Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8 Years of Age and Older (PAPAYA)

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2016 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01410890
First received: August 2, 2011
Last updated: May 26, 2016
Last verified: May 2016
  Purpose
  • The primary objective of this study is to characterize the pharmacokinetics of alglucosidase alfa manufactured at the 4000 L scale in patients 8 years of age and older 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: Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Official Title: A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8 Years of Age and Older

Resource links provided by NLM:


Further study details as provided by Sanofi:

Primary Outcome Measures:
  • Maximum observed concentration (Cmax) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Actual sampling time to reach maximum observed concentration (Tmax) [ Time Frame: Day 1 ] [ 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 ] [ 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 ] [ Designated as safety issue: No ]
  • Terminal elimination half-life (T1/2) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Total systemic clearance (CL) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Volume of distribution (Vd) [ Time Frame: Day 1 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Pharmacokinetic (PK) parameters assessed in relation to anti-rhGAA antibody titers [ Time Frame: Day 1 ] [ Designated as safety issue: No ]
  • Pharmacokinetic (PK) parameters assessed in relation to inhibitory/neutralizing anti-rhGAA antibody titers [ Time Frame: Day 1 ] [ Designated as safety issue: No ]

Estimated Enrollment: 20
Study Start Date: November 2014
Estimated Study Completion Date: January 2018
Estimated Primary Completion Date: January 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:   8 Years and older
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 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.
  • 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

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
Investigational Site Number 1026 Recruiting
Moscow, Russian Federation, 125412
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

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: May 26, 2016
Health Authority: United States: Food and Drug Administration
Germany: Federal Institute for Drugs and Medical Devices
Russia: Ministry of Health of the Russian Federation
Ukraine: Ministry of Health

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 May 30, 2016