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Phase 1 study of ABI‐009 (nab‐rapamycin) for surgically refractory epilepsy (RaSuRE).

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Title: Phase 1 study of ABI‐009 (nab‐rapamycin) for surgically refractory epilepsy (RaSuRE).
Authors: Hall, Koko (AUTHOR), Saneto, Russell (AUTHOR), Randle, Stephanie (AUTHOR), Wainwright, Mark S. (AUTHOR), Berry, Patricia (AUTHOR), Leary, Sarah (AUTHOR), Mirzaa, Ghayda (AUTHOR), Lockrow, Jason (AUTHOR), Goldstein, Hannah (AUTHOR), Ojemann, Jeffrey (AUTHOR), Thiessen, Rosita (AUTHOR), Willis, Hope (AUTHOR), Sullivan, Erin (AUTHOR), Rivers, Renee (AUTHOR), Klein, Jani (AUTHOR), Hauptman, Jason S. (AUTHOR)
Source: Epilepsia (Series 4). Nov2025, Vol. 66 Issue 11, p4246-4258. 13p.
Subjects: Epilepsy surgery, Treatment effectiveness, Rapamycin, mTOR inhibitors, Child patients, Patient safety, Clinical trials
Abstract: Objective: Seizures that are refractory to medical and surgical therapy increase the risk of morbidity and mortality in children with epilepsy. Novel therapeutic trials for this population remain sparse and suboptimal. This Phase 1 study evaluates the (1) safety, tolerability, and (2) preliminary efficacy of ABI‐009 (nab‐rapamycin) in children with medically and surgically refractory epilepsy. Methods: Children with medically intractable epilepsy and a history of resective surgery who experienced >8 seizures during the 30‐day baseline were included. Participants maintained their existing antiseizure medication regimens throughout the study. ABI‐009 was administered over 3 weeks using a 3 + 3 dose‐escalation design, with dosing cohorts of 5, 10, and 20 mg/m2. Treatment was then discontinued, and participants were observed for a 90‐day follow‐up period. Data were collected on seizure frequency, seizure type, adverse events, medical and laboratory assessments, the Quality of Life in Childhood Epilepsy (QOLCE), and the Nisonger Child Behavior Rating Form (NCBRF). Results: Twelve participants received ABI‐009 and completed all study visits. Seizure etiologies included tuberous sclerosis complex, focal cortical dysplasia, and hemimegalencephaly, although Mammalian target of rapamycin involvement was not required for inclusion. No doses were missed, and dose escalation progressed through all levels. The maximum tolerated dose was 20 mg/m2. All 12 participants experienced at least one treatment‐emergent adverse event (TEAE), with the majority being mild (80%). No participants withdrew from the study voluntarily or by necessity secondary to TEAEs, and there were no serious adverse events. The median change in the weekly seizure rate was a 37.6% reduction from baseline to treatment completion. The treatment response rate (≥25% seizure reduction) was 66.7%, and two participants achieved seizure freedom by follow‐up. Significance: ABI‐009 was deemed safe and tolerable in a pediatric cohort with medically and surgically refractory epilepsy, establishing a maximum tolerated dose of 20 mg/m2 and showing early signs of efficacy. [ABSTRACT FROM AUTHOR]
Copyright of Epilepsia (Series 4) is the property of Wiley-Blackwell and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)
Database: Psychology and Behavioral Sciences Collection
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PubType: Academic Journal
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  Data: Phase 1 study of ABI‐009 (nab‐rapamycin) for surgically refractory epilepsy (RaSuRE).
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  Data: <searchLink fieldCode="AR" term="%22Hall%2C+Koko%22">Hall, Koko</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Saneto%2C+Russell%22">Saneto, Russell</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Randle%2C+Stephanie%22">Randle, Stephanie</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Wainwright%2C+Mark+S%2E%22">Wainwright, Mark S.</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Berry%2C+Patricia%22">Berry, Patricia</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Leary%2C+Sarah%22">Leary, Sarah</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Mirzaa%2C+Ghayda%22">Mirzaa, Ghayda</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Lockrow%2C+Jason%22">Lockrow, Jason</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Goldstein%2C+Hannah%22">Goldstein, Hannah</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Ojemann%2C+Jeffrey%22">Ojemann, Jeffrey</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Thiessen%2C+Rosita%22">Thiessen, Rosita</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Willis%2C+Hope%22">Willis, Hope</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Sullivan%2C+Erin%22">Sullivan, Erin</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Rivers%2C+Renee%22">Rivers, Renee</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Klein%2C+Jani%22">Klein, Jani</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Hauptman%2C+Jason+S%2E%22">Hauptman, Jason S.</searchLink> (AUTHOR)
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  Data: <searchLink fieldCode="JN" term="%22Epilepsia+%28Series+4%29%22">Epilepsia (Series 4)</searchLink>. Nov2025, Vol. 66 Issue 11, p4246-4258. 13p.
