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New hope for MELAS: experimental drug tested for Long-Term safety

NCT ID NCT06961344

Disease control Sponsor: Tisento Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 08, 2026 · Updated 2 times

Summary

This study is testing the long-term safety of a daily oral drug called zagociguat in 44 adults with MELAS, a rare genetic disease that affects energy production in cells. All participants previously completed a lead-in study of the same drug. Researchers will monitor side effects over several years, with clinic visits every three to six months.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
zagociguat (15 mg oral tablet once daily)
What this could lead to
If successful, this study could show that zagociguat is safe enough for long-term use in MELAS, potentially leading to a treatment that helps manage this rare mitochondrial disease.
What could go wrong
This is a small, open-label extension study with no placebo group, so results are preliminary. It only includes people who already tolerated the drug in a prior trial, which may not reflect broader patient experiences.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 44 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2025

Expected to finish

Jul 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

lactic acidosis MELAS syndrome Mitochondrial encephalopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Mitochondrial encephalopathy, lactic acidosis and stroke-like episodes (melas syndrome)

Contacts and locations

Locations

  • Akron Children's Hospital

    Akron, Ohio, 44308, United States

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Children's Hospital of Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Fondazione Policlinico Universitario Agostino Gemelli

    Rome, 00168, Italy

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

    Munich, 80336, Germany

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Mount Sinai - Ichan School of Medicine

    New York, New York, 10029, United States

  • Neurologic Institute Carlo Besta of Milan

    Milan, 20133, Italy

  • Neuroscience Research Australia

    Sydney, New South Wales, NSW 2031, Australia

  • Rare Disease Research

    Atlanta, Georgia, 30329, United States

  • Royal Melbourne Hospital

    Melbourne, Victoria, 3052, Australia

  • The Newcastle upon Tyne NHS Foundation Trust

    Newcastle upon Tyne, NE14LP, United Kingdom

  • UC San Diego - Altman Clinical and Translational Research Institute

    La Jolla, California, 92037, United States

  • UT Health Houston

    Houston, Texas, 77030, United States

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

    London, WC1N 3BG, United Kingdom

More trials for these conditions

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