A Phase 2b Study of Zagociguat in Patients With MELAS (NCT06402123) | Clinical Trial Compass
Active — Not RecruitingPhase 2
A Phase 2b Study of Zagociguat in Patients With MELAS
United States, Australia, Canada43 participantsStarted 2024-09-10
Plain-language 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.
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Signed consent form.
. 18 to 75 years of age.
. Diagnosed with MELAS based on the presence of each of the following criteria:
. A documented pathogenic variant in a mitochondrial DNA (mtDNA) gene.
. History of one or more stroke-like episodes (SLEs) with magnetic resonance imaging (MRI) findings consistent with stroke-like lesions.
. Scores below normal average on the iDSST and GMLT.
. Reports fatigue due to MELAS.
. Can complete at least 1 sit-to-stand in the 30-second test interval.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
• PROMIS Fatigue MELAS Short Form scores• Groton Maze Learning Test scores • International Digit Symbol Substitution Test scores
Timeframe: Weeks 9 through 12 of each treatment period
2
Incidence of Treatment-emergent Adverse Events (TEAEs)
Timeframe: Weeks 1 through washout for treatment period 1 and Weeks 1 through follow-up for treatment period 2
. Systolic blood pressure (BP) 90 mmHg or diastolic BP 60 mmHg.
. Orthostatic hypotension when measured after standing from a semi-recumbent/supine position.
. Active cancer significant enough to confound the results of this study.
. Severe gastrointestinal dysmotility that may impact participation.
. 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.
. History of spontaneous fracture(s) that in the investigator's opinion represents a safety risk for trial participation.
. Current use of prohibited medication (reviewed by investigator).
. Any medical or other condition that the investigator thinks would preclude study participation.