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NeoGAA Extension Study

Information source: Sanofi
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Glycogen Storage Disease Type II Pompe Disease

Intervention: GZ402666 (Drug)

Phase: Phase 2/Phase 3

Status: Recruiting

Sponsored by: Genzyme, a Sanofi Company

Official(s) and/or principal investigator(s):
Clinical Sciences & Operations, Study Director, Affiliation: Sanofi

Overall contact:
For site information, send an email with site number to, Email: Contact-Us@sanofi.com

Summary

Primary Objective: Long-term safety and pharmacokinetics (PK) of neoGAA Secondary Objective: Long-term effect of neo-GAA on pharmacodynamic and exploratory efficacy variables

Clinical Details

Official title: An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of NeoGAA In Patients With Pompe Disease

Study design: Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome:

Assessment of adverse events (AEs) and treatment-emergent adverse events (TEAEs), including infusion-associated reactions (IARs) and deaths

Laboratory assessments including hematology, biochemistry and urinalysis

Vital signs

Secondary outcome:

Electrocardiogram

anti-neoGAA immunoglobulin G (IgG) antibodies, and neutralizing antibody formation in IgG seropositive patients; anti-alglucosidase alfa IgG antibodies

Cmax

AUC

t1/2

Skeletal muscle glycogen content

Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments

Urinary Hex4

plasma analyses of circulating mRNA and micro RNA

serum analyses of skeletal muscle RNA expression

Detailed description: The duration of the study will be 6 years from the date the first patient enters the study. Each patient will continue with the study until the patient withdraws, the Investigator withdraws the patient, or the Sponsor terminates the study.

Eligibility

Minimum age: N/A. Maximum age: N/A. Gender(s): Both.

Criteria:

Inclusion criteria: Patients with Pompe disease who previously completed a neoGAA study. The patient and/or their parent/legal guardian is willing and able to provide signed informed consent, and the patient, if <18 years of age, is willing to provide assent if deemed able to do so. The patient (and patient's legal guardian if patient is <18 years of age) must have the ability to comply with the clinical protocol. The patient, if female and of childbearing potential, must have a negative pregnancy test [urine beta-human chorionic gonadotropin] at baseline. Exclusion criteria: The patient is concurrently participating in another clinical study using investigational treatment. The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study. The patient has clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, precludes participation in the study or potentially decreases survival. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Locations and Contacts

For site information, send an email with site number to, Email: Contact-Us@sanofi.com

Investigational Site Number 056001, Leuven 3000, Belgium; Active, not recruiting

Investigational Site Number 208001, København Ø 2100, Denmark; Recruiting

Investigational Site Number 250003, Nice 06012, France; Active, not recruiting

Investigational Site Number 250002, Paris 75013, France; Active, not recruiting

Investigational Site Number 276003, Mainz 55131, Germany; Active, not recruiting

Investigational Site Number 276001, München 80336, Germany; Active, not recruiting

Investigational Site Number 276002, Münster 48149, Germany; Active, not recruiting

Investigational Site Number 528001, Rotterdam 3015 GJ, Netherlands; Active, not recruiting

Investigational Site Number 826003, Newcastle Upon Tyne NE1 4LP, United Kingdom; Active, not recruiting

Investigational Site Number 840006, Phoenix, Arizona 85013, United States; Recruiting

Investigational Site Number 840010, Jacksonville, Florida 32209, United States; Recruiting

Investigational Site Number 840001, Kansas City, Kansas 66160-7321, United States; Recruiting

Investigational Site Number 840008, St Louis, Missouri 63110, United States; Recruiting

Investigational Site Number 840002, Durham, North Carolina 27710, United States; Recruiting

Investigational Site Number 840009, Dallas, Texas 75390, United States; Recruiting

Investigational Site Number 840003, Fairfax, Virginia 22030, United States; Recruiting

Additional Information

Starting date: February 2014
Last updated: June 19, 2015

Page last updated: August 23, 2015

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