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

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ClinicalTrials.gov Identifier: NCT01898364
Recruitment Status : Completed
First Posted : July 12, 2013
Last Update Posted : March 11, 2015
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

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.


Condition or disease Intervention/treatment Phase
Pompe Disease Glycogen Storage Disease Type II (GSD II) Acid Maltase Deficiency Drug: GZ402666 Phase 1

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in naïve and Alglucosidase Alfa Treated Late-onset Pompe Disease Patients.
Study Start Date : July 2013
Primary Completion Date : February 2015
Study Completion Date : February 2015


Arm Intervention/treatment
Experimental: 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
Drug: GZ402666
Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous
Experimental: 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.
Drug: GZ402666
Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous
Experimental: 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.
Drug: GZ402666
Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous
Experimental: 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.
Drug: GZ402666
Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous
Experimental: 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.
Drug: GZ402666
Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous
Experimental: 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.
Drug: GZ402666
Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous



Primary Outcome Measures :
  1. Adverse events [ Time Frame: screening/baseline to Week 25 ]
  2. Laboratory assessments including hematology, biochemistry and urinalysis [ Time Frame: screening/baseline to Week 25 ]
  3. Vital signs [ Time Frame: screening/baseline to Week 25 ]

Secondary Outcome Measures :
  1. Electrocardiogram [ Time Frame: screening/baseline, Week 1, Week 13, Week 25 ]
  2. Immunogenicity assessments [ Time Frame: screening/baseline to Week 29 ]
  3. Cmax [ Time Frame: Week 1, Week 13, Week 25 ]
  4. AUC [ Time Frame: Week 1, Week 13, Week 25 ]
  5. t1/2 [ Time Frame: Week 1, Week 13, Week 25 ]
  6. Skeletal muscle glycogen content [ Time Frame: screening/baseline, Week 27 ]
  7. Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments. [ Time Frame: screening/baseline, Week 27 ]
  8. Urinary Hex4 [ Time Frame: screening/baseline to Week 25 ]
  9. Functional assessments including 6 Minute Walk Test (6MWT) [ Time Frame: screening/baseline, Week 13, Week 25 ]
    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)

  10. Quality of life assessments [ Time Frame: screening/baseline, Week 13, Week 25 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01898364


Locations
United States, Arizona
Investigational Site Number 840006
Phoenix, Arizona, United States, 85013
United States, Florida
Investigational Site Number 840010
Jacksonville, Florida, United States, 32209
United States, Kansas
Investigational Site Number 840001
Kansas City, Kansas, United States, 66160-7321
United States, Missouri
Investigational Site Number 840008
St Louis, Missouri, United States, 63110
United States, North Carolina
Investigational Site Number 840002
Durham, North Carolina, United States, 27710
United States, Texas
Investigational Site Number 840009
Dallas, Texas, United States, 75390
United States, Virginia
Investigational Site Number 840003
Fairfax, Virginia, United States, 22030
Belgium
Investigational Site Number 056001
Leuven, Belgium, 3000
Denmark
Investigational Site Number 208001
København Ø, Denmark, 2100
France
Investigational Site Number 250001
Marseille, France, 13385
Investigational Site Number 250003
Nice, France, 06012
Investigational Site Number 250002
Paris, France, 75013
Germany
Investigational Site Number 276003
Mainz, Germany, 55131
Investigational Site Number 276001
München, Germany, 80336
Investigational Site Number 276002
Münster, Germany, 48149
Netherlands
Investigational Site Number 528001
Rotterdam, Netherlands, 3015 GJ
United Kingdom
Investigational Site Number 826003
Newcastle Upon Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Clinical Sciences & Operations Sanofi

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT01898364     History of Changes
Other Study ID Numbers: TDR12857
2012-004167-42 ( EudraCT Number )
U1111-1144-7725 ( Other Identifier: (UTN) )
First Posted: July 12, 2013    Key Record Dates
Last Update Posted: March 11, 2015
Last Verified: March 2015

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Glycogen Storage Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases