Pegtibatinase for Homocystinuria

(ENSEMBLE Trial)

Recruiting at 4 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Travere Therapeutics, Inc.

Disqualifiers: Serious AE, Intolerance, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 104 weeks

100 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is Pegtibatinase safe for humans?

Research on PEGylated cystathionine β-synthase (a form of enzyme replacement therapy similar to Pegtibatinase) in mice showed improved liver symptoms and increased survival, suggesting it may be safe, but human safety data is not provided. ^{1 2 3 4 5}

How is the drug Pegtibatinase different from other treatments for homocystinuria?

Pegtibatinase is unique because it is a PEGylated enzyme replacement therapy designed to act as a metabolic sink, reducing high levels of homocysteine in the body, which is not effectively addressed by current treatments. This approach involves modifying the enzyme to improve its stability and effectiveness, offering a novel way to manage homocystinuria. ^{6 7 8 9 10}

What is the purpose of this trial?

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 1/2 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 104 weeks * a 4-week safety follow-up period

Research Team

Michael Imperiale, MD

Principal Investigator

Travere Therapeutics, Inc.

Eligibility Criteria

This trial is for people aged 12 to 65 with classical Homocystinuria (HCU) who are currently in the COMPOSE study or have finished the HARMONY study's initial 24 weeks. Participants will be involved for roughly 13 months, including a treatment phase of up to one year and a safety follow-up of four weeks.

Inclusion Criteria

Participants active in the Phase 1/2 COMPOSE study or who completed the 24-week blinded period in the Phase 3 HARMONY study

Exclusion Criteria

I was removed from a previous pegtibatinase study due to a severe side effect or intolerance.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive pegtibatinase treatment for long-term safety and efficacy assessment

104 weeks

Self-administration with regular clinical assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

Treatment Details

Interventions

• Pegtibatinase

Trial Overview The trial is testing Pegtibatinase's long-term effects on patients with HCU. It aims to understand how safe and effective this treatment is over an extended period when given weekly.

Participant Groups

Group I: pegtibatinase Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Travere Therapeutics, Inc.

Lead Sponsor

Trials

23

Recruited

103,000+

References

1. Canada pubmed.ncbi.nlm.nih.gov

Current and Novel Therapeutical Approaches of Classical Homocystinuria in Childhood With Special Focus on Enzyme Replacement Therapy, Liver-Directed Therapy and Gene Therapy. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Enzyme replacement with PEGylated cystathionine β-synthase ameliorates homocystinuria in murine model. [2018]

3. Netherlands pubmed.ncbi.nlm.nih.gov

Screening of homocysteine from newborn blood spots by high-performance liquid chromatography with coulometric array detection. [2019]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Diagnosis of Classic Homocystinuria in Two Boys Presenting with Acute Cerebral Venous Thrombosis and Neurologic Dysfunction after Normal Newborn Screening. [2021]

5. France pubmed.ncbi.nlm.nih.gov

[Homocystinuria in adulthood]. [2016]

6. Netherlands pubmed.ncbi.nlm.nih.gov

Pharmacokinetics and pharmacodynamics of PEGylated truncated human cystathionine beta-synthase for treatment of homocystinuria. [2019]

7. United States pubmed.ncbi.nlm.nih.gov

Engineering and Characterization of an Enzyme Replacement Therapy for Classical Homocystinuria. [2019]

8. United States pubmed.ncbi.nlm.nih.gov

Nutrition status of adults with phenylketonuria treated with pegvaliase. [2022]

9. United States pubmed.ncbi.nlm.nih.gov

Case-control study about the acceptance of Pegvaliase in Phenylketonuria. [2022]

10. United States pubmed.ncbi.nlm.nih.gov

Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial. [2022]