Trial intelligence / Profile preview

First-in-human multiple ascending dose intrathecal LTX-002 in adults with amyotrophic lateral sclerosis

2025-522039-33-00Phase 2Authorised

Sponsor
Leal Therapeutics
Start
Apr 2026
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Countries
5
01

Overview

Phase 2, randomised, double-blind trial in amyotrophic lateral sclerosis (ALS) comparing LTX-002 with Placebo (2 arms). Primary endpoints: Incidence and severity of adverse events, Clinical laboratory tests and 3 more. Planned start Apr 2026; status: authorised.

02

Drugs

LTX-002
03

Conditions

amyotrophic lateral sclerosis (ALS)

Beyond the preview

Go deeper on 2025-522039-33-00.

Study design, results and competitive context are available in the Gosset platform where reported.

Study design & results

Available in the Gosset platform where reported.

Competitive context

Available in the Gosset platform where reported.

Bring the full picture into focus.

See how Gosset can support your research on 2025-522039-33-00.

Gosset Free

Get started with Gosset.

Enter your corporate email.

Work email preferred.

Book a call