Pompe Disease (Late-onset)Phase 3NCT00158600

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

A Phase 3 study testing alglucosidase alfa in people with Pompe Disease (Late-onset) and Glycogen Storage Disease Type II (GSD-II), run by Genzyme, a Sanofi Company at 8 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Ages 8 years and older
Conditions the study is for: Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease
What is being tested?: alglucosidase alfa
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 90 participants
Where: 8 sitesin 3 countries
Study dates: Sep 2005to Sep 2007
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 8 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Patient must provide signed, informed consent prior to performing any study-related procedures.
Patient must have a diagnosis of Pompe disease based on deficient endogenous GAA activity in cultured skin fibroblasts of less than or equal to 40% of the normal mean of the testing laboratory and 2 confirmed GAA gene mutations;
Patient must be greater than or equal to 8 years of age at the time of enrollment;
Patient must be able to ambulate 40 meters (approximately 130 feet) in 6 minutes on each test performed on two consecutive days (use of assistive devices such as a walker, cane, or crutches, is permitted);
Patient must have an FVC of greater than or equal to 30% and < 80% predicted in the upright position;
Patient must have a postural drop in FVC (liters) of at least 10% from the upright to the supine position;

Show all 12Show fewer

Patient must have proximal muscle weakness in the lower limbs based on unilateral QMT of the knee extensors defined as < 80% of the predicted value based on age, gender and body size
Patient must be able to tolerate pulmonary function testing (PFT) and muscle testing in the supine position;
Patient must have testable muscle in bilateral knee flexors and knee extensors, and testable muscle in bilateral elbow flexors and elbow extensors;
Patient must be able to provide reproducible muscle and pulmonary function test results;
Patient (and patient's legal guardian if patient is < 18 years of age) must have the ability to comply with the clinical protocol;
A female patient of childbearing potential must have a negative pregnancy test (urine) at Baseline. Note: All female patients of childbearing potential and sexually mature males must use a medically accepted method of contraception throughout the study.

Who cannot join

Patient requires the use of invasive ventilatory support;
Patient requires the use of noninvasive ventilatory support while awake and in an upright position;
Patient has received enzyme replacement therapy with GAA from any source;
Patient has used an investigational product within 30 days prior to study enrollment, or is currently enrolled in another study which involves clinical evaluations, unless prior approval is given by Genzyme;
Patient has a major congenital anomaly, medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities;

Trial locations and contacts

8 sites in 3 countries.

Country

City, postcode or hospital

8 sites in 3 countries

United States5 sites

Tower Hematology Oncology Medical Group
Beverly Hills, California, 90211
Mount Sinai School of Medicine
New York, New York, 10029
University of Pittsburgh, Dept. of Neurology
Pittsburgh, Pennsylvania, 15213
Washington University Medical Center
St Louis, Missouri, 63110
Children's National Medical Center
Washington D.C., District of Columbia, 20010

Other trials in United States

Netherlands2 sites

Sophia Children's Hospital, Erasmus MC
Rotterdam, 3000 CB
Erasmus Medical Centre Rotterdam
Rotterdam, 3015 GD

Other trials in Netherlands

France1 site

Groupe Hospitalier Pitie-Salpetriere
Paris, 75651

Other trials in France

About this study

Official title: Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease.

Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective is to evaluate the safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa treatment in patients with late-onset Pompe disease as compared to placebo.

Conditions: Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
Being tested: alglucosidase alfa, Placebo
Keywords: Glycogen Storage Disease Type II, GSD-II, Pompe Disease
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.

alglucosidase alfaBiological
Also called Myozyme, Lumizyme
IV infusion of 20mg/kg; qow for 78 weeks.
PlaceboDrug
Placebo Comparator; qow for 78 weeks.

