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

Primary Purpose

Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis

Status

Recruiting

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

NRTX-1001

Sham Comparator

About this trial

This is an interventional treatment trial for Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

Male or Female, age ≥18 to 55
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, seizure focus is in the non-dominant hemisphere.
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

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Sham Comparator

Arm Label

NRTX-1001 (Stage 1)

NRTX-1001 (Stage 2)

Sham Comparator (Stage 2)

Arm Description

Up to 10 subjects.

Up to 20 subjects.

Up to 10 subjects.

Outcomes

Primary Outcome Measures

Frequency of serious or severe AEs

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

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

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.

Full Information

NCT ID

NCT05135091

First Posted

November 16, 2021

Last Updated

October 11, 2023

Sponsor

Neurona Therapeutics

Collaborators

California Institute for Regenerative Medicine (CIRM)

1. Study Identification

Unique Protocol Identification Number

NCT05135091

Brief Title

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

Official Title

A First-In-Human (FIH) Study of Inhibitory Interneurons (NRTX- 1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Recruiting

Study Start Date

June 16, 2022 (Actual)

Primary Completion Date

May 2025 (Anticipated)

Study Completion Date

May 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Neurona Therapeutics

Collaborators

California Institute for Regenerative Medicine (CIRM)

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Parallel Assignment

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

ParticipantInvestigatorOutcomes 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.

Allocation

Randomized

Enrollment

40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

NRTX-1001 (Stage 1)

Arm Type

Experimental

Arm Description

Up to 10 subjects.

Arm Title

NRTX-1001 (Stage 2)

Arm Type

Experimental

Arm Description

Up to 20 subjects.

Arm Title

Sham Comparator (Stage 2)

Arm Type

Sham Comparator

Arm Description

Up to 10 subjects.

Intervention Type

Biological

Intervention Name(s)

NRTX-1001

Other Intervention Name(s)

GABA-secreting interneurons

Intervention Description

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.

Intervention Type

Procedure

Intervention Name(s)

Sham Comparator

Intervention Description

Sham Comparator.

Primary Outcome Measure Information:

Title

Frequency of serious or severe AEs

Description

Time Frame

1 year

Secondary Outcome Measure Information:

Title

Change in seizure frequency

Description

Time Frame

1 year

Title

Responder rate

Description

Time Frame

1 year

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility 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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Sheri Madrid, BS, BA

Phone

949-500-0027

sheri@neuronatx.com

First Name & Middle Initial & Last Name or Official Title & Degree

C Priest, PhD

Phone

650-392-0372

cpriest@neuronatx.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

David Blum, MD

Organizational Affiliation

Neurona Therapeutics

Official's Role

Study Director

Facility Information:

