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

NCT ID: NCT06961344

Last Updated: 2025-12-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE2

Total Enrollment

44 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-08-18

Study Completion Date

2028-07-31

Brief Summary

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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.

Detailed Description

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On Screening Visit Day -1 (which corresponds to Period 2 Week 12 Visit from lead-in study TIS6463-203), participants will sign the informed consent form, be confirmed eligible for this open-label extension study and undergo Screening Visit assessments. Eligible participants will begin dosing the next day on Day 1 (there is no Day 0) at home. Participants will have a 3-month visit and 6-month study visit at the clinic and thereafter will have a clinic visit every 6 months. Participants should have a Follow-up Visit 4 (+1) weeks after zagociguat discontinuation.

Conditions

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

Keywords

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MELAS

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

An open-label, single-group assignment extension study.

Eligible patients will be entered into a single treatment sequence. The treatment period will last for the shortest of the following: 104 weeks; regulatory approval and commercial availability of zagociguat in the participant's respective country; or termination of the clinical development of zagociguat in MELAS
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Zagociguat 15mg

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

Group Type EXPERIMENTAL

zagociguat 15mg

Intervention Type DRUG

once daily oral tablets

Interventions

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zagociguat 15mg

once daily oral tablets

Intervention Type DRUG

Other Intervention Names

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IW-6463 CY6463

Eligibility Criteria

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Inclusion Criteria

1. Signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form (ICF).
2. Completed TIS6463-203 treatment through the Period 2 Week 12 Visit.
3. Agrees to follow lifestyle restrictions.
4. Other criteria per the protocol.

Exclusion Criteria

1\. Any medical condition or clinical finding that, per investigator judgement, would preclude safe study participation and/or completion of all trial requirements.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Tisento Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UC San Diego - Altman Clinical and Translational Research Institute

La Jolla, California, United States

Site Status

Children's Hospital of Colorado

Aurora, Colorado, United States

Site Status

Rare Disease Research

Atlanta, Georgia, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Mount Sinai - Ichan School of Medicine

New York, New York, United States

Site Status

Akron Children's Hospital

Akron, Ohio, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

Royal Melbourne Hospital

Melbourne, Victoria, Australia

Site Status

LMU Klinikum, Friedrich-Baur-Institute, Department of Neurology

Munich, , Germany

Site Status

University College London Hospital. The National Hospital for Neurology and Neurosurgery, Centre for Neuromuscular Diseases

London, , United Kingdom

Site Status

The Newcastle upon Tyne NHS Foundation Trust

Newcastle upon Tyne, , United Kingdom

Site Status

Countries

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United States Australia Germany United Kingdom

Other Identifiers

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TIS6463-204

Identifier Type: -

Identifier Source: org_study_id