Homocystinuria

• CLINICAL_TRIALS research · 2026-02-12
  Clinical Trial Update: Proof of Concept Creatine Supplementation for Homocystinuria Study [NA]

  A proof-of-concept clinical trial evaluating creatine supplementation for homocystinuria is currently active but not recruiting.

• CLINICAL_TRIALS research · 2025-04-10
  Clinical Trial Update: Health Related Quality of Life (HrQoL) in Classical Homocystinuria (CBS Deficiency)

  This clinical trial is investigating health-related quality of life in classical homocystinuria (CBS deficiency) and is currently recruiting…

• CLINICAL_TRIALS research · 2025-04-09
  Clinical Trial Update: A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment [Phase 3]

  Phase 3 study evaluating peg-tibatinase versus placebo for classical homocystinuria due to CBS deficiency; the trial status is active but no…

• CLINICAL_TRIALS research · 2024-11-22
  Clinical Trial Update: Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE) [Phase 1, Phase 2]

  Pegtibatinase is being studied in a Phase 1/Phase 2 trial for homocystinuria caused by cystathionine beta-synthase deficiency, sponsored by …

• CLINICAL_TRIALS research · 2024-08-13
  Clinical Trial Update: Incidence and Risk Factors of Ocular Complications Among Patients With Homocystinuria

  This clinical trial update investigates the incidence and risk factors of ocular complications in patients with homocystinuria and is curren…

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