Trial: HEALEY ALS Platform Trial - Regimen G DNL343

Quick Info

Status

Enrolling by invitation

Phase

2/3

Trial Type

Interventional

Treatment Type

Drug Trials

Randomization

3:1

Enrollment

240

Start Date

5/24/2023

Sponsor

Merit E. Cudkowicz, MD

Contact Information

833-425-8257 (HALT ALS)
healeyalsplatform@mgh.harvard.edu

Locations

United States, Arizona

Barrow Neurological Institute, AZ, United States
Contact: Whitney Dailey

United States, California

Cedars-Sinai Medical Center, CA, United States
Contact: Sophia Mostowy

Forbes Norris MDA/ALS Research Center, California Pacific Medical Center, CA, United States
Contact: Teji Dulai

Kaiser Permanente, Los Angeles Medical Center, CA, United States
Contact: Mary H. Berganza

Loma Linda University Health, CA, United States
Contact: David Borg

University of California, Irvine, CA, United States
Contact: Pola Gaid

University of California, San Francisco, CA, United States
Contact: Reshma Kolala

University of Southern California, CA, United States
Contact: Salma Akhter

United States, Colorado

University of Colorado Anschutz, CO, United States
Contact: Recruitment Coordinator

United States, Connecticut

Hospital for Special Care, CT, United States
Contact: Natalie Cartwright

United States, District of Columbia

Georgetown University Pasquerilla Health Center, DC, United States
Contact: Gabby Law

George Washington University, DC, United States
Contact: Grace Johnson

United States, Florida

Mayo Clinic Florida, FL, United States
Contact: Jany Paulett

Nova Southeastern University, FL, United States
Contact: Donovan Mott

Phil Smith Neuroscience Institute, Holy Cross Hospital, FL, United States
Contact: Ashley Stepler

University of Florida Gainesville, FL, United States
Contact: Jennifer Steshyn

University of Miami, FL, United States
Contact: Jessica Hernandez

University of South Florida College of Medicine, FL, United States
Contact: Jessica Shaw

United States, Georgia

Augusta University, GA, United States
Contact: Brandy Quarles

Emory University, GA, United States
Contact: N/A

United States, Idaho

Saint Alphonsus Regional Medical Center, ID, United States
Contact: Research Coordinator

United States, Illinois

Northwestern University, IL, United States
Contact: Emma Schmidt

University of Chicago, IL, United States
Contact: Deekshitha Turaka

United States, Iowa

University of Iowa Hospitals and Clinics, IA, United States
Contact: Emily Anderson

United States, Kansas

University of Kansas Medical Center, KS, United States
Contact: Katheryn Jennens

United States, Kentucky

University of Kentucky, KY, United States
Contact: Sura Al Hilfi

United States, Louisiana

Ochsner Health Systems, LA, United States
Contact: Lisa Edmond

United States, Maryland

Johns Hopkins University, MD, United States
Contact: Kristen Riley

University of Maryland, MD, United States
Contact: Vikram Nambiar

United States, Massachusetts

Beth Israel Deaconess Medical Center, MA, United States
Contact: Teresa Capella

Massachusetts General Hospital, MA, United States
Contact: Suhina Patel

University of Massachusetts Medical School, Worcester, MA, United States
Contact: Catherine Douthwright

Healey Center for ALS at Massachusetts General Hospital, Boston, MA, 02114, United States

United States, Michigan

Henry Ford Health System, MI, United States
Contact: Beverley Duthie

University of Michigan, MI, United States
Contact: Jayna Duell

Spectrum Health, MI, United States
Contact: Sehrish Khan

United States, Minnesota

Essentia Health, MN, United States
Contact: Brent Gavin

Mayo Clinic Rochester, MN, United States
Contact: Brenda Nelson

University of Minnesota Twin Cities ALS Research Consortium, MN, United States
Contact: Valerie Ferment

United States, Missouri

University of Missouri Health Care, MO, United States
Contact: Natalie Taylor

Saint Louis University, MO, United States
Contact: Susan Brown

Washington University School of Medicine, MO, United States
Contact: Rebecca Livigni

United States, Nebraska

Neurology Associates, P.C. Somnos Clinical Research, NE, United States
Contact: Desirae Eschiti

