NCT07618585Active, not recruitingPhase 1
Safety and Efficacy of Intrathecal NTF001 Injection in ALS
A Study of the Safety and Efficacy of Intrathecal NTF001 Injection in the Treatment of Amyotrophic Lateral Sclerosis
Source: ClinicalTrials.gov · Last updated 1 Jun 2026
Brief summary (registry, in English)
This is a single-arm, open-label, early-phase clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of intrathecal NTF001 injection, an AAV-mediated human neuron-derived neurotrophic factor gene therapy, in patients with amyotrophic lateral sclerosis (ALS). 12 patients with ALS will be enrolled. Each participant will receive a single intrathecal administration of NTF001 and will be followed for 52 weeks after treatment. The primary outcome measures include treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). Secondary outcome measures include changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), quality-of-life assessments, and neurological function. This study aims to provide preliminary clinical evidence regarding the safety and potential efficacy of intrathecal NTF001 injection in patients with ALS.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 12 (estimated)
- First submitted
- 25 May 2026
- Start date
- 1 Apr 2026
- Primary completion date
- 31 Mar 2028
- Lead sponsor
- Ruijin Hospital
- Diseases
- ALS (motor neurone disease)
- NCT number
- NCT07618585
What you commit to
- Study type: Interventional · Phase 1
- Planned participants: 12
- From 1 Apr 2026 to the primary completion date, 31 Mar 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Currently receiving antiviral treatment for hepatitis B or hepatitis C.
- Currently participating in another clinical trial, or participation in another clinical trial within 3 months before screening.
- Previous receipt of gene therapy before screening.
- Receipt of stem cell therapy within 6 months before screening.
- Use of other investigational drugs within 4 weeks before screening or within 5 half-lives of the investigational drug, whichever is longer, or use of any medication that, in the opinion of the investigator, may affect this study.
- Receipt of a live vaccine within 2 months before screening, or any vaccination within 30 days before screening.
Other illness
- Positive infectious disease screening, including positive HBsAg or HBV-DNA, positive HCV-RNA, positive HIV test, or positive syphilis serology.
- Current or previous history of malignant tumor.
- History of severe allergic reactions, allergy to contrast agents, or inability to undergo surgical anesthesia.
- History of alcohol dependence or drug addiction, and inability to stop alcohol consumption as instructed during the study.
- Patients allergic to the investigational intervention.
- Patients with obvious signs of dementia.
- Patients with other psychiatric disorders that may affect disease assessment.
Lab values
- Abnormal liver or renal function, defined as AST or ALT \> 1.5 × upper limit of normal (ULN), or serum creatinine (Cr) \> 1.5 × ULN.
- Unstable or severe systemic diseases, including active tuberculosis, cardiovascular, respiratory, gastrointestinal, urinary, psychiatric or neurological disorders, such as epilepsy, hematological disorders, immune system diseases, or abnormal laboratory findings that, in the opinion of the investigator, make the participant unsuitable for this study.
Other
- Mini-Mental State Examination (MMSE) score \< 24.
- Patient Health Questionnaire-9 (PHQ-9) score ≥ 16.
- Abnormal coagulation function or current use of anticoagulants.
- Female participants who are pregnant or breastfeeding.
- Participants considered unsuitable for enrollment by the investigator.
- Patients requiring ventilator-assisted ventilation.
- Severely obese patients, defined as BMI \> 35 kg/m².
Who the study is looking for
- Voluntarily participate in this study and sign the informed consent form.
- Agree to comply with study procedures and cooperate with all study-related assessments throughout the study.
- Male or female patients aged 18 to 65 years.
- Meet the diagnostic and exclusion criteria for amyotrophic lateral sclerosis according to the Chinese Expert Consensus on the Diagnosis and Treatment of Amyotrophic Lateral Sclerosis 2022 issued by the Neurology Branch of the Chinese Medical Association.
- Have a history of amyotrophic lateral sclerosis of no more than 5 years.
Age in the registry: 18 years to 65 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Ruijin Hospital, Shanghai Jiao Tong University School of Medicine Coordinator of this site? Confirm its status | Shanghai | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.