Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01515956 | Completed Phase 2 Results DMC

• 1 other identifier: MOR-007
• Study Type: interventional
• Target: 15
• Locations: 4 countries
• Sites: 5

Brief Summary

This open-label Phase 2 study will evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age at the time of administration of the first dose of study drug, diagnosed with MPS IVA (Morquio A Syndrome) for up to 208 weeks.

Conditions

Mucopolysaccharidosis IVA; Morquio A Syndrome; MPS IVA

Keywords

Mucopolysaccharidosis IVA type A; MPS IVA 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

Outcome Measures

Primary Outcomes (1)

• To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of First Study Drug Infusion

  Number of Participants Experiencing Adverse Events

  52 weeks

Secondary Outcomes (2)

• Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures

  Baseline to Week 52

• Change From Baseline in Normalized Growth Rate Z-Scores

  Baseline to Week 52

Study Arms (1)

BMN 110 Weekly

EXPERIMENTAL

Drug: BMN 110

Interventions

BMN 110 DRUG

Patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/wk over a period of approximately 4 hours every week for up to 208 weeks.

Also known as: N-acetylgalactosamine-6-sulfatase, N-acetylgalactosamine-6-sulfate, sulfatase, galactose-6-sulfatase, GALNS, enzyme replacement therapy, ERT

BMN 110 Weekly

Eligibility Criteria

• Age: Up to 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Less than 5 years of age at the time of the first study drug infusion
• 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
• Written informed consent provided by parent or legally authorized representative after the nature of the study has been explained and prior to any research-related procedures.

You may not qualify if:

• Previous hematopoietic stem cell transplant (HSCT).
• Previous treatment with BMN 110.
• Known hypersensitivity to any of the components of BMN 110.
• Major surgery within 3 months prior to stuy entry or planned major surgery during the 52-week treatment period.
• 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, 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 subject at high risk of poor treatment compliance or of not completing the study.

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (5)

Unknown Facility

Oakland, California, United States

Location

Unknown Facility

Manhasset, New York, United States

Location

Unknown Facility

Monza, Italy

Location

Unknown Facility

Taipei, Taiwan

Location

Unknown Facility

Central Manchester, United Kingdom

Location

Related Publications (1)

• Jones SA, Bialer M, Parini R, Martin K, Wang H, Yang K, Shaywitz AJ, Harmatz P. Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y. Pediatr Res. 2015 Dec;78(6):717-22. doi: 10.1038/pr.2015.169. Epub 2015 Sep 2.

  PMID: 26331768 DERIVED

MeSH Terms

Conditions

Mucopolysaccharidosis IV; Lysosomal Storage Diseases

Interventions

GALNS protein, human; Chondroitinases and Chondroitin Lyases; Galns protein, mouse; Sulfatases; Enzyme Replacement Therapy

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Esterases; Hydrolases; Enzymes; Enzymes and Coenzymes; Polysaccharide-Lyases; Carbon-Oxygen Lyases; Lyases; Enzyme Therapy; Drug Therapy; Therapeutics

Results Point of Contact

• Title: Peter Slasor/Sr Director, Biostatistics, Global Clinical Sciences
• Organization: BioMarin Pharmaceutical Inc.

PSlasor@bmrn.com

415-506-6765

Study Officials

• Debra Lounsbury

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2011
• First Posted: January 24, 2012
• Study Start: October 1, 2011
• Primary Completion: February 1, 2016
• Study Completion: February 1, 2016
• Last Updated: August 10, 2017
• Results First Posted: July 6, 2017

Record last verified: 2017-07

Locations

IT (1); GB (1); TW (1); US (2)