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 Pharmaceutical0 sites20 target enrollmentApril 2009

ConditionsMPS IV A

InterventionsBMN 110

DrugsBMN 110

Overview

Phase: Phase 1
Intervention: BMN 110
Conditions: MPS IV A
Sponsor: BioMarin Pharmaceutical
Enrollment: 20
Primary Endpoint: Subject Incidence of Treatment Emergent AEs
Status: Completed
Last Updated: 11 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Similar Trials

Overview

Brief 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).

Registry

clinicaltrials.gov

Start Date

April 2009

End Date

March 2011

Last Updated

11 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

BioMarin Pharmaceutical

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay, or documented result of molecular genetic testing confirming diagnosis of MPS IVA.
•Willing and able to provide written, signed informed consent, or in the case of subjects under the age of 16 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
•Between 5 and 18 years of age, inclusive.
•Sexually active subjects must be willing to use an acceptable method of contraception while participating in the study.
•Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study.
•Willing to perform all study procedures as physically possible.

Exclusion Criteria

•Previous hematopoietic stem cell transplant (HSCT).
•Has known hypersensitivity to BMN 110 or its excipients.
•Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
•Use of any investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
•Concurrent disease or condition that would interfere with study participation or safety, including, but not limited to, symptomatic cervical spine instability.
•Any condition that, in the view of the Principal Investigator (PI), places the subject at high risk of poor treatment compliance or of not completing the study.

Arms & Interventions

BMN 110

Within-patient Dose-Escalation

Intervention: BMN 110

Outcomes

Primary Outcomes

Subject Incidence of Treatment Emergent AEs

Time Frame: Entire Study, through week 84

The primary objective of the study was to evaluate the safety of weekly infusions of BMN 110 administered in escalating doses to subjects with MPS IVA. The safety variable incidence of TEAE is summarized.