Long-term Follow-Up for RGX-121

NCT04597385 | Unknown

• 1 other identifier: RGX-121-5101
• Study Type: observational
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

RGX-121-5101 is the long-term follow-up study to the RGX-121-101 first in human study where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study will evaluate the long-term safety and efficacy of RGX-121.

Conditions

Mucopolysaccharidosis II

Keywords

MPS II; Gene Therapy; Hunter

Outcome Measures

Primary Outcomes (2)

• Incidences of Adverse Events over time.

  Safety

  3 years

• Incidences of Serious Adverse Events over time

  Safety

  3 year

Secondary Outcomes (4)

• Biomarkers [Time frame: 1 Month, 12 Months, 24 Months, 36 Months]

  3 years

• Changes in neurodevelopmental parameters of cognitive function [Time Frame: 1 Month, 12 Months, 24 Months, 36 Months]

  3 years

• Changes in neurodevelopmental parameters of cognitive function [Time Frame: 1 Month, 12 Months, 24 Months, 36 Months]

  3 years

• Changes in neurodevelopmental parameters of adaptive behavior function [Time Frame: 1 Month, 12 Months, 24 Months, 36 Months]

  3 years

Study Arms (1)

Long-term Follow-Up

No intervention.

Other: Long-term Follow-Up

Interventions

Long-term Follow-Up OTHER

No intervention

Long-term Follow-Up

Eligibility Criteria

• Age: 28 Months+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Up to 12 subjects over the age of 28 months who have received RGX-121 in a previous study.

You may qualify if:

• 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

You may not qualify if:

• Patient has not received RGX-121 previously in a separate parent trial.

Sponsors & Collaborators

• REGENXBIO Inc.: lead

Study Sites (1)

Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders

Pittsburgh, Pennsylvania, 15224, United States

Location

MeSH Terms

Conditions

Mucopolysaccharidosis II; Sudden Infant Death

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Death, Sudden; Death; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Infant Death

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 15, 2020
• First Posted: October 22, 2020
• Study Start: March 14, 2021
• Primary Completion: September 1, 2025
• Study Completion: September 1, 2025
• Last Updated: August 10, 2021

Record last verified: 2021-08

Locations

US (1)