CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 20 enrolled
Drug / intervention
BMN 110drug
Likely dose
BMN 110 0.1 mg/kgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00884949
NCT00884949Phase 2Completed
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A Phase 1/2, Multicenter, Open-label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

BioMarin Pharmaceutical·interventional·Posted Apr 21, 2009·Updated Jun 30, 2014

In Brief

A Phase 2 clinical trial evaluating BMN 110 for MPS IV A. Completed, enrolled 20 participants across 3 sites.

Detailed Summary

This multicenter, open-label study is designed to assess safety, dose-response using pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in subjects between 5 and 18 years of age, diagnosed with Mucopolysaccharidosis IVA (MPS IVA).

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMPS IV A
CountriesUnited Kingdom
Collaborators--

Timeline

Phase 2CompletedFinished
200920102011201220132014201520162017201820192020202120222023202420252026
First PostedApr 21, 2009
Enrollment StartApr 1, 2009
Primary CompletionFeb 1, 2011
Study CompletionMar 1, 2011
TodayJul 2, 2026
Enrollment to primary: 1.8 yearsPosted 17.2 years ago

Interventions

BMN 110drug

Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 in 3 consecutive 12-week dosing intervals, using the following regimen: * Weeks 1-12: 0.1 mg/kg/week * Weeks 13-24: 1.0 mg/kg/week * Weeks 25-36: 2.0 mg/kg/week Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week.