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  Data: <searchLink fieldCode="DE" term="%22Epilepsy+surgery%22">Epilepsy surgery</searchLink><br /><searchLink fieldCode="DE" term="%22Treatment+effectiveness%22">Treatment effectiveness</searchLink><br /><searchLink fieldCode="DE" term="%22Rapamycin%22">Rapamycin</searchLink><br /><searchLink fieldCode="DE" term="%22mTOR+inhibitors%22">mTOR inhibitors</searchLink><br /><searchLink fieldCode="DE" term="%22Child+patients%22">Child patients</searchLink><br /><searchLink fieldCode="DE" term="%22Patient+safety%22">Patient safety</searchLink><br /><searchLink fieldCode="DE" term="%22Clinical+trials%22">Clinical trials</searchLink>
– Name: Abstract
  Label: Abstract
  Group: Ab
  Data: Objective: Seizures that are refractory to medical and surgical therapy increase the risk of morbidity and mortality in children with epilepsy. Novel therapeutic trials for this population remain sparse and suboptimal. This Phase 1 study evaluates the (1) safety, tolerability, and (2) preliminary efficacy of ABI‐009 (nab‐rapamycin) in children with medically and surgically refractory epilepsy. Methods: Children with medically intractable epilepsy and a history of resective surgery who experienced >8 seizures during the 30‐day baseline were included. Participants maintained their existing antiseizure medication regimens throughout the study. ABI‐009 was administered over 3 weeks using a 3 + 3 dose‐escalation design, with dosing cohorts of 5, 10, and 20 mg/m2. Treatment was then discontinued, and participants were observed for a 90‐day follow‐up period. Data were collected on seizure frequency, seizure type, adverse events, medical and laboratory assessments, the Quality of Life in Childhood Epilepsy (QOLCE), and the Nisonger Child Behavior Rating Form (NCBRF). Results: Twelve participants received ABI‐009 and completed all study visits. Seizure etiologies included tuberous sclerosis complex, focal cortical dysplasia, and hemimegalencephaly, although Mammalian target of rapamycin involvement was not required for inclusion. No doses were missed, and dose escalation progressed through all levels. The maximum tolerated dose was 20 mg/m2. All 12 participants experienced at least one treatment‐emergent adverse event (TEAE), with the majority being mild (80%). No participants withdrew from the study voluntarily or by necessity secondary to TEAEs, and there were no serious adverse events. The median change in the weekly seizure rate was a 37.6% reduction from baseline to treatment completion. The treatment response rate (≥25% seizure reduction) was 66.7%, and two participants achieved seizure freedom by follow‐up. Significance: ABI‐009 was deemed safe and tolerable in a pediatric cohort with medically and surgically refractory epilepsy, establishing a maximum tolerated dose of 20 mg/m2 and showing early signs of efficacy. [ABSTRACT FROM AUTHOR]
– Name: AbstractSuppliedCopyright
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  Data: <i>Copyright of Epilepsia (Series 4) is the property of Wiley-Blackwell and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.)
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=pbh&AN=189711233
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    Identifiers:
      – Type: doi
        Value: 10.1111/epi.18555
    Languages:
      – Code: eng
        Text: English
    PhysicalDescription:
      Pagination:
        PageCount: 13
        StartPage: 4246
    Subjects:
      – SubjectFull: Epilepsy surgery
        Type: general
      – SubjectFull: Treatment effectiveness
        Type: general
      – SubjectFull: Rapamycin
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      – SubjectFull: mTOR inhibitors
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      – SubjectFull: Child patients
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      – SubjectFull: Patient safety
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      – SubjectFull: Clinical trials
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      – TitleFull: Phase 1 study of ABI‐009 (nab‐rapamycin) for surgically refractory epilepsy (RaSuRE).
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              M: 11
              Text: Nov2025
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