Trial record 1 of 1 for:
NCT05135091
FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
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| ClinicalTrials.gov Identifier: NCT05135091 |
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Recruitment Status :
Recruiting
First Posted : November 26, 2021
Last Update Posted : January 22, 2024
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Sponsor:
Neurona Therapeutics
Collaborator:
California Institute for Regenerative Medicine (CIRM)
Information provided by (Responsible Party):
Neurona Therapeutics
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Brief Summary:
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 or disease | Intervention/treatment | Phase |
|---|---|---|
| Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis | Biological: NRTX-1001 Procedure: Sham Comparator | Phase 1 Phase 2 |
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.
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 40 participants |
| 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. |
| 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. |
| Primary Purpose: | Treatment |
| Official Title: | A First-In-Human (FIH) Study of Inhibitory Interneurons (NRTX- 1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE) |
| Actual Study Start Date : | June 16, 2022 |
| Estimated Primary Completion Date : | May 2025 |
| Estimated Study Completion Date : | May 2026 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: NRTX-1001 (Stage 1)
Up to 10 subjects.
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Biological: NRTX-1001
Biological: NRTX-1001 is an experimental neural cell therapy product candidate derived from an allogeneic human embryonic stem cell line. The stem cells were converted into inhibitory nerve cells that produce GABA.
Other Name: GABA-secreting interneurons |
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Experimental: NRTX-1001 (Stage 2)
Up to 20 subjects.
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Biological: NRTX-1001
Biological: NRTX-1001 is an experimental neural cell therapy product candidate derived from an allogeneic human embryonic stem cell line. The stem cells were converted into inhibitory nerve cells that produce GABA.
Other Name: GABA-secreting interneurons |
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Sham Comparator: Sham Comparator (Stage 2)
Up to 10 subjects.
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Procedure: Sham Comparator
Sham Comparator. |
Primary Outcome Measures :
- Frequency of serious or severe AEs [ Time Frame: 1 year ]The primary endpoint of the study is the frequency of serious or severe AEs over the period of 1 year after administration, comparing all subjects treated with NRTX-1001 (Stage 1 + Stage 2) with all subjects who receive sham treatment.
Secondary Outcome Measures :
- Change in seizure frequency [ Time Frame: 1 year ]The difference in the median percentage change from baseline in seizure frequency (seizures per 28 days) between subjects that receive NRTX-1001 and those who receive sham treatment, assessed in Stage 2.
- Responder rate [ Time Frame: 1 year ]The difference in the 75% responder rate, e.g., the difference between the fraction of subjects who receive NRTX-1001 and those that receive sham treatment who experience at least a 75% reduction from baseline in seizure frequency, assessed in Stage 2.
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| Ages Eligible for Study: | 18 Years to 65 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Key Inclusion Criteria:
- Male or Female, age ≥18 to 65
- Focal seizures, clinically defined as TLE
- Has failed to achieve seizure control despite adequate trials of at least 2 ASDs at appropriate doses
- Currently on stable doses (at least 1 month) of approved ASDs
- Single seizure focus confirmed as within one temporal lobe
- 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.
- Seizure frequency averages ≥2 per 28-day period over the 6 months prior to screening.
Key Exclusion Criteria:
- Epilepsy due to other and/or progressive neurologic disease
- Significant other medical condition which would impair safe participation
- Primary or secondary immunodeficiency
- Suicide attempt in the past year
- Severe psychiatric disorders
- Chronic indwelling intracranial device
- MRI indicating potential malignant lesion
- Pregnancy, or currently breastfeeding
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05135091
Contacts
| Contact: Sheri Madrid, BS, BA | 949-500-0027 | sheri@neuronatx.com | |
| Contact: C Priest, PhD | 650-392-0372 | cpriest@neuronatx.com |
Locations
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Sponsors and Collaborators
Neurona Therapeutics
California Institute for Regenerative Medicine (CIRM)
Investigators
| Study Director: | David Blum, MD | Neurona Therapeutics |
| Responsible Party: | Neurona Therapeutics |
| ClinicalTrials.gov Identifier: | NCT05135091 |
| Other Study ID Numbers: |
NTE001 |
| First Posted: | November 26, 2021 Key Record Dates |
| Last Update Posted: | January 22, 2024 |
| Last Verified: | January 2024 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Additional relevant MeSH terms:
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Epilepsy Epilepsy, Temporal Lobe Hippocampal Sclerosis Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Epilepsies, Partial Epileptic Syndromes Malformations of Cortical Development, Group I Malformations of Cortical Development Nervous System Malformations Congenital Abnormalities |