Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

This study has been completed.

Sponsor:

Collaborators:

Covance

PharmaNet

PRA Health Sciences

Information provided by (Responsible Party):

Shire

ClinicalTrials.gov Identifier:

NCT00607386

First received: January 22, 2008

Last updated: October 6, 2015

Last verified: February 2014

History of Changes

Results First Received: September 3, 2013

Study Type:	Interventional
Study Design:	Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
Conditions:	Hunter Syndrome Mucopolysaccharidosis II MPS II
Intervention:	Biological: Idursulfase

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
Idursulfase	Open-label treatment with idursulfase

Participant Flow: Overall Study

	Idursulfase
STARTED	28
COMPLETED	27
NOT COMPLETED	1
Physician Decision	1

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.
Safety population was defined as all enrolled participants who received at least one study dose (or any portion of a dose) of idursulfase.

Reporting Groups

	Description
Idursulfase	Open-label treatment with idursulfase

Baseline Measures

	Idursulfase
Overall Participants Analyzed [Units: Participants]	28

Age [Units: Years] Mean (Standard Deviation)	4.0 (1.62)

Gender [Units: Participants]
Female	0
Male	28

Baseline Normalized Urinary Glycosaminoglycan (GAG) Level [Units: Microgram per milligram creatinine] Mean (Standard Deviation)	738.3 (165.21)

Outcome Measures

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1. Primary:

Safety Evaluation [ Time Frame: From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks ]

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2. Secondary:

Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels [ Time Frame: Baseline, Weeks 18, 36 and 53 ]

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3. Secondary:

Single- and Repeat-Dose Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) [ Time Frame: Weeks 1 and 27 ]

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4. Secondary:

Single- and Repeat-Dose Pharmacokinetics - Time of Maximum Observed Serum Concentration (Tmax) [ Time Frame: Weeks 1 and 27 ]

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5. Secondary:

Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to the Final Time Point With a Concentration of at Least Lower Limit of Quantitation (AUClast) [ Time Frame: Weeks 1 and 27 ]

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6. Secondary:

Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to Infinity (AUCinf) [ Time Frame: Weeks 1 and 27 ]

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7. Secondary:

Single- and Repeat-Dose Pharmacokinetics - Elimination Half-Life (t1/2) [ Time Frame: Weeks 1 and 27 ]

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8. Secondary:

Single- and Repeat-Dose Pharmacokinetics - Mean Residence Time From Time 0 to Infinity (MRTinf) [ Time Frame: Weeks 1 and 27 ]

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9. Secondary:

Single- and Repeat-Dose Pharmacokinetics - Clearance (CL) [ Time Frame: Weeks 1 and 27 ]

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10. Secondary:

Single- and Repeat-Dose Pharmacokinetics - Volume of Distribution at Steady State (Vss) [ Time Frame: Weeks 1 and 27 ]

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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: Arian Pano, MD, MPH
Organization: Shire HGT
phone: 781-482-0875
e-mail: apano@shire.com

Publications of Results:

Pano A, Barbier AJ, Bielefeld B, Whiteman DA, Amato DA. Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome). Orphanet J Rare Dis. 2015 Apr 24;10:50. doi: 10.1186/s13023-015-0265-2.

Other Publications:

Muenzer J, Gucsavas-Calikoglu M, McCandless SE, Schuetz TJ, Kimura A. A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Mol Genet Metab. 2007 Mar;90(3):329-37. Epub 2006 Dec 20.

Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM, Vellodi A, Martin R, Ramaswami U, Gucsavas-Calikoglu M, Vijayaraghavan S, Wendt S, Puga AC, Ulbrich B, Shinawi M, Cleary M, Piper D, Conway AM, Kimura A. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med. 2006 Aug;8(8):465-73. Erratum in: Genet Med. 2006 Sep;8(9):599. Wendt, Suzanne [corrected to Wendt, Susanne]; Puga, Antonio [corrected to Puga, Ana Cristina]; Conway, Ann Marie [corrected to Conway, Anne Marie].

Responsible Party:	Shire
ClinicalTrials.gov Identifier:	NCT00607386 History of Changes
Other Study ID Numbers:	HGT-ELA-038 2007-006044-22 ( EudraCT Number )
Study First Received:	January 22, 2008
Results First Received:	September 3, 2013
Last Updated:	October 6, 2015

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