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

• Register: EUCTR
• Last refreshed on: 4 March 2024
• Main ID: EUCTR2016-000942-77-CZ
• Date of registration: 27/06/2016
• Prospective Registration: Yes
• Primary sponsor: Genzyme Corporation
• Public title: 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
• Scientific title: A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease
• Date of first enrolment: 05/10/2016
• Target sample size: 96
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-000942-77
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: yes Placebo: no Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Argentina, Australia, 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

• Name: www.sanofi.cz
• Affiliation: sanofi-aventis. s.r.o.

• Name: www.sanofi.cz
• Affiliation: sanofi-aventis. s.r.o.

Key inclusion & exclusion 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.
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

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 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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

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

Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Intervention(s)

Product Name: neoGAA
Product Code: GZ402666
Pharmaceutical Form: Powder for concentrate for solution for infusion
CAS Number: 1802558-87-7
Current Sponsor code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-

Trade Name: MYOZYME®
Product Name: Myozyme®
Pharmaceutical Form: Powder for concentrate for solution for infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
CAS Number: 420784-05-0
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-

Primary Outcome(s)

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).

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.

Primary end point(s): Change from baseline in percent predicted forced vital capacity (%FVC) in upright position

Secondary Outcome(s)

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 12 months

Secondary ID(s)

2016-000942-77-GB

EFC14028

Source(s) of Monetary Support

Genzyme Corporation

Ethics review

Status: Approved
Approval date: 13/09/2016
Contact:

Results

• Results available: Yes
• Date Posted: 12/06/2021
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2016-000942-77/results