EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment

Key facts

Sponsor

Bambino Gesù Hospital and Research Institute

Principal Investigator

Carlo Dionisi-Vici, MD

Bambino Gesù Hospital and Research Institute

Intervention

Epi-743(drug)

Enrollment

30 enrolled

Eligibility

1-20 years · All sexes

Timeline

2013 – 2017

Study locations (1)

• Bambino Gesù Hospital and Research Institute, Rome, Italy

Collaborators

Catholic University of the Sacred Heart

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.

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• ACTIVE NOT RECRUITINGNANCT06495567
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• 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)
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• 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
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• ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT03406611
  Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)
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• RECRUITINGNCT02998710
  Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
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See all trials for Homocystinuria →