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Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

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.
ClinicalTrials.gov Identifier: NCT01449240
Recruitment Status : Completed
First Posted : October 10, 2011
Results First Posted : December 5, 2014
Last Update Posted : October 28, 2015
Sponsor:
Information provided by (Responsible Party):
Shire

Study Type Observational
Study Design Observational Model: Case-Only;   Time Perspective: Prospective
Condition Hunter Syndrome
Intervention Other: No treatment
Enrollment 10
Recruitment Details  
Pre-assignment Details  
Arm/Group Title No Investigational Treatment or Control Group
Hide Arm/Group Description This was an observational study for the collection and study of cerebrospinal fluid (CSF) in patients with Hunter syndrome. No investigational treatment was given.
Period Title: Overall Study
Started 10
Completed 10
Not Completed 0
Arm/Group Title No Investigational Treatment or Control Group
Hide Arm/Group Description This was an observational study for the collection and study of CSF in patients with Hunter syndrome. No investigational treatment was given.
Overall Number of Baseline Participants 9
Hide Baseline Analysis Population Description
Safety Population: All patients who had undergone a procedure for CSF sample collection. This included a patient who underwent unsuccessful CSF sample collection. It did not include a patient who provided a retrospective CSF sample.
Age, Continuous   [1] 
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 9 participants
19.36
(4.1 to 36.8)
[1]
Measure Description: Age (years) at CSF sample collection
Age, Categorical   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
<=18 years
4
  44.4%
Between 18 and 65 years
5
  55.6%
>=65 years
0
   0.0%
[1]
Measure Description: Age (years) at CSF sample collection
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
0
   0.0%
Male
9
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 9 participants
United States 8
United Kingdom 1
1.Primary Outcome
Title Levels of Total Glycosaminoglycan (GAG) in CSF
Hide Description The concentration of total GAG, including heparan sulfate (HS) and dermatan sulfate (DS) oligosaccharides, in CSF was measured using an enzymatic assay.
Time Frame Day 1
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population: All patients for which an evaluable CSF sample was collected. This included a pediatric patient who was consented to provide a retrospective CSF sample.
Arm/Group Title No Investigational Treatment or Control Group
Hide Arm/Group Description:
This was an observational study for the collection and study of CSF in patients with Hunter syndrome. No investigational treatment was given.
Overall Number of Participants Analyzed 9
Mean (95% Confidence Interval)
Unit of Measure: ng/mL
816.750
(316.450 to 1317.050)
2.Secondary Outcome
Title Levels of GAG in Urine
Hide Description The levels of GAG (including sulfated DS/HS oligosaccharides) in urine were determined by the Blyscan sulfated GAG assay kit. The concentration of GAG in urine was normalized to the urine creatinine value and reported as mg GAG/mmol creatinine.
Time Frame Day 1
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population: All patients for which an evaluable CSF sample was collected. Urinary GAG was not measured in 2 patients: 1 pediatric patient who provided a retrospective CSF sample only (no urine sample was collected) and 1 adult patient whose CSF sample was not considered evaluable and therefore their urinary GAG was not measured.
Arm/Group Title No Investigational Treatment or Control Group
Hide Arm/Group Description:
This was an observational study for the collection and study of CSF in patients with Hunter syndrome. No investigational treatment was given.
Overall Number of Participants Analyzed 8
Mean (95% Confidence Interval)
Unit of Measure: mg GAG/mmol Creatinine
12.458
(3.282 to 21.633)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title No Investigational Treatment or Control Group
Hide Arm/Group Description This was an observational study for the collection and study of CSF in patients with Hunter syndrome. No investigational treatment was given. Safety analyses were performed in the Safety Population, which was defined as all patients who had undergone a procedure for CSF sample collection. This included a patient who underwent unsuccessful CSF sample collection; no CSF or urine GAG data were available for this adult patient.
All-Cause Mortality
No Investigational Treatment or Control Group
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
No Investigational Treatment or Control Group
Affected / at Risk (%) # Events
Total   1/9 (11.11%)    
Nervous system disorders   
Headache  1/9 (11.11%)  1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
No Investigational Treatment or Control Group
Affected / at Risk (%) # Events
Total   4/9 (44.44%)    
Gastrointestinal disorders   
Intermittent Lower Abdomen Spasms  1/9 (11.11%)  1
Injury, poisoning and procedural complications   
Pain Secondary to Surgical Procedure  1/9 (11.11%)  1
Difficulty Hearing in Right Ear Secondary to Neurosurgery  1/9 (11.11%)  1
Intermittent Headaches Post-Neurosurgery  1/9 (11.11%)  1
Intermittent Soreness Post-Surgery  1/9 (11.11%)  1
Musculoskeletal and connective tissue disorders   
Stiff Neck  1/9 (11.11%)  1
Nervous system disorders   
Headache  1/9 (11.11%)  1
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.
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
Results Point of Contact
Name/Title: Study Physician
Organization: Shire
Phone: +1 866 842 5335
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01449240     History of Changes
Other Study ID Numbers: HGT-HIT-072
First Submitted: October 6, 2011
First Posted: October 10, 2011
Results First Submitted: December 1, 2014
Results First Posted: December 5, 2014
Last Update Posted: October 28, 2015