Phoenix, AZNCT06247085Now EnrollingIRB Ready

Homocystinuria Clinical Trial in Phoenix, AZ

Access cutting-edge homocystinuria treatment through this clinical trial at a research site in Phoenix. Study-provided care at no cost to qualified participants.

Sponsored by Travere Therapeutics, Inc.

Quick Self-Assessment

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Expert Care in Phoenix

Access homocystinuria specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related homocystinuria treatment provided free

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Check if you qualify for this homocystinuria clinical trial in Phoenix, AZ

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Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Phoenix

    Convenient for AZ residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Phoenix site if eligible
  4. 4Begin participation

About This Homocystinuria Study in Phoenix

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)

Sponsor: Travere Therapeutics, Inc.

Who Can Participate

Inclusion Criteria

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
Participants who can become pregnant must have a negative pregnancy test before starting the study and must use a highly effective form of birth control (less than 1% risk of pregnancy per year) during the study and for at least 4 weeks after the last dose.
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 medical food for the duration of the study (unless changes are required based on medical/safety reasons)

Exclusion Criteria

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

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Phoenix?

Yes, this clinical trial (NCT06247085) has an active research site in Phoenix, AZ that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Homocystinuria Treatment Options in Phoenix, AZ

If you're searching for homocystinuria treatment options in Phoenix, AZ, this clinical trial (NCT06247085) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Phoenix research site is actively enrolling participants for this clinical trial. You'll receive care from experienced homocystinuria specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Ready to Join in Phoenix?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Phoenix, AZ