Pompe DiseasePhase 1NCT01898364
Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
A Phase 1 study testing GZ402666 in people with Pompe Disease and Glycogen Storage Disease Type II (GSD II), run by Genzyme, a Sanofi Company at 17 sites in 7 countries.
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pompe Disease, Glycogen Storage Disease Type II (GSD II), Acid Maltase Deficiency
- What is being tested?
- GZ402666
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 17 sitesin 7 countries
- Study dates
- 19 Aug 2013to 25 Feb 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
17 sites in 7 countries.
17 sites in 7 countries
United States7 sites
Investigational Site Number 840009
Dallas, Texas, 75390
Investigational Site Number 840002
Durham, North Carolina, 27710
Investigational Site Number 840003
Fairfax, Virginia, 22030
Investigational Site Number 840010
Jacksonville, Florida, 32209
Investigational Site Number 840001
Kansas City, Kansas, 66160-7321
Investigational Site Number 840006
Phoenix, Arizona, 85013
Investigational Site Number 840008
St Louis, Missouri, 63110
France3 sites
Investigational Site Number 250001
Marseille, 13385
Investigational Site Number 250003
Nice, 06012
Investigational Site Number 250002
Paris, 75013
Germany3 sites
Investigational Site Number 276003
Mainz, 55131
Investigational Site Number 276001
München, 80336
Investigational Site Number 276002
Münster, 48149
United Kingdom1 site
Investigational Site Number 826003
Newcastle upon Tyne, NE1 4LP
About this study
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.
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.
Read the 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
- Being tested
- GZ402666
- Medical subject headings
- Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GZ402666Drug
Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous
| Study group | Role | What they receive |
|---|---|---|
| GZ402666 (neoGAA) Group 1 - 5 mg | Experimental | Intravenous infusion of 5mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks Receives: Drug: GZ402666 |
| GZ402666 (neoGAA) Group 1 - 10 mg | Experimental | Intravenous infusion of 10mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks. Receives: Drug: GZ402666 |
| GZ402666 (neoGAA) Group 1 - 20 mg | Experimental | Intravenous infusion of 20mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks. Receives: Drug: GZ402666 |
| GZ402666 (neoGAA) Group 2 - 5 mg | Experimental | 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. Receives: Drug: GZ402666 |
| GZ402666 (neoGAA) Group 2 - 10 mg | Experimental | 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. Receives: Drug: GZ402666 |
| GZ402666 (neoGAA) Group 2 - 20 mg | Experimental | 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. Receives: Drug: GZ402666 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events
Measured: screening/baseline to Week 25
Laboratory assessments including hematology, biochemistry and urinalysis
Measured: screening/baseline to Week 25
Vital signs
Measured: screening/baseline to Week 25
Secondary outcomes (10)
Electrocardiogram
Measured: screening/baseline, Week 1, Week 13, Week 25
Immunogenicity assessments
Measured: screening/baseline to Week 29
Cmax
Measured: Week 1, Week 13, Week 25
AUC
Measured: Week 1, Week 13, Week 25
t1/2
Measured: Week 1, Week 13, Week 25
Skeletal muscle glycogen content
Measured: screening/baseline, Week 27
Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments.
Measured: screening/baseline, Week 27
Urinary Hex4
Measured: screening/baseline to Week 25
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)
Measured: screening/baseline, Week 13, Week 25
Quality of life assessments
Measured: screening/baseline, Week 13, Week 25
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2013 (estimated)
- Study start19 Aug 2013
- Primary completion25 Feb 2015
- Study completion25 Feb 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pompe DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01898364
- Sponsor's protocol ID
- TDR12857
- Other IDs
- 2012-004167-42, U1111-1144-7725
- Status last verified
- Mar 2023
- First submitted
- 2 Jul 2013
- Last updated
- 23 Mar 2023
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 35618441
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT01898364
- Acronym / sponsor ID
- TDR12857
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 19 Aug 2013 (actual)
- Primary completion
- 25 Feb 2015 (actual)
- Study completion
- 25 Feb 2015 (actual)
- First posted
- 12 Jul 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| GZ402666 (neoGAA) Group 1 - 5 mg | Experimental | Drug: GZ402666Intravenous infusion of 5mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks |
| GZ402666 (neoGAA) Group 1 - 10 mg | Experimental | Drug: GZ402666Intravenous infusion of 10mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks. |
| GZ402666 (neoGAA) Group 1 - 20 mg | Experimental | Drug: GZ402666Intravenous infusion of 20mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks. |
| GZ402666 (neoGAA) Group 2 - 5 mg | Experimental | Drug: GZ402666Intravenous 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. |
| GZ402666 (neoGAA) Group 2 - 10 mg | Experimental | Drug: GZ402666Intravenous 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. |
| GZ402666 (neoGAA) Group 2 - 20 mg | Experimental | Drug: GZ402666Intravenous 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GZ402666Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous | Drug | GZ402666 (neoGAA) Group 1 - 10 mg, GZ402666 (neoGAA) Group 1 - 20 mg, GZ402666 (neoGAA) Group 1 - 5 mg, GZ402666 (neoGAA) Group 2 - 10 mg, GZ402666 (neoGAA) Group 2 - 20 mg, GZ402666 (neoGAA) Group 2 - 5 mg |
Primary outcome
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
10 secondary outcomes
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
Quality of life assessments
Time frame: screening/baseline, Week 13, Week 25
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2023.
- Derived from this study: PubMed 35618441Dimachkie 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
17 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GZ402666 in people with Pompe Disease and Glycogen Storage Disease Type II (GSD II), run by Genzyme, a Sanofi Company at 17 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, France, Germany, Belgium, Denmark and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (time frame: screening/baseline to Week 25). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01898364, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2023; AzTrials last refreshed its copy on 7 Nov 2026.