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Enzyme Replacement Therapy

pegtibatinase for Homocystinuria (HARMONY Trial)

Phase 3

Recruiting

Research Sponsored by Travere Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Must have a diagnosis of classical HCU based on clinical, biochemical, and/or molecular genetic testing

Must be ≥12 to ≤65 years of age, at the time of signing the informed consent

Timeline

Screening 3 weeks

Treatment Varies

Follow Up weeks 16 - 24

Awards & highlights

HARMONY Trial Summary

This trial will test the effectiveness and safety of pegtibatinase compared to a placebo in individuals with classical HCU who have not reached target tHcy levels despite standard care. The study will

Who is the study for?

This trial is for people aged 12 to 65 with classical Homocystinuria (HCU) due to a specific enzyme deficiency, who are on standard treatments but haven't hit their target homocysteine levels. Participants must be stable on current treatment and able to follow the study's diet requirements.Check my eligibility

What is being tested?

The trial tests Pegtibatinase against a placebo in HCU patients. It lasts up to 38 weeks, including screening, diet standardization before treatment, a blinded treatment phase of 24 weeks with dose adjustments and evaluations, followed by safety monitoring.See study design

What are the potential side effects?

While the side effects of Pegtibatinase aren't detailed here, typical drug-related side effects could include allergic reactions, injection site discomfort, nausea or headaches. The exact risks will be explained by the research team.

HARMONY Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with classical homocystinuria.

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I am between 12 and 65 years old.

HARMONY Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ weeks 16 - 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~weeks 16 - 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and weeks 16 - 24 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from baseline in plasma tHcy levels

Secondary outcome measures

Plasma tHcy levels

HARMONY Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: pegtibatinaseExperimental Treatment1 Intervention

Group II: placeboPlacebo Group1 Intervention

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Travere Therapeutics, Inc.Lead Sponsor

20 Previous Clinical Trials

177,566 Total Patients Enrolled

2 Trials studying Homocystinuria

182 Patients Enrolled for Homocystinuria

Sergio Slawka, MD,MPH,GFMDStudy DirectorTravere Therapeutics, Inc.

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is it possible for me to enroll as a participant in this clinical trial?

"To qualify for participation in this scientific investigation, individuals should have homocystinuria and fall within the age range of 12 to 65 years. A total of 70 candidates will be enrolled in the research study."

Answered by AI

Are there ongoing efforts to actively enroll participants in this clinical trial?

"Per the information available on clinicaltrials.gov, this investigation is currently enrolling participants. The trial was first made public on December 28th, 2023, and its most recent update was on January 30th, 2024."

Answered by AI

What is the safety profile of pegtibatinase in individuals?

"Our assessments at Power indicate a safety rating of 3 for pegtibatinase due to the Phase 3 nature of this trial, symbolizing both initial efficacy data and repeated safety evaluations."

Answered by AI

How large is the patient population participating in this medical study?

"Yes, the data available on clinicaltrials.gov suggests that this trial is actively seeking volunteers. The study was first shared on December 28th, 2023 and last revised on January 30th, 2024. A total of 70 participants are sought at a single research site."

Answered by AI

Is the clinical trial inclusive of individuals below 20 years old?

"Patients eligible for participation in this scientific investigation must fall within the age bracket of 12 to 65 years. Of note, there are separate trials available for individuals under 18 and those above 65."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2023. "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". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06247085.

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