RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) (NCT04571970) | Clinical Trial Compass
CompletedPhase 1/2
RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)
United States, Canada6 participantsStarted 2021-03-11
Plain-language summary
RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who have severe MPS II.
Who can participate
Age range5 Years – 17 Years
SexMALE
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Inclusion criteria
✓. Has a documented diagnosis of MPS II AND a neurocognitive testing score ≤ 1 ½ standard deviation (SD) from the test normative mean (BSID-III: 77 and MSEL Visual Reception: 35), OR
✓. Has a documented diagnosis of MPS II AND has a decline of ≥ 1 standard deviation on serial neurocognitive testing administered between 3 to 36 months apart (BSID-III Cognitive or MSEL Visual Reception), OR
✓. Has a relative clinically diagnosed with neuronopathic MPS II who has the same IDS mutation as the participant AND the participant in the opinion of a geneticist has inherited a neuronopathic form of MPS II, OR
✓. Has documented mutation(s) in IDS that in the opinion of a geneticist is known to result in a neuronopathic phenotype AND in the opinion of a clinician has a neuronopathic form of MPS II
Exclusion criteria
✕. Has contraindications for intracisternal injection, intracerebroventricular injection, or lumbar puncture
✕. Has contraindications for immunosuppressive therapy
✕. Has any neurocognitive deficit not attributable to MPS II or diagnosis of a neuropsychiatric condition
✕. Has had prior treatment with an AAV-based gene therapy product
✕. If receiving ELAPRASE® via intrathecal (IT) administration, must agree to discontinue IT idursulfase for the duration of the study
✕. Has experienced a serious hypersensitivity reaction to intravenous (IV) ELAPRASE®
What they're measuring
1
Number of participants with treatment-related adverse events and serious adverse events