The study is designed in two stages to evaluate the safety (Stages 1 and 2) and efficacy (Stage 2) of NRTX-1001 for the treatment of drug-resistant unilateral mesial temporal lobe epilepsy with mesial temporal sclerosis (MTLE with MTS). In Stage 1, subjects will receive NRTX-1001 cells in open-label cohorts of two escalating doses. Selection of dose for Stage 2 will be based on the safety and tolerability of doses used in Stage 1. In Stage 2, subjects will be randomized to either surgical treatment with NRTX-1001 or sham surgery, combined with ongoing medical management. Subjects and members of the study team who conduct outcomes assessment will be blinded to treatment allocation. In Stage 2, the primary outcome measure will be reduction in seizure frequency, compared between the two treatment groups.

In both stages, subjects will be evaluated for safety as a primary outcome and efficacy as a secondary measure. Freedom from seizures, seizure frequency, seizure severity, responder rate, quality of life, adverse events (AEs), visual fields, effects on mood, and neuropsychological outcomes will be evaluated. We will also conduct immune system monitoring, measurement of relevant metabolites by magnetic resonance spectroscopy (MRS) and explore anti-seizure drug (ASD) dose reduction.

Sponsor

Neurona Therapeutics, Inc.

Status of enrollment

Open to accrual

Ages Eligible for Study

18 years old to 65 years old.

Genders Eligible for Study

Males or Females

Disease indication

Drug-resistant unilateral mesial temporal lobe epilepsy (MTLE)

Principal Investigator

Robert Knowlton, MD

Contact

Margaret Seaton: [email protected]

Additional study eligibility details can be found at UCSF Clinical Trials.