An extension of Global Phase III open-label, multicenter designed to evaluate the Long-term safety and efficacy of study drug for the treatment of the MPS II.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
80
IV infusion, 2.0 mg/kg/week
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Hospital Universitario Austral
Buenos Aires, Argentina
Change in heparan sulfate concentrations in cerebrospinal fluid from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study to each time point
Assessment will be mandatory for the first two years after starting JR-141 administration through the parent study or the extension study.
Time frame: [A-1, A-2, A-3: Week 157, 209, 261] [A-4: Week 131, 183, 235, 287]
Change in neurocognitive testing, calculated by the BSID-III or KABC-II, from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study
Time frame: [A-1: Week 157, 209, 261] [A-2: Week 131, 157, 183, 209, 261] [A-3: Week 131, 157, 209, 261 ] [A-4: Week 131, 157, 183, 235, 287]
Change in neurocognitive testing, calculated by the VABS-II from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study
Time frame: [A-1: Week 157, 209, 261] [A-2: Week 131, 157, 183, 209, 261] [A-3: Week 131, 157, 209, 261 ] [A-4: Week 131, 157, 183, 235, 287][B-1: Week 78, 105, 157, 209, 261] [B-2: Week 78, 105, 131, 157, 209, 261]
Change in neurocognitive testing, calculated by the Wechsler, from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study
Time frame: [B-1: Week 78, 105, 157, 209, 261] [B-2: Week 78, 105, 131, 157, 209, 261]
Change in liver and spleen volume from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study
Time frame: [A-1, A-3: Week 157, 209, 261] [A-2: Week 131, 157, 209, 261] [A-4: Week 131, 183, 235, 287] [B-1: Week 105, 157, 209, 261] [B-2: Week 78, 105, 157, 209, 261]
Change in 6-minute walk distance from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study
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Hospital de Clínicas de Porto Alegre
Porto Alegre, Brazil
Instituto de Genética e Erros Inatos do Metabolismo
São Paulo, Brazil
Hôpital Femme Mère Enfant
Bron, France
Universitätsklinikum Hamburg-Eppendorf
Hamburg, Germany
Osp. Pediatrico Bambino Gesù, IRCCS
Rome, Italy
Uniwersytecki Szpital Dziecięcy
Krakow, Poland
Hospital Sant Joan de Déu
Barcelona, Spain
Time frame: [B-1: Week 105, 157, 209, 261] [B-2: Week 78, 105, 157, 209, 261]