Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT01898364 |
|
Recruitment Status :
Completed
First Posted : July 12, 2013
Last Update Posted : March 23, 2023
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
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 |
| 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. |
| Actual Study Start Date : | August 19, 2013 |
| Actual Primary Completion Date : | February 25, 2015 |
| Actual Study Completion Date : | February 25, 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 |
- Adverse events [ Time Frame: screening/baseline to Week 25 ]
- Laboratory assessments including hematology, biochemistry and urinalysis [ Time Frame: screening/baseline to Week 25 ]
- Vital signs [ Time Frame: screening/baseline to Week 25 ]
- Electrocardiogram [ Time Frame: screening/baseline, Week 1, Week 13, Week 25 ]
- Immunogenicity assessments [ Time Frame: screening/baseline to Week 29 ]
- Cmax [ Time Frame: Week 1, Week 13, Week 25 ]
- AUC [ Time Frame: Week 1, Week 13, Week 25 ]
- t1/2 [ Time Frame: Week 1, Week 13, Week 25 ]
- Skeletal muscle glycogen content [ Time Frame: screening/baseline, Week 27 ]
- Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments. [ Time Frame: screening/baseline, Week 27 ]
- Urinary Hex4 [ Time Frame: screening/baseline to Week 25 ]
- 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)
- Quality of life assessments [ Time Frame: screening/baseline, Week 13, Week 25 ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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.
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
Show 17 study locations
| Study Director: | Clinical Sciences & Operations | Sanofi |
| Responsible Party: | Genzyme, a Sanofi Company |
| ClinicalTrials.gov Identifier: | NCT01898364 |
| 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 23, 2023 |
| Last Verified: | March 2023 |
|
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 |