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Trial record 1 of 1 for:    NCT00920647
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A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00920647
First Posted: June 15, 2009
Last Update Posted: October 28, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Shire
Results First Submitted: October 31, 2013  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Hunter Syndrome
Interventions: Other: Control
Drug: Idursulfase IT (1 mg)
Drug: Idursulfase IT (10 mg)
Drug: Idursulfase IT (30 mg)

  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

The first patient enrolled on 18 November 2009.

Patients were assigned randomly to active dose or no treatment. A total of 16 patients were randomized, 4 to each dose group and the no treatment arm.


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
Control Untreated Patients
Idursulfase IT (1 mg) monthly using an intrathecal drug delivery device (IDDD)
Idursulfase IT (10 mg) monthly using an intrathecal drug delivery device (IDDD)
Idursulfase IT (30 mg) monthly using an intrathecal drug delivery device (IDDD)

Participant Flow:   Overall Study
    Control   Idursulfase IT (1 mg)   Idursulfase IT (10 mg)   Idursulfase IT (30 mg)
STARTED   4   4   4   4 
COMPLETED   4   4   4   4 
NOT COMPLETED   0   0   0   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.
No text entered.

Reporting Groups
  Description
Control Untreated Patients
Idursulfase IT (1 mg) monthly using an intrathecal drug delivery device (IDDD)
Idursulfase IT (10 mg) monthly using an intrathecal drug delivery device (IDDD)
Idursulfase IT (30 mg) monthly using an intrathecal drug delivery device (IDDD)
Total Total of all reporting groups

Baseline Measures
   Control   Idursulfase IT (1 mg)   Idursulfase IT (10 mg)   Idursulfase IT (30 mg)   Total 
Overall Participants Analyzed 
[Units: Participants]
 4   4   4   4   16 
Age 
[Units: Participants]
         
<=18 years   4   4   4   4   16 
Between 18 and 65 years   0   0   0   0   0 
>=65 years   0   0   0   0   0 
Age 
[Units: Years]
Mean (Standard Deviation)
 8.64  (2.462)   5.61  (1.799)   4.34  (0.829)   6.91  (1.678)   6.38  (2.294) 
Gender 
[Units: Participants]
         
Female   0   0   0   0   0 
Male   4   4   4   4   16 
Region of Enrollment 
[Units: Participants]
         
United States   3   2   4   2   11 
United Kingdom   1   2   0   2   5 


  Outcome Measures
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1.  Primary:   Number of Serious Adverse Event (SAE)   [ Time Frame: 6 months ]

2.  Primary:   Number of Treatment Emergent Adverse Event (AE)   [ Time Frame: Baseline to week 23 ]

3.  Primary:   Safety Changes in Cerebrospinal Fluid (CSF)- White Blood Cells (WBC)   [ Time Frame: 6 months ]

4.  Primary:   Safety: Development of Anti-idursulfase Antibodies (CSF)   [ Time Frame: 6 months ]

5.  Primary:   Safety: Development of Anti-idursulfase Antibodies (Serum)   [ Time Frame: 6 months ]

6.  Primary:   Clinically Significant ECG Findings at Any Time During the Study.   [ Time Frame: 6 months ]

7.  Secondary:   Change From Baseline in CSF Glycosaminoglycans [GAGs] at Week 27   [ Time Frame: Baseline to Week 27 ]

8.  Secondary:   Level of Idursulfase in the CSF Compartment Resulting From Monthly Idursulfase IT Administrations   [ Time Frame: Week 27 (end of study) ]

9.  Secondary:   Concentration of Idursulfase in Serum After Single Administration (Week 3) in Conjunction With Elaprase   [ Time Frame: Weeks 3 ]

10.  Secondary:   Concentration of Idursulfase in Serum After Repeated Doses of Intrathecal Idursulfase-IT Given in Conjunction With Elaprase   [ Time Frame: Weeks 23 ]

11.  Secondary:   % Change From Baseline in Urinary GAG   [ Time Frame: Baseline to Week 27 ]


  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

Untreated control group were not implanted with IDDD.

Concentration of idursulfase in all CSF samples post single dose of idursulfase-IT were below LLOQ(Lower limit of quantitation) of the bioanalytical method therefore no results are reported.



  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 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: MedINfo
Organization: Shire Human Genetic Therapies Medical Information
phone: 1-866-888-0660 ext 2
e-mail: US_ShireHGT_Medicalinformation@shire.com


Publications:

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00920647     History of Changes
Other Study ID Numbers: HGT-HIT-045
2010-020048-36 ( EudraCT Number )
First Submitted: June 12, 2009
First Posted: June 15, 2009
Results First Submitted: October 31, 2013
Results First Posted: May 16, 2014
Last Update Posted: October 28, 2015