A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

NCT06247085 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: TVTX-TVT058-301
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standard of care who have not achieved tHcy target levels. Study details include:

• Total Study duration: up to 38 weeks
• Screening:
• Initial Screening duration: up to 4 weeks
• Pre-treatment Diet Standardization Period duration: up to 6 weeks
• Blinded Treatment Duration: 24 weeks
• 2-week blinded dose titration period
• 22-week blinded assessment period
• Safety Follow-Up: 4 weeks after last dose (as applicable for those not enrolling in the long term extension study, ENSEMBLE)

Conditions

Homocystinuria

Keywords

HCU; cystathionine beta synthase deficiency; Classical Homocystinuria

Outcome Measures

Primary Outcomes (1)

• Change from baseline in plasma tHcy levels

  The change from baseline in plasma tHcy levels averaged over Weeks 6 through 12 in participants receiving standard of care and treated with pegtibatinase as compared to participants receiving standard of care and placebo

  Weeks 6 - 12

Secondary Outcomes (1)

• Plasma tHcy levels

  Weeks 16 - 24

Study Arms (2)

pegtibatinase

EXPERIMENTAL

Drug: Pegtibatinase

placebo

PLACEBO COMPARATOR

Other: Placebo

Interventions

Pegtibatinase DRUG

Full Target Dose of pegtibatinase BIW

Also known as: OT-058, TVT-058

pegtibatinase

Placebo OTHER

volume-matched saline SC BIW

placebo

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Must be ≥12 to ≤65 years of age, at the time of signing the informed consent
• Must have a diagnosis of classical HCU based on clinical, biochemical, and/or molecular genetic testing
• Plasma tHcy ≥80 µM at Screening visit, with allowance for up to 18 participants who may be enrolled with a Screening plasma tHcy ≥50 to \<80 µM
• Willing to maintain a generally stable diet for the duration of the study (unless changes are required based on medical/safety reasons)
• Willing to maintain generally stable intake and doses of betaine, pyridoxine, and Met free formula for the duration of the study (unless changes are required based on medical/safety reasons)

You may not qualify if:

• Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) deficiency, or disorder of cobalamin metabolism
• Concurrent disease or condition (eg, history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurological, oncologic, or psychiatric disease) that would interfere with study participation or safety (excluding complications of HCU).
• History of major thrombotic event (eg, cerebrovascular accident, myocardial infarction, pulmonary embolism) in the previous 6 months.
• Body weight ≥160 kg.
• Use or planned use of any injectable drugs containing PEG (excluding PEG-containing vaccines)
• Any previous exposure to pegtibatinase and/or previous participation in a clinical study that included administration of pegtibatinase or pegtarviliase
• Prior severe immune reaction to a PEG-containing product

Sponsors & Collaborators

• Travere Therapeutics, Inc.: lead

Study Sites (1)

Travere Investigational Site - Virtual Site

Morrisville, North Carolina, 27560, United States

Location

MeSH Terms

Conditions

Homocystinuria

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hyperhomocysteinemia; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Michael Imperiale, MD

  Travere Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 30, 2024
• First Posted: February 7, 2024
• Study Start: December 28, 2023
• Primary Completion: December 1, 2025
• Study Completion: January 1, 2026
• Last Updated: April 9, 2025

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will share
• Time Frame: Requests can be made following completion of the study and full publication of the study data in a peer reviewed journal for up to 36 months following its publication.
• Access Criteria: Requires submission and approval of intended use and a data sharing agreement.

Locations

US (1)