New gene therapy targets Parkinson's in patients with specific gene flaw

NCT ID NCT04127578

Ongoing Disease control Sponsor: Prevail Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 17, 2026 · Updated 2 times

Summary

This study tests a new gene therapy called LY3884961 in 32 people with moderate to severe Parkinson's disease who have a specific genetic change (GBA1 mutation). The treatment is given as a single injection into the fluid around the brain and spinal cord. The main goal is to check safety and see if it can improve symptoms, but it is not expected to cure the disease. Participants will be followed for 5 years.

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Conditions

The condition(s) this trial relates to.

Gaucher disease Parkinson disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PARKINSON DISEASE

Contacts and locations

Locations

Esperanza Clinical, 25220 Hancock Avenue

Murrieta, California, 92562, United States
Hadassah Medical Center, Hadassah Ein Kerem, Dept. of Neurology, P.O. Box 12000

Jerusalem, 91120, Israel
Hospital of the University of Pennsylvania, 330 S. 9th Street

Philadelphia, Pennsylvania, 19107-6103, United States
Joan and Sanford I. Weill Department of Medicine, 525 E 68th Street

New York, New York, 10065, United States
K2 Medical Research, 101 Southhall Lane, Suite 150

Maitland, Florida, 32751-5669, United States
Mayo Clinic, 13400 E. Shea Boulevard

Scottsdale, Arizona, 85259, United States
Mayo Clinic, 200 First Street SW

Rochester, Minnesota, 55905, United States
Mount Sinai Beth Israel, 10 Union Square East, Suite 5H

New York, New York, 10003, United States
Northwestern University Feinberg School of Medicine, Dept. of Neurology, Parkinson's Disease & Movement Disorders Center, 710 N. Lake Shore Drive, 11th Floor

Chicago, Illinois, 60611, United States
PPD, 100 West Gore Street, Suite 202

Orlando, Florida, 32806, United States
Rocky Mountain Clinical Research - CenExel - PPDS, 701 East Hampden Avenue Suite 510

Englewood, Colorado, 80113-2776, United States
Shaare Zedek Medical Center, 12 Shmuel Biet Street

Jerusalem, 91031, Israel
Sheba Medical Center, Tel Hashomer

Ramat Gan, 52621, Israel
Tel Aviv Sourasky Medical Center, 6 Weizmann St.

Tel Aviv, 64239, Israel
Vanderbilt University Medical Center, 1500 21st Avenue South Suite 2600

Nashville, Tennessee, 37212, United States

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