HARMONY Study for Patients With HCU
Now Enrolling
Exploring a Phase 3 Study for People With Classical Homocystinuria (HCU)
Study Drug:
Pegtibatinase
Age Group:
12 - 65 years oold
Study Duration:
About 9 months total
Visits:
One full-day on-site visit, followed by a next-day follow-up on-site visit Additional remote visits through Week 24, scheduled every 2–4 weeks
Location:
United States, Europe, the Middle East, Asia Pacific, and South America
Compensation
Payment for time and travel may be available to participants and their caregivers
Study Overview
The HARMONY Study is a Phase 3 clinical trial designed to learn more about classical homocystinuria (HCU) and evaluate how safe and effective pegtibatinase may be in people with HCU. The study is sponsored by Travere Therapeutics and follows an earlier Phase 1/Phase 2 study called COMPOSE.
About 70 people with HCU are expected to take part in the study. After completing HARMONY, some participants may be able to join a long-term follow-on study and continue or begin receiving pegtibatinase.
Eligibility Criteria:
You may qualify for the HARMONY Study if you meet the following basic criteria:
- Diagnosed with HCU
- 12 to 65 years of age
- Additional factors and restrictions also apply
Study Process
- Screening Period (Up to 4 weeks)
To check if the study is right for you. - Pre-Treatment Period (Up to 6 weeks)
To understand your current diet and how to keep it stable during the study. - Study Treatment Period (24 weeks)
To receive pegtibatinase or placebo. - Follow-Up Period (Within 4 weeks)
To check your health after the Study Treatment Period.
Advance Rare Treatments.
Have a Question?
FAQ
We understand that participating in a clinical trial can raise a lot of questions. Here, you’ll find answers to some of the most common questions about our process, what to expect, and how we support our patients. If you don’t see the information you’re looking for, feel free to contact us directly for more details.
People with a diagnosis of HCU who are 12 to 65 years of age may be able to take part. Other factors and restrictions also apply.
The total time in the study is about 9 months, including screening, pre-treatment, study treatment, and follow-up periods.
Payment for time and travel may be available to participants and their caregivers.