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A Study to Evaluate the Tolerability, Safety and Efficacy of VGN-R13 in Patients with ALS | Clinical Trials | Clareo Health

RECRUITINGEARLY_PHASE1

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

An Open, Dose-Escalation Early Phase Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of Intrathecal of VGN-R13 in Patients with Amyotrophic Lateral Sclerosis (ALS)

NCT06849609•Hong Chen

View on ClinicalTrials.gov

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).

Detailed Description

The study will evaluate safety and efficacy of gene therapy in Amyotrophic Lateral Sclerosis (ALS) patients. ALS is a fatal central nervous system neurodegenerative disease. There is no effective treatment for ALS and current drug therapy has been unsuccessful in stabilizing or reversing this disease. Only supportive care is currently possible. This study consists of screening period, treatment period and follow-up period. Eligible subjects will be enrolled. The day of administration set to be D1. Prophylactic immunosuppressive therapy will be initiated on D1. During the follow-up period (up to 52 weeks after administration), all the examinations will be completed based on the evaluation time point specified in the Schedule of Assessments table for efficacy and safety assessments until the End of Trial. Unscheduled visits may occur if the PI determines that they are necessary.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Fully understand the purpose and risks of the study and voluntarily provide a signed and dated informed consent form.
•2. Aged ≥18 years, male or female;
•3. 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;
•4. The duration of the disease from the first symptom (any ALS symptom) prior to the screening must be less than 2 years (inclusive);
•5. Forced Vital Capacity (FVC) adjusted for gender, age, and height (sitting position) ≥50% of the predicted value.
•6. 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.
•7. 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.

Exclusion Criteria

•1. 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.
•2. 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).
•3. 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;
•4. Presence of active infection that needs system treatment;
•5. Presence or history of malignant tumors within 5 years prior to screening;
•6. 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.
•7. 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;
•8. 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;
•9. 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；
•10. Received systemic immunosuppressive therapy within 3 months prior to screening;
+12 more criteria

Locations (1)

TongJi Hospital

Wuhan, Hubei, China

Key Dates

Start Date

January 16, 2025

Primary Completion Date

May 1, 2026

Completion Date

June 1, 2026

Last Updated

March 17, 2025

Enrollment

ESTIMATED participants

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Interventions

VGN-R13

BIOLOGICAL

Contacts

Hong Chen

CONTACT

13296508243 chenhong1129@hotmail.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 17, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

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