Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT ID: NCT01415427
Last Updated: 2021-07-21
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
173 participants
INTERVENTIONAL
2011-07-31
2016-06-16
Brief Summary
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Detailed Description
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The last study visit assessments for MOR-004 will constitute Baseline for this study. The first study drug dose of this protocol will occur on Week 0 of MOR-005, which is the same as the last visit (Week 24) of MOR-004. Initially, the study will be double-blind with patients previously randomized to BMN 110 in MOR-004 remaining on their assigned BMN 110 dose regimen (qw or qow dosing). The MOR-004 placebo patients will be re-randomized (1:1 ratio) to one of the 2 BMN 110 dose regimen groups: 2.0 mg/kg/qw or 2.0 mg/kg/qow.
There will be two study parts:
* Part 1 - randomized double-blind until the optimal BMN 110 dose regimen has been determined, based on the final primary efficacy analysis from MOR-004
* Part 2 - open-label BMN 110 treatment with the single optimal dose regimen
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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BMN 110 Weekly
BMN 110 Weekly: In Part 1, patients will receive an intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
BMN 110 - Weekly
In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/qw administered over a period of approximately 4 hours once a week.
In Part 2, patients will continue to receive 2.0 mg/kg of BMN 110 every week, with no placebo.
BMN 110 Every Other Week
BMN 110 Every Other Week: In Part 1, patients will receive an intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and will receive infusions of placebo on alternating weeks.
BMN 110 - Every Other Week
In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week. Patients randomized to the 2.0 mg/kg/qow arm will receive infusions of placebo on alternating weeks, to mask active drug weeks.
In Part 2, patients will receive 2.0 mg/kg of BMN 110 every week, with no placebo.
Interventions
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BMN 110 - Weekly
In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/qw administered over a period of approximately 4 hours once a week.
In Part 2, patients will continue to receive 2.0 mg/kg of BMN 110 every week, with no placebo.
BMN 110 - Every Other Week
In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week. Patients randomized to the 2.0 mg/kg/qow arm will receive infusions of placebo on alternating weeks, to mask active drug weeks.
In Part 2, patients will receive 2.0 mg/kg of BMN 110 every week, with no placebo.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Is willing and able to provide written, signed informed consent. Or in the case of patients under the age of 18 (or other age as defined by regional law or regulation), provide written assent (if required) and have written informed consent, signed by a legally authorize representative, after the nature of the study has been explained, and prior to performance of research-related procedures.
* If sexually active, must be willing to use an acceptable method of contraception while participating in the study.
* If female, of childbearing potential, must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests done during the study.
Exclusion Criteria
* Has used any investigational product (other than BMN 110 in MOR-004), or investigational medical device, within 30 days prior to Baseline; or is required to use any investigational agent prior to completion of all scheduled study assessments.
* Was enrolled in a previous BMN 110 study, other than MOR-004.
* Has a 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 pose a safety risk, 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.
5 Years
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Debra Lounsbury
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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Phoenix, Arizona, United States
Oakland, California, United States
Orange, California, United States
Wilmington, Delaware, United States
Washington D.C., District of Columbia, United States
Orlando, Florida, United States
Honolulu, Hawaii, United States
Chicago, Illinois, United States
New York, New York, United States
Seattle, Washington, United States
Córdoba, , Argentina
Campina Grande, , Brazil
Porto Alegre, , Brazil
Rio de Janeiro, , Brazil
Montreal, , Canada
Sherbrooke, , Canada
Toronto, , Canada
Bogotá, , Colombia
Copenhagen, , Denmark
Lyon, , France
Marseille, , France
Paris, , France
Paris, , France
Mainz, , Germany
Monza, , Italy
Tokyo, , Japan
Amsterdam, , Netherlands
Oslo, , Norway
Coimbra, , Portugal
Lisbon, , Portugal
Doha, , Qatar
Riyadh, , Saudi Arabia
Seoul, , South Korea
Santiago de Compostela, , Spain
Taipei, , Taiwan
Ankara, , Turkey (Türkiye)
Belfast, , United Kingdom
Birmingham, , United Kingdom
Birmingham, , United Kingdom
London, , United Kingdom
London, , United Kingdom
London, , United Kingdom
Manchester, , United Kingdom
Countries
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References
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Hendriksz CJ, Parini R, AlSayed MD, Raiman J, Giugliani R, Mitchell JJ, Burton BK, Guelbert N, Stewart FJ, Hughes DA, Matousek R, Hawley SM, Decker C, Harmatz PR. Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study. Mol Genet Metab. 2018 Feb;123(2):127-134. doi: 10.1016/j.ymgme.2017.11.015. Epub 2017 Dec 5.
Hughes D, Giugliani R, Guffon N, Jones SA, Mengel KE, Parini R, Matousek R, Hawley SM, Quartel A. Clinical outcomes in a subpopulation of adults with Morquio A syndrome: results from a long-term extension study of elosulfase alfa. Orphanet J Rare Dis. 2017 May 23;12(1):98. doi: 10.1186/s13023-017-0634-0.
Long B, Tompkins T, Decker C, Jesaitis L, Khan S, Slasor P, Harmatz P, O'Neill CA, Schweighardt B. Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study. Clin Ther. 2017 Jan;39(1):118-129.e3. doi: 10.1016/j.clinthera.2016.11.017. Epub 2016 Dec 10.
Other Identifiers
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MOR-005
Identifier Type: -
Identifier Source: org_study_id
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