Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

NCT02998710 · ClinicalTrials.gov registry record

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

A clinical trial of Homocystinuria Due to CBS Deficiency, sponsored by Travere Therapeutics.

Recruiting
Registry status
150
Enrollment target
11
Study locations

NCT02998710: Recruiting study of Homocystinuria Due to CBS Deficiency, sponsored by Travere Therapeutics.

NCT02998710 is a study of Homocystinuria Due to CBS Deficiency that is actively recruiting participants, run by Travere Therapeutics. The registered enrollment target is 150 participants. The trial reports 11 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.

View on ClinicalTrials.gov ↗

View your shortlist →

The verdict

NCT02998710, a study of Homocystinuria Due to CBS Deficiency, is actively recruiting participants, sponsored by Travere Therapeutics.

RECRUITING
Registry status
150 participants
Enrollment target
11
Study locations

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

Primary Outcome

Changes in total homocysteine levels in micromoles. The met cycle metabolites levels and change from baseline will be summarized using descriptive statistics at each visit.

Study Locations (11)

Other

  • Travere Investigational Site - Dublin
  • Travere Investigational Site - Doha

California

  • Travere Investigational Site - Virtual Site - Culver City

Colorado

  • Travere Investigational Site (Enrolling 1 to <5 Year-olds Only) - Aurora

District of Columbia

  • Travere Investigational Site (Enrolling 1 to <5 Year-olds Only) - Washington D.C.

Georgia

  • Travere Investigational Site - Atlanta

Indiana

  • Travere Investigational Site - Indianapolis

Massachusetts

  • Travere Investigational Site - Boston

Pennsylvania

  • Travere Investigational Site (Enrolling 1 to <5 Year-olds Only) - Philadelphia

Trial Details

FieldValue
Enrollment Target 150 participants
Start Date 2017-01
Est. Completion 2026-08
Travere Therapeutics

12 total trials

What NCT02998710 shows while recruiting

NCT02998710 is an observational study that tracks outcomes without assigning an intervention. The registered 150 participants enrollment target is mid-sized for trials with a published cap.

The record links to 1 condition, with Homocystinuria Due to CBS Deficiency appearing as the primary indexed condition, and to 0 interventions.

NCT02998710 lists 11 locations in 10 states (Other, California, Colorado).

Frequently Asked Questions

What is clinical trial NCT02998710 about?

NCT02998710 is a clinical study titled "Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)". 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

What is the current status of trial NCT02998710?

This trial is currently recruiting. The enrollment target is 150 participants. The study started on 2017-01. Estimated completion is 2026-08.

What conditions does trial NCT02998710 study?

This clinical trial studies the following conditions: Homocystinuria Due to CBS Deficiency.

Who is sponsoring clinical trial NCT02998710?

This trial is sponsored by Travere Therapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT02998710 being conducted?

This trial has 11 study locations across California, Colorado, District of Columbia, Georgia, Indiana. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Nationwide trials with similar profiles

Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.

Similar enrollment target

  • NCT00412594 · 150 participants · Phase 2

    Cladribine and Rituximab in Treating Patients With Hairy Cell Leukemia

  • NCT01484730 · 150 participants

    A Pilot Study to Evaluate Multi-Spectral and Laser Speckle Imaging and Multiphoton Microscopy During Vascular Occlusion

  • NCT01985919 · 150 participants

    Protocol for Facilitating the Acquisition of Research Bone Marrow and Blood Samples

  • NCT02016248 · 150 participants · NA

    SBRT Boost for Unfavorable Prostate Cancer'

Similar registry start date

  • NCT05988463 · started 2027-01-01 · Phase 1

    Dose-Escalation Study of Artesunate Patients With IPF

  • NCT07219706 · started 2026-12-10 · NA

    Dietary Fiber and Time Restricted Eating

  • NCT02831335 · started 2026-12-01

    Technical Development of Multi-Parametric Renocerebral MRI

  • NCT05962346 · started 2026-12 · NA

    Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia

Source: ClinicalTrials.gov NCT02998710, the US trial registry maintained by the National Library of Medicine. NCT02998710 (mid enrollment · multi site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.