Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease
- Conditions
- Pompe disease (acid alpha-glucosidase deficiency)MedDRA version: 20.0Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2016-000942-77-SE
- Lead Sponsor
- Genzyme Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 100
-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 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: 2
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 94
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4
-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 =40% 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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Secondary Objective: To determine the safety and effect of neoGAA 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;Main Objective: To determine the effect of neoGAA (GZ402666) treatment on respiratory muscle strength measured by percent predicted forced vital capacity (% FVC) in the upright position, as compared to alglucosidase alfa.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Change from baseline in six-minute walk test scores<br>- Change from baseline in maximal inspiratory pressure in upright position<br>- Change from baseline in maximal expiratory pressure in upright position<br>- Change from baseline in hand-held dynamometry measurement<br>- Change from baseline in Quick Motor Function Test scores<br>- Change from baseline in 12- Item Short-form health survey scores<br>- Number of participants with adverse events;Timepoint(s) of evaluation of this end point: Baseline to 12 months