Morquio A Syndrome, MPS IV A, Mucopolysaccharidosis IVA
Conditions
Keywords
Mucopolysaccharidosis IV type A, MPS IV Type A, Mucopolysaccharidosis IVA, MPS IVA, Morquio A Syndrome, Lysosomal Storage Disorder, LSD, N-acetylgalactosamine-6-sulfatase, N-acetylgalactosamine-6-sulfate sulfatase, galactose-6-sulfatase, GALNS, enzyme replacement therapy, ERT
Brief summary
This multicenter, open-label extension study is designed to assess long-term efficacy and safety of 2.0 milligrams (mg)/kilogram(kg)/week of BMN 110 in patients diagnosed with Mucopolysaccharidosis IVA (MPS IVA). Patients with MPS IVA, who enrolled in a prior BioMarin sponsored clinical study of BMN 110 (NCT00884949; Study Identification Number MOR-002), were eligible to enroll in this study (except patients who enrolled in NCT01275066; Study Identification Number MOR-004).
Interventions
DRUGBMN 110
Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
Sponsors
BioMarin Pharmaceutical
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Willing and able to provide written, signed informed consent, or in the case of patients under the age of 18 (or 16 years, depending on the region), provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures. * Sexually active patients must be willing to use an acceptable method of contraception while participating in the study. * Females of childbearing potential must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests during the study. * Must have enrolled in a prior BioMarin sponsored clinical study of BMN 110.
Exclusion criteria
* Pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study. * Use of any investigational product (other than BMN 110 in a prior clinical study) or investigational medical device within 30 days prior to Baseline, or requirement for any investigational agent prior to completion of all scheduled study assessments. * Concurrent disease or condition, including but not limited to symptomatic cervical spine instability, clinically significant spinal cord compression, or severe cardiac disease that would interfere with study participation or safety as determined by the Investigator. * Any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study. * Were enrolled in MOR-004 (patients from MOR-004 may be eligible to participate in a separate, appropriately designed, extension study).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety Evaluation | Entire Study Period, up to 240 weeks | The primary objective of the study is to evaluate the safety of weekly infusions of BMN 110; the safety variables included Adverse Events (AEs). The primary outcome measure data is presented in more detail under the Adverse Events section. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial | Change from baseline in meters in 6-minute Walk Test. As a measure of endurance, a 6-minute walk test (6MWT) was performed according to the American Thoracic Society Guidelines. Patients were instructed to walk as far as possible in 6 minutes. |
| Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial | Change from baseline in the 3-minute Stair Climb Test (3MSCT). Patients walked up stairs that have a railing, which could be used for support, for 3 minutes, with the number of stairs climbed recorded. The test result was the number of steps climbed per minute. |
| Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 168 weeks during the MOR-100 extension trial | Percent Change from baseline in Normalized Urine KS. The percent change was calculated (Week X value - baseline value)/baseline value \*100% |
| Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial | Percent Change from baseline in Maximum Voluntary Ventilation. |
| Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial | Percent Change from baseline in Forced Vital Capacity. |
Countries
United Kingdom
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| BMN 110 BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks. | 20 |
| Total | 20 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Did not consent to MOR-100 follow-up | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | BMN 110 |
|---|---|
| Age, Continuous | 8.4 years STANDARD_DEVIATION 2.9 |
| Age, Customized >=10 to <=18 years | 5 participants |
| Age, Customized >=4 to < 8 years | 10 participants |
| Age, Customized >=8 to <10 years | 5 participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 20 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race/Ethnicity, Customized American Indian or Alaska Native | 0 participants |
| Race/Ethnicity, Customized Asian | 9 participants |
| Race/Ethnicity, Customized Black or African American | 0 participants |
| Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander | 0 participants |
| Race/Ethnicity, Customized Other | 2 participants |
| Race/Ethnicity, Customized White | 9 participants |
| Region of Enrollment United Kingdom | 20 participants |
| Sex: Female, Male Female | 8 Participants |
| Sex: Female, Male Male | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 20 / 20 |
| serious Total, serious adverse events | 19 / 20 |
Outcome results
Primary
Safety Evaluation
The primary objective of the study is to evaluate the safety of weekly infusions of BMN 110; the safety variables included Adverse Events (AEs). The primary outcome measure data is presented in more detail under the Adverse Events section.
