Avalglucosidase Alfa Extension Study

• Register: ClinicalTrials.gov
• Last refreshed on: 11 March 2024
• Main ID: NCT02032524
• Date of registration: 04/12/2013
• Prospective Registration: Yes
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: Avalglucosidase Alfa Extension Study NEO-EXT
• Scientific title: An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety and Pharmacokinetics of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) in Patients With Pompe Disease
• Date of first enrolment: February 27, 2014
• Target sample size: 19
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02032524
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Belgium, Denmark, France, Germany, Netherlands, United Kingdom, United States

Contacts

• Name: Clinical Sciences & Operations
• Affiliation: Sanofi

Key inclusion & exclusion criteria

Inclusion criteria:

Participants with Pompe disease who previously completed an avalglucosidase study.

The participant and/or their parent/legal guardian is willing and able to provide signed
informed consent, and the participant, if <18 years of age, was willing to provide assent
if deemed able to do so.

The participant (and participant's legal guardian if participant is <18 years of age) must
have had the ability to comply with the clinical protocol.

The participant, if female and of childbearing potential, had to have a negative pregnancy
test [urine beta-human chorionic gonadotropin] at baseline.

Exclusion criteria:

The participant was concurrently participating in another clinical study using
investigational treatment.

The participant, in the opinion of the Investigator, was unable to adhere to the
requirements of the study.

The participant had 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, precluded
participation in the study or potentially decreases survival.

The above information was not intended to contain all considerations relevant to a
participant's potential participation in a clinical trial.



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Glycogen Storage Disease Type II Pompe Disease

Intervention(s)

Drug: Avalglucosidase Alfa

Primary Outcome(s)

Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative [Time Frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks]

Terminal Half-Life (t1/2z) of Avalglucosidase Alfa [Time Frame: Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312]

Change From Baseline in Urine Hyaline Casts up to Last IMP Administration [Time Frame: Baseline (Day 1) and last on-treatment values (up to 454 weeks)]

Change From Baseline in Urine Leukocytes [White Blood Cell (WBC)] up to Last IMP Administration [Time Frame: Baseline (Day 1) and last on-treatment values (up to 454 weeks)]

Number of Participants With Body Weight Increased/Decreased [Time Frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks]

Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities [Time Frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks]

Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities [Time Frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks]

Time Corresponding to the Last Concentration (Tlast) of Avalglucosidase Alfa [Time Frame: Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312]

Apparent Total Body Clearance Steady-State (CLss) of Avalglucosidase Alfa [Time Frame: Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312]

Change From Baseline in Urine pH up to Last IMP Administration [Time Frame: Baseline (Day 1) and last on-treatment values (up to 454 weeks)]

Change From Baseline in Urine Specific Gravity up to Last IMP Administration [Time Frame: Baseline (Day 1) and last on-treatment values (up to 454 weeks)]

Maximum Observed Plasma Concentration (Cmax) of Avalglucosidase Alfa [Time Frame: Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312]

Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths [Time Frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks]

Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry [Time Frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks]

Number of Participants With Potentially Clinically Significant Abnormalities in Hematology [Time Frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks]

Apparent Volume of Distribution Steady-State (Vss) of Avalglucosidase Alfa [Time Frame: Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312]

Area Under the Plasma Concentration Versus Time Curve From Time Zero to the Real Time (AUClast) of Avalglucosidase Alfa [Time Frame: Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312]

Change From Baseline in Urine BUN up to Last IMP Administration [Time Frame: Baseline (Day 1) and last on-treatment values (up to 454 weeks)]

Number of Participants With Clinically Significant Physical Examination Abnormalities [Time Frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks]

Secondary Outcome(s)

Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 [Time Frame: Baseline (Day 1) and Weeks 27, 104, 208, 260 and 312]

Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 [Time Frame: Baseline (Day 1) and Weeks 104 and 442]

Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 [Time Frame: Baseline (Day 1) and Weeks 104 and 442]

Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 [Time Frame: Baseline (Day 1) and Weeks 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 52, 78, 104, 130, 156, 182, 208, 234, 260, 286, 312, 338, 364, 390, 416 and 442]

Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 [Time Frame: Baseline (Day 1) and Weeks 104 and 442]

Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 [Time Frame: Baseline (Day 1) and Weeks 104 and 442]

Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 [Time Frame: Baseline (Day 1) and Weeks 104 and 442]

Secondary ID(s)

LTS13769

U1111-1147-3439

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 01/03/2024
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02032524