New ALS drug targets genetic cause in early human trial
NCT ID NCT07660614
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests an investigational drug called LTX-002 in people with amyotrophic lateral sclerosis (ALS). The drug is designed to target a specific genetic message linked to the disease. The main goal is to check the drug's safety and tolerability, and to see how it moves through the body and affects disease markers. About 56 adults with ALS will receive either the drug or a placebo via injection into the spinal fluid.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
LTX-002 (an antisense oligonucleotide)
What this could lead to
If safe and effective, this could lead to a new treatment that slows or stops the progression of ALS by targeting a genetic cause.
What could go wrong
This is a very early, small study primarily testing safety. The drug may not work or could cause unexpected side effects. Results may not apply to all ALS patients.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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AOU Città della Salute e della Scienza di Torino
NOT_YET_RECRUITINGTorino, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Istituto Neurologico "Carlo Besta"
NOT_YET_RECRUITINGMilan, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Karolinska Universitetssjukhuset
RECRUITINGStockholm, Sweden
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Universitair Medisch Centrum Utrecht
RECRUITINGUtrecht, Netherlands
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University Hospital Schleswig-Holstein, Campus Lübeck
NOT_YET_RECRUITINGLübeck, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••