Gene therapy injection hopes to slow ALS
NCT ID NCT07618585
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early study tests a single injection of a gene therapy called NTF001 in 12 people with ALS. The therapy aims to deliver a protein that supports nerve cell health. The main goal is to check safety, but researchers will also look for signs of benefit over 52 weeks.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- NTF001 (a gene therapy that delivers a human neuron-derived neurotrophic factor via a single spinal injection)
- What this could lead to
- If it works, this could point toward a treatment that slows or stabilizes ALS progression by protecting nerve cells.
- What could go wrong
- This is a very early, small Phase 1 trial with only 12 people, focused on safety. It may not show any benefit, and gene therapies can have unexpected side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Apr 2026
- Expected to finish
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Mar 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 65 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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. Exclusion Criteria: * Mini-Mental State Examination (MMSE) score \< 24. * Patient Health Questionnaire-9 (PHQ-9) score ≥ 16. * Abnormal liver or renal function, defined as AST or ALT \> 1.5 × upper limit of normal (ULN), or serum creatinine (Cr) \> 1.5 × ULN. * Abnormal coagulation function or current use of anticoagulants. * Positive infectious disease screening, including positive HBsAg or HBV-DNA, positive HCV-RNA, positive HIV test, or positive syphilis serology. * Currently receiving antiviral treatment for hepatitis B or hepatitis C. * 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. * Current or previous history of malignant tumor. * History of severe allergic reactions, allergy to contrast agents, or inability to undergo surgical anesthesia. * 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. * History of alcohol dependence or drug addiction, and inability to stop alcohol consumption as instructed during the study. * Female participants who are pregnant or breastfeeding. * Participants considered unsuitable for enrollment by the investigator. * Patients requiring ventilator-assisted ventilation. * Patients allergic to the investigational intervention. * Patients with obvious signs of dementia. * Patients with other psychiatric disorders that may affect disease assessment. * Severely obese patients, defined as BMI \> 35 kg/m².
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, 200025, China
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Other studies related to the condition(s) this trial covers.
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