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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01697319
First received: September 17, 2012
Last updated: December 20, 2013
Last verified: December 2013

September 17, 2012
December 20, 2013
August 2012
July 2016   (final data collection date for primary outcome measure)
Efficacy will be determined/summarized by percent changes of the domain scores for upper extremity function, dexterity, mobility, pain, and self-care and functional ability testing from baseline. [ Time Frame: Up to 144 weeks ] [ Designated as safety issue: No ]
  • Upper extremity function will be tested using the Grip-Pinch Test.
  • Dexterity will be tested using the Functional Dexterity Test.
  • Mobility will be tested using the 25 Foot Walk test
  • Pain will be determined by the Brief Pain Inventory-Short Form questionnaire or the Adolescent Pediatric Pain Tool
  • Functional/self care abilities will be determined using Pediatric Outcomes Data Collection Instrument (PODCI)or the SF-36 questionnaire.

Efficacy will be measured at the following timepoints: Baseline, Weeks: 12, 24, 36, 48, 72, 96, 120, 144 / ETV. Weeks: 60, 84, 108, & 132 (Pain Inventory & PODCI only.)

Efficacy will be determined/ summarized by percent changes of the domain scores for upper extremity function, dexterity, mobility, pain, and self-care and functional ability testing from baseline. [ Time Frame: Up to 48 weeks ] [ Designated as safety issue: No ]
  • Upper extremity function will be tested using the Grip-Pinch Test.
  • Dexterity will be tested using the Functional Dexterity Test.
  • Mobility will be tested using the 25 Foot Walk test
  • Pain will be determined by the Brief Pain Inventory-Short Form questionnaire or the Adolescent Pediatric Pain Tool
  • Functional/self care abilities will be determined using Pediatric Outcomes Data Collection Instrument (PODCI)or the SF-36 questionnaire.

Efficacy will be measured at the following timepoints: Baseline, Week 12, Week 24, Week 36,and Week 48/ ETV

Complete list of historical versions of study NCT01697319 on ClinicalTrials.gov Archive Site
  • Change in respiratory function using a summarized analysis of the percentage change in FET, FIVC, FVC, FEV1, MVV and optional TLC values from the Baseline visit. [ Time Frame: Up to 144 weeks ] [ Designated as safety issue: No ]
    Timepoints: Baseline, Weeks: 24, 48, 96, 144 / ETV
  • Change in urinary KS over time as determined by descriptive statistics. [ Time Frame: Up to 144 weeks ] [ Designated as safety issue: No ]
    Timepoints: Baseline, Weeks: 2, 4, 6, 12, 24, 36, 48, 72, 96, 120, 144 / ETV
  • Assessment of effect on sleep apnea results as calculated using the Apnea-hypopnea index(AHI). [ Time Frame: Up to 144 weeks ] [ Designated as safety issue: No ]
    A subset consisting of any subjects who have abnormal overnight pulse oximetry readings will be assessed for sleep apnea. Only selected sites are participating in the sleep apnea study.
  • Change in respiratory function using a summarized analysis of the percentage change in FET, FIVC, FVC, FEV1, MVV and optional TLC values from the Baseline visit. [ Time Frame: Up to 48 weeks ] [ Designated as safety issue: No ]
    Timepoints: Baseline, Week 24, and Week 48
  • Change in plasma and urinary KS over time as determined by descriptive statistics. [ Time Frame: Up to 48 weeks ] [ Designated as safety issue: No ]
    Timepoints: Baseline, Week 2, Week 4, Week 6, Week 12, Week 24, Week 36, Week 48/ ETV
  • Assessment of effect on sleep apnea results as calculated using the Apnea-hypopnea index(AHI). [ Time Frame: Up to 48 weeks ] [ Designated as safety issue: No ]
    A subset of approximately 5 patients at selected sites who have abnormal overnight pulse oximetry readings and who provide additional informed consent will be assessed for sleep apnea.
Descriptive summary of clinical safety assessments [ Time Frame: Continuously for up to 144 weeks or more ] [ Designated as safety issue: Yes ]
Incidence of AEs and changes in neurologic examinations, vital signs, ECHOs, ECGs, cervical spine radiographs, immunogenicity tests, clinical laboratory tests, and concomitant medications.
Descriptive summary of clinical safety assessments [ Time Frame: Continuously for up to 48 weeks or more ] [ Designated as safety issue: Yes ]
Incidence of AEs and changes in neurologic examinations, vital signs, ECHOs, ECGs, cervical spine radiographs, immunogenicity tests, clinical laboratory tests, and concomitant medications.
 
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation

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.

Effect is defined by the following domains:

  • Upper extremity function and dexterity
  • Mobility
  • Pain
  • Self care and functional abilities
Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Mucopolysaccharidosis IVA
  • Morquio A Syndrome
  • MPS IVA
Drug: 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.
Other Names:
  • N-acetylgalactosamine-6-sulfatase
  • N-acetylgalactosamine-6-sulfate
  • sulfatase
  • galactose-6-sulfatase
  • GALNS
  • enzyme replacement therapy
  • ERT
Experimental: 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.
Intervention: Drug: BMN 110
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
15
July 2016
July 2016   (final data collection date for primary outcome measure)

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.
Both
5 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States,   Germany,   United Kingdom
 
NCT01697319
MOR-006, 2011-005703-33
Yes
BioMarin Pharmaceutical
BioMarin Pharmaceutical
Not Provided
Study Director: Celeste Decker, M.D. BioMarin Pharmaceutical
BioMarin Pharmaceutical
December 2013

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