ACTIVE_NOT_RECRUITING

A Phase 2b Study of Zagociguat in Patients With MELAS

Conditions

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

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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

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.

Official Title

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

Quick Facts

Study Start:2024-09-10

Study Completion:2026-06-20

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT06402123

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:18 Years to 75 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	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.

Inclusion Criteria

Exclusion 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.

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.

Contacts and Locations

Study Locations (Sites)

UC San Diego - Altman Clinical and Translational Research Institute

La Jolla, California, 92037

United States

Children's Hospital of Colorado

Aurora, Colorado, 80045

United States

Rare Disease Research

Atlanta, Georgia, 30329

United States

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21287

United States

Massachusetts General Hospital

Boston, Massachusetts, 02114

United States

Mayo Clinic Rochester

Rochester, Minnesota, 55905

United States

Columbia University Irving Medical Center

New York, New York, 10027

United States

Mount Sinai - Ichan School of Medicine

New York, New York, 10029

United States

Akron Children's Hospital

Akron, Ohio, 44308

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

United States

Baylor College of Medicine

Houston, Texas, 77030

United States

University of Texas Medical School at Houston

Houston, Texas, 77030

United States

Collaborators and Investigators

Sponsor: Tisento Therapeutics

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 Date2024-09-10

Study Completion Date2026-06-20

Study Record Updates

Study Start Date2024-09-10

Study Completion Date2026-06-20

Terms related to this study

Keywords Provided by Researchers

MELAS

Additional Relevant MeSH Terms

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