ACTIVE_NOT_RECRUITING

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)

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Conditions

Amyotrophic Lateral SclerosisAmyotrophic Lateral Sclerosis Participants With Fused in Sarcoma MutationsSarcoma Mutations

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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).

Official Title

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)

Quick Facts

Study Start:2021-06-14
Study Completion:2028-03
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT04768972

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.

Ages Eligible for Study:10 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Locations (Sites)

University of California San Diego
La Jolla, California, 92037
United States
Stanford University Medical Center
Palo Alto, California, 94304
United States
Johns Hopkins University
Baltimore, Maryland, 21205
United States
Massachusetts General Hospital
Boston, Massachusetts, 02114
United States
Washington University School of Medicine
Saint Louis, Missouri, 63110
United States
Columbia University Medical Center
New York, New York, 10032
United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
United States
University of Utah
Salt Lake City, Utah, 84132
United States

Collaborators and Investigators

Sponsor: Ionis Pharmaceuticals, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-06-14
Study Completion Date2028-03

Study Record Updates

Study Start Date2021-06-14
Study Completion Date2028-03

Terms related to this study

Keywords Provided by Researchers

  • Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations
  • Amyotrophic Lateral Sclerosis
  • Sarcoma Mutations

Additional Relevant MeSH Terms

  • Amyotrophic Lateral Sclerosis