HARMONY

• Research type

  Research Study

• Full title

  A phase 3, parallel-group treatment, blinded, randomized, placebo-controlled study to assess the efficacy and safety of pegtibatinase administered subcutaneously in addition to standard of care in participants with classical homocystinuria due to cystathionine beta synthase deficiency (HARMONY).

• IRAS ID

  1010489

• Contact name

  Maryam Doucette

• Contact email

  maryam.doucette@travere.com

• Sponsor organisation

  Travere Therapeutics, Inc.

• Clinicaltrials.gov Identifier

  NCT06247085

• Research summary

  Classical homocystinuria (HCU) is a rare inherited condition. In HCU, a gene (inherited material in our body cells that carries instructions for the body to grow and develop) that is not working prevents an enzyme from working correctly in the body. When this enzyme is not working, a substance called homocysteine (Hcy) builds up in the blood, spreads into different tissues, and stops them from working normally. With untreated HCU, damaged tissues can cause vision problems, weak bones and fractures, joint problems, learning disabilities, brain damage, life-threatening blood clots (gel-like clumps of blood that may reduce blood flow to critical parts of the body), and strokes.
  The study is being conducted to find out if pegtibatinase is safe and can lower the amount of Hcy in the blood. This will be assessed by regular blood tests throughout the study to measure Hcy levels and by comparing the effects of pegtibatinase to placebo. Placebo is inactive and offers no benefit. Participants will continue standard HCU therapies, which may include betaine, vitamins B6/B12/D, folate, methionine-free formulas, and a methionine-restricted diet.
  Approximately 70 eligible participants will be recruited and placed into 1 of 2 groups by chance (like flipping a coin) to receive either pegtibatinase or placebo. The study is “blinded,” which means that neither participants nor the study doctor will know which treatment is given. The study will last 38 weeks (about 9 months) and has four parts: a 4-week screening period to check eligibility; a 6-week pre-treatment period to understand diet and keep it stable; a 24-week treatment period where participants receive pegtibatinase or placebo 2 times each week injected under the skin; and a safety follow-up visit 4 weeks after the last dose.

• REC name

  East Midlands - Nottingham 2 Research Ethics Committee

• REC reference

  26/EM/0010

• Date of REC Opinion

  27 Mar 2026

• REC opinion

  Further Information Favourable Opinion