- |||||||||| tralesinidase alfa (AX 250) / Allievex Corp, lesinidase alfa (SBC-103) / AstraZeneca
Preclinical, Journal: Femoral Structure and Biomechanical Characteristics in Sanfilippo Syndrome Type-B Mice. (Pubmed Central) - Sep 28, 2023
Here, we establish some osteogenic manifestations of MPS IIIB within the mouse model by radiographic and biomechanical tests, which are also differentially affected by age and sex. This suggests that some skeletal features of the MPS IIIB mouse model may be used as biomarkers of peripheral disease correction for preclinical treatment of MPS IIIB.
- |||||||||| tralesinidase alfa (AX 250) / Allievex Corp, lesinidase alfa (SBC-103) / AstraZeneca
Preclinical, Journal: Tralesinidase alfa enzyme replacement therapy prevents disease manifestations in a canine model of mucopolysaccharidosis type IIIB. (Pubmed Central) - Aug 22, 2022
These findings demonstrate the ability of TA to prevent or limit the biochemical, pathological, and cognitive manifestations of canine MPS IIIB disease, thus providing support of its potential long-term tolerability and efficacy in MPS IIIB subjects. Significance Statement This work illustrates the efficacy and tolerability of tralesinidase alfa as a potential therapeutic for MPS IIIB patients by documenting that direct CNS administration to MPS IIIB dogs prevents accumulation of disease-associated GAGs in lysosomes, hepatomegaly, cerebellar atrophy, and cognitive decline.
- |||||||||| lesinidase alfa (SBC-103) / AstraZeneca
Trial completion, Trial primary completion date: Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB) (clinicaltrials.gov) - Jul 12, 2018
P1/2, N=11, Completed,
Changes in heparan sulfate levels in CSF were small and were not maintained from earlier study time points, there was no clear evidence overall of clinically meaningful improvement in neurocognitive function at the higher doses investigated, and no dose-dependent effects were observed. Active, not recruiting --> Completed | Trial primary completion date: Jul 2018 --> Oct 2017
- |||||||||| lesinidase alfa (SBC-103) / AstraZeneca
Trial completion date, Trial termination, Trial primary completion date: CL01-T: A Open Label Study in Previously Studied, SBC-103 Treatment Na (clinicaltrials.gov) - Apr 18, 2018
P1/2, N=3, Terminated,
Active, not recruiting --> Completed | Trial primary completion date: Jul 2018 --> Oct 2017 Trial completion date: Dec 2018 --> Aug 2017 | Active, not recruiting --> Terminated | Trial primary completion date: Dec 2018 --> Aug 2017; The Sponsor's decision to terminate the SBC-103 program was reached after review of the data from all interventional clinical studies of SBC-103.
- |||||||||| lesinidase alfa (SBC-103) / AstraZeneca
Enrollment closed: Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB) (clinicaltrials.gov) - Aug 11, 2015
P1/2, N=9, Active, not recruiting,
Trial completion date: Dec 2018 --> Aug 2017 | Active, not recruiting --> Terminated | Trial primary completion date: Dec 2018 --> Aug 2017; The Sponsor's decision to terminate the SBC-103 program was reached after review of the data from all interventional clinical studies of SBC-103. Recruiting --> Active, not recruiting
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