Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

NCT ID: NCT01898364

Last Updated: 2023-03-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-08-19
• Study Completion Date: 2015-02-25

Brief Summary

Primary Objective:

To evaluate the safety and tolerability of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.

Secondary Objective:

To evaluate the pharmacokinetics, pharmacodynamics of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.

To evaluate the effect of neoGAA on exploratory efficacy endpoints in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.

Detailed Description

Screening: within 90 days Period of treatment: 24 weeks (including 13 bi-weekly infusions) Post treatment evaluation visit: 2 weeks after last neoGAA infusion (at Week 27) End of study visit: 4 weeks after last neoGAA infusion (at Week 29) Total duration: approximately 41 weeks

Conditions

Pompe Disease; Glycogen Storage Disease Type II (GSD II); Acid Maltase Deficiency

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

GZ402666 (neoGAA) Group 1 - 5 mg

Intravenous infusion of 5mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks

Group Type: EXPERIMENTAL

GZ402666

Intervention Type: DRUG

Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous

GZ402666 (neoGAA) Group 1 - 10 mg

Intravenous infusion of 10mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks.

Group Type: EXPERIMENTAL

GZ402666

Intervention Type: DRUG

Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous

GZ402666 (neoGAA) Group 1 - 20 mg

Intravenous infusion of 20mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks.

Group Type: EXPERIMENTAL

GZ402666

Intervention Type: DRUG

Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous

GZ402666 (neoGAA) Group 2 - 5 mg

Intravenous infusion of 5mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa.

Group Type: EXPERIMENTAL

GZ402666

Intervention Type: DRUG

Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous

GZ402666 (neoGAA) Group 2 - 10 mg

Intravenous infusion of 10mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa.

Group Type: EXPERIMENTAL

GZ402666

Intervention Type: DRUG

Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous

GZ402666 (neoGAA) Group 2 - 20 mg

Intravenous infusion of 20mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa.

Group Type: EXPERIMENTAL

GZ402666

Intervention Type: DRUG

Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous

Interventions

GZ402666

Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

For both Group 1 and Group 2:

• Male or female patients with confirmed acid α-glucosidase (GAA) enzyme deficiency from any tissue source and/or confirmed GAA gene mutation and without known cardiac hypertrophy.
• Patient willing and able to provide signed informed consent
• Patient is able to ambulate 50 meters (approximately 160 feet) without stopping and without an assistive device. Use of assistive device for community ambulation is appropriate.
• Patient has a forced vital capacity (FVC) in upright position of ≥50% predicted.
• The patient, if female and of childbearing potential, must have a negative pregnancy test [urine beta-human chorionic gonadotropin (β-hCG)] at baseline.

Group 2 patients only:

• The patient has been previously treated with alglucosidase alfa for at least 9 months.

Exclusion Criteria

For both Group 1 and Group 2:

• Patient is wheelchair dependent.
• Patient requires invasive-ventilation (non-invasive ventilation is allowed).
• Patient is participating in another clinical study using investigational treatment.
• Patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
• 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.
• Patient cannot submit to MRI examination because of a formal contraindication such as a pacemaker, implanted ferromagnetic metals, anxiety disorder, etc.

Group 1 only:

• Patient has had previous treatment with alglucosidase alfa or any other enzyme replacement therapy (ERT) for Pompe disease.

Group 2 only:

• Patient has a high risk for a severe allergic reaction to neoGAA (i.e. previous moderate to severe anaphylactic reaction to alglucosidase alfa and/or patient has immunoglobulin (Ig) E antibodies to alglucosidase alfa, and/or a history of sustained high immunoglobulin G (IgG) antibody titers to alglucosidase alfa that in the opinion of the investigator suggest a high risk for an allergic reaction to neoGAA).

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

Investigational Site Number 840006

Phoenix, Arizona, United States

Investigational Site Number 840010

Jacksonville, Florida, United States

Investigational Site Number 840001

Kansas City, Kansas, United States

Investigational Site Number 840008

St Louis, Missouri, United States

Investigational Site Number 840002

Durham, North Carolina, United States

Investigational Site Number 840009

Dallas, Texas, United States

Investigational Site Number 840003

Fairfax, Virginia, United States

Investigational Site Number 056001

Leuven, , Belgium

Investigational Site Number 208001

København Ø, , Denmark

Investigational Site Number 250001

Marseille, , France

Investigational Site Number 250003

Nice, , France

Investigational Site Number 250002

Paris, , France

Investigational Site Number 276003

Mainz, , Germany

Investigational Site Number 276001

München, , Germany

Investigational Site Number 276002

Münster, , Germany

Investigational Site Number 528001

Rotterdam, , Netherlands

Investigational Site Number 826003

Newcastle upon Tyne, , United Kingdom

Countries

United States; Belgium; Denmark; France; Germany; Netherlands; United Kingdom

References

Dimachkie MM, Barohn RJ, Byrne B, Goker-Alpan O, Kishnani PS, Ladha S, Laforet P, Mengel KE, Pena LDM, Sacconi S, Straub V, Trivedi J, Van Damme P, van der Ploeg AT, Vissing J, Young P, Haack KA, Foster M, Gilbert JM, Miossec P, Vitse O, Zhou T, Schoser B; NEO-EXT investigators. Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease. Neurology. 2022 Aug 1;99(5):e536-e548. doi: 10.1212/WNL.0000000000200746.

Reference Type: DERIVED

PMID: 35618441 (View on PubMed)

Other Identifiers

2012-004167-42

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1144-7725

Identifier Type: OTHER

Identifier Source: secondary_id

TDR12857

Identifier Type: -

Identifier Source: org_study_id