Mucopolysaccharidosis VI
Conditions
Brief summary
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
Interventions
DRUGPlacebo/rhASB
Sponsors
BioMarin Pharmaceutical
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
7 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patient consent * Patient must have successfully completed study ASB-03-05, including having received at least 20 of 24 scheduled weekly infusions and having missed no more then two consecutive infusions * If female of childbearing potential, patient must have a negative pregnancy test
Exclusion criteria
* Pregnant or lactating patient * Patient has received an investigational drug within 30 days prior to study enrollment * Patient is unwilling or unable to travel to the primary site for periodic assessments
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 12-Minute Walk Test | Baseline of ASB-03-05 through week 96 of ASB-03-06 | Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 3-Minute Stair Climb | Baseline ASB-03-05 through week 96 of ASB-03-06. | Mean change in number of stairs climbed per minute in 3 minutes. Mean change is the mean difference between the 3-Minute Stair Climb at 96 weeks and that measured before first ever treatment with rhASB. Mean change is calculated for Week 96 - Baseline for the rhASB/rhASB group and for Week 96 - Week 24 for the placebo/rhASB group. |
| Change in Urinary Glycosaminoglycans (GAG) Level | 72 weeks | Mean change in urinary GAG level for the first 72 weeks of rhASB treatment. For the rhASB/rhASB group, mean change was calculated for Week 72 -Baseline. For the placebo/rhASB, mean change was calculated for Week 96 - Week 24. |
Countries
United States
Participant flow
Recruitment details
Global study sites were hospitals and medical clinics. First Enrollment: 05FEB04 Last Dose: 28SEP06
Participants by arm
| Arm | Count |
|---|---|
| rhASB/rhASB Patients received rhASB in the double-blind study(ASB-03-05, NCT00067470)and continued to receive rhASB in the extension study(ASB-03-06). | 19 |
| Placebo/rhASB Patients received placebo in the double-blind study (ASB-03-05, NCT00067470)then received rhASB in the extension study (ASB-03-06). | 20 |
| Total | 39 |
Baseline characteristics
| Characteristic | Total | rhASB/rhASB | Placebo/rhASB |
|---|---|---|---|
| Age Continuous Age | 12.2 years STANDARD_DEVIATION 5.62 | 13.7 years STANDARD_DEVIATION 6.47 | 10.7 years STANDARD_DEVIATION 4.35 |
| Race/Ethnicity, Customized Asian | 2 participants | 1 participants | 1 participants |
| Race/Ethnicity, Customized Black | 3 participants | 1 participants | 2 participants |
| Race/Ethnicity, Customized Hispanic | 4 participants | 1 participants | 3 participants |
| Race/Ethnicity, Customized Indigenous | 2 participants | 1 participants | 1 participants |
| Race/Ethnicity, Customized Other | 4 participants | 0 participants | 4 participants |
| Race/Ethnicity, Customized White, non-Hispanic | 24 participants | 15 participants | 9 participants |
| Region of Enrollment Brazil | 8 participants | 4 participants | 4 participants |
| Region of Enrollment France | 5 participants | 2 participants | 3 participants |
| Region of Enrollment Germany | 8 participants | 4 participants | 4 participants |
| Region of Enrollment Portugal | 6 participants | 4 participants | 2 participants |
| Region of Enrollment United Kingdom | 6 participants | 3 participants | 3 participants |
| Region of Enrollment United States | 6 participants | 2 participants | 4 participants |
| Sex: Female, Male Female | 26 Participants | 12 Participants | 14 Participants |
| Sex: Female, Male Male | 13 Participants | 7 Participants | 6 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 38 / 38 |
| serious Total, serious adverse events | 14 / 38 |
Outcome results
Primary
12-Minute Walk Test
Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24.
Time frame: Baseline of ASB-03-05 through week 96 of ASB-03-06
Population: The efficacy analysis included all subjects who continued in the extension study (ASB-03-06) except the 1 subject from the rhASB/rhASB group and 1 subject from the placebo/rhASB group who missed the Week 96 measurement.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| rhASB/rhASB | 12-Minute Walk Test | 187 meters | Standard Deviation 187 |
| Placebo/rhASB | 12-Minute Walk Test | 118 meters | Standard Deviation 127 |
Secondary
3-Minute Stair Climb
Mean change in number of stairs climbed per minute in 3 minutes. Mean change is the mean difference between the 3-Minute Stair Climb at 96 weeks and that measured before first ever treatment with rhASB. Mean change is calculated for Week 96 - Baseline for the rhASB/rhASB group and for Week 96 - Week 24 for the placebo/rhASB group.
Time frame: Baseline ASB-03-05 through week 96 of ASB-03-06.
Population: The efficacy analysis included all subjects who continued in the extension study (ASB-03-06) except 1 subject from the placebo/rhASB group who missed the Week 96 measurement.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| rhASB/rhASB | 3-Minute Stair Climb | 13.1 stairs/min | Standard Deviation 16.1 |
| Placebo/rhASB | 3-Minute Stair Climb | 11.1 stairs/min | Standard Deviation 10 |
Secondary
Change in Urinary Glycosaminoglycans (GAG) Level
Mean change in urinary GAG level for the first 72 weeks of rhASB treatment. For the rhASB/rhASB group, mean change was calculated for Week 72 -Baseline. For the placebo/rhASB, mean change was calculated for Week 96 - Week 24.
Time frame: 72 weeks
Population: rhASB/rhASB and placebo/rhASB groups were combined for the analysis, representing 72 weeks of rhASB treatment in each group.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| rhASB/rhASB | Change in Urinary Glycosaminoglycans (GAG) Level | -231 microgram/mg creatinine | Standard Deviation 91 |