Safety and efficacy of pegtibatinase enzyme replacement therapy in adults with classical homocystinuria in the COMPOSE phase 1/2 randomized trial.

Can Ficicioglu¹, Janet A Thomas², Jaya Ganesh³, David Kudrow⁴, Melissa Lah⁵, Wendy E Smith⁶, Jalé Güner⁷, Sharon McDermott⁸, Sagar A Vaidya⁸, Liz Wilkening⁷, Harvey L Levy⁹

¹Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA. Electronic address: ficicioglu@chop.edu.

²University of Colorado School of Medicine, Aurora, CO.

³Icahn School of Medicine at Mount Sinai, New York, NY.

⁴Science 37, Culver City, CA.

⁵Indiana University School of Medicine, Indianapolis, IN.

⁶MaineHealth Maine Medical Center, Portland, ME.

⁷Travere Therapeutics, Inc., San Diego, CA.

⁸Former employee, Travere Therapeutics, Inc., San Diego, CA.

⁹Boston Children's Hospital, Harvard Medical School, Boston, MA.

PubMed DOI

Abstract

PURPOSE: Because the standard-of-care treatment for classical homocystinuria (HCU) often cannot achieve adequate metabolic control, the phase 1/2 COMPOSE trial (NCT03406611) evaluated pegtibatinase enzyme replacement therapy. METHODS: Participants with HCU aged 12 to 65 years with elevated total plasma homocysteine (tHcy) receiving standard-of-care treatment were randomized 3:1 into 6 increasing…

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