Avalglucosidase Alfa Extension Study

• Register: EUCTR
• Last refreshed on: 1 February 2020
• Main ID: EUCTR2013-003321-28-BE
• Date of registration: 17/02/2014
• Prospective Registration: Yes
• Primary sponsor: Genzyme Corporation
• Public title: Avalglucosidase Alfa Extension Study
• Scientific title: An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of Avalglucosidase Alfa (NeoGAA, GZ402666) In Patients With Pompe Disease
• Date of first enrolment: 12/05/2014
• Target sample size: 21
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-003321-28
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; 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
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, Denmark, France, Germany, Italy, Netherlands, United Kingdom, United States

Contacts

• Name: Medical Information Genzyme Europe
• Affiliation: Genzyme Europe B.V.

• Name: Medical Information Genzyme Europe
• Affiliation: Genzyme Europe B.V.

Key inclusion & exclusion criteria

Inclusion criteria:
Patients with Pompe disease who previously completed an avalglucosidase alfa study.
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 [urine beta-human chorionic gonadotropin] at baseline

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 17
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4

Exclusion criteria:
The patient is concurrently participating in another clinical study using investigational treatment.
The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
The patient has clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, precludes participation in the study or potentially decreases survival.



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: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1 Level: PT Classification code 10053185 Term: Glycogen storage disease type II System Organ Class: 10010331 - Congenital, familial and genetic disorders

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

Intervention(s)

Product Name: Avalglucosidase Alfa
Product Code: GZ402666
Pharmaceutical Form: Powder for concentrate for solution for infusion
Current Sponsor code: GZ402666
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-

Primary Outcome(s)

Main Objective: Long-term safety and pharmacokinetics (PK) of avalglucosidase alfa

Primary end point(s): 1) Assessment of adverse events (AEs) and treatment-emergent adverse events (TEAEs), including infusion-associated reactions (IARs) and deaths
2) Laboratory assessments including hematology, biochemistry and urinalysis
3) Vital signs

Timepoint(s) of evaluation of this end point: 1) and 3) Screening/baseline until year 8
2) Monthly, from baseline until Year 3 then quaterly until Year 8

Secondary Objective: Long-term effect of avalglucosidase alfa on pharmacodynamic and exploratory efficacy variables.

Secondary Outcome(s)

Secondary end point(s): 1) Electrocardiogram
2) Anti-avalglucosidase alfa antibodies,and neutralizing antibody formation in anti-avalglucosidase alfa positive patients;
anti-alglucosidase alfa IgG antibodies
3) Cmax (Maximal concentration of the compound in the blood)
4) AUC (Area under the curve, relates to the quantity of compound that produces an effect)
5) t1/2 (half-life, which is the time needed to eliminate half of the compound)
6) Skeletal muscle glycogen content
7) Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments
8) Urinary Hex4
9) Serum analyses of skeletal muscle RNA expression
10) Plasma analyses of circulating mRNA and micro RNA

Timepoint(s) of evaluation of this end point: 1) Every 6 months, from baseline until year 8
2) Monthly, from baseline up to 6 months, then every 3 months until Year 8 for Anti-avalglucosidase alfa antibodies,and neutralizing antibody formation in anti-avalglucosidase alfa positive patients; every 6 months from baseline until Year 8 for anti-alglucosidase alfa IgG antibodies
3), 4), 5), 8), 9) and 10) at 6 months, then yearly until Year 8
6) and 7) Every 2 years, from baseline until Year 8

Secondary ID(s)

LTS13769

Source(s) of Monetary Support

Genzyme Corporation

Ethics review

Status: Approved
Approval date: 12/05/2014
Contact: