Trial Outcomes & Findings for Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome (NCT NCT01449240)
NCT ID: NCT01449240
Last Updated: 2021-06-09
Results Overview
The concentration of total GAG, including heparan sulfate (HS) and dermatan sulfate (DS) oligosaccharides, in CSF was measured using an enzymatic assay.
COMPLETED
10 participants
Day 1
2021-06-09
Participant Flow
Participant milestones
| Measure |
No Investigational Treatment or Control Group
This was an observational study for the collection and study of cerebrospinal fluid (CSF) in patients with Hunter syndrome. No investigational treatment was given.
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome
Baseline characteristics by cohort
| Measure |
No Investigational Treatment or Control Group
n=9 Participants
This was an observational study for the collection and study of CSF in patients with Hunter syndrome. No investigational treatment was given.
|
|---|---|
|
Age, Categorical
<=18 years
|
4 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
19.36 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
8 Participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 1Population: 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.
The concentration of total GAG, including heparan sulfate (HS) and dermatan sulfate (DS) oligosaccharides, in CSF was measured using an enzymatic assay.
Outcome measures
| Measure |
No Investigational Treatment or Control Group
n=9 Participants
This was an observational study for the collection and study of CSF in patients with Hunter syndrome. No investigational treatment was given.
|
|---|---|
|
Levels of Total Glycosaminoglycan (GAG) in CSF
|
816.750 ng/mL
Interval 316.45 to 1317.05
|
SECONDARY outcome
Timeframe: Day 1Population: 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.
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.
Outcome measures
| Measure |
No Investigational Treatment or Control Group
n=8 Participants
This was an observational study for the collection and study of CSF in patients with Hunter syndrome. No investigational treatment was given.
|
|---|---|
|
Levels of GAG in Urine
|
12.458 mg GAG/mmol Creatinine
Interval 3.282 to 21.633
|
Adverse Events
No Investigational Treatment or Control Group
Serious adverse events
| Measure |
No Investigational Treatment or Control Group
n=9 participants at risk
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.
|
|---|---|
|
Nervous system disorders
Headache
|
11.1%
1/9 • Number of events 1
|
Other adverse events
| Measure |
No Investigational Treatment or Control Group
n=9 participants at risk
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.
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Stiff Neck
|
11.1%
1/9 • Number of events 1
|
|
Nervous system disorders
Headache
|
11.1%
1/9 • Number of events 1
|
|
Injury, poisoning and procedural complications
Pain Secondary to Surgical Procedure
|
11.1%
1/9 • Number of events 1
|
|
Injury, poisoning and procedural complications
Difficulty Hearing in Right Ear Secondary to Neurosurgery
|
11.1%
1/9 • Number of events 1
|
|
Injury, poisoning and procedural complications
Intermittent Headaches Post-Neurosurgery
|
11.1%
1/9 • Number of events 1
|
|
Gastrointestinal disorders
Intermittent Lower Abdomen Spasms
|
11.1%
1/9 • Number of events 1
|
|
Injury, poisoning and procedural complications
Intermittent Soreness Post-Surgery
|
11.1%
1/9 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER