A Phase 2b Study of Zagociguat in Patients with MELAS

Description

PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period, crossover study evaluating the efficacy and safety of oral zagociguat 15 and 30 mg vs. placebo when administered daily for 12 weeks in participants with genetically and phenotypically defined MELAS.

Conditions

Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)

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Study Overview

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Study Details

Study overview

PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period, crossover study evaluating the efficacy and safety of oral zagociguat 15 and 30 mg vs. placebo when administered daily for 12 weeks in participants with genetically and phenotypically defined MELAS.

Phase 2b Randomized, Double-blind, Placebo-controlled Crossover Study Evaluating the Efficacy and Safety of Zagociguat in Participants with MELAS (PRIZM)

A Phase 2b Study of Zagociguat in Patients with MELAS

Condition
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
Intervention / Treatment

-

Contacts and Locations

La Jolla

UC San Diego - Altman Clinical and Translational Research Institute, La Jolla, California, United States, 92037

Aurora

Children's Hospital of Colorado, Aurora, Colorado, United States, 80045

Washington

Children's National, Washington, District of Columbia, United States, 20010

Atlanta

Rare Disease Research, Atlanta, Georgia, United States, 30329

Baltimore

Johns Hopkins University School of Medicine, Baltimore, Maryland, United States, 21287

Boston

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

Rochester

Mayo Clinic Rochester, Rochester, Minnesota, United States, 55905

New York

Columbia University Irving Medical Center, New York, New York, United States, 10027

New York

Mount Sinai - Ichan School of Medicine, New York, New York, United States, 10029

Akron

Akron Children's Hospital, Akron, Ohio, United States, 44308

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

  • 1. Signed consent form.
  • 2. 18 to 75 years of age.
  • 3. Diagnosed with MELAS based on the presence of each of the following criteria:
  • 1. A documented pathogenic variant in a mitochondrial DNA (mtDNA) gene.
  • 2. History of one or more stroke-like episodes (SLEs) with magnetic resonance imaging (MRI) findings consistent with stroke-like lesions.
  • 4. Scores below normal average on composite memory score from One Back and One Card Learning tests.
  • 5. Reports fatigue due to MELAS.
  • 6. Can complete at least 1 sit-to-stand in the 30-second test interval.
  • 7. Completes all at-home weekly activities independently during the Screening Period (caregiver may help set up device/app, log-in, etc.).
  • 8. Other criteria per the protocol.
  • 1. Systolic blood pressure (BP) 90 mmHg or diastolic BP 60 mmHg.
  • 2. Orthostatic hypotension when measured after standing from a semi-recumbent/supine position.
  • 3. Active cancer significant enough to confound the results of this study.
  • 4. Severe gastrointestinal dysmotility that may impact participation.
  • 5. Recent history (within last 6 months) of platelet dysfunction, hemophilia, von Willebrand disease, coagulation disorder, other bleeding diathesis condition(s), or significant, nontraumatic bleeding episodes.
  • 6. History of spontaneous fracture(s) that in the investigator's opinion represents a safety risk for trial participation.
  • 7. Current use of prohibited medication (reviewed by investigator).
  • 8. Any medical or other condition that the investigator thinks would preclude study participation.
  • 9. Other exclusion criteria per protocol.

Ages Eligible for Study

18 Years to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Tisento Therapeutics,

Study Record Dates

2025-09-01