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NCT02655614

A Study of GDC-0134 to Determine Initial Safety, Tolerability, and Pharmacokinetic Parameters in Participants With Amyotrophic Lateral Sclerosis

Completed Phase 1 Last updated 6 August 2020
What this trial tests

Phase 1 trial testing GDC-0134 in Amyotrophic Lateral Sclerosis in 54 participants. Completed in 16 March 2020.

Timeline
31 May 2016
Primary endpoint
16 March 2020
16 March 2020

Quick facts

Lead sponsorGenentech, Inc.
PhasePhase 1
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designsequential
Maskingdouble
Primary purposetreatment
Enrollment54
Start date31 May 2016
Primary completion16 March 2020
Estimated completion16 March 2020
Sites10 locations across Netherlands, Canada, United States

Drugs / interventions tested

Conditions studied

Sponsor

Genentech, Inc. — full company profile →

Who can join

18 and older, any sex, with Amyotrophic Lateral Sclerosis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This first-in-human, double-blind, placebo-controlled Phase I study will be conducted in participants with amyotrophic lateral sclerosis (ALS) to explore safety, tolerability, and pharmacokinetic (PK) properties of GDC-0134. It will include three components: a Single-Ascending-Dose (SAD) stage, a Multiple-Ascending-Dose (MAD) stage, and an Open-Label Safety Expansion (OSE) stage.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Cell Death and Neurodegeneration.
    Andreone BJ, Larhammar M, Lewcock JW. · · 2020 · cited 85× · PMID 31451511 · DOI 10.1101/cshperspect.a036434
  2. Induced pluripotent stem cell-derived motor neurons of CMT type 2 patients reveal progressive mitochondrial dysfunction.
    Van Lent J, Verstraelen P, Asselbergh B, Adriaenssens E, et al · · 2021 · cited 53× · PMID 34128983 · DOI 10.1093/brain/awab226
  3. New developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022.
    Jiang J, Wang Y, Deng M. · · 2022 · cited 48× · PMID 36518658 · DOI 10.3389/fphar.2022.1054006
  4. A Phase 1 study of GDC-0134, a dual leucine zipper kinase inhibitor, in ALS.
    Katz JS, Rothstein JD, Cudkowicz ME, Genge A, et al · · 2022 · cited 37× · PMID 35014217 · DOI 10.1002/acn3.51491
  5. Clinically Precedented Protein Kinases: Rationale for Their Use in Neurodegenerative Disease.
    Benn CL, Dawson LA. · · 2020 · cited 36× · PMID 33117143 · DOI 10.3389/fnagi.2020.00242
  6. Advances and challenges in modeling Charcot-Marie-Tooth type 2A using iPSC-derived models.
    Rizzuti M, Pagliari E, D'Agostino M, Ottoboni L, et al · · 2025 · PMID 41237781 · DOI 10.1016/j.stemcr.2025.102711
  7. Programmable self-replicating JEV nanotherapeutics redefine RNA delivery in ALS.
    Loo YS, Yusoh NA, Yap K, Ng CS. · · 2025 · PMID 40858858 · DOI 10.1038/s42003-025-08579-7

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Other recruiting trials for Amyotrophic Lateral Sclerosis

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Other Genentech, Inc. trials

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Data sources for this page

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