Avalglucosidase Alfa Extension Study
Glycogen Storage Disease Type II Pompe Disease
Bottom Line
View on ClinicalTrials.gov: NCT02032524 ↗Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Avalglucosidase Alfa (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Genzyme, a Sanofi Company
- Primary completion
- Dec 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths |
4; 3; 3; 4; 4; 6 | — |
| PRIMARY Number of Participants With Clinically Significant Physical Examination Abnormalities |
0; 0; 0; 0; 0; 1 | — |
| PRIMARY Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry |
2; 2; 2; 1; 2; 1 | — |
| PRIMARY Number of Participants With Potentially Clinically Significant Abnormalities in Hematology |
1; 0; 1; 0; 0; 1 | — |
| PRIMARY Change From Baseline in Urine BUN up to Last IMP Administration |
60.8; -17.6; 179.5; 72.9; 37.1; 118.8 | — |
| PRIMARY Change From Baseline in Urine Hyaline Casts up to Last IMP Administration |
2.0 | — |
| PRIMARY Change From Baseline in Urine Leukocytes [White Blood Cell (WBC)] up to Last IMP Administration |
-1.0 | — |
| PRIMARY Change From Baseline in Urine Specific Gravity up to Last IMP Administration |
0.0; 0.0; 0.0; 0.0; 0.0; 0.0 | — |
| PRIMARY Change From Baseline in Urine pH up to Last IMP Administration |
-0.4; 0.2; -0.3; 0.1; -0.8; -0.3 | — |
| PRIMARY Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities |
2; 2; 2; 2; 3; 2 | — |
| PRIMARY Number of Participants With Body Weight Increased/Decreased |
1; 0; 0; 1; 0; 2 | — |
| PRIMARY Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities |
1; 0; 0; 0; 1; 0 | — |
| PRIMARY Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative |
0; 1; 0; 1; 1; 3 | — |
| PRIMARY Maximum Observed Plasma Concentration (Cmax) of Avalglucosidase Alfa |
269; 234 | — |
| PRIMARY Area Under the Plasma Concentration Versus Time Curve From Time Zero to the Real Time (AUClast) of Avalglucosidase Alfa |
1350; 1290 | — |
| PRIMARY Time Corresponding to the Last Concentration (Tlast) of Avalglucosidase Alfa |
25.84; 27.6 | — |
| PRIMARY Terminal Half-Life (t1/2z) of Avalglucosidase Alfa |
1.62; 1.79 | — |
| PRIMARY Apparent Total Body Clearance Steady-State (CLss) of Avalglucosidase Alfa |
0.90; 1.06 | — |
| PRIMARY Apparent Volume of Distribution Steady-State (Vss) of Avalglucosidase Alfa |
3.04; 3.8 | — |
| SECONDARY Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 |
57.4; -0.9; 46.1; 26.7; 123.0; -135.7 | — |
| SECONDARY Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 |
0.9; 1.4; -0.7; 0.3; 5.7; -0.2 | — |
| SECONDARY Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 |
44.8; -13.8; -5.8; 21.8; -131.6; -100.9 | — |
| SECONDARY Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 |
-0.6; 0.5; 0.8; 0.0; 0.7; 1.6 | — |
| SECONDARY Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 |
-0.5; 0.5; 1.4; 0.3; -0.4; 2.3 | — |
| SECONDARY Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 |
0.8; 0.9; -3.6; 0.5; -0.4; -4.8 | — |
| SECONDARY Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 |
0.5; -0.6; -1.9; -0.9; -0.9; -0.5 | — |
Summary
Eligibility 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.
Data sourced from ClinicalTrials.gov (NCT02032524). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.