Ignite Creation Date:
2024-10-26 @ 3:43 PM
Last Modification Date:
2024-10-26 @ 3:43 PM
Study NCT ID:
NCT06644534
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-09-29
Brief Title:
A Study to Assess TTI-0102 vs Placebo in MELAS Patients
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Multi-Center Randomized Double-Blind Placebo-Controlled Study to Assess the Efficacy Safety Tolerability Pharmacokinetics and Pharmacodynamics of Oral TTI-0102 for Treatment of Patients With Mitochondrial Encephalomyopathy Lactic Acidosis and Stroke-like Episodes MELAS
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a randomized double-blind placebo-controlled study Prior to treatment patients will undergo a screening visit If eligible each subject will return for a Day 1 visit and will receive their first dose of investigational product TTI-0102 or placebo At the end of the first week of treatment subjects will return for a Week 1Day 8 study visit to assess study drug dosingtolerance and instruct on dosing for the upcoming second week of treatment
For the first 8 weeks of treatment subjects will alternate between returning to the clinic for detailed assessments Weeks 4 and 8 and receiving a telephone call from the Investigator team to assess safety and TTI-0102 dose Weeks 2 and 6 and the potential need for an immediate unscheduled study visit
After the first 8 weeks of treatment subjects will continue to return to the clinic for monthly assessments at Weeks 12 16 20 The Study Exit visit will occur at Week 24 and subjects will be offered to continue on an open-label extension study of TTI-0102 If a subject does not complete the study they will be asked to return for a Study Exit visit 4 weeks after last study drug dose
Primary Objective The primary objective of this study is to assess the efficacy safety and tolerability of oral TTI 0102 compared to placebo for up to 6 months in patients with MELAS
Secondary Objective The secondary objectives of this study are to assess the efficacy pharmacokinetics PK and pharmacodynamics PD of cysteamine after oral administration of TTI-0102 at steady state in patients with MELAS on a stable dose of TTI-0102
This is a randomized double-blind placebo-controlled study Prior to treatment patients will undergo a screening visit If eligible each subject will return for a Day 1 visit and will receive their first dose of investigational product TTI-0102 or placebo At the end of the first week of treatment subjects will return for a Week 1Day 8 study visit to assess study drug dosingtolerance and instruct on dosing for the upcoming second week of treatment
For the first 8 weeks of treatment subjects will alternate between returning to the clinic for detailed assessments Weeks 4 and 8 and receiving a telephone call from the Investigator team to assess safety and TTI-0102 dose Weeks 2 and 6 and the potential need for an immediate unscheduled study visit
After the first 8 weeks of treatment subjects will continue to return to the clinic for monthly assessments at Weeks 12 16 20 The Study Exit visit will occur at Week 24 and subjects will be offered to continue on an open-label extension study of TTI-0102 If a subject does not complete the study they will be asked to return for a Study Exit visit 4 weeks after last study drug dose
Study Drug Dosing To prevent any manifestation of intolerance at the initiation of drug treatment only half a dose 275 grams will be given once a day for the first week of treatment During the following weeks of treatment patients will be given a full dose of 55 grams once a day
Interim Data Review After nine 9 patients have completed three months of treatment the Week 12 visit an interim data cut will take place to assess safety and potential efficacy signals Even if no indications of efficacy are detected at this early stage the trial itself will not be terminated unless there is a serious safety concern ie protocol-defined Stopping Criteria are met
For the first 8 weeks of treatment subjects will alternate between returning to the clinic for detailed assessments Weeks 4 and 8 and receiving a telephone call from the Investigator team to assess safety and TTI-0102 dose Weeks 2 and 6 and the potential need for an immediate unscheduled study visit
After the first 8 weeks of treatment subjects will continue to return to the clinic for monthly assessments at Weeks 12 16 20 The Study Exit visit will occur at Week 24 and subjects will be offered to continue on an open-label extension study of TTI-0102 If a subject does not complete the study they will be asked to return for a Study Exit visit 4 weeks after last study drug dose
Primary Objective The primary objective of this study is to assess the efficacy safety and tolerability of oral TTI 0102 compared to placebo for up to 6 months in patients with MELAS
Secondary Objective The secondary objectives of this study are to assess the efficacy pharmacokinetics PK and pharmacodynamics PD of cysteamine after oral administration of TTI-0102 at steady state in patients with MELAS on a stable dose of TTI-0102
This is a randomized double-blind placebo-controlled study Prior to treatment patients will undergo a screening visit If eligible each subject will return for a Day 1 visit and will receive their first dose of investigational product TTI-0102 or placebo At the end of the first week of treatment subjects will return for a Week 1Day 8 study visit to assess study drug dosingtolerance and instruct on dosing for the upcoming second week of treatment
For the first 8 weeks of treatment subjects will alternate between returning to the clinic for detailed assessments Weeks 4 and 8 and receiving a telephone call from the Investigator team to assess safety and TTI-0102 dose Weeks 2 and 6 and the potential need for an immediate unscheduled study visit
After the first 8 weeks of treatment subjects will continue to return to the clinic for monthly assessments at Weeks 12 16 20 The Study Exit visit will occur at Week 24 and subjects will be offered to continue on an open-label extension study of TTI-0102 If a subject does not complete the study they will be asked to return for a Study Exit visit 4 weeks after last study drug dose
Study Drug Dosing To prevent any manifestation of intolerance at the initiation of drug treatment only half a dose 275 grams will be given once a day for the first week of treatment During the following weeks of treatment patients will be given a full dose of 55 grams once a day
Interim Data Review After nine 9 patients have completed three months of treatment the Week 12 visit an interim data cut will take place to assess safety and potential efficacy signals Even if no indications of efficacy are detected at this early stage the trial itself will not be terminated unless there is a serious safety concern ie protocol-defined Stopping Criteria are met
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None