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A study to evaluate the safety and effectiveness of pegtibatinase in patients aged 12 to 65 years with classical homocystinuria.

Fast replyInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 2 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Study participation and medication administration

    You will receive either pegtibatinase or a placebo (a saline solution that contains no active medicine) in addition to your current standard of care treatment.

    If pegtibatinase is assigned, it is given as a subcutaneous injection, which means it is injected into the layer of fat just under the skin.

    The dose for pegtibatinase is 2.5 mg/kg (milligrams per kilogram of body weight).

  2. Step 2

    Monitoring and evaluation

    During the first 12 weeks of the study, your plasma tHcy (total homocysteine, a substance in the blood) and plasma Met (methionine, an amino acid in the blood) levels will be measured to track changes.

    Measurements will be taken to assess the levels at weeks 6, 8, 10, and 12.

    After the initial 12 weeks, monitoring will continue through weeks 16, 20, and 24 to observe the long-term effects on your blood levels.

Who can join the trial?

5 criteria

  • You must have a diagnosis of Classical Homocystinuria, which is a rare condition, confirmed through medical exams, blood tests, or molecular genetic testing (tests that look at your DNA to find changes in your genes).
  • Your plasma tHcy levels, which is the amount of a substance called homocysteine found in your blood, must be measured during the screening process. Most participants will have a level of 80 µM (micromoles per liter) or higher, but some may be included if their level is between 50 and 80 µM.
  • You must be between 12 and 65 years old at the time of the initial screening.
  • You must be willing to follow a stable diet, meaning you keep your food intake consistent, specifically regarding DIPI (dietary intake of protein and specific nutrients).
  • You must be willing to continue your current HCU-related therapies, such as taking betaine, pyridoxine (a form of vitamin B6), or using medical food (specialized nutritional formulas designed for your condition) as part of your regular treatment.

Who cannot join the trial?

7 criteria

  • You cannot participate if you have Marfan syndrome, which is a genetic condition that affects the body's connective tissues, methylenetetrahydrofolate reductase (MTHFR) deficiency, which is a genetic issue affecting how your body processes certain nutrients, or disorders of cobalamin metabolism, which means your body has trouble using vitamin B12.
  • You cannot participate if your body weight is 160 kg (352 pounds) or more.
  • You cannot participate if you have previously used pegtibatinase or pegtarviliase.
  • You cannot participate if you are currently using or plan to use any injectable medicine that contains PEG (polyethylene glycol, a common ingredient in many medicines), unless it is the study medicine or a vaccine containing PEG.
  • You cannot participate if you have a history of a severe immune reaction (a strong, harmful reaction by your body's defense system) to any product containing PEG.
  • You cannot participate if you have had an organ transplant.
  • You cannot participate if you have received intensive chronic immunosuppressive treatment (strong medicine used to lower your body's ability to fight infections) within the 6 months before the screening process began.
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Investigated drugs

Pegtibatinase is an experimental medication given as a shot under the skin. In this study, it is used alongside standard care to see if it can help lower levels of certain substances in the blood, specifically homocysteine and methionine, in people with a condition called classical homocystinuria.

What is already known about the treatment

  • 0.9 % sodium chloride saline solution

    This substance is a simple salt-water solution administered as a placebo to compare against the active treatment. It is widely used in medical practice to provide fluids or as a carrier for other medications. It does not have a specific medicinal effect on the underlying disease being studied.

  • Pegtibatinase

    This medication is an experimental enzyme treatment administered as an injection under the skin. It is currently being studied in clinical trials to treat a rare metabolic disorder called classical homocystinuria. The drug works by helping the body break down specific substances in the blood that build up due to a missing enzyme, thereby helping to balance metabolic levels. It is classified as an enzyme replacement therapy.

Investigated diseases

Classical Homocystinuria - This is a genetic metabolic disorder caused by a deficiency in an enzyme needed to break down the amino acid methionine. Because this process is interrupted, high levels of homocysteine build up in the blood and urine. As the condition progresses, the excess homocysteine can affect various bodily systems. This imbalance often leads to changes in the structure of connective tissues and physical development. The metabolic buildup can also impact the eyes and the central nervous system over time.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-504135-40-00Protocol codeTVTX-TVT058-301Estimated enrolment70 patientsSponsorTravere Therapeutics Inc.

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