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-NL
- Lead Sponsor
- Genzyme Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 96
-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.
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 88
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 stopping 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 (and patient’s legal guardian if patient is legally minor as defined by local regulation) is (are) not able to comply with the clinical protocol.
-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
- The patient, if female and of childbearing potential, has a positive pregnancy test (beta-human chorionic gonadotropin) at baseline.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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 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).<br>;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
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Change from baseline in six-minute walk test scores <br><br>- Change from baseline in maximal inspiratory pressure in upright position<br><br>- Change from baseline in maximal expiratory pressure in upright position<br><br>- Change from baseline in hand-held dynamometry measurement<br><br>- Change from baseline in Quick Motor Function Test scores<br><br>- Change from baseline in 12- Item Short-form health survey scores<br><br>- Number of participants with adverse events <br><br> <br>;Timepoint(s) of evaluation of this end point: Baseline to 12 months