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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01898364
Other study ID # TDR12857
Secondary ID 2012-004167-42U1
Status Completed
Phase Phase 1
First received
Last updated
Start date August 19, 2013
Est. completion date February 25, 2015

Study information

Verified date March 2023
Source Sanofi
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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


Recruitment information / eligibility

Status Completed
Enrollment 24
Est. completion date February 25, 2015
Est. primary completion date February 25, 2015
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion criteria : For both Group 1 and Group 2: - Male or female patients with confirmed acid a-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 (ß-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.

Study Design


Intervention

Drug:
GZ402666
Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous

Locations

Country Name City State
Belgium Investigational Site Number 056001 Leuven
Denmark Investigational Site Number 208001 København Ø
France Investigational Site Number 250001 Marseille
France Investigational Site Number 250003 Nice
France Investigational Site Number 250002 Paris
Germany Investigational Site Number 276003 Mainz
Germany Investigational Site Number 276001 München
Germany Investigational Site Number 276002 Münster
Netherlands Investigational Site Number 528001 Rotterdam
United Kingdom Investigational Site Number 826003 Newcastle Upon Tyne
United States Investigational Site Number 840009 Dallas Texas
United States Investigational Site Number 840002 Durham North Carolina
United States Investigational Site Number 840003 Fairfax Virginia
United States Investigational Site Number 840010 Jacksonville Florida
United States Investigational Site Number 840001 Kansas City Kansas
United States Investigational Site Number 840006 Phoenix Arizona
United States Investigational Site Number 840008 Saint Louis Missouri

Sponsors (1)

Lead Sponsor Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

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

Outcome

Type Measure Description Time frame Safety issue
Primary Adverse events screening/baseline to Week 25
Primary Laboratory assessments including hematology, biochemistry and urinalysis screening/baseline to Week 25
Primary Vital signs screening/baseline to Week 25
Secondary Electrocardiogram screening/baseline, Week 1, Week 13, Week 25
Secondary Immunogenicity assessments screening/baseline to Week 29
Secondary Cmax Week 1, Week 13, Week 25
Secondary AUC Week 1, Week 13, Week 25
Secondary t1/2 Week 1, Week 13, Week 25
Secondary Skeletal muscle glycogen content screening/baseline, Week 27
Secondary Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments. screening/baseline, Week 27
Secondary Urinary Hex4 screening/baseline to Week 25
Secondary Functional assessments including 6 Minute Walk Test (6MWT) Functional Assessment includes - pulmonary function testing (PFT) endpoints, Gait, Stair, Gower's Maneuver, Chair (GSGC), Gross Motor Function Measure-88 (GMFM-88), Quick Motor Function Test (QMFT), hand-held dynamometer testing, Pediatric Quality of Life Inventory Multidimensional Fatigue Scale (PedsQL) screening/baseline, Week 13, Week 25
Secondary Quality of life assessments screening/baseline, Week 13, Week 25
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