Actively Recruiting
An Open, Dose-Escalation Early Phase Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of Intrathecal VGN-R13 in Patients with Amyotrophic Lateral Sclerosis (ALS)
Led by Hong Chen · Updated on 2025-03-17
6
Participants Needed
1
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of intrathecal delivery of VGN-R13, a gene therapy, for treating Amyotrophic Lateral Sclerosis (ALS), a fatal neurodegenerative disease with no current effective treatments beyond supportive care. This early phase clinical trial aims to assess how well patients tolerate the therapy and its potential benefits in ALS patients. Participants will receive a one-time intrathecal injection of VGN-R13, with up to two different dose levels planned. The study includes a screening period, a treatment day (D1) when the gene therapy and prophylactic immunosuppressive therapy start, followed by a follow-up period lasting up to 52 weeks. During follow-up, patients will undergo regular evaluations to monitor safety and treatment effects, with unscheduled visits possible if needed. Throughout the study, participants will have scheduled assessments including functional rating scales, lung function tests, muscle strength measurements, and antibody or immunogenicity tests. Researchers will closely track adverse events, disease progression, and survival outcomes over one year. This comprehensive monitoring aims to understand the therapy's safety profile and impact on ALS progression.
CONDITIONS
Brief Title
A Study to Evaluate the Tolerability, Safety and Efficacy of VGN-R13 in Patients with ALS
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Fully understand the study purpose and risks and provide signed informed consent
- Aged 18 years or older, male or female
- Diagnosed with ALS per World Federation of Neurology El Escorial criteria and confirmed by genetic testing excluding SOD1 and FUS mutations
- Disease duration less than or equal to 2 years from first ALS symptom
- Forced Vital Capacity (FVC) at least 50% of predicted value adjusted for gender, age, and height
- Discontinued riluzole for more than five half-lives before screening or on stable riluzole dose for at least 30 days and maintaining it during the study
- Discontinued edaravone for more than five half-lives before screening or on stable edaravone regimen for at least 60 days and maintaining it during the study
You will not qualify if you...
- Other diseases causing motor neuron dysfunction that may confuse ALS diagnosis
- Increased or uncontrolled bleeding risk or bleeding disorders not well managed
- Severe liver, kidney, cardiovascular disease, coagulation dysfunction, autoimmune deficiency or uncontrolled autoimmune disease
- Active infection requiring systemic treatment
- History or presence of malignant tumors within 5 years prior to screening
- Significant cognitive impairment, clinical dementia, or unstable psychiatric illness
- Use of medications strongly affecting CYP3A4 or P-glycoprotein
- Use of other investigational ALS treatments or biologics within one month before dosing
- Anticipated need for antiplatelet or anticoagulant medications around lumbar puncture
- Recent systemic immunosuppressive therapy within 3 months prior to screening
- Presence of implanted shunt or CNS catheter
- Prior gene therapy or stem cell transduction therapy
- Positive tests for HIV, syphilis, active hepatitis B or C, or tuberculosis
- Clinically significant blood, chemistry, or ECG abnormalities
- History of hypersensitivity to investigational product or immunosuppressants
- Contraindicated use of corticosteroids or sirolimus
- Recent drug abuse or alcoholism within 6 months
- Alcohol abuse exceeding 14 units per week or smoking more than 5 cigarettes daily in past 6 months
- Pregnant or breastfeeding women
- Other reasons deemed unsuitable by Investigator or Sponsor
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 1 day
Participants receive a one-time intrathecal injection of the gene therapy VGN-R13 along with prophylactic immunosuppressive therapy starting on the day of administration.
1 treatment visit (in-person)
Duration - Up to 52 weeks
Participants are monitored for safety and efficacy through various examinations and assessments up to 52 weeks after treatment administration.
Multiple follow-up visits based on evaluation schedule; unscheduled visits may occur if necessary
Trial Site Locations
Total: 1 location
1
TongJi Hospital
Wuhan, Hubei, China
Actively Recruiting
Research Team
H
Hong Chen
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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