FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

Description

The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).

Conditions

Amyotrophic Lateral Sclerosis

Study Overview

Study Details

Study overview

The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).

A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients With Fused in Sarcoma Mutations (FUS-ALS)

FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

Condition
Amyotrophic Lateral Sclerosis
Intervention / Treatment

-

Contacts and Locations

La Jolla

University of California San Diego, La Jolla, California, United States, 92037

Palo Alto

Stanford University Medical Center, Palo Alto, California, United States, 94304

Baltimore

Johns Hopkins University, Baltimore, Maryland, United States, 21205

Boston

Massachusetts General Hospital, Boston, Massachusetts, United States, 02114

Saint Louis

Washington University School of Medicine, Saint Louis, Missouri, United States, 63110

New York

Columbia University Medical Center, New York, New York, United States, 10032

Columbus

The Ohio State University Wexner Medical Center, Columbus, Ohio, United States, 43210

Salt Lake City

University of Utah, Salt Lake City, Utah, United States, 84132

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

    Ages Eligible for Study

    10 Years to

    Sexes Eligible for Study

    ALL

    Accepts Healthy Volunteers

    No

    Collaborators and Investigators

    Ionis Pharmaceuticals, Inc.,

    Study Record Dates

    2028-03