A Phase I, Double-Blind, Randomized, Placebo-Controlled, Multicenter, Single- and Multiple-Ascending-Dose Study to Determine Initial Safety, Tolerability, and Pharmacokinetics of GDC-0134 in Patients With Amyotrophic Lateral Sclerosis

Study Purpose:

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.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

GDC-0134, Placebo, Rabeprazole, Midazolam, Caffeine




Phase 1

Study Chair(s)/Principal Investigator(s):

Clinical Trials, Hoffmann-La Roche

Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information

United States

Study Sponsor:

Genentech, Inc.

Estimated Enrollment:


Estimated Study Start Date:

05 / 31 / 2016

Estimated Study Completion Date:

03 / 16 / 2020

Posting Last Modified Date:

08 / 06 / 2020

Date Study Added to neals.org:

01 / 14 / 2016

Minimum Age:

18 Years

Maximum Age:


Can participants use Riluzole?


Inclusion Criteria:

- Male or female participants with a diagnosis of possible, laboratory-supported probable, probable, or definite ALS according to modified El Escorial criteria

- Upright forced vital capacity of at least 50 percent (%)

- Ability to fast from food for 8 hours prior to dosing and 2 hours after dosing

Exclusion Criteria:

- Currently taking riluzole unless on a stable dose for the 3 months prior to Day -1 and without current liver enzyme or liver function abnormalities

- Currently taking edaravone unless after completion of at least the second 14-day drug-treatment period, as long as Day 1 occurs during a drug-free period at least 24 hours after the last edaravone dose and at least 5 days prior to the first dose of the next cycle

- Positive for hepatitis C antibody, hepatitis B surface antigen, or human immunodeficiency virus (HIV) antibody

- Clinically significant thrombocytopenia

- Currently taking nutritional/herbal supplements, except for over-the-counter vitamins that are within Recommended Dietary Allowance (RDA), unless discontinued at least 7 days prior to Day -1, except upon approval of both the investigator and Sponsor

- For participants participating in a designated drug-drug interaction (DDI) cohort in the MAD stage of the study, who require midazolam/caffeine administration: known allergy, religious prohibition, or other condition limiting midazolam or caffeine administration

Forbes Norris Mda/als Ctr; Research Center

San Francisco, California 94115
United States

Mayo Clinic Hospital - Florida

Jacksonville, Florida 32224
United States

University of Miami Miller School of Medicine

Miami, Florida 33136
United States

The Emory ALS Clinic

Atlanta, Georgia 30322
United States

Johns Hopkins University School of Medicine

Baltimore, Maryland 21205
United States

Massachusetts General Hospital

Boston, Massachusetts 02114
United States

Wake Research Associates

Raleigh, North Carolina 27612
United States

New Orleans Center for Clinical Research

Knoxville, Tennessee 37920
United States

MUCH - Montreal Neurological Institute & Hospital

Montreal, Quebec H3A 2B4

UMC Utrecht

Utrecht 3508 GA