FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
Purpose
This clinical trial is designed to test whether a single stereotactic intracerebral administration of inhibitory nerve cells into subjects with drug-resistant mesial temporal lobe epilepsy is safe (frequency of adverse events) and effective (seizure frequency).
Condition
- Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis
Eligibility
- Eligible Ages
- Between 18 Years and 65 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Male or Female, age ≥18 to 65 2. Focal seizures, clinically defined as TLE 3. Has failed to achieve seizure control despite adequate trials of at least 2 ASDs at appropriate doses 4. Currently on stable doses (at least 1 month) of approved ASDs 5. Single seizure focus confirmed as within one temporal lobe 6. For subjects entering Stage 1, the seizure focus is either a) in the non-dominant hemisphere, or b) in the dominant hemisphere and the subject has a Rey Auditory Verbal Learning Test (RAVLT) assessed within one year of screening, or at the screening visit, that is at least 1.5 standard deviations below the population mean. 7. Seizure frequency averages ≥2 per 28-day period over the 6 months prior to screening.
Exclusion Criteria
- Epilepsy due to other and/or progressive neurologic disease 2. Significant other medical condition which would impair safe participation 3. Primary or secondary immunodeficiency 4. Suicide attempt in the past year 5. Severe psychiatric disorders 6. Chronic indwelling intracranial device 7. MRI indicating potential malignant lesion 8. Pregnancy, or currently breastfeeding
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Intervention Model Description
- This is a two-stage study. Stage 1 is an open-label, single arm, sequential dose escalation. Stage 2 is a parallel, randomized, 2-arm, sham controlled study.
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Investigator, Outcomes Assessor)
- Masking Description
- This is a two-stage study. Stage 1 is open-label and unmasked. Stage 2 is masked with participant, part of investigator team, and outcomes assessor masked to treatment assignment.
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental NRTX-1001 (Stage 1) |
Up to 10 subjects. |
|
|
Experimental NRTX-1001 (Stage 2) |
Up to 20 subjects. |
|
|
Sham Comparator Sham Comparator (Stage 2) |
Up to 10 subjects. |
|
Recruiting Locations
Houston, Texas 77030
More Details
- Status
- Recruiting
- Sponsor
- Neurona Therapeutics
Detailed Description
Subjects will undergo a single stereotactic intracerebral administration of neural cells, called interneurons, that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Subjects will then take medicines to partially suppress their immune system (aimed to prevent the body from rejecting the cells) for 1 year. Safety, tolerability, evidence of neural cell viability and local inflammation (using MRI scans of the brain), and effects on epilepsy disease symptoms will be assessed for 2 years post-transplant. Subjects will be followed for an additional 13 years with quarterly phone contact and annual visits.