long-term efficacy and safety extension study of bmn 110 in patients with mucopolysaccharidosis iva (morquio a syndrome)

Conditions: Mucopolysaccharidosis IV A; Morquio A Syndrome; MPS IVA

Summary: this phase 3 extension study will evaluate the long-term efficacy and safety of bmn 110 2.0 mg/kg/week and/or bmn 110 2.0 mg/kg/every other week in patients with mucopolysaccharidosis iva (morquio a syndrome).

this is a multi-center, multinational, extension study to evaluate 2 dose regimens of bmn 110 treatment in patients with mps iva who completed mor-004. the last study visit assessments for mor-004 will constitute baseline for this study. the first study drug dose of this protocol will occur on week 0 of mor-005, which is the same as the last visit (week 24) of mor-004. initially, the study will be double-blind with patients previously randomized to bmn 110 in mor-004 remaining on their assigned bmn 110 dose regimen (qw or qow dosing). the mor-004 placebo patients will be re-randomized (1:1 ratio) to one of the 2 bmn 110 dose regimen groups: 2.0 mg/kg/qw or 2.0 mg/kg/qow. there will be two study parts: * part 1 - randomized double-blind until the optimal bmn 110 dose regimen has been determined, based on the final primary efficacy analysis from mor-004 * part 2 - open-label bmn 110 treatment with the single optimal dose regimen

Eligibility criteria:
inclusion criteria: * must have completed mor-004 * is willing and able to provide written, signed informed consent. or in the case of patients under the age of 18 (or other age as defined by regional law or regulation), provide written assent (if required) and have written informed consent, signed by a legally authorize representative, after the nature of the study has been explained, and prior to performance of research-related procedures. * if sexually active, must be willing to use an acceptable method of contraception while participating in the study. * if female, of childbearing potential, must have a negative pregnancy test at baseline and be willing to have additional pregnancy tests done during the study. exclusion criteria: * is pregnant or breastfeeding, at baseline, or planning to become pregnant (self or partner) at any time during the study. * has used any investigational product (other than bmn 110 in mor-004), or investigational medical device, within 30 days prior to baseline; or is required to use any investigational agent prior to completion of all scheduled study assessments. * was enrolled in a previous bmn 110 study, other than mor-004. * has a 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 pose a safety risk, as determined by the investigator. * has 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.

Keywords: mucopolysaccharidosis iv type a mps iv type a mucopolysaccharidosis iva mps iva morquio a syndrome lysosomal storage disorder lsd n-acetylgalactosamine-6-sulfatase n-acetylgalactosamine-6-sulfate sulfatase galactose-6-sulfatase galns enzyme replacement therapy ert