Gene therapy injection hopes to slow ALS

NCT ID NCT07618585

Ongoing Disease control Sponsor: Ruijin Hospital Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ALS - AMYOTROPHIC LATERAL SCLEROSIS

Contacts and locations

Locations

Ruijin Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, 200025, China

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