Time frame: Entire Study Period, up to 240 weeks
Population: The primary outcome measure data is presented in more detail under the Adverse Events section.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| BMN 110 | Safety Evaluation | Serious Adverse Events | 19 participants |
| BMN 110 | Safety Evaluation | Other (Not Including Serious) Adverse Events | 20 participants |
Secondary
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
Change from baseline in meters in 6-minute Walk Test. As a measure of endurance, a 6-minute walk test (6MWT) was performed according to the American Thoracic Society Guidelines. Patients were instructed to walk as far as possible in 6 minutes.
Time frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial
Population: Intent-to-Treat population (all subjects who enrolled in the study). Two patients were either physically (score was designated as 0 m) or developmentally (score was set to missing) unable to perform the 6MWT. The analysis was based on observed cases.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Week 12 Change from Baseline (n=19) | -20.7 meters | Standard Deviation 85.95 |
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Week 24 Change from Baseline (n=17) | 16.3 meters | Standard Deviation 71.74 |
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Week 36 Change from Baseline (n=17) | 13.8 meters | Standard Deviation 63.25 |
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Week 48 Change from Baseline (n=17) | -4.8 meters | Standard Deviation 64.7 |
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Week 72 Change from Baseline (n=17) | 4.0 meters | Standard Deviation 87.24 |
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 0 Change from Baseline (n=16) | 15.7 meters | Standard Deviation 89 |
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 24 Change from Baseline (n=16) | 24.5 meters | Standard Deviation 101.23 |
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 48 Change from Baseline (n=16) | 6.8 meters | Standard Deviation 98.66 |
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 72 Change from Baseline (n=17) | -49.8 meters | Standard Deviation 132.63 |
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 96 Change from Baseline (n=16) | 11.2 meters | Standard Deviation 85.24 |
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 120 Change from Baseline (n=16) | 4.2 meters | Standard Deviation 94.09 |
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 144 Change from Baseline (n=13) | 3.1 meters | Standard Deviation 106.82 |
| BMN 110 | Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 192 Change from Baseline (n=9) | -37.1 meters | Standard Deviation 103.96 |
Secondary
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
Change from baseline in the 3-minute Stair Climb Test (3MSCT). Patients walked up stairs that have a railing, which could be used for support, for 3 minutes, with the number of stairs climbed recorded. The test result was the number of steps climbed per minute.
Time frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial
Population: Intent-to-Treat population (all subjects who enrolled in the study). One patient was developmentally unable to perform the 3MSCT and the test scores were set to missing. The analysis was based on observed cases.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Week 12 Change from Baseline (n=19) | 0.3 steps/min | Standard Deviation 14.07 |
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Week 24 Change from Baseline (n=17) | 6.1 steps/min | Standard Deviation 8.66 |
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Week 36 Change from Baseline (n=17) | 7.8 steps/min | Standard Deviation 13.69 |
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Week 48 Change from Baseline (n=17) | 9.7 steps/min | Standard Deviation 14.42 |
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Week 72 Change from Baseline (n=17) | 9.7 steps/min | Standard Deviation 13.91 |
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 0 Change from Baseline (n=16) | 12.7 steps/min | Standard Deviation 13.96 |
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 24 Change from Baseline (n=16) | 13.4 steps/min | Standard Deviation 17.07 |
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 48 Change from Baseline (n=16) | 6.6 steps/min | Standard Deviation 16.87 |
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 72 Change from Baseline (n=16) | -1.4 steps/min | Standard Deviation 21.11 |
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 96 Change from Baseline (n=16) | 9.9 steps/min | Standard Deviation 18.84 |
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 120 Change from Baseline (n=16) | 6.2 steps/min | Standard Deviation 14.41 |
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 144 Change from Baseline (n=12) | 5.4 steps/min | Standard Deviation 11.93 |
| BMN 110 | Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Week 192 Change from Baseline (n=7) | -0.2 steps/min | Standard Deviation 10.34 |
Secondary
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
Percent Change from baseline in Forced Vital Capacity.
