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Trial record 1 of 1 for:
NCT01410890
Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease (PAPAYA)
This study is currently recruiting participants.
Verified September 2017 by Sanofi ( Genzyme, a Sanofi Company )
Sponsor:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01410890
First received: August 2, 2011
Last updated: September 13, 2017
Last verified: September 2017
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Purpose
- 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.
| Condition | Intervention | Phase |
|---|---|---|
| Pompe Disease (Late-Onset) Glycogen Storage Disease Type II (GSD II) | Biological: alglucosidase alfa | Phase 4 |
| Study Type: | Interventional |
| Study Design: | Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Other |
| Official Title: | A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease |
Resource links provided by NLM:
Further study details as provided by Sanofi ( Genzyme, a Sanofi Company ):
Primary Outcome Measures:
- 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 ]
Secondary Outcome Measures:
- 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 ]
| Estimated Enrollment: | 20 |
| Study Start Date: | November 2014 |
| Estimated Study Completion Date: | August 2018 |
| Estimated Primary Completion Date: | August 2018 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: alglucosidase alfa
alglucosidase alfa intravenous (IV) infusion of 20mg/kg body weight
|
Biological: alglucosidase alfa
Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)
Other Name: Lumizyme
|
Detailed Description:
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).
Eligibility| Ages Eligible for Study: | Child, Adult, Senior |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
A patient must meet all of the following criteria to be eligible for this study:
- The patient and/or the patient's parent/legal guardian is willing and able to provide signed informed consent.
- The patient has a confirmed GAA enzyme deficiency from skin, blood, or muscle tissue and/or 2 confirmed GAA gene mutations.
- Infant and toddler Pompe disease patients can be included in the study only under condition (minimal body weight) that the trial-related blood loss (including any losses in the maneuver) will not exceed 3 percent of the total blood volume during a period of 4 weeks and will not exceed 1 percent at any single time.
- The patient, if female and of childbearing potential, must have a negative pregnancy test (urine beta-human chorionic gonadotropin) at screening. Note: All female patients of childbearing potential and sexually mature males must agree to use a medically accepted method of contraception throughout the study.
- For patients previously treated with alglucosidase alfa the patient has received alglucosidase alfa for at least 6 months.
Exclusion Criteria:
A patient who meets any of the following criteria will be excluded from this study:
- The patient is participating in another clinical study using an investigational product.
- The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01410890
Please refer to this study by its ClinicalTrials.gov identifier: NCT01410890
Contacts
| Contact: For site information, send an email with site number to | Contact-Us@sanofi.com | ||
| Contact: Medical Information | 617-252-7832 | medinfo@genzyme.com |
Locations
| Bulgaria | |
| Investigational Site Number 1028 | Recruiting |
| Sofia, Bulgaria, 1113 | |
| Russian Federation | |
| Investigational Site Number 643001 | Recruiting |
| Moscow, Russian Federation, 125367 | |
| Investigational Site Number 643002 | Recruiting |
| Moscow, Russian Federation, 125412 | |
| Ukraine | |
| Investigational Site Number 804001 | Recruiting |
| Kiev, Ukraine, 01135 | |
| Investigational Site Number 804002 | Recruiting |
| Vynnitsa, Ukraine, 21000 | |
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
| Study Director: | Medical Monitor | Genzyme, a Sanofi Company |
More Information
| Responsible Party: | Genzyme, a Sanofi Company |
| ClinicalTrials.gov Identifier: | NCT01410890 History of Changes |
| Other Study ID Numbers: |
AGLU07710 2010-022231-11 ( EudraCT Number ) MSC12790 ( Other Identifier: Sanofi ) |
| Study First Received: | August 2, 2011 |
| Last Updated: | September 13, 2017 |
Additional relevant MeSH terms:
|
Glycogen Storage Disease Type II Glycogen Storage Disease Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases |
Nervous System Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Carbohydrate Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases |
ClinicalTrials.gov processed this record on September 20, 2017