Ignite Creation Date:
2024-06-16 @ 11:49 AM
Last Modification Date:
2024-10-26 @ 3:30 PM
Study NCT ID:
NCT06431893
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2024-05-29
First Post:
2024-05-08
Brief Title:
A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants 12 to 65 Years of Age with Classical Homocystinuria HCU
Sponsor:
Travere Therapeutics Inc
Organization:
Travere Therapeutics Inc
Study Overview
Official Title:
An Open-Label Phase 3 Long-Term Extension LTE Study to Assess the Safety Tolerability and Efficacy of Treatment with Pegtibatinase in Participants with Classical Homocystinuria HCU Due to Cystathionine Beta Synthase Deficiency ENSEMBLE
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ENSEMBLE
Brief Summary:
The goal of this long-term extension LTE study is to evaluate the safety and efficacy of pegtibatinase in patients with classical homocystinuria HCU Patients who are active in the Phase 12 COMPOSE study or those who complete the 24 weeks of treatment in the Phase 3 HARMONY are eligible to participate
Participants will be in this clinical study for up to about 13 months including
a treatment period of up to 52 weeks
a 4-week safety follow-up period
Participants will be in this clinical study for up to about 13 months including
a treatment period of up to 52 weeks
a 4-week safety follow-up period
Detailed Description:
Overall Design
This is a global multicenter single-arm open-label study Participants who meet all eligibility criteria may transition from other pegtibatinase studies and enroll into this LTE study for long-term safety efficacy and clinical assessments of pegtibatinase treatment
All participants will follow a protocol of self-administration of pegtibatinase unless exempted During the ENSEMBLE study an optional protein tolerance modification PTM sub-study will also be conducted for eligible participants
This is a global multicenter single-arm open-label study Participants who meet all eligibility criteria may transition from other pegtibatinase studies and enroll into this LTE study for long-term safety efficacy and clinical assessments of pegtibatinase treatment
All participants will follow a protocol of self-administration of pegtibatinase unless exempted During the ENSEMBLE study an optional protein tolerance modification PTM sub-study will also be conducted for eligible participants
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None