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Enzyme Replacement Therapy
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
Phase 4
Waitlist Available
Research Sponsored by BioMarin Pharmaceutical
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up minimum 52 weeks of dosing
Awards & highlights
Pivotal Trial
All Individual Drugs Already Approved
No Placebo-Only Group
Drug Has Already Been Approved
Summary
The purpose of the study is to evaluate the safety and efficacy of two dose levels of Naglazyme in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.
Eligible Conditions
- Maroteaux-Lamy Syndrome
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ minimum 52 weeks of dosing
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~minimum 52 weeks of dosing
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Secondary study objectives
Change in Urinary Glycosaminoglycan Levels
Side effects data
From 2009 Phase 4 trial • 4 Patients • NCT00299000100%
Ear infection
100%
Diarrhea
100%
Rhinitis
100%
Vomiting
50%
Nasal congestion
50%
Upper respiratory tract infections
50%
Scoliosis
50%
Febrile convulsion
50%
Umbilical hernia
50%
Pyrexia
50%
Adenoidal hypertrophy
50%
Cast application
50%
Tonsillitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Naglazyme, 2.0 mg/kg
Naglazyme, 1.0 mg/kg
Awards & Highlights
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Drug Has Already Been Approved
The FDA has already approved this drug, and is just seeking more data.
Trial Design
2Treatment groups
Experimental Treatment
Group I: Naglazyme, 2.0 mg/kgExperimental Treatment1 Intervention
Dose Comparison
Group II: Naglazyme, 1.0 mg/kgExperimental Treatment1 Intervention
Dose comparison
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Galsulfase
FDA approved
Find a Location
Who is running the clinical trial?
BioMarin PharmaceuticalLead Sponsor
159 Previous Clinical Trials
115,046 Total Patients Enrolled
Celeste Decker, MDStudy DirectorBioMarin Pharmaceutical
4 Previous Clinical Trials
418 Total Patients Enrolled