Phase 2b randomized, double-blind, placebo-controlled crossover study evaluating the efficacy and safety of zagociguat in participants with MELAS (PRIZM)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-515389-15-00

Acronym

TIS6463-203

Enrollment

Registered

2024-10-16

Start date

2024-12-13

Completion date

Unknown

Last updated

2025-10-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

and stroke-like episodes (MELAS), lactic acidosis, Mitochondrial encephalomyopathy

Brief summary

For Week 9 through 12 • PROMIS Fatigue MELAS Short Form (PFM-SF) scores • Groton Maze Learning Test (GMLT) scores • International Digit Symbol Substitution Test (iDSST) scores These 3 endpoints (with weights of 0.4, 0.3, and 0.3, respectively) will be combined using a global statistical test (GST), Incidence of treatment-emergent adverse events (TEAEs) from study drug initiation through the Follow-up Visit or end of treatment if a participant continues to the open-label extension study.

Detailed description

Number of repetitions completed during the 30-second sit-to-stand test at Week 12, PROMIS Cognitive Function MELAS Short Form (PCFM-SF) score for Week 12, Concentrations of GDF-15 at Week 12, Memory composite scores (One Card Learning and One Back Tests) for Week 9 through 12

Interventions

DRUGPlacebo-to-match (ptm) zagociguat 7.5mg tablets

DRUGPlacebo-to-match (ptm) zagociguat 15mg tablets

DRUGzagociguat 15 mg

DRUGzagociguat 7.5 mg

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
For Week 9 through 12 • PROMIS Fatigue MELAS Short Form (PFM-SF) scores • Groton Maze Learning Test (GMLT) scores • International Digit Symbol Substitution Test (iDSST) scores These 3 endpoints (with weights of 0.4, 0.3, and 0.3, respectively) will be combined using a global statistical test (GST), Incidence of treatment-emergent adverse events (TEAEs) from study drug initiation through the Follow-up Visit or end of treatment if a participant continues to the open-label extension study.	—

Measure

Time frame

—

Secondary

Measure	Time frame
Number of repetitions completed during the 30-second sit-to-stand test at Week 12, PROMIS Cognitive Function MELAS Short Form (PCFM-SF) score for Week 12, Concentrations of GDF-15 at Week 12, Memory composite scores (One Card Learning and One Back Tests) for Week 9 through 12	—

Countries

Germany, Italy

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026