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FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

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ClinicalTrials.gov Identifier: NCT05135091
Recruitment Status : Recruiting
First Posted : November 26, 2021
Last Update Posted : March 21, 2023
Sponsor:
Collaborator:
California Institute for Regenerative Medicine (CIRM)
Information provided by (Responsible Party):
Neurona Therapeutics

Tracking Information
First Submitted Date  ICMJE November 16, 2021
First Posted Date  ICMJE November 26, 2021
Last Update Posted Date March 21, 2023
Actual Study Start Date  ICMJE June 16, 2022
Estimated Primary Completion Date May 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 16, 2021)		 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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 16, 2021)
  • 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
Official Title  ICMJE A First-In-Human (FIH) Study of Inhibitory Interneurons (NRTX- 1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)
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).
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE 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
Condition  ICMJE Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis
Intervention  ICMJE
  • 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
  • Procedure: Sham Comparator
    Sham Comparator.
Study Arms  ICMJE
  • Experimental: NRTX-1001 (Stage 1)
    Up to 10 subjects.
    Intervention: Biological: NRTX-1001
  • Experimental: NRTX-1001 (Stage 2)
    Up to 20 subjects.
    Intervention: Biological: NRTX-1001
  • Sham Comparator: Sham Comparator (Stage 2)
    Up to 10 subjects.
    Intervention: Procedure: Sham Comparator
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: November 16, 2021)		 40
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 2026
Estimated Primary Completion Date May 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  1. Male or Female, age ≥18 to 55
  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.

Key Exclusion Criteria:

  1. 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 55 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Sheri Madrid, BS, BA 650-255-6885 sheri.madrid@neuronatx.com
Contact: C Priest, PhD 650-392-0372 cpriest@neuronatx.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05135091
Other Study ID Numbers  ICMJE NTE001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Neurona Therapeutics
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Neurona Therapeutics
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE California Institute for Regenerative Medicine (CIRM)
Investigators  ICMJE
Study Director: David Blum, MD Neurona Therapeutics
PRS Account Neurona Therapeutics
Verification Date March 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP