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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01242111
First received: October 28, 2010
Last updated: July 26, 2013
Last verified: July 2013
  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 Phase
MPS IV A
Drug: BMN 110
Phase 2

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 48 weeks ] [ Designated as safety issue: No ]
  • Change from baseline in respiratory function tests [ Time Frame: Baseline and every 48 weeks ] [ Designated as safety issue: No ]
  • Change from baseline in urine keratan sulfate (KS) levels (normalized to creatinine) [ Time Frame: Baseline and every 24 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 24 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 48 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 24 weeks ] [ Designated as safety issue: Yes ]
  • Change from baseline in safety as determined by evaluation of physical examinations [ Time Frame: Baseline and every 24 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 72 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 20
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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: Adam Shaywitz, 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: July 26, 2013
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
United Kingdom: Research Ethics Committee

Keywords provided by BioMarin Pharmaceutical:
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

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 July 22, 2014