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<p>Intrathecal onasemnogene abeparvovec (OAV101 IT) may enable a one-time gene transfer therapy, addressing an unmet need across the broader spinal muscular atrophy (SMA) population. STRENGTH (<a href="http://clinicaltrials.gov/study/NCT05386680">NCT05386680</a>) was a 52-week, phase 3b, single-arm, open-label, multicenter study evaluating OAV101 IT in participants with SMA aged 2 to &lt;18 years who discontinued nusinersen or risdiplam. The primary objective was safety/tolerability. Twenty-seven participants were enrolled (mean (s.d.) age at OAV101 IT, 7.4 (3.35) years; range, 2.4–17.7 years) with prior nusinersen or risdiplam (mean (s.d.) duration of 4.3 (1.07) years; range, 1.86–6.18 years or 3.0 (2.02); range, 0.39–6.28 years). All (<i>n</i> = 27) experienced at least one adverse event (AE), most frequently nasopharyngitis (<i>n</i> = 15, 55.6%), pyrexia (<i>n</i> = 14, 51.9%) and vomiting (<i>n</i> = 13, 48.1%). Thirteen participants (48.1%) experienced treatment-related AEs, most frequently vomiting (<i>n</i> = 6, 22.2%), headache (<i>n</i> = 4, 14.8%) and pyrexia (<i>n</i> = 3, 11.1%). Serious adverse events (SAEs) were reported for four participants (<i>n</i> = 4, 14.8%) (mostly respiratory infection related). No AEs leading to death or study discontinuation were reported. Adverse events of special interest (AESI) reported were in categories of transient thrombocytopenia (<i>n</i> = 8, 29.6%), hepatotoxicity (<i>n</i> = 4, 14.8%) and signs and symptoms that may be suggestive of dorsal root ganglia toxicity (<i>n</i> = 2, 7.4%). The OAV101 IT safety profile was consistent with findings in treatment-naïve patients. Trial registration: ClinicalTrials.gov identifier: <a href="http://clinicaltrials.gov/study/NCT05386680">NCT05386680</a>.</p>

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Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial

  • 期刊论文

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  • 发表日期：

    2025年12月8日

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  • Jennifer M. Kwon,
  • Francina Munell,
  • Laure Le Goff,
  • Kotaro Yuge,
  • Tamaki Kato,
  • Claude Cances,
  • Liesbeth De Waele,
  • Ian R. Woodcock,
  • Eugenio M. Mercuri,
  • Crystal M. Proud,
  • Basil T. Darras,
  • Leslie H. Hayes,
  • Maryam Oskoui,
  • Jeannie Visootsak,
  • Gemma Williams,
  • Andreja Ilić,
  • Lina Yang,
  • W. Ludo van der Pol

摘要

Intrathecal onasemnogene abeparvovec (OAV101 IT) may enable a one-time gene transfer therapy, addressing an unmet need across the broader spinal muscular atrophy (SMA) population. STRENGTH (NCT05386680) was a 52-week, phase 3b, single-arm, open-label, multicenter study evaluating OAV101 IT in participants with SMA aged 2 to <18 years who discontinued nusinersen or risdiplam. The primary objective was safety/tolerability. Twenty-seven participants were enrolled (mean (s.d.) age at OAV101 IT, 7.4 (3.35) years; range, 2.4–17.7 years) with prior nusinersen or risdiplam (mean (s.d.) duration of 4.3 (1.07) years; range, 1.86–6.18 years or 3.0 (2.02); range, 0.39–6.28 years). All (n = 27) experienced at least one adverse event (AE), most frequently nasopharyngitis (n = 15, 55.6%), pyrexia (n = 14, 51.9%) and vomiting (n = 13, 48.1%). Thirteen participants (48.1%) experienced treatment-related AEs, most frequently vomiting (n = 6, 22.2%), headache (n = 4, 14.8%) and pyrexia (n = 3, 11.1%). Serious adverse events (SAEs) were reported for four participants (n = 4, 14.8%) (mostly respiratory infection related). No AEs leading to death or study discontinuation were reported. Adverse events of special interest (AESI) reported were in categories of transient thrombocytopenia (n = 8, 29.6%), hepatotoxicity (n = 4, 14.8%) and signs and symptoms that may be suggestive of dorsal root ganglia toxicity (n = 2, 7.4%). The OAV101 IT safety profile was consistent with findings in treatment-naïve patients. Trial registration: ClinicalTrials.gov identifier: NCT05386680.