Facility Name

University of California Los Angeles

City

Los Angeles

State/Province

California

ZIP/Postal Code

90095

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Suchi Tiwari, MBBS

Phone

310-825-4321

stiwari@mednet.ucla.edu

First Name & Middle Initial & Last Name & Degree

Daisy J Mercado, BS

Phone

310-825-8851

dmercado@mednet.ucla.edu

First Name & Middle Initial & Last Name & Degree

John M Stern, MD

Facility Name

Stanford University

City

Palo Alto

State/Province

California

ZIP/Postal Code

94304

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Jordan Seliger, CRC, MA

Phone

650-460-9260

jseliger@stanford.edu

First Name & Middle Initial & Last Name & Degree

Ruba Shaik, CRC, BS

Phone

650-546-1436

rubas@stanford.edu

First Name & Middle Initial & Last Name & Degree

Kevin Graber, MD

First Name & Middle Initial & Last Name & Degree

Vivek Buch, MD

First Name & Middle Initial & Last Name & Degree

Jaimie Henderson, MD

First Name & Middle Initial & Last Name & Degree

Richard Lafayette, MD

First Name & Middle Initial & Last Name & Degree

Jordan Seliger

Facility Name

University of California San Diego

City

San Diego

State/Province

California

ZIP/Postal Code

92037

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Christian P Fulinara, BS

Phone

858-249-3038

chfulinara@health.ucsd.edu

First Name & Middle Initial & Last Name & Degree

Alpha Stem Cell Clinic

Phone

844-317-7836

alphastemcellclinic@ucsd.edu

First Name & Middle Initial & Last Name & Degree

Jerry J Shih, MD

Facility Name

University of California San Francisco

City

San Francisco

State/Province

California

ZIP/Postal Code

94143

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Vanessa R Anderson, BA

vanessa.anderson@ucsf.edu

First Name & Middle Initial & Last Name & Degree

Robert C Knowlton, MD

Phone

(415) 353-2437

Robert.Knowlton@ucsf.edu

First Name & Middle Initial & Last Name & Degree

Robert C Knowlton, MD

First Name & Middle Initial & Last Name & Degree

Edward F Chang, MD

First Name & Middle Initial & Last Name & Degree

Paul A Garcia, MD

First Name & Middle Initial & Last Name & Degree

Jeffrey M Gelfand, MD, MAS

First Name & Middle Initial & Last Name & Degree

Brandon E Kopald, PsyD

Facility Name

University of Colorado Anschutz Medical Campus

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Trey Jouard, MS

Phone

970-817-4272

Trey.Jouard@cuanschutz.edu

First Name & Middle Initial & Last Name & Degree

Christopher Mizenko, MS

Phone

303-724-4117

Christopher.Mizenko@cuanschutz.edu

First Name & Middle Initial & Last Name & Degree

Lesley Kaye, MD

First Name & Middle Initial & Last Name & Degree

Steven Ojemann, MD

First Name & Middle Initial & Last Name & Degree

Daniel Kramer, MD

First Name & Middle Initial & Last Name & Degree

Amanda Piquet, MD

First Name & Middle Initial & Last Name & Degree

Naveed Chaudhry, MD

First Name & Middle Initial & Last Name & Degree

Thomas Wodushek, PhD

First Name & Middle Initial & Last Name & Degree

Christopher Mizenko

First Name & Middle Initial & Last Name & Degree

Trey Jouard

First Name & Middle Initial & Last Name & Degree

Michelle Sandoval

Facility Name

Rush University Medical Center

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60612

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Amanda Sremac, BS

Phone

312-942-0539

Amanda_C_Sremac@rush.edu

First Name & Middle Initial & Last Name & Degree

Rebecca McWilliams, BS

Phone

312-563-1025

Rebecca_McWilliams@rush.edu

First Name & Middle Initial & Last Name & Degree

Rebecca O'Dwyer, MD

First Name & Middle Initial & Last Name & Degree

Travis Stoub, PhD

First Name & Middle Initial & Last Name & Degree

Sepehr B Sani, MD

Facility Name

University of Chicago

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60637

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Agnieszka Stadnik, MS

Phone

773-702-8996

astadnik@bsd.uchicago.edu

First Name & Middle Initial & Last Name & Degree

Justine Hsu, BSN, RN

Phone

773-834-1485

justinelee89@bsd.uchicago.edu

First Name & Middle Initial & Last Name & Degree

Peter Warnke, MD

Facility Name

NYU Langone Comprehensive Epilepsy Center

City

New York

State/Province

New York

ZIP/Postal Code

10016

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Jack C Carter, BS

Phone

646-558-0841

jack.carter@nyulangone.org

First Name & Middle Initial & Last Name & Degree

Jacob Sloane, BA

Phone

646-558-0818

Jacob.sloane@nyulangone.org

First Name & Middle Initial & Last Name & Degree

Orrin Devinsky, MD

First Name & Middle Initial & Last Name & Degree

Daniel Friedman, MD

First Name & Middle Initial & Last Name & Degree

Werner Doyle, MD

First Name & Middle Initial & Last Name & Degree

Thomas Wisniewski, MD

