal dose level to evaluate GAG levels in the cerebrospinal fluid (CSF) and evaluate the effect of RGX-121 on neurodevelopmental function.
- If you have a patient less than 5 years old who has or may have neuronopathic MPS II, contact a study site or reach out to email@example.com for more information.
Natural History Data in MPS II
An analysis of natural history data from 32 patients (age range at first assessment: 1 to 117 months) provides a comprehensive, multi-domain developmental picture of neuronopathic MPS II.
The data demonstrate that:
- Developmental skill acquisition slows in all Mullen Scales of Early Learning (MSEL) when compared to peer development (delay stage) and that the expressive language scale provides the earliest indication of delay.3
- Mean developmental age-equivalent score (AEq) trajectories begin to deviate as early as 12.8 months.3
- The rate of skill acquisition slows early in development and the delay becomes more substantial over time.3
Data from the natural history analysis were presented as a poster at the 2022 annual meeting of the Society for the Study of Inborn Errors of Metabolism (SSIEM).3 Click here to view the poster.
Current Enrollment Opportunities
REGENXBIO is enrolling boys with neuronopathic MPS II, aged 4 months up to 5 years of age, in the CAMPSIITE Study. The primary objectives of this pivotal trial are to evaluate cerebrospinal fluid (CSF) GAGs and the effect of RGX-121 on neurodevelopmental function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) or Mullen Scales of Early Learning (MSEL) in pediatric neuronopathic MPS II. Safety, caregiver-reported outcomes, and MRI parameters will also be assessed.
Below are key eligibility criteria for the CAMPSIITE trial. For a more complete list of eligibility criteria, a list of CAMPSIITE Study locations, and more information, visit ClinicalTrials.gov (identifier # NCT03566043).
|Key Eligibility Criteria||
Eligible participants must be a male between 4 months and 5 years of age on Day 1 of trial and have a documented diagnosis of neuronopathic MPS II. For a full list of eligibility criteria and additional information, please visit clinicaltrials.gov.
If your patient’s MPS II phenotype is unknown, you can refer your patient to a study site for assessment.
If you have any patients in your practice who may be eligible to participate in the CAMPSIITE Study, we encourage you to contact a study site.
Primary Study Endpoints
Participants will be assessed (safety and efficacy) for two years following treatment with RGX-121 and will then be encouraged to join a long-term follow-up study. The primary endpoints of the pivotal expansion are:
- CSF GAG levels
- Neurodevelopmental function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) or Mullen Scales of Early Learning (MSEL)
- Giugliani R. RGX-121 gene therapy for the treatment of severe mucopolysaccharidisos type II: CAMPSIITE™ Phase I/II/III: A clinical study. Presented at 2022 annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM), Freiburg, Germany, August 31, 2022. Abstract SSIEM22-2719.
- Giugliani R. RGX-121 gene therapy for the treatment of severe mucopolysaccharidosis type II: interim analysis of the first in human study. Presented at WORLDSymposium 2022, 18th Annual Research Meeting, San Diego, CA, February 9, 2022.
- Escolar M, Phillips D, Cho Y, Mulatya C, Nevoret M-L, Poe M. Natural history of neurodevelopment in neuronopathic mucopolysaccharidosis type II (MPS II): Mullen Scales of Early Learning (MSEL) visual reception, expressive and receptive language and fine motor scale developmental trajectories. Presented at 2022 annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM), Freiburg, Germany, August 2022. Abstract SSIEM22-2814.