A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: SNUG01.
- Who it may be relevant to
- Registry conditions: Amyotrophic Lateral Sclerosis (ALS). Basic parameters: 18 years — 80 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Multicenter, Open-label, Single-arm, Dose Escalation and Expansion, Phase I/IIa Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of SNUG01 in Adult Subjects With Amyotrophic Lateral Sclerosis (ALS)
Overview
The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of SNUG01 in in adult subjects with Amyotrophic Lateral Sclerosis (ALS).
Detailed description
This is a 52-week, multicenter, open-label, single-arm, dose escalation and expansion Phase Ⅰ/Ⅱa study of SNUG01 in adults with ALS. Safety will be the primary focus, with secondary emphasis on immunogenicity, PK and preliminary clinical efficacy of SNUG01.
Interventions
- Drug SNUG01
AAV (adeno-associated virus) Gene therapy
Primary outcome measures
- The safety and tolerability of SNUG01 administered by intrathecal. [Time frame: up to 1 year after administration]
- To recommend the optimal expansion dose of SNUG01 that demonstrates acceptable safety with maximum preliminary efficacy administered by IT. [Time frame: up to 1 years]
Secondary outcome measures (3)
- Immunogenicity of SNUG01 [Time frame: up to 1 years]
- PK of SNUG01 (Biodistribution and Viral Shedding) [Time frame: up to 1 years]
- Preliminary Clinical Efficacy of SNUG01 [Time frame: up to 1 years]
Eligibility criteria
- Key Inclusion Criteria\*\*:
- Subjects who are able to provide written informed consent form (ICF).
- Subjects who are males or females must be ≥ 18 years and ≤ 80 years of age at the screening visit.
- Subjects who have clinically definite ALS, clinically probable ALS, or clinically probable-laboratory supported ALS as specified in the revised version of the El Escorial World Federation of Neurology criteria.
- Subjects must have an ALS disease duration (from first symptom onset to the screening visit) ≤ 2 years.
- Subjects with a body mass index (BMI) ≥ 19 kg/m2 at the screening visit.
- Subjects whose percent-predicted Forced Vital Capacity (%FVC) is ≥ 70% or percent-predicted Slow Vital Capacity (%SVC) is ≥ 60%, adjusted for sex, age, and height at the screening visit.
- The ALSFRS-R score ≥ 30 during the screening period, and the three respiratory scores (dyspnea, upright respiration, and respiratory insufficiency) must be full marks.
- Key Exclusion Criteria\*\*:
- Serum Anti-AAV9 neutralizing antibody titer ≥ 1:100.
- Current or previous exposure to gene therapy, stem cell products, and solid organ transplantation.
- Subjects who have implanted or are estimated to require a diaphragmatic pacing system during the study period.
- Any thromboembolic event, such as deep vein thrombosis, pulmonary arteriovenous embolism, and jugular vein embolism, has occurred within 6 months before the administration.
- Suffering from autoimmune diseases or ongoing immune-related therapy, except intranasal, inhalation, ocular, topical, intra-articular corticosteroid therapy or corticosteroid physiological replacement therapy.
- Active or chronic uncontrolled infection within 4 weeks before the administration, deemed unacceptable in the discretion of the investigator.
- Evidence of human immunodeficiency virus (HIV) and treponema pallidum (TP) infection, as documented by the treatment for HIV or TP, or by HIV or TP antibodies positivity at the screening visit.
- Has a positive serum pregnancy test at screening (females of childbearing potential only), a positive urine or serum pregnancy test at baseline (Day -1. females of childbearing potential only), or is nursing.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 3 centers
- Peking University Third Hospital — Beijing
- Fujian Medical University Union Hospital — Fuzhou
- Second Affiliated Hospital Zhejiang University School of Medicine — Hangzhou
United States · 1 center
- Massachusetts General Hospital — Boston
Identifiers
NCT: NCT07169175 · SNUG01-101