Mucopolysaccharidosis Type I
1
Pipeline Programs
3
Companies
4
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
1
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
Monoclonal Antibody
1100%
+ 3 programs with unclassified modality
Competitive Landscape
3 companies ranked by most advanced pipeline stage
Innovation PharmaceuticalsMA - Wakefield
1 program1
AdalimumabPhase 1/2Monoclonal Antibody1 trial
Active Trials
Rare Disease TherapeuticsTN - Franklin
2 programsLongitudinal Studies of Brain Structure and Function in MPS DisordersN/A1 trial
MRS to Determine Neuroinflammation and Oxidative Stress in MPS IN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Innovation PharmaceuticalsAdalimumab
Rare Disease TherapeuticsMRS to Determine Neuroinflammation and Oxidative Stress in MPS I
CENTOGENEBiomarker for Hurler Disease (BioHurler)
Rare Disease TherapeuticsLongitudinal Studies of Brain Structure and Function in MPS Disorders
Clinical Trials (4)
Total enrollment: 132 patients across 4 trials
Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI
Start: May 2015Est. completion: Jul 20162 patients
Phase 1/2Completed
NCT03576729Rare Disease TherapeuticsMRS to Determine Neuroinflammation and Oxidative Stress in MPS I
MRS to Determine Neuroinflammation and Oxidative Stress in MPS I
Start: Nov 2018Est. completion: Aug 201930 patients
N/ACompleted
Biomarker for Hurler Disease (BioHurler)
Start: Aug 2018Est. completion: Feb 20210
N/AWithdrawn
NCT01870375Rare Disease TherapeuticsLongitudinal Studies of Brain Structure and Function in MPS Disorders
Longitudinal Studies of Brain Structure and Function in MPS Disorders
Start: Sep 2009Est. completion: Aug 2019100 patients
N/ACompleted
Related Jobs
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
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.