Long-term Follow-Up for RGX-121
Launched by REGENXBIO INC. · Oct 15, 2020
Trial Information
Current as of June 26, 2025
Enrolling by invitation
Keywords
ClinConnect Summary
This is a prospective follow-up study to evaluate the long-term safety and efficacy after a single administration of RGX-121. Eligible participants are those who previously have enrolled in clinical study RGX-121-101 and received a single intracisternal (IC) or intracerebroventricular (ICV) infusion of RGX-121. Enrollment of each participant will occur the same day or after the participant has completed the end of study (EOS) visit or early termination visit (ET) from the previous (parent) study. Participants will be followed in this study cumulatively for up to 5 years after RGX-121 admini...
Gender
MALE
Eligibility criteria
- Inclusion Criteria:
- • To be eligible, a participant must have previously received RGX-121 in a separate parent trial.
- • Participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent
- Exclusion Criteria:
- • Patient has not received RGX-121 previously in a separate parent trial.
About Regenxbio Inc.
Regenxbio Inc. is a leading biotechnology company focused on developing innovative gene therapies for the treatment of genetic diseases and neurological disorders. Utilizing its proprietary AAV8 (adeno-associated virus) delivery platform, Regenxbio aims to provide transformative therapeutic solutions that address unmet medical needs. The company is committed to advancing its clinical programs through rigorous research and development, leveraging its expertise in gene editing and vector design to enhance patient outcomes. With a robust pipeline of candidates in various stages of clinical trials, Regenxbio is dedicated to pioneering advancements in gene therapy and improving the lives of patients worldwide.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Pittsburgh, Pennsylvania, United States
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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