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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

150 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-01-31

Study Completion Date

2026-08-31

Brief Summary

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The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical management practices

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Detailed Description

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Conditions

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Homocystinuria Due to CBS Deficiency

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

* Patients who are clinically diagnosed with homocystinuria
* Male/female patients aged 1 to 65 years
* Patients who consented and/or assented
* Patients who are willing and able to comply with all study-related procedures.

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

Minimum Eligible Age

1 Year

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No