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A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | Clinical Trials | Clareo Health

TERMINATEDPHASE1/PHASE2

A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

A Multicenter, Multinational, Open-Label, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01242111•BioMarin Pharmaceutical

View on ClinicalTrials.gov

Summary

This multicenter, open-label extension study is designed to assess long-term efficacy and safety of 2.0 milligrams (mg)/kilogram(kg)/week of BMN 110 in patients diagnosed with Mucopolysaccharidosis IVA (MPS IVA). Patients with MPS IVA, who enrolled in a prior BioMarin sponsored clinical study of BMN 110 (NCT00884949; Study Identification Number MOR-002), were eligible to enroll in this study (except patients who enrolled in NCT01275066; Study Identification Number MOR-004).

Eligibility

Age

All ages

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Willing and able to provide written, signed informed consent, or in the case of patients under the age of 18 (or 16 years, depending on the region), 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.
•Sexually active patients 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 Baseline and be willing to have additional pregnancy tests during the study.
•Must have enrolled in a prior BioMarin sponsored clinical study of BMN 110.

Exclusion Criteria

•Pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study.
•Use of any investigational product (other than BMN 110 in a prior clinical study) or investigational medical device within 30 days prior to Baseline, or requirement for any investigational agent prior to completion of all scheduled study assessments.
•Concurrent disease or condition, including but not limited to symptomatic cervical spine instability, clinically significant spinal cord compression, or severe cardiac disease that would interfere with study participation or safety as determined by the Investigator.
•Any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.
•Were enrolled in MOR-004 (patients from MOR-004 may be eligible to participate in a separate, appropriately designed, extension study).

Locations (4)

Birmingham, United Kingdom, United Kingdom

Dumfries, United Kingdom, United Kingdom

London, United Kingdom, United Kingdom

Manchester, United Kingdom, United Kingdom

Key Dates

Start Date

November 1, 2010

Primary Completion Date

July 1, 2014

Completion Date

July 1, 2014

Last Updated

September 30, 2015

Enrollment

ACTUAL participants

Conditions

MPS IV AMucopolysaccharidosis IVAMorquio A Syndrome

Interventions

BMN 110

DRUG

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

NCT03632213

Mucopolysaccharidosis IV AMucopolysaccharidosis VI+7 more

View study

COMPLETEDPHASE3

BMN 110 Phase 3B in Australian Patients

NCT01966029

Mucopolysaccharidosis IVA (Morquio A Syndrome)

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 30, 2015Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Inclusion Criteria

Exclusion Criteria

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

BMN 110 Phase 3B in Australian Patients

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome