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Homocystinuria

2
Pipeline Programs
3
Companies
5
Clinical Trials
2 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
1
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

March Biosciences
March BiosciencesTX - Houston
1 program
1
N-acetylcysteinePhase 21 trial
Active Trials
NCT00483314Completed5Est. Feb 2009
Colorado Therapeutics
Colorado TherapeuticsCO - Louisville
1 program
1
taurinePhase 1/21 trial
Active Trials
NCT01192828Completed15Est. Dec 2017
Travere Therapeutics
3 programs
PegtibatinasePHASE_1_21 trial
PegtibatinasePHASE_31 trial
PegtibatinasePHASE_31 trial
Active Trials
NCT03406611Recruiting39Est. Jul 2027
NCT06247085Recruiting70Est. Sep 2027
NCT06431893Enrolling By Invitation100Est. Aug 2030

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Travere TherapeuticsPegtibatinase
Travere TherapeuticsPegtibatinase
March BiosciencesN-acetylcysteine
Travere TherapeuticsPegtibatinase
Colorado Therapeuticstaurine

Clinical Trials (5)

Total enrollment: 229 patients across 5 trials

NCT06431893Travere TherapeuticsPegtibatinase

A 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)

Start: Apr 2024Est. completion: Aug 2030100 patients
Phase 3Enrolling By Invitation
NCT06247085Travere TherapeuticsPegtibatinase

Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment

Start: Dec 2023Est. completion: Sep 202770 patients
Phase 3Recruiting
NCT00483314March BiosciencesN-acetylcysteine

Homocystinuria: Treatment With N-Acetylcysteine

Start: Nov 2007Est. completion: Feb 20095 patients
Phase 2Completed
NCT03406611Travere TherapeuticsPegtibatinase

Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)

Start: Jan 2019Est. completion: Jul 202739 patients
Phase 1/2Recruiting
NCT01192828Colorado Therapeuticstaurine

Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine

Start: Jan 2010Est. completion: Dec 201715 patients
Phase 1/2Completed

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

2 actively recruiting trials targeting 229 patients
3 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.