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: June 9, 2014
Last verified: February 2014
Results First Received: September 3, 2013  
Study Type: Interventional
Study Design: Endpoint Classification: Safety Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Hunter Syndrome
Mucopolysaccharidosis II
MPS II
Intervention: Biological: Idursulfase

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

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
  Hide Baseline Characteristics

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: All enrolled patients 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  
Number of Participants  
[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: ug/mg¬†creatinine]
Mean ± Standard Deviation
  738.3  ± 165.21  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Safety Evaluation   [ Time Frame: 53 weeks ]

2.  Secondary:   Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels (μg/mg Creatinine)   [ Time Frame: 53 weeks ]

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

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

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 ≥ LOQ (AUClast)   [ Time Frame: Weeks 1 and 27 ]

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 ]

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

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

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

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


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide 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.


  More Information
  Hide More Information

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:
unchecked 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.
unchecked 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.


Results Point of Contact:  
Name/Title: Arian Pano, MD, MPH
Organization: Shire HGT
phone: 781-482-0875
e-mail: apano@shire.com


Publications:

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00607386     History of Changes
Other Study ID Numbers: HGT-ELA-038, 2007-006044-22
Study First Received: January 22, 2008
Results First Received: September 3, 2013
Last Updated: June 9, 2014
Health Authority: United States: Food and Drug Administration
Taiwan: Department of Health
Brazil: National Health Surveillance Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products