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BMN 110 Phase 3B in Australian Patients

NCT01966029 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 13

Last updated 2019-09-26

No results posted yet for this study

Summary

There is currently no treatment for MPS IVA other than supportive care for the clinical manifestations of the disease. Enzyme replacement therapy (ERT) with BMN 110 to replace the deficient GALNS is a potential new treatment option for MPS IVA patients. BMN 110, containing recombinant human GALNS (rhGALNS) developed by BioMarin is expected to reduce the progressive, pathologic accumulation of KS, and improve signs and symptoms of the disease.

The objective of this Phase 3B open label study (110-502) will be to evaluate the safety and tolerability of 2.0 mg/kg/week (qw) of BMN 110 in Australian patients with MPS IVA. In addition, a number of secondary and tertiary efficacy endpoints will also be investigated. The dose and regimen of BMN 110 have been selected on the basis of data from a Phase 1/2 clinical study with BMN 110, nonclinical and in vitro studies with BMN 110, and clinical and nonclinical data from other enzyme replacement therapies.

Extension Phase is included per amendment dated 10Mar 2014: To provide patients enrolled in the Initial Phase access to BMN 110 until commercial product becomes available in Australia and continue to assess long-term safety

Conditions

  • Mucopolysaccharidosis IVA (Morquio A Syndrome)

Interventions

DRUG

BMN 110

All pateints receive treatment with BMN 110

Sponsors & Collaborators

Principal Investigators

  • Tristan Zhang, MD · BioMarin Pharmaceutical

  • Eric He, MD · BioMarin Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-07-31
Primary Completion
2016-07-31
Completion
2016-12-31

Countries

  • Australia

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01966029 on ClinicalTrials.gov