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

  • STATUS
    Recruiting
  • End date
    Dec 4, 2024
  • participants needed
    100
  • sponsor
    Orphan Technologies Ltd
Updated on 4 March 2021
Investigator
Travere Medical Information
Primary Contact
Temple Street Children's University Hospital (1.0 mi away) Contact
+7 other location

Summary

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

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

Eligibility

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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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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