Homocystinuria – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Wed, 19 Aug 2026 04:04:14 +0000 en-US hourly 1 https://wordpress.org/?v=7.0.4 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Homocystinuria – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Safety and Tolerability Study of Subcutaneous Pegtibatinase in Adults and Children with Classical Homocystinuria (Phase 1/2) https://clinicaltrials.eu/trial/safety-and-tolerability-study-of-subcutaneous-pegtibatinase-in-adults-and-children-with-classical-homocystinuria-phase-1-2/ Mon, 03 Aug 2026 10:45:51 +0000 https://clinicaltrials.eu/trial/safety-and-tolerability-study-of-subcutaneous-pegtibatinase-in-adults-and-children-with-classical-homocystinuria-phase-1-2/ In this research, a rare metabolic disorder called Classical Homocystinuria is being studied. The condition causes the body to build up certain amino acids, leading to problems with the eyes, bones, and brain. The investigational medicine being tested is a protein called pegtibatinase, also known by the code TVT-058, which is given by a subcutaneous injection (an injection placed under the skin) to help break down the excess substances.

The purpose of the study is to determine how safe and well‑tolerated the medication is for people with this disorder. Participants will be randomly assigned to receive either the active drug or a placebo in a double-blind manner (meaning neither the participants nor the study staff know which treatment is being given) and will be followed for several weeks with regular check‑ups, blood tests, and heart rhythm recordings. Researchers will also look at whether the body creates an immune response to the drug, known as immunogenicity (the tendency to trigger antibodies).

During the study, volunteers will attend clinic visits where they will receive the assigned injection, have blood drawn to measure drug levels and metabolic markers, and undergo simple safety assessments such as blood pressure checks and an electrocardiogram (a test that records the heart’s electrical activity). Any side effects or changes in laboratory results will be recorded, and the overall health and quality‑of‑life information will be collected throughout the treatment period.

]]> A study to evaluate the safety and effectiveness of pegtibatinase in patients aged 12 to 65 years with classical homocystinuria. https://clinicaltrials.eu/trial/a-study-to-evaluate-the-safety-and-effectiveness-of-pegtibatinase-in-patients-aged-12-to-65-years-with-classical-homocystinuria/ Mon, 03 Aug 2026 10:44:23 +0000 https://clinicaltrials.eu/trial/a-study-to-evaluate-the-safety-and-effectiveness-of-pegtibatinase-in-patients-aged-12-to-65-years-with-classical-homocystinuria/ This study is being conducted to evaluate the effectiveness and safety of a medication called pegtibatinase in individuals living with Classical Homocystinuria. This condition is caused by a deficiency in the cystathionine beta synthase enzyme, which leads to high levels of certain substances in the blood. Participants in the study will continue to receive their usual standard of care treatments while also receiving either pegtibatinase or a placebo via subcutaneous injection, which is a method of delivering medicine through a needle just under the skin.

The goal of the study is to see how pegtibatinase affects levels of total homocysteine (tHcy) and methionine (Met) in the blood. High levels of these substances are common in people with this disease. During the study, blood samples will be taken to monitor these levels and to ensure that the treatment is being tolerated well by the body.

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