Safety Study of Replagal® Therapy in Children With Fabry Disease

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Shire

ClinicalTrials.gov Identifier:

NCT01363492

First received: March 31, 2011

Last updated: April 17, 2014

Last verified: April 2014

History of Changes

Results First Received: March 25, 2014

Study Type:	Interventional
Study Design:	Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
Condition:	Fabry Disease
Intervention:	Biological: Replagal (agalsidase alfa)

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
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Reporting Groups

	Description
Replagal® (0.2 mg/kg)	0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)

Participant Flow: Overall Study

	Replagal® (0.2 mg/kg)
STARTED	14
COMPLETED	14
NOT COMPLETED	0

Baseline Characteristics

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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.
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Reporting Groups

	Description
Replagal (0.2 mg/kg)	0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW

Baseline Measures

Replagal (0.2 mg/kg)

Overall Participants Analyzed
[Units: Participants]

Age
[Units: Years]
Mean (Standard Deviation)

12.16 (2.992)

Age, Customized ^[1]
[Units: Participants]

<=18 years

^[1]	Age at informed consent

Gender
[Units: Participants]

Female

Male

Region of Enrollment
[Units: Participants]

UNITED STATES

Heart rate variability parameter SDNN
[Units: Msec]
Mean (Standard Deviation)

103.46 (32.928)

Heart rate variability parameter rMSSD
[Units: Msec]
Mean (Standard Deviation)

75.92 (45.747)

Heart rate variability parameter pNN50
[Units: Msec]
Mean (Standard Deviation)

32.79 (19.997)

Left Ventricular Mass Index (LVMI)
[Units: (g/m^2.7)]
Mean (Standard Deviation)

35.37 (10.129)

Midwall Fractional Shortening (MFS)
[Units: (%)]
Mean (Standard Deviation)

18.63 (2.891)

Plasma Gb3
[Units: (nmol/mL)]
Mean (Standard Deviation)

14.79 (12.228)

Urine Gb3
[Units: (nmol/g creatinine)]
Mean (Standard Deviation)

1775.08 (3691.087)

Outcome Measures

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1. Primary:

Number of Serious Adverse Event (SAE) [ Time Frame: Baseline to week 55 ]

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2. Primary:

Number of Treatment Emergent Adverse Event (TEAE) [ Time Frame: Baseline to week 55 ]

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3. Primary:

Development of IgG Anti-Agalsidase Alfa Antibody [ Time Frame: Baseline to Week 55 ]

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4. Primary:

Change From Baseline in Heart Rate Variability Parameter SDNN [ Time Frame: Baseline to week 55 ]

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5. Primary:

Change From Baseline in Heart Rate Variability Parameter rMSSD [ Time Frame: Baseline to week 55 ]

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6. Primary:

Change From Baseline in Heart Rate Variability Parameter pNN50 [ Time Frame: Baseline to week 55 ]

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7. Secondary:

Change From Baseline in LVMI [ Time Frame: Baseline to week 55 ]

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8. Secondary:

Change From Baseline in MFS [ Time Frame: Baseline to week 55 ]

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9. Secondary:

Change From Baseline in Plasma Gb3 [ Time Frame: Baseline to week 55 ]

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10. Secondary:

Change From Baseline in Urine Gb3 [ Time Frame: Baseline to week 55 ]

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Serious Adverse Events

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Other Adverse Events

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Limitations and Caveats

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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
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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: Shire's agreements with investigators vary. All agreements provide Shire the right to embargo communications regarding trial results prior to public release for a period ≤180 days from the time submitted to Shire for review. Shire does not prohibit publication, but can require the removal of confidential information (excluding trial results) and can request postponement of a single-center publication until after disclosure of the trial's multi-center publication

Results Point of Contact:

Name/Title: Study Physician
Organization: Shire Development LLC
phone: +1 866 842 5335

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Goker-Alpan O, Longo N, McDonald M, Shankar SP, Schiffmann R, Chang P, Shen Y, Pano A. An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy. Drug Des Devel Ther. 2016 May 25;10:1771-81. doi: 10.2147/DDDT.S102761. eCollection 2016.

Responsible Party:	Shire
ClinicalTrials.gov Identifier:	NCT01363492 History of Changes
Other Study ID Numbers:	HGT-REP-084
Study First Received:	March 31, 2011
Results First Received:	March 25, 2014
Last Updated:	April 17, 2014

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