Study groups in NCT00158600
Study group	Role	What they receive
alglucosidase alfa	Active comparator	Intravenous (IV) infusions of alglucosidase alfa at 20 milligrams (mg)/kilogram (kg) of body weight every other week (qow) for 78 weeks. Receives: Biological: alglucosidase alfa
Placebo	Placebo comparator	Intravenous (IV) infusions of placebo every other week (qow) for 78 weeks. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Summary of Patients Reporting Treatment-Emergent Adverse Events
Overall safety summary of patients experiencing Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, and Infusion Associated Reactions (IARs). Summary is based on Treatment-emergent AEs (TEAEs), defined as AEs that occurred following the initiation of study treatment, i.e., alglucosidase alfa or placebo.
Measured: weeks 0-78
Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline
Mean distance walked gives an indication of functional endurance. The greater the distance, the greater the endurance. Mean values of distance walked in a six-minute walk test are offered for baseline, week 78 (or last available observation), and the mean change from baseline (at week 78 or last available post-baseline observation).
Measured: weeks 0, 78
Percent of Predicted Forced Vital Capacity (FVC)
Forced vital capacity is a standard pulmonary function test used to quantify respiratory muscle weakness. Forced vital capacity (FVC) is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100%.
Measured: weeks 0, 78
Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC)
Area under the plasma concentration versus time curve from time zero (pre-dose) to 16 hours after the end of infusion. Blood sample time points were 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes.
Measured: weeks 0, 12 and 52
Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Maximum Plasma Concentration(Cmax)
Maximum plasma concentration observed in blood samples taken at the following time points: 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes.
Measured: weeks 0, 12, 52
Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Time to Maximum Plasma Concentration(Tmax)
Time to maximum plasma concentration observed in blood samples taken at the following time points: 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes.
Measured: weeks 0, 12, 52

Secondary outcomes (2)

Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT)
Quantitative muscle testing (QMT) is a standardized system to measure muscle force production during maximal voluntary isometric contraction. QMT data were collected directly from sensors into laptop computers. Predicted normal values for QMT are based on a formula using sex, age and body mass index of a person, and is an estimate of healthy muscle force. Percent of predicted QMT = (observed value)/(predicted value) * 100%. The QMT Leg Score is the average of the bilateral means for percent predicted knee flexors and extensors. A value of 100% indicates 'normal' muscle strength.
Measured: weeks 0, 78
Health-related Quality of Life Survey Values Related to Physical Components as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey
The Medical Outcomes Study Short Form (MOS SF)-36 questionnaire consists of 36 items grouped into 8 domains designed to assess generic health-related quality of life in healthy and ill adult populations. Physical Component Scores (PCS) report the four domains of physical functioning, role-physical, bodily pain, and general health. Higher scores are associated with better quality of life. All questions are scored on a scale from 0 to 100, with 100 representing the highest level of functioning possible. The PCS scores are reported.
Measured: weeks 0, 78

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Sep 2005Sep 2007

First posted12 Sep 2005 (estimated)
Study startSep 2005
Primary completionSep 2007
Study completionSep 2007

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Pompe Disease (Late-onset)Studies 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 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 90 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00158600
Sponsor's protocol ID: AGLU02704
Other IDs: 2005-002759-42, 2005
Status last verified: Apr 2015
First submitted: 8 Sep 2005
Last updated: 28 Apr 2015
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Expanded access: Available

Publications

Forsha D, Li JS, Smith PB, van der Ploeg AT, Kishnani P, Pasquali SK; Late-Onset Treatment Study Investigators. Cardiovascular abnormalities in late-onset Pompe disease and response to enzyme replacement therapy. Genet Med. 2011 Jul;13(7):625-31. doi: 10.1097/GIM.0b013e3182142966.
PubMed 21543987
van der Ploeg AT, Clemens PR, Corzo D, Escolar DM, Florence J, Groeneveld GJ, Herson S, Kishnani PS, Laforet P, Lake SL, Lange DJ, Leshner RT, Mayhew JE, Morgan C, Nozaki K, Park DJ, Pestronk A, Rosenbloom B, Skrinar A, van Capelle CI, van der Beek NA, Wasserstein M, Zivkovic SA. A randomized study of alglucosidase alfa in late-onset Pompe's disease. N Engl J Med. 2010 Apr 15;362(15):1396-406. doi: 10.1056/NEJMoa0909859.
PubMed 20393176

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingObservational study
Pompe Disease Registry Protocol
Glycogen Storage Disease Type II, Pompe Disease
Not yet recruitingPhase 2
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
Pompe Disease
RecruitingPhase 1/2
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
Pompe Disease (Late-onset), Pompe Disease Late-Onset
Active, not recruiting
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
Pompe Disease (Late-onset)

