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

BioMarin Pharmaceutical

Status and phase

Terminated

Phase 2

Phase 1

Conditions

MPS IV A

Mucopolysaccharidosis IVA

Morquio A Syndrome

Treatments

Drug: BMN 110

Study type

Interventional

Funder types

Industry

Identifiers

NCT01242111

MOR-100

Details and patient eligibility

About

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).

Enrollment

20 patients

Sex

All

Volunteers

No 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).