Time frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial
Population: Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BMN 110 | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 72 Percent Change from Baseline (n=15) | 16.1 percentage change | Standard Deviation 21.96 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 96 Percent Change from Baseline (n=14) | 14.8 percentage change | Standard Deviation 17.36 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Wk 12 Percent Change from Baseline (n=18) | 3.4 percentage change | Standard Deviation 10.85 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Wk 24 Percent Change from Baseline (n=16) | 0.2 percentage change | Standard Deviation 16.6 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Wk 36 Percent Change from Baseline (n=16) | 10.7 percentage change | Standard Deviation 20.81 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Wk 72 Percent Change from Baseline (n=16) | 12.5 percentage change | Standard Deviation 14.88 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 0 Percent Change from Baseline (n=15) | 11.8 percentage change | Standard Deviation 14.97 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 24 Percent Change from Baseline (n=14) | 15.3 percentage change | Standard Deviation 16.31 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 48 Percent Change from Baseline (n=13) | 15.8 percentage change | Standard Deviation 16.56 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 120 Percent Change from Baseline (n=16) | 22.8 percentage change | Standard Deviation 21.14 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 144 Percent Change from Baseline (n=12) | 17.5 percentage change | Standard Deviation 24.32 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 192 Percent Change from Baseline (n=7) | 18.6 percentage change | Standard Deviation 30.98 |
Secondary
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
Percent Change from baseline in Maximum Voluntary Ventilation.
Time frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trial
Population: Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BMN 110 | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Wk 12 Percent Change from Baseline (n=14) | 9.9 percentage change | Standard Deviation 21.29 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Wk 24 Percent Change from Baseline (n=13) | 11.0 percentage change | Standard Deviation 21.48 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Wk 36 Percent Change from Baseline (n=14) | 10.5 percentage change | Standard Deviation 17.43 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Wk 72 Percent Change from Baseline (n=14) | 18.4 percentage change | Standard Deviation 20.77 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 0 Percent Change from Baseline (n=13) | 11.1 percentage change | Standard Deviation 16.44 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 24 Percent Change from Baseline (n=13) | 9.8 percentage change | Standard Deviation 22.25 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 48 Percent Change from Baseline (n=12) | 3.5 percentage change | Standard Deviation 17.78 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 72 Percent Change from Baseline (n=13) | 10.1 percentage change | Standard Deviation 27.83 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 96 Percent Change from Baseline (n=12) | -4.9 percentage change | Standard Deviation 35.53 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 120 Percent Change from Baseline (n=13) | -3.3 percentage change | Standard Deviation 28.35 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 144 Percent Change from Baseline (n=11) | -3.5 percentage change | Standard Deviation 34.61 |
| BMN 110 | Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 192 Percent Change from Baseline (n=6) | 28.2 percentage change | Standard Deviation 38.78 |
Secondary
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
Percent Change from baseline in Normalized Urine KS. The percent change was calculated (Week X value - baseline value)/baseline value \*100%
Time frame: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 168 weeks during the MOR-100 extension trial
Population: Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BMN 110 | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Wk 12 Percent Change from Baseline (n=19) | -23.2 percentage change | Standard Deviation 19.04 |
| BMN 110 | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Wk 24 Percent Change from Baseline (n=18) | -27.9 percentage change | Standard Deviation 17.92 |
| BMN 110 | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Wk 36 Percent Change from Baseline (n=18) | -40.6 percentage change | Standard Deviation 20.16 |
| BMN 110 | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-002 Wk 72 Percent Change from Baseline (n=17) | -32.2 percentage change | Standard Deviation 17.1 |
| BMN 110 | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 0 Percent Change from Baseline (n=17) | -30.0 percentage change | Standard Deviation 19.23 |
| BMN 110 | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 24 Percent Change from Baseline (n=17) | -43.6 percentage change | Standard Deviation 19.56 |
| BMN 110 | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 48 Percent Change from Baseline (n=16) | -41.9 percentage change | Standard Deviation 19.29 |
| BMN 110 | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 72 Percent Change from Baseline (n=12) | -36.4 percentage change | Standard Deviation 36.7 |
| BMN 110 | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 96 Percent Change from Baseline (n=17) | -49.7 percentage change | Standard Deviation 19.93 |
| BMN 110 | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 120 Percent Change from Baseline (n=16) | -49.3 percentage change | Standard Deviation 22.28 |
| BMN 110 | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 144 Percent Change from Baseline (n=16) | -56.6 percentage change | Standard Deviation 19.54 |
| BMN 110 | Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100). | MOR-100 Wk 168 Percent Change from Baseline (n=15) | -58.9 percentage change | Standard Deviation 16.02 |