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Avalglucosidase Alfa Extension Study (NEO-EXT)

Primary Purpose

Glycogen Storage Disease Type II Pompe Disease

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
GZ402666
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Glycogen Storage Disease Type II Pompe Disease

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Investigational Site Number 840006
  • Investigational Site Number 840010
  • Investigational Site Number 840001
  • Investigational Site Number 840008
  • Investigational Site Number 840002
  • Investigational Site Number 840011
  • Investigational Site Number 840009
  • Investigational Site Number 840003
  • Investigational Site Number 056001
  • Investigational Site Number 208001
  • Investigational Site Number 250003
  • Investigational Site Number 250002
  • Investigational Site Number 276003
  • Investigational Site Number 276001
  • Investigational Site Number 276002
  • Investigational Site Number 528001
  • Investigational Site Number 826003

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Avalglucosidase alfa

Arm Description

administered intravenously every 2 weeks

Outcomes

Primary Outcome Measures

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 Measures

Electrocardiogram
Anti-avalglucosidase alfa antibodies, and neutralizing antibody formation in anti-avalglucosidase alfa positive patients; anti-alglucosidase alfa IgG antibodies
Cmax (maximal concentration of the compound in the blood)
AUC (area under the curve, relates to the quantity of compound that produces an effect)
t1/2 (half-life, which is the time needed to eliminate half of the compound)
Skeletal muscle glycogen content
Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments
Urinary Hex4
Plasma analyses of circulating RNA
Serum analyses of skeletal muscle RNA expression

Full Information

First Posted
December 4, 2013
Last Updated
December 14, 2022
Sponsor
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT02032524
Brief Title
Avalglucosidase Alfa Extension Study
Acronym
NEO-EXT
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
December 2022
Overall Recruitment Status
Completed
Study Start Date
February 27, 2014 (Actual)
Primary Completion Date
December 12, 2022 (Actual)
Study Completion Date
December 12, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Glycogen Storage Disease Type II Pompe Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
19 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Avalglucosidase alfa
Arm Type
Experimental
Arm Description
administered intravenously every 2 weeks
Intervention Type
Drug
Intervention Name(s)
GZ402666
Other Intervention Name(s)
avalglucosidase alfa
Intervention Description
Pharmaceutical form: lyophilized powder reconstituted for infusion Route of administration: intravenous
Primary Outcome Measure Information:
Title
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
Title
Laboratory assessments including hematology, biochemistry and urinalysis
Time Frame
Monthly, from baseline until Year 3 then quarterly until approximately Year 8
Title
Vital signs
Time Frame
screening/baseline until approximately Year 8
Secondary Outcome Measure Information:
Title
Electrocardiogram
Time Frame
every 6 months, from baseline until approximately Year 8
Title
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
Title
Cmax (maximal concentration of the compound in the blood)
Time Frame
at 6 months, then yearly until approximately Year 8
Title
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
Title
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
Title
Skeletal muscle glycogen content
Time Frame
every 2 years, from baseline until approximately Year 8
Title
Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments
Time Frame
every 2 years, from baseline until approximately Year 8
Title
Urinary Hex4
Time Frame
at 6 months, then yearly until approximately Year 8
Title
Plasma analyses of circulating RNA
Time Frame
at 6 months, then yearly until approximately Year 8
Title
Serum analyses of skeletal muscle RNA expression
Time Frame
at 6 months, then yearly until approximately Year 8

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Sciences & Operations
Organizational Affiliation
Sanofi
Official's Role
Study Director
Facility Information:
Facility Name
Investigational Site Number 840006
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85013
Country
United States
Facility Name
Investigational Site Number 840010
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32209
Country
United States
Facility Name
Investigational Site Number 840001
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160-7321
Country
United States
Facility Name
Investigational Site Number 840008
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Investigational Site Number 840002
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Investigational Site Number 840011
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45219
Country
United States
Facility Name
Investigational Site Number 840009
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Investigational Site Number 840003
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Facility Name
Investigational Site Number 056001
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Investigational Site Number 208001
City
København Ø
ZIP/Postal Code
2100
Country
Denmark
Facility Name
Investigational Site Number 250003
City
Nice
ZIP/Postal Code
06000
Country
France
Facility Name
Investigational Site Number 250002
City
Paris
ZIP/Postal Code
75013
Country
France
Facility Name
Investigational Site Number 276003
City
Mainz
ZIP/Postal Code
55131
Country
Germany
Facility Name
Investigational Site Number 276001
City
München
ZIP/Postal Code
80336
Country
Germany
Facility Name
Investigational Site Number 276002
City
Münster
ZIP/Postal Code
48149
Country
Germany
Facility Name
Investigational Site Number 528001
City
Rotterdam
ZIP/Postal Code
3015 GJ
Country
Netherlands
Facility Name
Investigational Site Number 826003
City
Newcastle Upon Tyne
ZIP/Postal Code
NE1 4LP
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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

Learn more about this trial

Avalglucosidase Alfa Extension Study

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