A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

• Register: EUCTR
• Last refreshed on: 9 March 2015
• Main ID: EUCTR2015-000512-18-Outside-EU/EEA
• Date of registration: 02/03/2015
• Prospective Registration: Yes
• Primary sponsor: Genzyme Corporation Inc
• Public title: A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
• Scientific title: A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale
• Target sample size: 114
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-000512-18
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1

Countries of recruitment

United States

Contacts

• Name: Trial Transparency Team
• Affiliation: Genzyme, a Sanofi Company

• Name: Trial Transparency Team
• Affiliation: Genzyme, a Sanofi Company

Key inclusion & exclusion criteria

Inclusion criteria:
1.The patient and/or their parent/legal guardian is willing and able to provide signed informed consent.
2.The patient must be at least 1 year of age at the time of informed consent.
3.The patient has a diagnosis of Pompe disease and must have received treatment with 160 L alglucosidase alfa prior to screening.
4.The patient, if female and of childbearing potential, must have a negative pregnancy test (urine beta-human chorionic gonadotropin) at baseline. Note: all female patients of childbearing potential and sexually mature males must agree to use a medically accepted method of contraception throughout the study.

Are the trial subjects under 18? yes
Number of subjects for this age range: 113
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1.The patient has within the past 3 months received or is currently receiving any investigational product other than 160 L alglucosidase alfa or is currently participating in another clinical treatment study.
2.The patient, in the opinion of the Investigator, is clinically unstable and would not be expected to survive to completion of the 52-week treatment period.
3.The patient and/or their parent/legal guardian, in the opinion of the Investigator, is unable to adhere to the requirements of the study.



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

Health Condition(s) or Problem(s) studied

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

Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 17.1 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 100000004850

Intervention(s)

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

Primary Outcome(s)

Secondary Objective: Not applicable

Timepoint(s) of evaluation of this end point: Percentage of participants who are clinically stable or improved at Week 52 [ Time Frame: Week 52 ]

Main Objective: The objective of this study is to evaluate the efficacy and safety of treatment with 4000 L alglucosidase alfa (Lumizyme®) in Pompe patients.

Primary end point(s): Percentage of participants who are clinically stable or improved at Week 52

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Percent survival at Week 52 [ Time Frame: Week 52 ]
Percentage of participants who are invasive ventilator-free at Week 52 [ Time Frame: Week 52 ]
Change from baseline on left ventricular mass Z-score (LVM-Z) [ Time Frame: Baseline and Week 52 ]
Change from baseline on Gross Motor Function Measure-88 (GMFM-88) Total Percent Score [ Time Frame: Baseline and Week 52 ]
Change from baseline in forced vital capacity (FVC) % predicted [ Time Frame: Baseline and Week 52 ]

Secondary end point(s): Percent survival

Percentage of participants who are invasive ventilator-free

Change from baseline on left ventricular mass Z-score (LVM-Z)

Change from baseline on Gross Motor Function Measure-88 (GMFM-88) Total Percent Score

Change from baseline in forced vital capacity (FVC) % predicted

Secondary ID(s)

AGLU09411/EFC12720

NCT01526785

Source(s) of Monetary Support

Genzyme Corporation Inc