Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients with Late-onset Pompe Disease

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-000942-77-GB

Enrollment

100

Registered

2016-05-04

Start date

2016-07-15

Completion date

Unknown

Last updated

2019-06-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 100000004850

Interventions

Product Name: neoGAA or avalglucosidase alfa Product Code: GZ402666 Pharmaceutical Form: Powder for concentrate for solution for infusion CAS Number: 18

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: -The patient has confirmed GAA enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations. -The patient must provide signed, informed consent prior to performing any study related procedures. Consent of a legally authorized guardian(s) is (are) required for legally minor patients as defined by local regulation. If the patient is legally minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from patients, if applicable. -The patient (and patient's legal guardian if patient is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol. -The patient, if female and of childbearing potential, must have a negative pregnancy test (betahuman chorionic gonadotropin) at baseline. Are the trial subjects under 18? yes Number of subjects for this age range: 7 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 122 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 17

Exclusion criteria

Exclusion criteria: -The patient is <3 years of age. -The patient has known Pompe specific cardiac hypertrophy. -The patient is wheelchair dependent. -The patient is not able to ambulate 40 meters (approximately 130 feet) without topping and without an assistive device. -The patient requires invasive-ventilation (non-invasive ventilation is allowed). -The patient is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of =30% predicted and =85% predicted. -The patient has had previous treatment with alglucosidase alfa or any investigational therapy for Pompe disease. -The patient has prior or current use of immune tolerance induction therapy

Design outcomes

Primary

Measure	Time frame
Main Objective: To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength measured by percent predicted forced vital capacity (% FVC) in the upright position, as compared to alglucosidase alfa.;Secondary Objective: To determine the safety and effect of avalglucosidase alfa treatment on functional endurance (6-minute walk test [6MWT], inspiratory muscle strength (maximum inspiratory pressure [MIP]), expiratory muscle strength (maximum expiratory pressure [MEP]), lower extremity muscle strength (hand-held dynamometry [HHD]), motor function (Quick Motor Function Test [QMFT]), and health-related quality of life (SF-12).;Primary end point(s): Change from baseline in percent predicted forced vital capacity (%FVC) in upright position;Timepoint(s) of evaluation of this end point: Baseline to 12 months	—

Measure

Time frame

Main Objective: To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength measured by percent predicted forced vital capacity (% FVC) in the upright position, as compared to alglucosidase alfa.;Secondary Objective: To determine the safety and effect of avalglucosidase alfa treatment on functional endurance (6-minute walk test [6MWT], inspiratory muscle strength (maximum inspiratory pressure [MIP]), expiratory muscle strength (maximum expiratory pressure [MEP]), lower extremity muscle strength (hand-held dynamometry [HHD]), motor function (Quick Motor Function Test [QMFT]), and health-related quality of life (SF-12).;Primary end point(s): Change from baseline in percent predicted forced vital capacity (%FVC) in upright position;Timepoint(s) of evaluation of this end point: Baseline to 12 months

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Secondary

Measure	Time frame
Secondary end point(s): Change from baseline in six-minute walk test scores Change from baseline in maximal inspiratory pressure in upright position Change from baseline in maximal expiratory pressure in upright position Change from baseline in hand-held dynamometry measurement Change from baseline in Quick Motor Function Test scores Change from baseline in 12-Item Short-form health survey scores Number of participants with adverse events ; Timepoint(s) of evaluation of this end point: Baseline to 49 weeks Baseline to 49 weeks Baseline to 49 weeks Baseline to 49 weeks Baseline to 49 weeks Baseline to 49 weeks Baseline to 49 weeks Baseline to 49 weeks and up to 6 years	—

Measure

Time frame

Secondary end point(s): Change from baseline in six-minute walk test scores Change from baseline in maximal inspiratory pressure in upright position Change from baseline in maximal expiratory pressure in upright position Change from baseline in hand-held dynamometry measurement Change from baseline in Quick Motor Function Test scores Change from baseline in 12-Item Short-form health survey scores Number of participants with adverse events ; Timepoint(s) of evaluation of this end point: Baseline to 49 weeks Baseline to 49 weeks Baseline to 49 weeks Baseline to 49 weeks Baseline to 49 weeks Baseline to 49 weeks Baseline to 49 weeks Baseline to 49 weeks and up to 6 years

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Countries

Argentina, Austria, Belgium, Brazil, Bulgaria, Canada, Colombia, Czech Republic, Denmark, France, Germany, Hungary, Italy, Japan, Korea, Republic of, Mexico, Netherlands, Poland, Portugal, Russian Federation, Spain, Sweden, Switzerland, Taiwan, Turkey, United Kingdom, United States

Contacts

Public ContactMedical Information Genzyme Europe

Genzyme Europe B.V.

eumedinfo@genzyme.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026