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NCT02998710RECRUITING

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

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
Interventions / Drugs
View on ClinicalTrials.gov ↗
Sponsor
TVTX
Travere Therapeutics
Nephrology
C61/100
Study Details
Enrollment150 participants
Primary Completion—
Lead SponsorTravere Therapeutics, Inc.
Data Retrieved2026-04-11 04:30:13.942993+00:00
What This Trial Means for Biotech Investors
Trial Phase Context
Status & Expected Data

Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.

Enrolled: 150 participants
Sponsor Catalyst Profile

Travere Therapeutics (TVTX) is the sponsoring company for this trial. BiotechSign currently grades this company C (61/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

TVTX Full Pipeline →All Companies →
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