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A Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum (LSS)

NCT06990984 · Status: NOT_YET_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2025-07-11

No results posted yet for this study

Summary

The goal of this clinical trial is to investigate oral TTI-0102 to evaluate its pharmacokinetics, safety, and preliminary efficacy in patients with Leigh syndrome spectrum, in three sequences. First sequence will be placebo-controlled and include 12 adolescents and adults weighing more than 40 kg. Second sequence will be open-label and include 3 adults and 3 children weighing 25-40 kg. Third sequence will also be open-label and will include 3 children weighing 20-25 kg. The main questions clinical trial aims to answer are:

* Characterize the relationship between TTI-0102 drug levels by measuring

1. cysteamine pharmacokinetic (PK) parameters
2. pharmacodynamic (PD) efficacy measures
* Generate a population PK model and determine dose level for later dosing groups
* collect data to inform the design of a subsequent phase 2b/3 study of TTI-0102 in this patient population

Participants will be asked to:

* Take the study drug twice daily for 12 weeks
* Visit the clinic for checkups and testing 7 times in total: once prior to study drug dosing, then 5 times over the 12-week treatment period and one more time a month after the last study drug dose.

Conditions

  • Leigh Syndrome

Interventions

DRUG

TTI-0102: cysteamine-pantetheine disulfide

TTI-0102: cysteamine-pantetheine disulfide, powder for dissolution in water for oral administration

DRUG

D-Mannitol

D-Mannitol

Sponsors & Collaborators

  • Thiogenesis Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Zarazuela Zolkipli-Cunningham, MBChB, MRCP · Children's Hospital of Philadelphia

  • Patrice Rioux, MD, PhD · Thiogenesis Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
5 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-10-31
Primary Completion
2026-09-30
Completion
2026-09-30
FDA Drug
Yes

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06990984 on ClinicalTrials.gov