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Avalglucosidase Alfa Extension Study (NEO-EXT)

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.
 
ClinicalTrials.gov Identifier: NCT02032524
Recruitment Status : Completed
First Posted : January 10, 2014
Last Update Posted : December 15, 2022
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Tracking Information
First Submitted Date  ICMJE December 4, 2013
First Posted Date  ICMJE January 10, 2014
Last Update Posted Date December 15, 2022
Actual Study Start Date  ICMJE February 27, 2014
Actual Primary Completion Date December 12, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 16, 2019)
  • Assessment of adverse events (AEs) and treatment-emergent adverse events (TEAEs), including infusion-associated reactions (IARs) and deaths [ Time Frame: screening/baseline until approximately Year 8 ]
  • Laboratory assessments including hematology, biochemistry and urinalysis [ Time Frame: Monthly, from baseline until Year 3 then quarterly until approximately Year 8 ]
  • Vital signs [ Time Frame: screening/baseline until approximately Year 8 ]
Original Primary Outcome Measures  ICMJE
 (submitted: January 8, 2014)
  • Assessment of adverse events (AEs) and treatment-emergent adverse events (TEAEs) [ 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 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 16, 2019)
  • Electrocardiogram [ Time Frame: every 6 months, from baseline until approximately Year 8 ]
  • Anti-avalglucosidase alfa antibodies, and neutralizing antibody formation in anti-avalglucosidase alfa positive patients; anti-alglucosidase alfa IgG antibodies [ Time Frame: monthly, from baseline up to 6 months, then every 3 months until approximately Year 8; every 6 months from baseline until approximately Year 8 ]
  • Cmax (maximal concentration of the compound in the blood) [ Time Frame: at 6 months, then yearly until approximately Year 8 ]
  • AUC (area under the curve, relates to the quantity of compound that produces an effect) [ Time Frame: at 6 months, then yearly until approximately Year 8 ]
  • t1/2 (half-life, which is the time needed to eliminate half of the compound) [ Time Frame: at 6 months, then yearly until approximately Year 8 ]
  • Skeletal muscle glycogen content [ Time Frame: every 2 years, from baseline until approximately Year 8 ]
  • Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments [ Time Frame: every 2 years, from baseline until approximately Year 8 ]
  • Urinary Hex4 [ Time Frame: at 6 months, then yearly until approximately Year 8 ]
  • Plasma analyses of circulating RNA [ Time Frame: at 6 months, then yearly until approximately Year 8 ]
  • Serum analyses of skeletal muscle RNA expression [ Time Frame: at 6 months, then yearly until approximately Year 8 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: January 8, 2014)
  • Electrocardiogram [ Time Frame: every 6 months, from baseline until year 6 ]
  • Immunogenicity assessments (anti-neoGAA immunoglobulin G (IgG) antibodies, neutralizing antibody formation in IgG seropositive patients, and anti-alglucosidase alfa IgG antibodies) [ Time Frame: 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Avalglucosidase Alfa Extension Study
Official Title  ICMJE An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of Avalglucosidase Alfa In Patients With Pompe Disease
Brief Summary

Primary Objective:

Long-term safety and pharmacokinetics (PK) of avalglucosidase alfa

Secondary Objective:

Long-term effect of avalglucosidase alfa on pharmacodynamic and exploratory efficacy variables

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.

An additional follow-up phase will begin after the patient's last planned visit in the 6-year study and last until avalglucosidase alfa is approved in the patient's country for all patients.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Glycogen Storage Disease Type II Pompe Disease
Intervention  ICMJE Drug: GZ402666
Pharmaceutical form: lyophilized powder reconstituted for infusion Route of administration: intravenous
Other Name: avalglucosidase alfa
Study Arms  ICMJE Experimental: Avalglucosidase alfa
administered intravenously every 2 weeks
Intervention: Drug: GZ402666
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 3, 2021)
19
Original Estimated Enrollment  ICMJE
 (submitted: January 8, 2014)
18
Actual Study Completion Date  ICMJE December 12, 2022
Actual Primary Completion Date December 12, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion criteria:

Patients with Pompe disease who previously completed a an avalglucosidase 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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   Denmark,   France,   Germany,   Netherlands,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02032524
Other Study ID Numbers  ICMJE LTS13769
U1111-1147-3439 ( Other Identifier: UTN )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Current Responsible Party Sanofi ( Genzyme, a Sanofi Company )
Original Responsible Party Sanofi
Current Study Sponsor  ICMJE Genzyme, a Sanofi Company
Original Study Sponsor  ICMJE Sanofi
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Clinical Sciences & Operations Sanofi
PRS Account Sanofi
Verification Date December 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP