Replagal Enzyme Replacement Therapy for Children With Fabry Disease

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Shire

ClinicalTrials.gov Identifier:

NCT00084084

First received: June 5, 2004

Last updated: March 25, 2014

Last verified: March 2014

History of Changes

Results First Received: August 1, 2013

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: Open Label; Primary Purpose: Treatment
Condition:	Fabry Disease
Intervention:	Drug: 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
No patient was enrolled in Cohort 2 (ie, no treatment-naive patients were enrolled).

Reporting Groups

	Description
Agalsidase Alfa (Cohort 1)	Cohort 1 was composed of patients who had completed TKT023. Patients received 0.2 mg/kg agalsidase alfa, IV, every other week.

Participant Flow for 2 periods

Period 1: Phase 1 (Treatment With Replagal RB)

	Agalsidase Alfa (Cohort 1)
STARTED	17
COMPLETED	16
NOT COMPLETED	1
Lost to Follow-up	1

Period 2: Phase 2 (Transition to Replagal AF)

	Agalsidase Alfa (Cohort 1)
STARTED	11 ^[1]
COMPLETED	10
NOT COMPLETED	1
Failure to visit clinic as scheduled	1

^[1]	The 11 patients remaining in TKT029 at the time Replagal AF was made available continued to Phase 2.

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
Agalsidase Alfa (Cohort 1)	0.2 mg/kg agalsidase alfa infused by IV over 40 (+/- 10) minutes every other week

Baseline Measures

	Agalsidase Alfa (Cohort 1)
Overall Participants Analyzed [Units: Participants]	17

Age [Units: Years] Mean (Standard Deviation)	11.99 (3.2464)

Gender [Units: Participants]
Female	1
Male	16

Race/Ethnicity, Customized [Units: Participants]
White	15
Hispanic	2

Baseline Heart Rate Variability (SDNN) [Units: Msec] Mean (Standard Deviation)	98.947 (32.324)

Outcome Measures

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1. Primary:

Patients Who Experienced At Least One Adverse Event (AE) [ Time Frame: 362 weeks ]

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2. Secondary:

Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) [ Time Frame: 341 weeks ]

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3. Secondary:

Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) [ Time Frame: 341 weeks ]

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4. Other Pre-specified:

Heart Rate Variability - Change From Baseline at Week 185 in SDNN [ Time Frame: Week 185 ]

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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: Anna Wijatyk, MD, Medical Director
Organization: Shire Human Genetic Therapies
phone: 781-482-9622
e-mail: awijatyk@shire.com

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

Schiffmann R, Pastores GM, Lien YH, Castaneda V, Chang P, Martin R, Wijatyk A. Agalsidase alfa in pediatric patients with Fabry disease: a 6.5-year open-label follow-up study. Orphanet J Rare Dis. 2014 Nov 26;9:169. doi: 10.1186/s13023-014-0169-6.

Responsible Party:	Shire
ClinicalTrials.gov Identifier:	NCT00084084 History of Changes
Other Study ID Numbers:	TKT029
Study First Received:	June 5, 2004
Results First Received:	August 1, 2013
Last Updated:	March 25, 2014

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