Long-term Follow-up of RGX-121 in Mucopolysaccharidosis Type II (MPS II) Patients

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This study will follow Mucopolysaccharidosis Type II (MPS II) patients after enrolling and receiving study drug RGX 121 in the parent study RGX 121-101. The purpose of this study is to determine drug safety and efficacy of RGX 121. No study drug is being given in this study.

If you agree to take part, your participation will last for up to 3 years after having completed participation in the parent studies. It will include up to 7 visits: up to 4 on-site study visits and 3 check-ins (e.g., over the phone or by internet).  The impact of RGX-121 on biomarkers in patients' cerebrospinal fluid (CSF), blood plasma, and urine will also be evaluated in the long-term. 

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at julcad [at] chop.edu or 267-426-1368.

ClinicalTrials.gov Identifier

NCT04597385

View full trial information on ClinicalTrials.gov

Eligibility & Criteria

IRB #:
20-017984
Official Title:
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-121
Study Phase:
Not Applicable
Eligible Age Range:
2 - 17 Years
Gender:
All
Study Categories:

Visit Criteria

The primary study procedures are:

  • Research blood tests
  • Hearing assessments
  • Cognitive (Neurodevelopmental and Adaptive & Behavioral function testing)
  • Physical and Neurological exams
  • ECGs
  • Echocardiograms
  • Auditory capacity testing
  • Lumbar puncture (LPs)
  • Brain and Abdominal Magnetic Resonance Imaging (MRIs). Abdominal Ultrasound (as alternative to Abdominal MRI)
  • Caregiver questionnaires
  • Video capture
  • Some of the procedures may involve the administration of general anesthesia for the research. 
Leader
Can Ficicioglu

Can Ficicioglu, MD, PhD