The five most common treatment-emergent adverse events (TEAEs) were headache, pyrexia, upper respiratory tract infection, cough, and nasopharyngitis. The incidence and severity of adverse events were consistent with the underlying patient population and nusinersen therapy. No safety risks were identified over 24 months of treatment.
Topaz simplify trial trial#
These findings complement previously reported data from the TOPAZ trial 24-month extension period that demonstrated sustained and durable improvements in motor function as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE) and RULM in patients with nonambulatory Types 2 and 3 SMA.
The trends of improvement with ESBBT are consistent with the previously reported increases in RULM scores observed in the TOPAZ trial at 24 months. Additionally, these patients also experienced trends in improvements in fatigability and endurance measures based on mean change in ESBBT activities. Nonambulatory Types 2 and 3 patients (aged five to 21 who began receiving nusinersen maintenance therapy at or after age five) reported stabilization or increases in ADL up to a mean change from baseline of 0.7 points (n=8) in PEDI-CAT scores, and less fatigue up to a mean change from baseline of 3.5 points (n=2) in PROMIS scores over 24 months of apitegromab. Nonambulatory Type 2 patients (aged two or older who began receiving nusinersen maintenance therapy before age five) reported stabilization or continuous improvements in ADL up to a mean change from baseline of 3 points (n=14) in PEDI-CAT scores and fatigue up to a mean change from baseline of 5 points (n=10) in PROMIS scores over 24 months of apitegromab. Limitations of these exploratory quality-of-life data analyses include small patient sample sizes in an open-label study, and further exploration is warranted. These data are relevant for informing the therapeutic hypotheses being evaluated in the Phase 3 SAPPHIRE trial. The tertiary endpoint data from these measures show trends of continuous improvement over 24 months. Patient Reported Outcome Measurement Information System (PROMIS) measures mild subjective feelings of tiredness to debilitating and sustained feelings of exhaustion, with lower scores reflecting less fatigue 4,5 andĮndurance Shuttle Box and Block Test (ESBBT), a muscle endurance measurement tool, evaluates how fast a patient fatigues with the added measure of endurance 6 and may be complementary to outcome measures that focus on arm motor function, such as the Revised Upper Limb Module (RULM) assessment. The Pediatric Evaluation of Disability Inventory-Computer Adaptive Test (PEDI-CAT) measures pediatric abilities through three functional domains, daily activities, mobility, and social cognition 3
"As the Phase 3 SAPPHIRE trial advances, these additional TOPAZ trial analyses coupled with the previously reported measures of motor function, such as HFMSE and RULM, continue to yield positive, consistent results, building the case for apitegromab as a promising new treatment option for patients with SMA." 1,2 These positive TOPAZ data indicate sustained improvements of quality-of-life measures over 24 months in the patient population studied," said Jay Backstrom, M.D., M.P.H., Chief Executive Officer of Scholar Rock. "SMA can have a significant impact on the ability to perform daily activities but there is limited research on potential interventions to improve quality-of-life measures, such as increasing muscle endurance and reducing fatigue. The data were featured in a podium presentation today by Basil Darras, M.D., Associate Neurologist-in-Chief, Boston Children’s Hospital and Professor of Neurology, Harvard Medical School, at the 3rd International Scientific Congress on SMA in Barcelona, Spain (SMA Europe 2022). These data indicate the potential for sustained improvement in quality-of-life measures for patients with symptomatic SMA and offer further evidence of possible durable effects of apitegromabĬAMBRIDGE, Mass., October 22, 2022-( BUSINESS WIRE)-Scholar Rock (NASDAQ: SRRK), a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced new quality-of-life (QoL) data from its Phase 2 TOPAZ trial extension period evaluating patient outcomes after 24 months of treatment which indicate stabilization or continued improvement with apitegromab for nonambulatory patients with Types 2 and 3 spinal muscular atrophy (SMA) receiving an SMN-targeted therapy. Tertiary endpoint data show trends of continuous improvement in activities of daily living, fatigue, and endurance over 24 months
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