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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 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 70

Last updated 2026-05-08

No results posted yet for this study

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. 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

Interventions

DRUG

Pegtibatinase

Pegtibatinase BIW

OTHER

Placebo

volume-matched saline SC BIW

Sponsors & Collaborators

  • Travere Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Michael Imperiale, MD · Travere Therapeutics, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
12 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-12-28
Primary Completion
2027-08-31
Completion
2027-09-30
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • France
  • Germany
  • Ireland
  • Italy
  • Poland
  • Portugal
  • Qatar
  • Saudi Arabia
  • Spain
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

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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 NCT06247085 on ClinicalTrials.gov