Actively Recruiting
A Study to Evaluate the Tolerability, Safety and Efficacy of VGN-R13 in Patients with ALS
Led by Hong Chen · Updated on 2025-03-17
6
Participants Needed
1
Research Sites
71 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official 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...
- Understand the study purpose and risks and provide signed informed consent.
- Age 18 years or older, any gender.
- Diagnosed with ALS by World Federation of Neurology El Escorial criteria and confirmed by genetic testing excluding SOD1 or FUS mutations.
- Disease duration less than or equal to 2 years from first symptom.
- Forced Vital Capacity (FVC) adjusted for gender, age, and height is at least 50% of predicted.
- Either stopped riluzole more than five half-lives before screening and not expected to resume, or on a stable riluzole dose for at least 30 days maintained during the study.
- Either stopped edaravone more than five half-lives before screening and not expected to resume, or on stable edaravone treatment for at least 60 days maintained during the study.
You will not qualify if you...
- Presence of other motor neuron diseases or conditions that could confuse ALS diagnosis.
- Risk of increased or uncontrolled bleeding not well managed, including anatomical or coagulation disorders.
- Severe liver, kidney, cardiovascular disease, coagulation dysfunction, autoimmune disease, poorly controlled diabetes (HbA1c greater than 7%), high blood pressure, severe gastrointestinal ulcers, or history of gastrointestinal bleeding.
- Active infection requiring systemic treatment.
- History or presence of malignant tumors within 5 years before screening.
- Significant cognitive impairment, dementia, or unstable psychiatric illness including recent severe depression.
- Use of medications that strongly affect CYP3A4 or P-glycoprotein.
- Recent use of other ALS treatments or investigational drugs within specified washout periods.
- Expected need for antiplatelet or anticoagulant therapy around lumbar puncture.
- Systemic immunosuppressive therapy within 3 months before screening.
- Presence of implanted cerebrospinal fluid shunts or CNS catheters.
- Prior participation in gene or stem cell therapy for ALS.
- Positive tests for HIV, syphilis, active hepatitis B or C, or tuberculosis.
- Clinically significant abnormal lab or ECG findings.
- History of systemic allergic reactions to study products or immunosuppressants.
- Contraindicated corticosteroid or sirolimus use.
- Recent drug abuse or alcoholism limiting participation.
- Excessive alcohol or smoking use in past 6 months.
- Pregnant or breastfeeding women.
- Other investigator or sponsor-determined reasons making participation unsuitable.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
TongJi Hospital
Wuhan, Hubei, China
Actively Recruiting
Research Team
H
Hong Chen
CONTACT
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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