Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-08-31

Study Completion Date

2014-10-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

NCT06280209

Duchenne Muscular Dystrophy

RECRUITING PHASE1/PHASE2

A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

NCT01957059

Duchenne Muscular Dystrophy

TERMINATED PHASE1/PHASE2

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

NCT02851797

Duchenne Muscular Dystrophy

COMPLETED PHASE3

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1

NCT06667453

Myotonic Dystrophy 1

RECRUITING PHASE2

Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)

NCT02958202

Duchenne Muscular Dystrophy

TERMINATED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Effect is defined by the following key domains:

* Upper extremity function and dexterity
* Mobility

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Mucopolysaccharidosis IVA Morquio A Syndrome MPS IVA

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

BMN 110 at 2.0 mg/kg/week

Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for up to 144 weeks.

Group Type EXPERIMENTAL

BMN 110

Intervention Type DRUG

Drug will be delivered through a 4 hour (approximate) IV infusion at a dosage amount of 2.0 mg/kg/week for up to 144 weeks of treatment.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

BMN 110

Drug will be delivered through a 4 hour (approximate) IV infusion at a dosage amount of 2.0 mg/kg/week for up to 144 weeks of treatment.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

N-acetylgalactosamine-6-sulfatase N-acetylgalactosamine-6-sulfate sulfatase galactose-6-sulfatase GALNS enzyme replacement therapy ERT elosulfase alfa

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Is willing and able to provide written, signed informed consent (or their legally authorized representative) after the nature of the study has been explained and prior to performance of any research-related procedure. Patients who do not meet country and local age requirements for informed consent must be willing and able to provide written assent after the nature of the study has been explained and prior to performance of any research-related procedure.
* Has documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA.
* Is ≥ 5 years of age.
* If sexually active, is willing to use an acceptable method of contraception while participating in the study.
* Females of childbearing potential must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study.
* Is willing and able to perform all study procedures as physically possible.

Exclusion Criteria

* Is able to walk farther than a specified distance as assessed by the 6MWT.
* Has previous hematopoietic stem cell transplant (HSCT).
* Has received previous treatment with BMN 110.
* Has a known hypersensitivity to any of the components of BMN 110.
* Has had major surgery within 3 months prior to study entry or is planning to have a major surgery during the first 24 weeks of the study.
* Has used any other investigational product or investigational medical device within 30 days prior to the Screening Visit or requires any investigational agent prior to completion of all scheduled study assessments.
* Is pregnant or breastfeeding at the Screening Visit or planning to become pregnant (self or partner) at any time during the study.
* Has a concurrent disease or condition, including but not limited to symptomatic cervical spine instability or severe cardiac disease or complete paralysis due to a spinal cord injury (defined as an inability to move arms and legs), that would interfere with study participation or safety 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.

Minimum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No