University of Nebraska Medical Center, NE, United States
Contact: Katelyn Hilz

United States, Nevada

Las Vegas Clinic, NV, United States
Contact: Richa Priya Jha

United States, New Hampshire

Dartmouth-Hitchcock Medical Center, NH, United States
Contact: Jessica Kemmet

United States, New Jersey

Hackensack University Medical Center, NJ, United States
Contact: Michelle Williams

United States, New York

Columbia University, NY, United States
Contact: Oneika Blackman

Stony Brook University, NY, United States
Contact: Christine Pol

SUNY Upstate, NY, United States
Contact: Lena Deb

United States, North Carolina

Duke University, NC, United States
Contact: Michelle Ward

Wake Forest University Health Sciences, NC, United States
Contact: Mozhdeh Marandi

United States, Ohio

Cleveland Clinic, OH, United States
Contact: Matt Tainer or

Ohio State University, OH, United States
Contact: Cheyenne Grove

University of Cincinnati, OH, United States
Contact: Ashley Smith

United States, Oregon

Providence Brain and Spine Institute ALS Center, OR, United States
Contact: Ashley Adamo

United States, Pennsylvania

Jefferson Weinberg ALS Center, Thomas Jefferson University, PA, United States
Contact: Daniel Kelly

Lehigh Valley Health Network, PA, United States
Contact: Kevin Stanley

Lewis Katz School of Medicine, Temple University, PA, United States
Contact: Kathleen Hatala

Penn State Hershey, PA, United States
Contact: Michele Hare

University of Pennsylvania, PA, United States
Contact: Adreeja Guharay

University of Pittsburgh Medical Center, PA, United States
Contact: Jaquelynn Jones

United States, Tennessee

Vanderbilt University Medical Center, TN, United States
Contact: Diana Davis

United States, Texas

Houston Methodist, TX, United States
Contact: Anjana Singh

Texas Neurology, TX, United States
Contact: Mohamad Asaad Nasri

University of Texas Health Science Center at San Antonio (UTHSCSA), TX, United States
Contact: Randee Kent-Baron

United States, Utah

University of Utah, UT, United States
Contact: Crystal Neate

United States, Virginia

University of Virginia, VA, United States
Contact: Mary Wagoner

Virginia Commonwealth University, VA, United States
Contact: LaVon Smith

United States, Washington

Swedish Medical Center, WA, United States
Contact: Janae Rahiman

University of Washington, WA, United States
Contact: Kassie Chu

United States, Wisconsin

Medical College of Wisconsin, WI, United States
Contact: Marie Mejaki

United States,

Indiana University, United States
Contact: Sandy Guingrich

Enrollment Criteria

Breathing Ability

Percent lung function (FVC) or (SVC)

≥ 50%

Months Since Onset

Number of months since first symptoms of ALS.

N/A

Non-Invasive Ventilation (NIV)

Can PALS use a BiPAP in the trial?

Yes

Diaphragm Pacer (DPS)

Can PALS use a DPS in the trial?

N/A

Edaravone Usage

Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?

Not Available

Open Label

Yes

Update Notes

Press release that 1st participant dosed

5/30/2023

Updated outcome measures

5/22/2023

Other Information

Purpose

Eligibility

Inclusion Criteria:
- No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).
Exclusion Criteria:
- The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
1. Diagnosis of epilepsy or seizure within 6 months of randomization 2. Hypersensitivity to DNL343 or any of the excipients contained within the DNL343 drug product 3. The concomitant use of prescription or over-the-counter (OTC) medications that are inducers of certain cytochrome P450 enzymes, substrates of certain cytochrome P450 enzymes, or substrates of certain drug transporters.

Details

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683. Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen. If a participant is randomized to Regimen G DNL343, the participant will complete a screening visit to assess additional Regimen G eligibility criteria. Once Regimen G eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active DNL343 or matching placebo. Regimen G will enroll by invitation, as participants may not choose to enroll in Regimen G. Participants must first enroll into the Master Protocol and be eligible to participate in the Master Protocol before being able to be randomly assigned to Regimen G. For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.

Collaborator(s)

Denali Therapeutics

Trial Protocol as Published on Clinicaltrials.gov

NCT05842941 (First Published: 4/24/2023)