A Phase 1, Multicenter, Open-Label, Single-Arm, Multiple Dose Study to Evaluate the the Pharmacokinetics and Pharmacodynamics of Natalizumab in Pediatric Subjects With Relapsing Remitting Multiple Sclerosis (RMS)
Trial Snapshot
- Phase
- Phase 1
- Status
- Completed
- Sponsor
- Biogen
- Enrollment
- 13
- Locations
- 5
- Primary Endpoint
- predose (trough) concentrations from multiple dosing (Cpredose)
Study Overview
Brief Summary
The primary objective of the study is to determine the pharmacokinetic (PK) profile of multiple doses of natalizumab in pediatric subjects with relapsing-remitting multiple sclerosis (RRMS). The secondary objectives are as follows: to characterize the pharmacodynamic (PD) profile of natalizumab (as defined by α4 integrin binding) and to explore the safety and tolerability of multiple doses of natalizumab in the pediatric population.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 10 Years to 17 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Rapidly evolving severe relapsing remitting multiple sclerosis, defined by 2 or more disabling relapses in 1 year, and with 1 or more gadolinium-enhancing lesions on brain MRI or a significant increase in T2 lesion load, as compared to a previous recent magnetic resonance imaging (MRI)
Exclusion Criteria
- •History of, or abnormal laboratory values indicative of, significant medical, neurologic (other than MS), or psychiatric disorders that might preclude participation in the study in the opinion of the Investigator.
- •Prior natalizumab therapy.
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Outcomes
Primary Outcomes
predose (trough) concentrations from multiple dosing (Cpredose)
Time Frame: Up to week 16
maximum plasma concentration (Cmax)
Time Frame: Up to Week 16
time to maximum plasma concentration (Tmax)
Time Frame: Up to Week 16
area under the plasma concentration curve from time of first dose to infinity (AUCinf)
Time Frame: Up to Week 16
apparent clearance (Cl/F)
Time Frame: Up to Week 16
volume of distribution
Time Frame: Up to Week 16
elimination half-life (t1/2)
Time Frame: Up to Week 16
Secondary Outcomes
- the average and minimum saturation values of α4 integrin over the dosing interval(Up to Week 16)
- incidence of serious adverse events (SAEs), infusion and hypersensitivity reactions, and other AEs(Up to Week 16)
- the presence of anti-natalizumab antibodies(Up to Week 16)