Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
Conditions
Keywords
MELAS
Brief summary
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
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.
Interventions
DRUGzagociguat 15mg
once daily oral tablets
Sponsors
Tisento Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
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
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment-emergent Adverse Events (TEAEs) | Day 1 through the Follow-up for treatment period 4 weeks after last dose of study medication. | TEAEs are any untoward event that may or may not be related to study medication. |
Countries
Australia, Germany, Italy, United Kingdom, United States
Outcome results
None listed