All Pompe Disease (Late-onset) trials

Study at a glance

From the registry record, last updated by the sponsor on 28 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease.
ClinicalTrials.gov ID: NCT00158600
Acronym / sponsor ID: AGLU02704
Conditions: Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
Keywords: Glycogen Storage Disease Type II, GSD-II, Pompe Disease
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 90 participants (actual)
Lead sponsor: Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
Study start: Sep 2005
Primary completion: Sep 2007 (actual)
Study completion: Sep 2007 (actual)
First posted: 12 Sep 2005 (estimated)
Results: Posted 23 Jul 2010

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
alglucosidase alfa	Active comparator	Biological: alglucosidase alfaIntravenous (IV) infusions of alglucosidase alfa at 20 milligrams (mg)/kilogram (kg) of body weight every other week (qow) for 78 weeks.
Placebo	Placebo comparator	Drug: PlaceboIntravenous (IV) infusions of placebo every other week (qow) for 78 weeks.

Who may qualify

Ages: 8 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (12 listed)

Patient must provide signed, informed consent prior to performing any study-related procedures.
Patient must have a diagnosis of Pompe disease based on deficient endogenous GAA activity in cultured skin fibroblasts of less than or equal to 40% of the normal mean of the testing laboratory and 2 confirmed GAA gene mutations;
Patient must be greater than or equal to 8 years of age at the time of enrollment;
Patient must be able to ambulate 40 meters (approximately 130 feet) in 6 minutes on each test performed on two consecutive days (use of assistive devices such as a walker, cane, or crutches, is permitted);
Patient must have an FVC of greater than or equal to 30% and < 80% predicted in the upright position;

Key exclusion criteria (5 listed)

Patient requires the use of invasive ventilatory support;
Patient requires the use of noninvasive ventilatory support while awake and in an upright position;
Patient has received enzyme replacement therapy with GAA from any source;
Patient has used an investigational product within 30 days prior to study enrollment, or is currently enrolled in another study which involves clinical evaluations, unless prior approval is given by Genzyme;
Patient has a major congenital anomaly, medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities;

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
alglucosidase alfaAlso: Myozyme, LumizymeIV infusion of 20mg/kg; qow for 78 weeks.	Biological	alglucosidase alfa
PlaceboPlacebo Comparator; qow for 78 weeks.	Drug	Placebo

Primary outcome

Summary of Patients Reporting Treatment-Emergent Adverse Events
Time frame: weeks 0-78
Overall safety summary of patients experiencing Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, and Infusion Associated Reactions (IARs). Summary is based on Treatment-emergent AEs (TEAEs), defined as AEs that occurred following the initiation of study treatment, i.e., alglucosidase alfa or placebo.
Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline
Time frame: weeks 0, 78
Mean distance walked gives an indication of functional endurance. The greater the distance, the greater the endurance. Mean values of distance walked in a six-minute walk test are offered for baseline, week 78 (or last available observation), and the mean change from baseline (at week 78 or last available post-baseline observation).
Percent of Predicted Forced Vital Capacity (FVC)
Time frame: weeks 0, 78
Forced vital capacity is a standard pulmonary function test used to quantify respiratory muscle weakness. Forced vital capacity (FVC) is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) * 100%.
Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC)
Time frame: weeks 0, 12 and 52
Area under the plasma concentration versus time curve from time zero (pre-dose) to 16 hours after the end of infusion. Blood sample time points were 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes.
Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Maximum Plasma Concentration(Cmax)
Time frame: weeks 0, 12, 52
Maximum plasma concentration observed in blood samples taken at the following time points: 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes.
Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Time to Maximum Plasma Concentration(Tmax)
Time frame: weeks 0, 12, 52
Time to maximum plasma concentration observed in blood samples taken at the following time points: 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes.

2 secondary outcomes

Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT)
Time frame: weeks 0, 78
Health-related Quality of Life Survey Values Related to Physical Components as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey
Time frame: weeks 0, 78

Results and safety

Results posted to the registry on 23 Jul 2010, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Alglucosidase Alfa	60	55
Placebo	30	26

Primary outcome: Summary of Patients Reporting Treatment-Emergent Adverse Events

Time frame: weeks 0-78. Number. Unit: participants

Measure	Alglucosidase Alfa n = 60	Placebo n = 30
Patients with Any AEs	60	30
Patients with Treatment-Related AEs	32	17
Patients with Infusion-Associated Reactions	17	7
Patients with SAEs	13	6
Patients with Severe AEs	14	10
Patients who Discontinued Due to AEs (incl death)	3	1
Patients who Died	1	0

Population analysed: All patients who received any amount of study treatment comprise the safety population. Patients were considered, for safety analysis, to be in the treatment group of the treatment they actually received. Missing or invalid safety or resource utilization data were not replaced.

