A Study to Evaluate the Tolerability, Safety and Efficacy of VGN-R13 in Patients with ALS

NCT06849609 | Recruiting Early Phase 1

• 1 other identifier: VGN-R13-001
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of VGN-R13 as a treatment of Amyotrophic Lateral Sclerosis (ALS).

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of adverse events

  An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment.

  up to 52 weeks

Secondary Outcomes (6)

• Change From Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score

  up to 52 weeks

• Change From Baseline in Percent Predicted Forced Vital Capacity (FVC)

  up to 52 weeks

• Time to Death or Permanent Ventilation

  up to 52 weeks

• Change From Baseline in Handheld Dynamometry (HHD) Megascore as Measured by the HHD Device

  up to 52 weeks

• The antibody titer

  up to 52 weeks

Study Arms (1)

VGN-R13

EXPERIMENTAL

Up to two sequential VGN-R13 dose levels are planned. Each subject will receive one-time administration of VGN-R13 through intrathecal injection.

Biological: VGN-R13

Interventions

VGN-R13 BIOLOGICAL

An adeno-associated viral vector 9 gene therapy product.

VGN-R13

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Fully understand the purpose and risks of the study and voluntarily provide a signed and dated informed consent form.
• Aged ≥18 years, male or female;
• A diagnosis of ALS according to the World Federation of Neurology El Escorial criteria (revised according to the Airlie House Conference 1998 \[Brooks 2000\]), and confirmed through genetic diagnosis to exclude pathogenic mutations in the superoxide dismutase 1 (SOD1) and/or FUS genes;
• The duration of the disease from the first symptom (any ALS symptom) prior to the screening must be less than 2 years (inclusive);
• Forced Vital Capacity (FVC) adjusted for gender, age, and height (sitting position) ≥50% of the predicted value.
• Discontinued riluzole for more than five half-lives prior to screening (and is not expected to resume during the study) or has been on a stable dose of riluzole for ≥30 days and continue to maintain this dose during the study period.
• Discontinued edaravone for more than five half-lives prior to screening (and is not expected to resume edaravone during the study) or is receiving the standard edaravone treatment regimen at screening and has maintained a stable dose for ≥60 days (two treatment cycles) prior to administration, and continue to maintain this dose during the study period.

You may not qualify if:

• There are other diseases related to motor neuron dysfunction (progressive bulbar palsy, primary lateral sclerosis, cervical spondylosis, lumbar spondylosis, etc., idiopathic inflammatory myopathy), which may confuse or cover up the diagnosis of ALS.
• Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally and could place a participant at an increased risk for intraoperative or postoperative bleeding. These could include, but are not limited to, anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms, or other abnormalities) and underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., hemophilia, Von Willebrand's disease, liver disease).
• Current severe liver or kidney or cardiovascular disease or coagulation dysfunction, autoimmune deficiency, or uncontrolled autoimmune disease or need immunosuppressive long-term treatment, poorly controlled diabetes (HBA1C ≥7% at screening) or high blood pressure, presence of sever gastrointestinal ulcers or history of gastrointestinal bleeding;
• Presence of active infection that needs system treatment;
• Presence or history of malignant tumors within 5 years prior to screening;
• Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression ≤ 90 days, as determined by the Investigator.
• Current using medications including, drugs, herbal or OTC medications that strongly inhibit or induce CYP3A4 or P-glycoprotein (P-gp), e.g., metoclopramide, grapefruit juice, ketoconazole, erythromycin;
• Received another drug for the treatment of ALS disease (including but not limited to sodium phenylbutyrate (PB), taurine diol (TURSO), tauroursodeoxycholic acid (TUDCA) within 1 month before the first dose) or ursodeoxycholic acid (UDCA), biologics or Other investigational drugs with a discontinuation period shorter than five half-lives;
• Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication (e.g., clopidogrel) for 7 days before or 48 hours after an LP；
• Received systemic immunosuppressive therapy within 3 months prior to screening;
• Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter；
• Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter；
• Participation in gene therapy or stem cell transduction therapy at any time prior to screening；
• Positive test result for HIV, positive treponemal antibody for syphilis, Active hepatitis B or hepatitis C infection, Active tuberculosis infection;
• Clinically significant abnormalities in hematology or clinical chemistry parameters, as determined by the Investigator, which would render the participant unsuitable for enrollment;

Sponsors & Collaborators

• Hong Chen: lead

Study Sites (1)

TongJi Hospital

Wuhan, Hubei, China

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Hong Chen

CONTACT

13296508243; chenhong1129@hotmail.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: February 23, 2025
• First Posted: February 27, 2025
• Study Start: January 16, 2025
• Primary Completion: May 1, 2026
• Study Completion: June 1, 2026
• Last Updated: March 17, 2025

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)