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NCT06402123 | RECRUITING | Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)

A Phase 2b Study of Zagociguat in Patients With MELAS
Sponsor:

Tisento Therapeutics

Brief Summary:

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.

Condition or disease

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

Intervention/treatment

zagociguat 15mg

zagociguat 30mg

Placebo

Phase

PHASE2

Detailed Description:

The goal of this clinical trial is to learn about the effectiveness of zagociguat in patients with MELAS. The main questions it aims to answer are: * Does zagociguat improve fatigue in patients with MELAS? * Does zagociguat improve cognitive performance in patients with MELAS? * What is the safety and tolerability profile of zagociguat? The PRIZM study is evaluating 2 dose levels of zagociguat in a crossover design consisting of two 12-week treatment periods separated by a 4-week washout. Patients will be screened and if eligible, randomly assigned either to receive placebo in period 1 followed by active drug in period 2 OR to receive active drug in period 1 followed by placebo in period 2. Study medication is a once daily oral tablet and will be provided at the clinic and/or shipped to the participant's home. Clinic visits will occur at screening and Week 1 and Week 12 of each treatment period. Visits at Week 4 and Week 8 of both crossover periods will either be in clinic or optionally at the participant's home. Study assessments will be conducted weekly on a phone app and a separate tablet and additional assessments will be conducted during visits. Patients who complete the study will be eligible for an open label extension study.

Study Type : INTERVENTIONAL
Estimated Enrollment : 44 participants
Masking : QUADRUPLE
Masking Description : This a double-blind study in which investigators, participants/caregivers, Sponsor, and all study personnel will be blinded.
Primary Purpose : TREATMENT
Official Title : Phase 2b Randomized, Double-blind, Placebo-controlled Crossover Study Evaluating the Efficacy and Safety of Zagociguat in Participants With MELAS (PRIZM)
Actual Study Start Date : 2024-09-10
Estimated Primary Completion Date : 2026-03-01
Estimated Study Completion Date : 2026-03-01

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 18 Years to 75 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
Inclusion 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 the iDSST and GMLT.
    • 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.
    Exclusion Criteria
    • 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.
A Phase 2b Study of Zagociguat in Patients With MELAS

Location Details

NCT06402123

Please Choose a site

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

RECRUITING

United States, California

UC San Diego - Altman Clinical and Translational Research Institute

THE JOLLA, California, United States, 92037

RECRUITING

United States, Colorado

Children's Hospital of Colorado

Aurora, Colorado, United States, 80045

RECRUITING

United States, District of Columbia

Children's National

Washington, District of Columbia, United States, 20010

RECRUITING

United States, Georgia

Rare Disease Research

Atlanta, Georgia, United States, 30329

NOT YET RECRUITING

United States, Maryland

Johns Hopkins University School of Medicine

Baltimore, Maryland, United States, 21287

RECRUITING

United States, Massachusetts

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

RECRUITING

United States, Minnesota

Mayo Clinic Rochester

Rochester, Minnesota, United States, 55905

NOT YET RECRUITING

United States, New York

Columbia University Irving Medical Center

New York, New York, United States, 10027

RECRUITING

United States, New York

Mount Sinai - Ichan School of Medicine

New York, New York, United States, 10029

RECRUITING

United States, Ohio

Akron Children's Hospital

Akron, Ohio, United States, 44308

NOT YET RECRUITING

United States, Pennsylvania

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

RECRUITING

United States, Pennsylvania

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States, 15224

RECRUITING

United States, Texas

Baylor College of Medicine

Houston, Texas, United States, 77030

RECRUITING

United States, Texas

University of Texas Medical School at Houston

Houston, Texas, United States, 77030

NOT YET RECRUITING

Australia, New South Wales

Neuroscience Research Australia

Sydney, New South Wales, Australia, NSW 2031

RECRUITING

Australia, Victoria

Royal Melbourne Hospital

Melbourne, Victoria, Australia, 3052

RECRUITING

Canada, Manitoba

Shared Health/University of Manitoba

Winnipeg, Manitoba, Canada, Raa 1 stack

NOT YET RECRUITING

Canada, Ontario

McMaster University Medical Center

Hamilton, Ontario, Canada, Live Binging

RECRUITING

Germany,

University Hospital Bonn

Bonn, Germany, 53127

NOT YET RECRUITING

Germany,

Ludwig Maximilians-University of Munich

Munich, Germany, 80336

RECRUITING

Italy,

Neurologic Institute Carlo Besta of Milan

Milan, Italy, 20133

NOT YET RECRUITING

Italy,

University of Pisa Neurological Clinic

Pisa, Italy, 56100

NOT YET RECRUITING

Italy,

Agostino Gemelli Polyclinic University Foundation

Roma, Italy, 00168

RECRUITING

United Kingdom,

UCL Queen Square Institute of Neurology

London, United Kingdom, WC1N 3BG

RECRUITING

United Kingdom,

Newcastle University

Newcastle Upon Tyne, United Kingdom, Ne14LP