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NeoGAA Extension Study (NEO-EXT)

This study is enrolling participants by invitation only.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02032524
First Posted: January 10, 2014
Last Update Posted: June 2, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
  Purpose

Primary Objective:

Long-term safety and pharmacokinetics (PK) of neoGAA

Secondary Objective:

Long-term effect of neo-GAA on pharmacodynamic and exploratory efficacy variables


Condition Intervention Phase
Glycogen Storage Disease Type II Pompe Disease Drug: GZ402666 Phase 2 Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Sanofi ( Genzyme, a Sanofi Company ):

Primary Outcome Measures:
  • Assessment of adverse events (AEs) and treatment-emergent adverse events (TEAEs), including infusion-associated reactions (IARs) and deaths [ Time Frame: screening/baseline until year 6 ]
  • Laboratory assessments including hematology, biochemistry and urinalysis [ Time Frame: Monthly, from baseline until year 6 ]
  • Vital signs [ Time Frame: screening/baseline until year 6 ]

Secondary Outcome Measures:
  • Electrocardiogram [ Time Frame: every 6 months, from baseline until year 6 ]
  • anti-neoGAA immunoglobulin G (IgG) antibodies, and neutralizing antibody formation in IgG seropositive patients; anti-alglucosidase alfa IgG antibodies [ Time Frame: monthly, from baseline up to 6 months, then every 3 months until year 6; every 6 months from baseline until year 6 ]
  • Cmax [ Time Frame: at 6 months, then yearly until year 6 ]
  • AUC [ Time Frame: at 6 months, then yearly until year 6 ]
  • t1/2 [ Time Frame: at 6 months, then yearly until year 6 ]
  • Skeletal muscle glycogen content [ Time Frame: every 2 years, from baseline until year 6 ]
  • Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments [ Time Frame: every 2 years, from baseline until year 6 ]
  • Urinary Hex4 [ Time Frame: every 6 months, from baseline until year 6 ]
  • plasma analyses of circulating mRNA and micro RNA [ Time Frame: every 6 months, from baseline until year 6 ]
  • serum analyses of skeletal muscle RNA expression [ Time Frame: every 2 years, from baseline until year 6 ]

Estimated Enrollment: 21
Study Start Date: February 2014
Estimated Study Completion Date: May 2020
Estimated Primary Completion Date: April 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: neoGAA 5mg/kg
5 mg/kg every 2 weeks, intravenous
Drug: GZ402666
Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous
Experimental: neoGAA 10mg/kg
10 mg/kg every 2 weeks, intravenous
Drug: GZ402666
Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous
Experimental: neoGAA 20mg/kg
20 mg/kg every 2 weeks, intravenous
Drug: GZ402666
Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous

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

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02032524


Locations
United States, Arizona
Investigational Site Number 840006
Phoenix, Arizona, United States, 85013
United States, Florida
Investigational Site Number 840010
Jacksonville, Florida, United States, 32209
United States, Kansas
Investigational Site Number 840001
Kansas City, Kansas, United States, 66160-7321
United States, Missouri
Investigational Site Number 840008
Saint Louis, Missouri, United States, 63110
United States, North Carolina
Investigational Site Number 840002
Durham, North Carolina, United States, 27710
United States, Texas
Investigational Site Number 840009
Dallas, Texas, United States, 75390
United States, Virginia
Investigational Site Number 840003
Fairfax, Virginia, United States, 22030
Belgium
Investigational Site Number 056001
Leuven, Belgium, 3000
Denmark
Investigational Site Number 208001
København Ø, Denmark, 2100
France
Investigational Site Number 250003
Nice, France, 06000
Investigational Site Number 250002
Paris, France, 75013
Germany
Investigational Site Number 276003
Mainz, Germany, 55131
Investigational Site Number 276001
München, Germany, 80336
Investigational Site Number 276002
Münster, Germany, 48149
Netherlands
Investigational Site Number 528001
Rotterdam, Netherlands, 3015 GJ
United Kingdom
Investigational Site Number 826003
Newcastle Upon Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Clinical Sciences & Operations Sanofi
  More Information

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT02032524     History of Changes
Other Study ID Numbers: LTS13769
U1111-1147-3439 ( Other Identifier: UTN )
First Submitted: December 4, 2013
First Posted: January 10, 2014
Last Update Posted: June 2, 2017
Last Verified: June 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