An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)

Conditions

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

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Interventions

• zagociguat 15mg — DRUG
  once daily oral tablets

Study Details

The goal of this clinical trial is to evaluate the long-term safety and tolerability of zagociguat in patients with MELAS who completed study medication treatment in the lead-in study TIS6463-203. TIS6463-204 is evaluating zagociguat in an open-label extension study at the daily dose of 15mg. The study medication is a once daily oral table and will be provided at the clinic and/or shipped to the participant's home. Study assessments will be conducted during clinic visits which will occur at three months and then at six months. Thereafter, clinic visits will occur every six months.

Key Dates

Start date

Aug 18, 2025

Status verified

Mar 2026

Primary completion

Jul 31, 2028

Completion

Jul 31, 2028

Study Design

Enrollment

44 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Zagociguat 15mg
  Participants receive zagociguat 15 mg once a day (QD) for the duration of the study.

Primary Outcome Measure

Incidence of Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Day 1 through the Follow-up for treatment period 4 weeks after last dose of study medication. ]

Locations (9)

Find similar trials in La Jolla, CA