Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT00069641
First received: September 29, 2003
Last updated: April 27, 2015
Last verified: January 2014
Results First Received: January 16, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Investigator);   Primary Purpose: Treatment
Condition: Mucopolysaccharidosis II
Interventions: Biological: Iduronate-2-sulfatase enzyme replacement therapy
Biological: iduronate-2-sulfatase enzyme replacement therapy
Biological: 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
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 Weekly (0.5 mg/kg) Idursulfase 0.5 milligram per kilogram (mg/kg) administered once-weekly by intravenous infusion for one year (52 infusions).
Idursulfase Every Other Week (EOW) (0.5 mg/kg) Idursulfase 0.5 mg/kg administered every other week by intravenous infusion for one year (26 infusions) along with placebo matching to idursulfase every other week (alternating with idursulfase) by intravenous infusion for one year (26 infusions).
Placebo Placebo matching to idursulfase administered once-weekly by intravenous infusion for one year (52 infusions).

Participant Flow:   Overall Study
    Idursulfase Weekly (0.5 mg/kg)     Idursulfase Every Other Week (EOW) (0.5 mg/kg)     Placebo  
STARTED     32     32     32  
COMPLETED     31     32     31  
NOT COMPLETED     1     0     1  
Death                 1                 0                 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.
Intent-to-treat (ITT) population included all randomized participants.

Reporting Groups
  Description
Idursulfase Weekly (0.5 mg/kg) Idursulfase 0.5 mg/kg administered once-weekly by intravenous infusion for one year (52 infusions).
Idursulfase EOW (0.5 mg/kg) Idursulfase 0.5 mg/kg administered every other week by intravenous infusion for one year (26 infusions) along with placebo matching to idursulfase every other week (alternating with idursulfase) by intravenous infusion for one year (26 infusions).
Placebo Placebo matching to idursulfase administered once-weekly by intravenous infusion for one year (52 infusions).
Total Total of all reporting groups

Baseline Measures
    Idursulfase Weekly (0.5 mg/kg)     Idursulfase EOW (0.5 mg/kg)     Placebo     Total  
Number of Participants  
[units: participants]
  32     32     32     96  
Age [1]
[units: years]
Mean (Standard Deviation)
  15.14  (6.293)     14.40  (7.019)     13.12  (6.908)     14.22  (6.729)  
Age, Customized [2]
[units: participants]
       
5 to 11 years     14     14     15     43  
12 to 18 years     10     9     10     29  
19 to 25 years     7     7     5     19  
greater than equal to (>=) 26 years     1     2     2     5  
Gender  
[units: participants]
       
Female     0     0     0     0  
Male     32     32     32     96  
Region of Enrollment  
[units: participants]
       
North America     11     11     12     34  
South America     7     7     7     21  
Europe     14     14     13     41  
[1] Age at randomization.
[2]

Age at randomization. The participant who was less than (<) 5 years old was considered for randomization to be in 5 to 11 years category.

Participants who were greater than (>) 25 years old were considered for randomization to be in 19 to 25 years category.




  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Ranked Adjusted 2-Component Composite Variable Score Based on Change From Baseline to Week 53   [ Time Frame: Baseline, Week 53 ]

2.  Secondary:   Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53   [ Time Frame: Baseline, Week 53 ]

3.  Secondary:   Mean Combined Liver and Spleen Volume at Baseline   [ Time Frame: Baseline ]

4.  Secondary:   Percent Change From Baseline in Mean Combined Liver and Spleen Volume at Week 53   [ Time Frame: Baseline, Week 53 ]

5.  Secondary:   Change From Baseline in Mean Normalized Urine Glycosaminoglycan (GAG) Levels at Week 53   [ Time Frame: Baseline, Week 53 ]

6.  Secondary:   Mean Cardiac Left Ventricular Mass Index (LVMI) at Baseline   [ Time Frame: Baseline ]

7.  Secondary:   Percent Change From Baseline in Mean Cardiac Left Ventricular Mass Index (LVMI) at Week 53   [ Time Frame: Baseline, Week 53 ]


  Serious Adverse Events


  Other Adverse Events


  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:
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 Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Study Physician
Organization: Shire
phone: 1 866-842-5335


No publications provided by Shire

Publications automatically indexed to this study:

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00069641     History of Changes
Other Study ID Numbers: TKT024
Study First Received: September 29, 2003
Results First Received: January 16, 2014
Last Updated: April 27, 2015
Health Authority: United States: Food and Drug Administration
Brazil: National Health Surveillance Agency
Germany: Federal Institute for Drugs and Medical Devices
United Kingdom: Medicines and Healthcare Products Regulatory Agency