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

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ClinicalTrials.gov Identifier: NCT01242111
Recruitment Status : Terminated
First Posted : November 16, 2010
Results First Posted : September 30, 2015
Last Update Posted : September 30, 2015
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Tracking Information
First Submitted Date  ICMJE October 28, 2010
First Posted Date  ICMJE November 16, 2010
Results First Submitted Date  ICMJE July 10, 2015
Results First Posted Date  ICMJE September 30, 2015
Last Update Posted Date September 30, 2015
Study Start Date  ICMJE November 2010
Actual Primary Completion Date July 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 28, 2015)
Safety Evaluation [ Time Frame: Entire Study Period, up to 240 weeks ]
The primary objective of the study is to evaluate the safety of weekly infusions of BMN 110; the safety variables included Adverse Events (AEs). The primary outcome measure data is presented in more detail under the Adverse Events section.
Original Primary Outcome Measures  ICMJE
 (submitted: November 13, 2010)
  • Change from baseline in endurance as measured by the 6-minute Walk Test [ Time Frame: Baseline and every 12 weeks ]
  • Change from baseline in respiratory function tests [ Time Frame: Baseline and every 24 weeks ]
  • Change from baseline in urine keratan sulfate (KS) levels (normalized to creatinine) [ Time Frame: Baseline and every 12 weeks ]
  • 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 ]
  • Bone growth as evaluated by X-rays of the lumbar spine and lower extremity [ Time Frame: Baseline ]
  • Change from baseline in self-care and mobility skills as assessed by the Mucopolysaccharidosis Health Assessment Questionnaire [ Time Frame: Baseline and every 24 weeks ]
  • Change from baseline in safety as determined by evaluation of adverse events [ Time Frame: Baseline, week 0, and weekly ]
  • Change from baseline in safety as determined by evaluation of clinical laboratory tests [ Time Frame: Baseline and every 12 weeks ]
  • Change from baseline in safety as determined by evaluation of electrocardiograms [ Time Frame: Baseline and every 48 weeks ]
  • Change from baseline in endurance as measured by the 3-minute Stair-Climb Test [ Time Frame: Baseline and every 12 weeks ]
  • Change from baseline in safety as determined by evaluation of vital signs measurements [ Time Frame: Baseline, week 0, and weekly ]
  • Change from baseline in safety as determined by evaluation of concomitant medications [ Time Frame: Baseline, week 0, and weekly ]
  • Change from baseline in safety as determined by evaluation of immunogenicity tests [ Time Frame: Baseline and every 12 weeks ]
  • Change from baseline in safety as determined by evaluation of physical examinations [ Time Frame: Baseline and every 12 weeks ]
  • Change from baseline in safety as determined by evaluation of echocardiograms [ Time Frame: Baseline and every 48 weeks ]
  • Change from baseline in safety as determined by evaluation of cervical spine X-rays [ Time Frame: Baseline and every 48 weeks ]
Change History Complete list of historical versions of study NCT01242111 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 28, 2015)
  • Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). [ Time Frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial ]
    Change from baseline in meters in 6-minute Walk Test. As a measure of endurance, a 6-minute walk test (6MWT) was performed according to the American Thoracic Society Guidelines. Patients were instructed to walk as far as possible in 6 minutes.
  • Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). [ Time Frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial ]
    Change from baseline in the 3-minute Stair Climb Test (3MSCT). Patients walked up stairs that have a railing, which could be used for support, for 3 minutes, with the number of stairs climbed recorded. The test result was the number of steps climbed per minute.
  • Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). [ Time Frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 168 weeks during the MOR-100 extension trial ]
    Percent Change from baseline in Normalized Urine KS. The percent change was calculated (Week X value - baseline value)/baseline value *100%
  • Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). [ Time Frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial ]
    Percent Change from baseline in Maximum Voluntary Ventilation.
  • Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). [ Time Frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial ]
    Percent Change from baseline in Forced Vital Capacity.
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Official Title  ICMJE 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)
Brief Summary 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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • MPS IV A
  • Mucopolysaccharidosis IVA
  • Morquio A Syndrome
Intervention  ICMJE Drug: BMN 110
Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
Study Arms  ICMJE Experimental: BMN 110
Intervention: Drug: BMN 110
Publications * Hendriksz C, Santra S, Jones SA, Geberhiwot T, Jesaitis L, Long B, Qi Y, Hawley SM, Decker C. Safety, immunogenicity, and clinical outcomes in patients with Morquio A syndrome participating in 2 sequential open-label studies of elosulfase alfa enzyme replacement therapy (MOR-002/MOR-100), representing 5 years of treatment. Mol Genet Metab. 2018 Apr;123(4):479-487. doi: 10.1016/j.ymgme.2018.02.011. Epub 2018 Feb 19.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: August 28, 2015)
20
Original Estimated Enrollment  ICMJE
 (submitted: November 13, 2010)
200
Actual Study Completion Date  ICMJE July 2014
Actual Primary Completion Date July 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01242111
Other Study ID Numbers  ICMJE MOR-100
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party BioMarin Pharmaceutical
Study Sponsor  ICMJE BioMarin Pharmaceutical
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Celeste Decker, MD BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date August 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP