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

Tisento Therapeutics

Status and phase: Begins enrollment in 1 month; Phase 2

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

Treatments: Drug: zagociguat 15mg

Study type: Interventional

Funder types: Industry

Identifiers

NCT06961344

TIS6463-204

Details and patient eligibility

About

The goal of this clinical trial is to evaluate the long-term safety and tolerability of zagociguat in patients with MELAS who completed 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.

Full description

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.

Enrollment: 44 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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44 participants in 1 patient group

Zagociguat 15mg

Experimental group

Description:

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

Treatment:

Drug: zagociguat 15mg