Avalglucosidase Alfa Extension Study (NEO-EXT)
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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 |
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Recruitment Status :
Completed
First Posted : January 10, 2014
Last Update Posted : December 15, 2022
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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 |
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 : | 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 |
|---|---|
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Experimental: Avalglucosidase alfa
administered intravenously every 2 weeks
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Drug: GZ402666
Pharmaceutical form: lyophilized powder reconstituted for infusion Route of administration: intravenous
Other Name: avalglucosidase alfa |
- 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 ]
- 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 ]
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, 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.
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
Show 17 study locations
| Study Director: | Clinical Sciences & Operations | Sanofi |
| 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 |
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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 |