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

This study has been completed.

Sponsor:

Information provided by:

Genzyme

ClinicalTrials.gov Identifier:

NCT00158600

First received: September 8, 2005

Last updated: June 24, 2010

Last verified: June 2010

History of Changes

Results First Received: June 24, 2010

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Conditions:	Pompe Disease (Late-onset) Glycogen Storage Disease Type II (GSD-II) Acid Maltase Deficiency Disease Glycogenosis 2
Interventions:	Biological: alglucosidase alfa Drug: Placebo

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
One hundred patients screened and 90 enrolled.

Reporting Groups

	Description
Alglucosidase Alfa	Intravenous (IV) infusions of alglucosidase alfa at 20 milligrams (mg)/kilogram (kg) of body weight every other week (qow) for 78 weeks.
Placebo	Intravenous (IV) infusions of placebo every other week (qow) for 78 weeks.

Participant Flow: Overall Study

	Alglucosidase Alfa	Placebo
STARTED	60	30
COMPLETED	55	26
NOT COMPLETED	5	4
unable to commit time to study	0	1
Adverse Event	2	1
Death	1	0
Withdrawal by Subject	2	2

Baseline Characteristics

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Reporting Groups

	Description
Alglucosidase Alfa	Intravenous (IV) infusions of alglucosidase alfa at 20 milligrams (mg)/kilogram (kg) of body weight every other week (qow) for 78 weeks.
Placebo	Intravenous (IV) infusions of placebo every other week (qow) for 78 weeks.
Total	Total of all reporting groups

Baseline Measures

	Alglucosidase Alfa	Placebo	Total
Number of Participants [units: participants]	60	30	90
Age ^[1] [units: years] Mean ± Standard Deviation	45.3 ± 12.37	42.6 ± 11.63	44.4 ± 12.14
Gender [units: participants]
Female	26	19	45
Male	34	11	45
Race/Ethnicity, Customized [units: participants]
Hispanic	1	1	2
Asian	1	1	2
Black or African American	0	0	0
White	57	27	84
Unknown or not reported	1	1	2

[1]	Age at First Infusion

Outcome Measures

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1. Primary:

Summary of Patients Reporting Treatment-Emergent Adverse Events [ Time Frame: weeks 0-78 ]

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Measure Type	Primary
Measure Title	Summary of Patients Reporting Treatment-Emergent Adverse Events
Measure Description	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.
Time Frame	weeks 0-78
Safety Issue	Yes

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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.

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.

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.

Reporting Groups

	Description
Alglucosidase Alfa	Intravenous (IV) infusions of alglucosidase alfa at 20 milligrams (mg)/kilogram (kg) of body weight every other week (qow) for 78 weeks.
Placebo	Intravenous (IV) infusions of placebo every other week (qow) for 78 weeks.

Measured Values

	Alglucosidase Alfa	Placebo
Number of Participants Analyzed [units: participants]	60	30
Summary of Patients Reporting Treatment-Emergent Adverse Events [units: participants]
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

No statistical analysis provided for Summary of Patients Reporting Treatment-Emergent Adverse Events

2. Primary:

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 ]

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3. Primary:

Percent of Predicted Forced Vital Capacity (FVC) [ Time Frame: weeks 0, 78 ]

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4. Primary:

Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC) [ Time Frame: weeks 0, 12 and 52 ]

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5. Primary:

Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Maximum Plasma Concentration(Cmax) [ Time Frame: weeks 0, 12, 52 ]

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6. Primary:

Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Time to Maximum Plasma Concentration(Tmax) [ Time Frame: weeks 0, 12, 52 ]

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7. Secondary:

Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) [ Time Frame: weeks 0, 78 ]

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8. Secondary:

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 ]

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Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: In multi-site studies, PI can publish after Genzyme publishes or 18 months after study completion. PI gives Genzyme a draft 60 days before publication. Genzyme can ask that confidential information be removed, and can defer publication another 60 days upon notifying PI that it will file a patent application on inventions contained in the draft.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.

Results Point of Contact:

Name/Title: Genzyme Medical Information
Organization: Genzyme Corporation
phone: 800-745-4447

No publications provided by Genzyme

Publications automatically indexed to this study:

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.

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.

Responsible Party:	Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier:	NCT00158600 History of Changes
Other Study ID Numbers:	AGLU02704, 2005-002759-42
Study First Received:	September 8, 2005
Results First Received:	June 24, 2010
Last Updated:	June 24, 2010
Health Authority:	United States: Food and Drug Administration European Union: European Medicines Agency France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) Netherlands: College ter Beoordeling van Geneesmiddelen Medicines Evaluation Board (CBGMEB)

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