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Trial record 1 of 1 for:    NEO-EXT | Pompe Disease
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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
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

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

Condition or disease Intervention/treatment Phase
Glycogen Storage Disease Type II Pompe Disease Drug: GZ402666 Phase 2 Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Allocation: N/A
Intervention Model: Single Group 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 Avalglucosidase Alfa In Patients With Pompe Disease
Actual Study Start Date : February 27, 2014
Actual Primary Completion Date : December 12, 2022
Actual Study Completion Date : December 12, 2022

Arm Intervention/treatment
Experimental: Avalglucosidase alfa
administered intravenously every 2 weeks
Drug: GZ402666
Pharmaceutical form: lyophilized powder reconstituted for infusion Route of administration: intravenous
Other Name: avalglucosidase alfa

Primary Outcome Measures :
  1. 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 ]
  2. Laboratory assessments including hematology, biochemistry and urinalysis [ Time Frame: Monthly, from baseline until Year 3 then quarterly until approximately Year 8 ]
  3. Vital signs [ Time Frame: screening/baseline until approximately Year 8 ]

Secondary Outcome Measures :
  1. Electrocardiogram [ Time Frame: every 6 months, from baseline until approximately Year 8 ]
  2. 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 ]
  3. Cmax (maximal concentration of the compound in the blood) [ Time Frame: at 6 months, then yearly until approximately Year 8 ]
  4. 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 ]
  5. 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 ]
  6. Skeletal muscle glycogen content [ Time Frame: every 2 years, from baseline until approximately Year 8 ]
  7. Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments [ Time Frame: every 2 years, from baseline until approximately Year 8 ]
  8. Urinary Hex4 [ Time Frame: at 6 months, then yearly until approximately Year 8 ]
  9. Plasma analyses of circulating RNA [ Time Frame: at 6 months, then yearly until approximately Year 8 ]
  10. Serum analyses of skeletal muscle RNA expression [ Time Frame: at 6 months, then yearly until approximately Year 8 ]

Information from the National Library of Medicine

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

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.

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

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Sponsors and Collaborators
Genzyme, a Sanofi Company
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Study Director: Clinical Sciences & Operations Sanofi
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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT02032524    
Other Study ID Numbers: LTS13769
U1111-1147-3439 ( Other Identifier: UTN )
First Posted: January 10, 2014    Key Record Dates
Last Update Posted: December 15, 2022
Last Verified: December 2022
Individual Participant Data (IPD) Sharing Statement:
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
Additional relevant MeSH terms:
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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
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors