Clinical Trial: Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
Brief Summary:
- The primary objective of this study is to characterize the pharmacokinetics of alglucosidase alfa manufactured at the 4000 L scale in patients who have a confirmed diagnosis of Pompe disease.
- A secondary objective of this study is to evaluate and explore the relationship between anti-rhGAA antibody titers and the pharmacokinetics of alglucosidase alfa.
Detailed Summary: The total study duration per patient will be 4 to 9 weeks that consist of a screening period (from 2 days to 4 weeks), treatment visit (1 day), and a follow up call (≥30 days).
Sponsor: Genzyme, a Sanofi Company
Current Primary Outcome:
- Maximum observed concentration (Cmax) [ Time Frame: Day 1 ]
- Actual sampling time to reach maximum observed concentration (Tmax) [ Time Frame: Day 1 ]
- Area under the concentration-time curve from 0 to the time of the last quantifiable concentration (AUC last) [ Time Frame: Day 1 ]
- Area under the concentration-time curve from time 0 and extrapolated to infinite time (AUC inf) [ Time Frame: Day 1 ]
- Terminal elimination half-life (T1/2) [ Time Frame: Day 1 ]
- Total systemic clearance (CL) [ Time Frame: Day 1 ]
- Volume of distribution (Vd) [ Time Frame: Day 1 ]
Original Primary Outcome:
- Maximum observed concentration (Cmax) [ Time Frame: Day 1, Week 12, Week 26 ]
- Actual sampling time to reach maximum observed concentration (Tmax) [ Time Frame: Day 1, Week 12, Week 26 ]
- Area under the concentration-time curve from 0 to the time of the last quantifiable concentration (AUC last) [ Time Frame: Day 1, Week 12, Week 26 ]
- Area under the concentration-time curve from time 0 and extrapolated to infinite time (AUC inf) [ Time Frame: Day 1, Week 12, Week 26 ]
- Terminal elimination half-life (T1/2) [ Time Frame: Day 1, Week 12, Week 26 ]
- Total systemic clearance (CL) [ Time Frame: Day 1, Week 12, Week 26 ]
- Volume of distribution (Vd) [ Time Frame: Day 1, Week 12, Week 26 ]
Current Secondary Outcome:
- Pharmacokinetic (PK) parameters assessed in relation to anti-rhGAA antibody titers [ Time Frame: Day 1 ]
- Pharmacokinetic (PK) parameters assessed in relation to inhibitory/neutralizing anti-rhGAA antibody titers [ Time Frame: Day 1 ]
Original Secondary Outcome:
- Impact of anti-rhGAA IgG antibodies on pharmacokinetic (PK) profile as measured by change in assessed PK parameters including clearance [ Time Frame: Day 1, Week 12, Week 26 ]
- Impact of inhibitory/neutralizing antibodies on pharmacokinetic (PK) profile as measured by change in assessed PK parameters including clearance [ Time Frame: Day 1, Week 12, Week 26 ]
Information By: Sanofi
Dates:
Date Received: August 2, 2011
Date Started: November 2014
Date Completion: August 2018
Last Updated: March 16, 2017
Last Verified: March 2017