Health Related Quality of Life (HrQoL) in Classical Homocystinuria (CBS Deficiency)

Key facts

Sponsor

University Children's Hospital, Zurich

Intervention

Interview(behavioral)

Enrollment

80 enrolled

Eligibility

8 years · All sexes

Timeline

2025 – 2025

Study locations (1)

• Kinderspital Zürich, Zurich, Switzerland

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Other trials for Homocystinuria

Additional recruiting or active studies for the same condition.

• ACTIVE NOT RECRUITINGNANCT06495567
  Proof of Concept Creatine Supplementation for Homocystinuria Study
  University of British Columbia
• ENROLLING BY INVITATIONPHASE3NCT06431893
  A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)
  Travere Therapeutics, Inc.
• ACTIVE NOT RECRUITINGPHASE3NCT06247085
  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
  Travere Therapeutics, Inc.
• ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT03406611
  Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)
  Travere Therapeutics, Inc.
• RECRUITINGNCT02998710
  Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
  Travere Therapeutics, Inc.

See all trials for Homocystinuria →