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Condition: Homocystinuria Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Homocystinuria

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Ag
Phase 3 · 52 location(s) · Travere Therapeutics, Inc. · NCT06247085Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
Phase N/A · 11 location(s) · Travere Therapeutics, Inc. · NCT02998710Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
Phase N/A · 1 location(s) · Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta · NCT04880356Health Related Quality of Life (HrQoL) in Classical Homocystinuria (CBS Deficiency)
Phase N/A · 1 location(s) · University Children's Hospital, Zurich · NCT06556615
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.