Clinical trial NCT00884949: A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
Official title: A Phase 1/2, Multicenter, Open-label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: MPS IV A
Keywords: 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
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2009-04-21
Enrollment: 20
Sex: ALL
Minimum age (years): 5
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This multicenter, open-label study is designed to assess safety, dose-response using pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in subjects between 5 and 18 years of age, diagnosed with Mucopolysaccharidosis IVA (MPS IVA).


Eligibility criteria: Inclusion Criteria:

* Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay, or documented result of molecular genetic testing confirming diagnosis of MPS IVA.
* Willing and able to provide written, signed informed consent, or in the case of subjects under the age of 16 years, 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.
* Between 5 and 18 years of age, inclusive.
* Sexually active subjects 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 Screening and be willing to have additional pregnancy tests during the study.
* Willing to perform all study procedures as physically possible.

Exclusion Criteria:

* Previous hematopoietic stem cell transplant (HSCT).
* Has known hypersensitivity to BMN 110 or its excipients.
* Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
* Use of any investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
* Concurrent disease or condition that would interfere with study participation or safety, including, but not limited to, symptomatic cervical spine instability.
* Any condition that, in the view of the Principal Investigator (PI), places the subject at high risk of poor treatment compliance or of not completing the study.

Primary outcomes:
measure: Subject Incidence of Treatment Emergent AEs
timeFrame: Entire Study, through week 84
Secondary outcomes:
measure: Change From Baseline in 6MWT
timeFrame: Baseline to Weeks 12, 24, 36, 48, 72
measure: Change From Baseline in 3MSCT
timeFrame: Baseline to Weeks 12, 24, 36, 48, 72
measure: Percent Change From Baseline in uKS
timeFrame: Baseline to Weeks 12, 24, 36, 72
measure: Percent Change From Baseline in MVV
timeFrame: Baseline to Weeks 12, 24, 36, 72
measure: Percent Change From Baseline in FVC
timeFrame: Baseline to Weeks 12, 24, 36, 72
Other outcomes:
Locations:
city: Birmingham
country: United Kingdom
city: London
country: United Kingdom
city: Manchester
country: United Kingdom
Study officials:
affiliation: BioMarin Pharmceutical Inc.
name: Celeste Decker, MD
role: STUDY_DIRECTOR
References:
citation: 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.