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

NCT02998710 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 150

Last updated 2024-11-22

No results posted yet for this study

Summary

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

Conditions

  • Homocystinuria Due to CBS Deficiency

Sponsors & Collaborators

  • Travere Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Michael Imperiale, MD · Travere Therapeutics, Inc.

Eligibility

Min Age
1 Year
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-01-31
Primary Completion
2026-04-30
Completion
2026-08-31

Countries

  • United States
  • Ireland
  • Qatar
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02998710 on ClinicalTrials.gov