Long-term Follow-up Study of Patients Who Received EG110A, a Non-replicative Herpes Simplex Virus 1-derived Gene Therapy

Long-term Follow-up Study of Patients Who Received EG110A, a Non-replicative Herpes Simplex Virus 1-derived Gene Therapy Expressing a Transgene Encoding the Light Chain of Botulinum Toxin F

The aim of this study is to continue following participants who have received EG110A during five years to know how it is tolerated in humans, any side-effects it may cause and what might be an effective dose over five years. All participants who have received at least one dose/injection of EG110A in a previous clinical study of EG110A will be asked to roll-over to this long-term follow-up study upon either early discontinuation from, or completion of, that study.

Study Overview

• Status: Not yet recruiting
• Conditions: Urinary Bladder, Overactive; Spinal Cord Injury
• Intervention / Treatment: Other: Urodynamics; Drug: EG110A; Other: Patient-reported Outcome

Detailed Description

The objective of this long-term follow-up study is to continue following participants who have received EG110A for safety, and to observe long-term efficacy via a disease-specific quality of life questionnaire, time to participant request for alternative treatment, and locally-evaluated urodynamics.

This study aims to identify any potential long-term risks and will capture any delayed adverse events (AEs) in participants following the last study visit of the preceding study. Additionally, it will allow the Sponsor to further understand the long-term effectiveness of EG110A in this patient population.

• Study Type: Interventional
• Enrollment (Estimated): 16
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Cornelia Haag-Molkenteller, MD; Email: Cornelia@eg427.com
• Study Contact Backup: Name: Sandrine Zourbas, Ph.D.; Phone Number: 33 (0)625622802; Email: szourbas@eg427.com

Study Locations

• United States
  • California
    • Downey, California, United States, 90242
      • Rancho Los Amigos National Rehabilitation Center
      • Contact: Evgeniy Kreydin, MD
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Rogel Cancer Center
      • Contact: John Stoffel, MD
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • Sidney Kimmel Medical College
      • Contact: Patrick Shenot, MD
  • Texas
    • Houston, Texas, United States, 77030
      • UTHealth Houston / TIRR Memorial Hermann
      • Contact: Argyrios Stampas, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

- All participants who received at least one dose/injection of EG110A in Study EG110A-001-01 and have prematurely discontinued or completed that study.

Exclusion Criteria:

• Participant plans to participate in another investigational gene therapy study
• Participant has an uncontrolled intercurrent illness, any disorder, or current substance abuse that would limit compliance with study requirements in the opinion of the investigator

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Patients who received EG110A in a clinical study, regardless of dose	Other: Urodynamics; standardized local evaluation of urodynamic parameters; Drug: EG110A; Patients already treated with EG110A; Other: Patient-reported Outcome; assessment of participant's perception of the level of impairment in functioning and well-being associated with NDO symptoms

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of treatment emergent serious adverse events (TESAEs)	Any participant who has a reported treatment emergent serious adverse event (TESAE)	5 years
Incidence of treatment emergent adverse events (TEAEs) including treatment emergent averse events of special interest (TEAESIs)	Any participant who has a reported emergent adverse event (TEAE) including treatment emergent averse events of special interest (TEAESIs)	5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient-reported outcome (PRO) questionnaire	Participantes I-QOL total summary scores throughout the study compared with baseline from EG110A-001-01 study	5 years
Duration of response to EG110A	Time from initial treatment with EG110A to request an alternative treatment for NDO by the participant at any time during the long-term follow-up study	5 years

Collaborators and Investigators

• Sponsor: EG 427
• Investigators: Study Director: Cornelia Haag-Molkenteller, MD, EG 427

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2026
• Primary Completion (Estimated): April 1, 2031
• Study Completion (Estimated): April 1, 2031

Study Registration Dates

• First Submitted: November 10, 2025
• First Submitted That Met QC Criteria: November 10, 2025
• First Posted (Actual): November 12, 2025

Study Record Updates

• Last Update Posted (Actual): November 12, 2025
• Last Update Submitted That Met QC Criteria: November 10, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urogenital Diseases; Central Nervous System Diseases; Nervous System Diseases; Wounds and Injuries; Male Urogenital Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Lower Urinary Tract Symptoms; Urological Manifestations; Trauma, Nervous System; Urinary Bladder Diseases; Spinal Cord Diseases; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Spinal Cord Injuries; Urinary Bladder, Overactive; Health Services Administration; Health Care Quality, Access, and Evaluation; Investigative Techniques; Epidemiologic Methods; Data Collection; Health Care Evaluation Mechanisms; Quality of Health Care; Public Health; Environment and Public Health; Health Care Economics and Organizations; Outcome Assessment, Health Care; Outcome and Process Assessment, Health Care; Surveys and Questionnaires; Health Planning; Reproductive and Urinary Physiological Phenomena; Health Care Surveys; Health Services Research; Patient Outcome Assessment; Urinary Tract Physiological Phenomena; Patient Reported Outcome Measures; Urodynamics
• Other Study ID Numbers: EG110A-001-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No