First Name & Middle Initial & Last Name & Degree

Bruce Solitar, MD

Facility Name

SUNY Upstate Medical University

City

Syracuse

State/Province

New York

ZIP/Postal Code

13210

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Lena F Deb, BA

Phone

315-464-9756

debl@upstate.edu

First Name & Middle Initial & Last Name & Degree

Marielle N De Masi, BS

Phone

315-464-9756

demasim@upstate.edu

First Name & Middle Initial & Last Name & Degree

Robert L Beach, MD, PhD

First Name & Middle Initial & Last Name & Degree

Corey A McGraw, MD

First Name & Middle Initial & Last Name & Degree

Harish Babu, MD, PhD

First Name & Middle Initial & Last Name & Degree

Shahram Izadyar, MD

First Name & Middle Initial & Last Name & Degree

Xiangping Zhou, MD, PhD

Facility Name

Duke University Hospital

City

Durham

State/Province

North Carolina

ZIP/Postal Code

27710

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Jodi Lennon, BSN, RN

Phone

919-613-9129

jodi.lennon@duke.edu

First Name & Middle Initial & Last Name & Degree

Beth Perry, RN, CCRP

Phone

919-681-2695

beth.perry@duke.edu

First Name & Middle Initial & Last Name & Degree

Derek Southwell, MD, PhD

First Name & Middle Initial & Last Name & Degree

Joanne Kurtzberg, MD

First Name & Middle Initial & Last Name & Degree

Austin Dixon, MD, MBA

First Name & Middle Initial & Last Name & Degree

Sidney Gospe III, MD, PhD

First Name & Middle Initial & Last Name & Degree

Jill Stuart, PhD

First Name & Middle Initial & Last Name & Degree

Mitchell E Horwitz, MD

First Name & Middle Initial & Last Name & Degree

Beth Shaz, MD, MBA

First Name & Middle Initial & Last Name & Degree

Matthew Luedke, MD

Facility Name

Oregon Health and Science University

City

Portland

State/Province

Oregon

ZIP/Postal Code

97239

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Claire Dorfman, BA

Phone

971-413-9201

dorfman@ohsu.edu

First Name & Middle Initial & Last Name & Degree

Celena Byerlee-Dixon, MS

Phone

971-334-1942

byerlee@ohsu.edu

First Name & Middle Initial & Last Name & Degree

David C Spencer, MD

First Name & Middle Initial & Last Name & Degree

Kim Burchiel, MD

First Name & Middle Initial & Last Name & Degree

Paul Motika, MD

First Name & Middle Initial & Last Name & Degree

Lia Ernst, MD

First Name & Middle Initial & Last Name & Degree

David Pettersson, MD

First Name & Middle Initial & Last Name & Degree

Michael Lane, MD

First Name & Middle Initial & Last Name & Degree

Tyler Duffield, PhD

Facility Name

Thomas Jefferson University Hospital

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19107

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Latasha Adams, MBS

Phone

215-955-2606

Latasha.adams2@jefferson.edu

First Name & Middle Initial & Last Name & Degree

Emily Patunas, BS

Phone

215-503-3213

Emily.patunas@jefferson.edu

First Name & Middle Initial & Last Name & Degree

Michael Sperling, MD

Facility Name

UTHealth Houston

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Eliana M Klier, PhD

Phone

713-500-5442

Eliana.Klier@uth.tmc.edu

First Name & Middle Initial & Last Name & Degree

Jessica A Johnson, MSN, NP-C

Phone

713-500-5443

Jessica.A.Johnson@uth.tmc.edu

First Name & Middle Initial & Last Name & Degree

Nitin Tandon, MD

Facility Name

University of Utah Health

City

Salt Lake City

State/Province

Utah

ZIP/Postal Code

84108

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Laura Beeler, BS

Phone

801-585-9266

laura.beeler@hsc.utah.edu

First Name & Middle Initial & Last Name & Degree

Marissa Castillo, BS

Phone

801-585-9266

marissa.castillo@hsc.utah.edu

First Name & Middle Initial & Last Name & Degree

Amir Arain, MD

First Name & Middle Initial & Last Name & Degree

Sindhu Richard, MD

First Name & Middle Initial & Last Name & Degree

Brian Johnson, MD

First Name & Middle Initial & Last Name & Degree

Blake Newman, MD

First Name & Middle Initial & Last Name & Degree

Angela Peters, MD

First Name & Middle Initial & Last Name & Degree

Katherine Krulisky, MD

First Name & Middle Initial & Last Name & Degree

Shervin Rahimpour, MD

First Name & Middle Initial & Last Name & Degree

Michelle Miranda, PhD, MPH

First Name & Middle Initial & Last Name & Degree

Jonathan Galli, MD

Facility Name

Medical College of Wisconsin

City

Milwaukee

State/Province

Wisconsin

ZIP/Postal Code

53226

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Sarah Cornell, MS

Phone

414-955-0989

scornell@mcw.edu

First Name & Middle Initial & Last Name & Degree

Kaitlin Goetschel, BS

Phone

414-955-7398

kgoetschel@mcw.edu

First Name & Middle Initial & Last Name & Degree

Sean Lew, MD

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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

Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial