Clinical Trial: Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Olipudase Alfa in Pediatric Patients Ag
Brief Summary:
Primary Objective:
To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric patients every 2 weeks for 52 weeks.
Secondary Objective:
To characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory efficacy of olipudase alfa administered intravenously in pediatric patients every 2 weeks for 52 weeks.
Detailed Summary: The maximum study duration per patient is approximately 18 months (screening period: up to 60 days; treatment period: 64 weeks; post-treatment period: up to 37 days, not applicable if patient enrolls in a long term extension treatment trial).
Sponsor: Genzyme, a Sanofi Company
Current Primary Outcome:
- Number of adverse events [ Time Frame: From screening through Week 64 ]
- Clinically significant changes in laboratory parameters (complete blood count (CBC), clinical chemistry, and urinalysis) [ Time Frame: From screening through Week 64 ]
- Clinically significant changes in physical examinations (vital signs, electrocardiogram (ECG), Doppler echocardiography, and liver ultrasound Doppler) [ Time Frame: From screening through Week 64 ]
Original Primary Outcome:
- Number of adverse events [ Time Frame: From screening through Week 52 ]
- Clinically significant changes in laboratory parameters (complete blood count (CBC), clinical chemistry, and urinalysis) [ Time Frame: From screening through Week 52 ]
- Clinically significant changes in physical examinations (vital signs, electrocardiogram (ECG), doppler echocardiography, and liver ultrasound doppler) [ Time Frame: From screening through Week 52 ]
Current Secondary Outcome:
- Maximum concentration (Cmax) [ Time Frame: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52 ]
- Area under the curve until the last measurable concentration (AUClast) [ Time Frame: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52 ]
- Area under the curve extrapolated to infinity (AUC) [ Time Frame: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52 ]
- Half-life (t1/2) [ Time Frame: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52 ]
- Clearance (CL) [ Time Frame: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52 ]
- Volume of distribution (Vss) [ Time Frame: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52 ]
- Change in sphingomyelin levels [ Time Frame: From Day 1 through Week 64 ]
- Change in sphingomyelin metabolite levels [ Time Frame: From Day 1 through Week 64 ]
Original Secondary Outcome:
- Maximum concentration (Cmax) [ Time Frame: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52 ]
- Area under the curve until the last measurable concentration (AUClast) [ Time Frame: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52 ]
- Area under the curve extrapolated to infinity (AUC) [ Time Frame: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52 ]
- Half-life (t1/2) [ Time Frame: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52 ]
- Clearance (CL) [ Time Frame: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52 ]
- Volume of distribution (Vss) [ Time Frame: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at Week 52 ]
- Change in sphingomyelin levels [ Time Frame: From Day 1 through Week 52 ]
- Change in sphingomyelin metabolite levels [ Time Frame: From Day 1 through Week 52 ]
Information By: Sanofi
Dates:
Date Received: November 7, 2014
Date Started: May 2015
Date Completion: July 2018
Last Updated: February 9, 2017
Last Verified: February 2017