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Menkes Disease

2
Pipeline Programs
3
Companies
3
Clinical Trials
0
Approved Products

Pipeline by Development Stage

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

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Human BioSciences
Human BioSciencesWV - Martinsburg
1 program
1
Copper HistidinePhase 3
Cyprium Therapeutics
Cyprium TherapeuticsFL - Bay Harbor Islands
1 program
1
Copper HistidinePhase 31 trial
Active Trials
NCT00811785Completed93Est. Aug 2020
Sentynl Therapeutics
Sentynl TherapeuticsCA - Solana Beach
2 programs
Long Term Follow-UpN/A1 trial
copper histidinateN/A1 trial
Active Trials
NCT04337684Active Not Recruiting50Est. Aug 2026
NCT04074512Approved For Marketing

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Cyprium TherapeuticsCopper Histidine
Sentynl TherapeuticsLong Term Follow-Up

Clinical Trials (3)

Total enrollment: 143 patients across 3 trials

Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency

Start: Feb 2009Est. completion: Aug 202093 patients
Phase 3Completed

Copper Histidinate Treatment for Menkes Disease

N/AApproved For Marketing

Long Term Follow-up on Menkes Disease Patients

Start: Dec 2019Est. completion: Aug 202650 patients
N/AActive Not Recruiting

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
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.