Trial record 1 of 1 for:
NCT01410890
Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8-18 Years of Age (PAPAYA)
This study is currently recruiting participants.
(see Contacts and Locations)
Verified February 2015 by Sanofi
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: February 4, 2015
Last verified: February 2015
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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 ranging from 8 to 18 years of age who have a confirmed diagnosis of Pompe disease and are naïve to treatment with alglucosidase alfa. Eligible patients will receive an intravenous (IV) infusion of alglucosidase alfa of 20 mg/kg of body weight every other week (qow) for 26 weeks.
| Condition | Intervention | Phase |
|---|---|---|
|
Pompe Disease (Late-Onset) Glycogen Storage Disease Type II (GSD II) Glycogenesis 2 Acid Maltase Deficiency |
Biological: alglucosidase alfa |
Phase 4 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Pharmacokinetics Study Intervention Model: Single Group Assignment Masking: Open Label |
| Official Title: | A Phase IV Prospective Study to Characterize the Pharmacokinetics of Alglucosidase in Patients Aged 8-18 Years of Age |
Resource links provided by NLM:
Genetics Home Reference related topics:
glycogen storage disease type IX
Pompe disease
Schindler disease
succinic semialdehyde dehydrogenase deficiency
Drug Information available for:
Alglucosidase Alfa
U.S. FDA Resources
Further study details as provided by Sanofi:
Primary Outcome Measures:
- Maximum observed concentration (Cmax) [ Time Frame: Day 1, Week 12, Week 26 ] [ Designated as safety issue: No ]
- Actual sampling time to reach maximum observed concentration (Tmax) [ Time Frame: Day 1, Week 12, Week 26 ] [ Designated as safety issue: No ]
- 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 ] [ Designated as safety issue: No ]
- Area under the concentration-time curve from time 0 and extrapolated to infinite time (AUC inf) [ Time Frame: Day 1, Week 12, Week 26 ] [ Designated as safety issue: No ]
- Terminal elimination half-life (T1/2) [ Time Frame: Day 1, Week 12, Week 26 ] [ Designated as safety issue: No ]
- Total systemic clearance (CL) [ Time Frame: Day 1, Week 12, Week 26 ] [ Designated as safety issue: No ]
- Volume of distribution (Vd) [ Time Frame: Day 1, Week 12, Week 26 ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- 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 ] [ Designated as safety issue: No ]
- 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 ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 20 |
| Study Start Date: | August 2013 |
| Estimated Study Completion Date: | April 2017 |
| Estimated Primary Completion Date: | April 2017 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: alglucosidase alfa
alglucosidase alfa intravenous (IV) infusion of 20mg/kg body weight every other week (qow)
|
Biological: alglucosidase alfa
Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)
Other Name: Lumizyme
|
Eligibility| Ages Eligible for Study: | 8 Years to 18 Years |
| Genders Eligible for Study: | Both |
| 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 is ≥8 and ≤18 years of age with confirmed acid α-glucosidase [GAA] enzyme deficiency from skin, blood, or muscle tissue and/or 2 confirmed GAA gene mutations.
- The patient, if female and of childbearing potential, must have a negative pregnancy test (urine beta-human chorionic gonadotropin) at baseline. Note: All female patients of childbearing potential and sexually mature males must agree to use a medically accepted method of contraception throughout the study.
Exclusion Criteria:
A patient who meets any of the following criteria will be excluded from this study.
- The patient has had previous treatment with alglucosidase alfa.
- 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.
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 | |
| Germany | |
| Investigational Site Number 1079 | Recruiting |
| Mainz, Germany, 55131 | |
| Russian Federation | |
| Investigational Site Number 1024 | Recruiting |
| Moscow, Russian Federation, 119991 | |
| Ukraine | |
| Investigational Site Number 1076 | Recruiting |
| Vynnitsa, Ukraine, 21000 | |
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
| Study Director: | Medical Monitor | Genzyme, a Sanofi Company |
More Information
No publications provided
| Responsible Party: | Sanofi ( Genzyme, a Sanofi Company ) |
| ClinicalTrials.gov Identifier: | NCT01410890 History of Changes |
| Other Study ID Numbers: | AGLU07710, 2010-022231-11, MSC12790 |
| Study First Received: | August 2, 2011 |
| Last Updated: | February 4, 2015 |
| Health Authority: | United States: Food and Drug Administration Germany: Federal Institute for Drugs and Medical Devices (Bundesinstitut für Arzneimittel und Medizinprodukte (BfArM)) Russia: Ministry of Health of Russian Federation Ukraine: The State Expert Center of the Ministry of Health of Ukraine |
Additional relevant MeSH terms:
|
Glycogen Storage Disease Glycogen Storage Disease Type II Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases |
Genetic Diseases, Inborn Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases |
ClinicalTrials.gov processed this record on March 03, 2015