Access cutting-edge homocystinuria treatment through this phase 1/2 clinical trial at a research site in Boston. Study-provided care at no cost to qualified participants.
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This study follows strict safety protocols and ethical guidelines
All study-related homocystinuria treatment provided free
Check if you qualify for this homocystinuria clinical trial in Boston, MA
No-Cost Study Care
Local to Boston
Convenient for MA 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.
Researchers are looking for a better way to treat people who have classical homocystinuria (HCU), a rare condition that is passed down by parents (or "genetic condition"). It is caused by changes in the cystathionine beta-synthase (or "CBS") gene and prevents an enzyme from working correctly in the body. This enzyme breaks down a substance called homocysteine (from dietary methionine found in protein) and keeps both homocysteine and methionine at normal levels. When this enzyme is not working, homocysteine and methionine build up in the blood, which spreads into different tissues of the body and stops these body tissues from working normally. People with HCU can experience problems with vision, bones, blood vessels, and cognitive function (the ability to think, learn, and remember). Treatments available for HCU, such as a low protein diet and betaine (Cystadane®), help reduce homocysteine levels. The diet is a low methionine diet and a methionine-free protein supplement (a product that provides extra protein to help meet daily protein needs). These treatments are either not sufficient or are hard to take for many patients. Pegtibatinase was developed by scientists to be a version of the CBS enzyme that can be given to people with HCU. Researchers believe that giving pegtibatinase to people with HCU already getting medical treatment (or "standard of care") may reduce their homocysteine levels. This study is split into 7 different groups getting different amounts of drug. The first 6 groups have already finished the study. Group 7 plans to enroll participants from the US (virtual and in-person), France, and Qatar.
Sponsor: Travere Therapeutics, Inc.
Yes, this clinical trial (NCT03406611) has an active research site in Boston, MA that is currently enrolling participants.
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.
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.
Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.
If you're searching for homocystinuria treatment options in Boston, MA, this clinical trial (NCT03406611) 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 Boston research site is actively enrolling participants for this phase 1/2 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.