2026 American Academy of Neurology Abstract Website

Agnies van Eeghen¹, Sarah M.L. Wilson², Elizabeth A. Thiele³, Stevie Roszkowski⁴, Maria Dunaway-Bryant⁴, Kasia Wajer⁵, Teresa Greco⁴, Joanne Stevens⁴, Lisa Moore-Ramdin⁵, Petrus J. de Vries⁶
¹Amsterdam University Medical Centers; Advisium, ’s Heeren Loo, ²McGovern Medical School, University of Texas Health Science Center at Houston, ³Massachusetts General Hospital, ⁴Jazz Pharmaceuticals, Inc., ⁵Jazz Pharmaceuticals, UK Ltd., ⁶Centre for Autism Research in Africa, University of Cape Town

Objective:

This prespecified 6-month analysis of the ongoing EpiCom phase 3b/4 study (NCT05864846) investigated TAND outcomes after adjunctive cannabidiol (CBD; Epidiolex^®[US]/Epidyolex^® [EU], 100 mg/mL oral solution) initiation in participants with TSC-associated seizures.

Background:

TAND reduce quality of life in a majority of people with TSC, yet treatment options remain limited. Anecdotal evidence suggests some neuropsychiatric benefits of CBD.

Design/Methods:

Participants with TSC and moderate/severe behavioral challenges received CBD ≤25 mg/kg/day plus standard of care (SOC) for 26 weeks, followed by CBD+SOC or SOC alone for ≤26 additional weeks. Most problematic behavior (MPB), TAND Self-Report Quantified Checklist (TAND-SQ), Aberrant Behavior Checklist (ABC), Caregiver Global Impression of Severity (CareGI-S), and Clinician Global Impression of Severity (CGI-S) scales were assessed at baseline, Week (W) 13, and W26.

Results:

Of 79 participants enrolled at 6 months, 62 had ≥1 postbaseline assessment. At baseline, median (range) age was 16.0 (3–42) years; 2.0 (1–6) antiseizure medications were used and median MPB numerical rating scale (NRS) value was 9.0 of 10, suggesting severe TAND. At W26, median change from baseline in MPB NRS was −2.0. Greatest changes among TAND-SQ clusters were eat/sleep (−2.0) and overactive/impulsive (−1.3); among ABC subscales, irritability (−8.0) and hyperactive noncompliance (−7.0). Compared with baseline, fewer caregivers and clinicians rated behavioral problems as severe/very severe post-treatment at W13 and W26, respectively (CareGI-S severe: 38% vs 14% and 13%; very severe: 18% vs 4% and 13%; CGI-S severe: 47% vs 10% and 0%; very severe: 10% vs 0% and 0%). Treatment-emergent adverse events occurred in 49 participants (62%). Of 6 serious adverse events (7.6%) reported, 4 were deemed treatment-related (resolved).

Conclusions:

In this intermediate analysis, reductions were seen in TAND-SQ, ABC scores, and severity of caregiver- and clinician-reported behavioral problems at 26 weeks after CBD initiation. The safety profile remained consistent with previous studies.