Primary outcome: Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline

Time frame: weeks 0, 78. Mean (Standard Deviation). Unit: meters

Measure	Alglucosidase Alfa n = 60	Placebo n = 30
Distance Walked at Baseline	332.20 (126.69)	317.93 (132.29)
Distance Walked at Last Available Observation	357.85 (141.32)	313.07 (144.69)
Change at Last Available Observation from Baseline	26.08 (64.41)	-4.87 (45.24)

Alglucosidase Alfa vs Placebo: Difference: 28.12 (95% CI 2.07 to 54.17), p = 0.0347, ANCOVA

Population analysed: Intent-to-Treat (ITT) population. Last observation carried forward. The last available distance walked for one patient was the Baseline visit; therefore, this patient was excluded from the change from baseline calculation.

Primary outcome: Percent of Predicted Forced Vital Capacity (FVC)

Time frame: weeks 0, 78. Mean (Standard Deviation). Unit: percent predicted FVC

Measure	Alglucosidase Alfa n = 60	Placebo n = 30
Baseline (week 0)	55.43 (14.44)	53.00 (15.66)
Week 78 (or last observation)	56.71 (16.30)	50.70 (14.88)
Change at Week 78 from Baseline	1.25 (5.55)	-2.30 (4.33)

Alglucosidase Alfa vs Placebo: Difference: 3.40 (95% CI 1.03 to 5.77), p = 0.0055, ANCOVA

Population analysed: ITT population. Last observation carried forward.

Primary outcome: Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC)

Time frame: weeks 0, 12 and 52. Mean (Standard Deviation). Unit: ug*h/mL

Measure	Alglucosidase Alfa n = 32
Week 0	2672.47 (1139.85)
Week 12	2386.76 (555.09)
Week 52	2699.28 (999.97)
Pooled	2586.17 (933.28)

Population analysed: The subgroup of patients for whom pharmacokinetic samples were obtained was based on those study sites that could accommodate pharmacokinetic sampling needs.

2 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Alglucosidase Alfa	13 of 60 (21.7%)	60 of 60 (100%)	Not reported
Placebo	6 of 30 (20%)	30 of 30 (100%)	Not reported
Overall	19 of 90 (21.1%)	90 of 90 (100%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2015.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 21543987Forsha D, Li JS, Smith PB, van der Ploeg AT, Kishnani P, Pasquali SK; Late-Onset Treatment Study Investigators. Cardiovascular abnormalities in late-onset Pompe disease and response to enzyme replacement therapy. Genet Med. 2011 Jul;13(7):625-31. doi: 10.1097/GIM.0b013e3182142966.
Derived from this study: PubMed 20393176van der Ploeg AT, Clemens PR, Corzo D, Escolar DM, Florence J, Groeneveld GJ, Herson S, Kishnani PS, Laforet P, Lake SL, Lange DJ, Leshner RT, Mayhew JE, Morgan C, Nozaki K, Park DJ, Pestronk A, Rosenbloom B, Skrinar A, van Capelle CI, van der Beek NA, Wasserstein M, Zivkovic SA. A randomized study of alglucosidase alfa in late-onset Pompe's disease. N Engl J Med. 2010 Apr 15;362(15):1396-406. doi: 10.1056/NEJMoa0909859.

Related trials for Pompe Disease (Late-onset)

Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingObservational study
Pompe Disease Registry Protocol
Glycogen Storage Disease Type II, Pompe Disease
Not yet recruitingPhase 2
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
Pompe Disease
RecruitingPhase 1/2
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
Pompe Disease (Late-onset), Pompe Disease Late-Onset
Active, not recruiting
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
Pompe Disease (Late-onset)

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.

Medical Monitor study director

Genzyme, a Sanofi Company

8 sites in 3 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing alglucosidase alfa in people with Pompe Disease (Late-onset) and Glycogen Storage Disease Type II (GSD-II), run by Genzyme, a Sanofi Company at 8 sites in 3 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 12 inclusion and 5 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 28 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in United States, Netherlands, France. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 23 Jul 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Summary of Patients Reporting Treatment-Emergent Adverse Events (time frame: weeks 0-78). The study lists 6 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 NCT00158600, which also lists 2 linked publications.

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 28 Apr 2015.