A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This study is enrolling participants by invitation only.
Sponsor:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01242111
First received: October 28, 2010
Last updated: May 10, 2012
Last verified: May 2012
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Purpose
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).
| Condition | Intervention |
|---|---|
|
MPS IV A |
Drug: BMN 110 |
| Study Type: | Interventional |
| Study Design: | Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | 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) |
Resource links provided by NLM:
Further study details as provided by BioMarin Pharmaceutical:
Primary Outcome Measures:
- Change from baseline in endurance as measured by the 6-minute Walk Test [ Time Frame: Baseline and every 12 weeks ] [ Designated as safety issue: No ]
- Change from baseline in respiratory function tests [ Time Frame: Baseline and every 24 weeks ] [ Designated as safety issue: No ]
- Change from baseline in urine keratan sulfate (KS) levels (normalized to creatinine) [ Time Frame: Baseline and every 12 weeks ] [ Designated as safety issue: No ]
- Change from baseline in growth and development as measured by anthropometric measurements (standing height, length, sitting height, and weight) [ Time Frame: Baseline and every 24 weeks ] [ Designated as safety issue: No ]
- Change from baseline in bone growth as evaluated by X-rays of the lumbar spine and lower extremity [ Time Frame: Baseline and every 18 months ] [ Designated as safety issue: No ]
- Change from baseline in self-care and mobility skills as assessed by the Mucopolysaccharidosis Health Assessment Questionnaire [ Time Frame: Baseline and every 24 weeks ] [ Designated as safety issue: No ]
- Change from baseline in safety as determined by evaluation of adverse events [ Time Frame: Baseline, week 0, and weekly ] [ Designated as safety issue: Yes ]
- Change from baseline in safety as determined by evaluation of clinical laboratory tests [ Time Frame: Baseline and every 12 weeks ] [ Designated as safety issue: Yes ]
- Change from baseline in safety as determined by evaluation of electrocardiograms [ Time Frame: Baseline and every 48 weeks ] [ Designated as safety issue: Yes ]
- Change from baseline in endurance as measured by the 3-minute Stair-Climb Test [ Time Frame: Baseline and every 12 weeks ] [ Designated as safety issue: No ]
- Change from baseline in safety as determined by evaluation of vital signs measurements [ Time Frame: Baseline, week 0, and weekly ] [ Designated as safety issue: Yes ]
- Change from baseline in safety as determined by evaluation of concomitant medications [ Time Frame: Baseline, week 0, and weekly ] [ Designated as safety issue: Yes ]
- Change from baseline in safety as determined by evaluation of immunogenicity tests [ Time Frame: Baseline and every 12 weeks ] [ Designated as safety issue: Yes ]
- Change from baseline in safety as determined by evaluation of physical examinations [ Time Frame: Baseline and every 12 weeks ] [ Designated as safety issue: Yes ]
- Change from baseline in safety as determined by evaluation of echocardiograms [ Time Frame: Baseline and every 48 weeks ] [ Designated as safety issue: Yes ]
- Change from baseline in safety as determined by evaluation of cervical spine X-rays [ Time Frame: Baseline and every 48 weeks ] [ Designated as safety issue: Yes ]
| Estimated Enrollment: | 100 |
| Study Start Date: | November 2010 |
| Estimated Primary Completion Date: | November 2015 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: BMN 110 |
Drug: BMN 110
Patients will receive a weekly 4-hour intravenous infusion of 2.0 mg/kg BMN 110 for up to 240 weeks.
|
Eligibility| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01242111
Locations
| United Kingdom | |
| Birmingham, United Kingdom | |
| Dumfries, United Kingdom | |
| London, United Kingdom | |
| Manchester, United Kingdom | |
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
| Study Director: | Celeste Decker, MD | BioMarin Pharmaceutical |
More Information
Additional Information:
No publications provided
| Responsible Party: | BioMarin Pharmaceutical |
| ClinicalTrials.gov Identifier: | NCT01242111 History of Changes |
| Other Study ID Numbers: | MOR-100 |
| Study First Received: | October 28, 2010 |
| Last Updated: | May 10, 2012 |
| Health Authority: | United Kingdom: Medicines and Healthcare Products Regulatory Agency United Kingdom: Research Ethics Committee |
Keywords provided by BioMarin Pharmaceutical:
|
Mucopolysaccharidosis IV type A Mucopolysaccharidosis IVA MPS IV Type A 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 |
Additional relevant MeSH terms:
|
Mucopolysaccharidoses Mucopolysaccharidosis IV Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn |
Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases |
ClinicalTrials.gov processed this record on May 22, 2013