Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency

  • End date
    Dec 4, 2024
  • participants needed
  • sponsor
    Orphan Technologies Ltd
Updated on 4 March 2021
Travere Medical Information
Primary Contact
Temple Street Children's University Hospital (1.0 mi away) Contact
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The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 5 to 65 years under current clinical management practices

Condition Homocystinuria Due to CBS Deficiency
Clinical Study IdentifierNCT02998710
SponsorOrphan Technologies Ltd
Last Modified on4 March 2021


Yes No Not Sure

Inclusion Criteria

Patients who are clinically diagnosed with homocystinuria
Male/female patients aged 5 to 65 years
Patients who consented and/or assented

Exclusion Criteria

Medically significant postnatal complications or congenital anomalies that are not associated with homocystinuria
Received any experimental therapy for homocystinuria during the 6 months prior to enrollment or expected to receive any such therapy during duration of the study
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