Classic Homocystinuria — Clinical Trials

MONDO:00093525 active·2 recruitingLast updated: Mar 20, 2026, 10:49 AM

Trial Landscape

Classic Homocystinuria has an emerging clinical trial program, driven by drug treatment.

Recruiting

Active Trials

By Approach

Drug Treatment3

3 additional trials involve behavioral, dietary, diagnostic, or observational approaches.

By Phase

Phase 1

Phase 2

Phase 3

2 trials are observational or have no phase designation.

Sponsor Types

Industry4Academic/Other1

Top Sponsors

Travere Therapeutics, Inc.

Trial Locations

United States4 trials

Ireland1 trial

Qatar1 trial

United Kingdom1 trial

Switzerland1 trial

Trials often recruit across multiple countries, so totals exceed the trial count.

3 of 5 Trials

Phase 3Enrolling by Invitation

NCT06431893

A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

Sponsor:Travere Therapeutics, Inc.

United StatesStarted: Apr 2024Est. completion: Jan 2027Updated: Apr 2025

Phase 3Active, Not Recruiting

NCT06247085

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

Sponsor:Travere Therapeutics, Inc.

United StatesStarted: Dec 2023Est. completion: Jan 2026Updated: Apr 2025

Phase 1Active, Not Recruiting

NCT03406611

Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)

Sponsor:Travere Therapeutics, Inc.

United StatesStarted: Jan 2019Est. completion: Dec 2026Updated: Nov 2024

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Data sourced from ClinicalTrials.gov via Kisho's automated pipeline. Trials refreshed every 4 hours for monitored diseases.

Last sync: March 20, 2026 at 10:49 AM

Classic Homocystinuria — Clinical Trials

Trial Landscape

By Approach

By Phase

Sponsor Types

Top Sponsors

Trial Locations

3 of 5 Trials

Open Trials1

A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

Active Trials2

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)