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: 19 February 2018
• Main ID: EUCTR2016-000942-77-IT
• Date of registration: 06/02/2018
• Prospective Registration: No
• 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 Treatmentnaïve Patients with Late-onset Pompe Disease - COMET
• Date of first enrolment: 26/08/2016
• Target sample size: 90
• Recruitment status: Authorised-recruitment may be ongoing or finished
• 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

Austria, Belgium, Czech Republic, Denmark, Germany, Italy, Netherlands, Poland, Spain, Sweden, United Kingdom

Contacts

• Name: CONTACT POINT
• Affiliation: SANOFI S.p.A.

• Name: CONTACT POINT
• Affiliation: SANOFI S.p.A.

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 and/or their parent/legal guardian is willing and able to provide signed informed consent, and the patient, if <18 years of age, is willing to provide assent if deemed able to do so.
-The patient (and patient’s legal guardian if patient is <18 years of age) 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: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 84
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 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


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: Concentrate for solution for infusion
CAS Number: 1802558-87-7
Current Sponsor code: GZ402666
Other descriptive name: a-glucosidasi acida umana ricombinante coniugata con multiple copie di bis-mannosio-6- fosfato-tetra-mannosio glicano sintetico (NEOGAA)
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-

Trade Name: MYOZYME®
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
CAS Number: 420784-05-0
Current Sponsor code: .
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-

Primary Outcome(s)

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 Objective: To determine the safety and effect of neoGAA treatment on inspiratory muscle strength (maximum inspiratory pressure [MIP]), expiratory muscle strength (maximum expiratory pressure [MEP]), unctional endurance (6-minute walk test[6MWT]), 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

Secondary Outcome(s)

Secondary end point(s): Change from baseline in maximal
inspiratory pressure in upright position
Change from baseline in maximal expiratory pressure in upright position
Change from baseline in six-minute walk test scores
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

Timepoint(s) of evaluation of this end point: Baseline to 12 months

Secondary ID(s)

EFC14028

2016-000942-77-GB

Source(s) of Monetary Support

Genzyme Corporation