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HARMONY phase 3 trial of pegtibatinase in classical homocystinuria due to cystathionine beta synthase deficiency
2023-504135-40-00Phase 3Authorised
- Sponsor
- Travere Therapeutics
- Start
- Jul 2026
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 14
01
Overview
Phase 3, randomised, double-blind trial in Cystathionine beta-synthase deficiency (CBS) comparing Pegtibatinase + standard of care with Placebo + standard of care (2 arms). Primary endpoint: Change from baseline in plasma total homocysteine averaged over Weeks 6 through 12. Planned start Jul 2026; status: authorised.
02
Drugs
pegtibatinase
03
Conditions
Cystathionine beta-synthase deficiency (CBS)
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