Trial record 1 of 1 for:    NCT01410890
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Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8-18 Years of Age (PAPAYA)

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01410890
First received: August 2, 2011
Last updated: September 15, 2014
Last verified: September 2014
  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:


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: February 2017
Estimated Primary Completion Date: February 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

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
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: September 15, 2014
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 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 18, 2014