Trial Outcomes & Findings for Examining the Effect of Burosumab on Muscle Function (NCT NCT04146935)
NCT ID: NCT04146935
Last Updated: 2023-08-24
Results Overview
Rates of mitochondrial phosphorylation activity were assessed in the soleus/gastrocnemius muscle complex of the right calf by 31P magnetic resonance spectroscopy saturation transfer technique (micro-mol/g/min)
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
10 participants
Primary outcome timeframe
2.5 months
Results posted on
2023-08-24
Participant Flow
Participant milestones
| Measure |
Patients With XLH
Patients will receive Burosumab monthly at visits 1,2 and 3 subcutaneously at a dose of 1.0 mg/kg. Dose may be adjusted as needed.
Burosumab Injection \[Crysvita\]: Burosumab/Crysvita SC injection monthly
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Examining the Effect of Burosumab on Muscle Function
Baseline characteristics by cohort
| Measure |
Patients With XLH
n=10 Participants
Patients will receive Burosumab monthly at visits 1,2 and 3 subcutaneously at a dose of 1.0 mg/kg. Dose may be adjusted as needed.
Burosumab Injection \[Crysvita\]: Burosumab/Crysvita SC injection monthly
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
10 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
39.7 years
STANDARD_DEVIATION 11.2 • n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
10 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
8 Participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
2 Participants
n=5 Participants
|
|
Fasting serum phosphate
|
1.74 mg/dL
STANDARD_DEVIATION 0.43 • n=5 Participants
|
|
Serum calcium
|
9.30 mg/dL
STANDARD_DEVIATION 0.32 • n=5 Participants
|
|
Parathyroid hormone (PTH)
|
68.78 pg/mL
STANDARD_DEVIATION 24.40 • n=5 Participants
|
|
Renal phosphate threshold
|
1.59 mg/100 ml GF
STANDARD_DEVIATION 0.44 • n=5 Participants
|
|
Fibroblast growth factor (FGF) 23 levels
|
53.4 pg/mL
STANDARD_DEVIATION 14.5 • n=5 Participants
|
|
25-hydroxy vitamin D
|
34.23 ng/mL
STANDARD_DEVIATION 15.53 • n=5 Participants
|
|
1,25-dihydroxy vitamin D
|
71.29 pg/mL
STANDARD_DEVIATION 19.05 • n=5 Participants
|
|
Serum creatinine
|
0.63 mg/dL
STANDARD_DEVIATION 0.15 • n=5 Participants
|
PRIMARY outcome
Timeframe: 2.5 monthsRates of mitochondrial phosphorylation activity were assessed in the soleus/gastrocnemius muscle complex of the right calf by 31P magnetic resonance spectroscopy saturation transfer technique (micro-mol/g/min)
Outcome measures
| Measure |
Patients With XLH
n=10 Participants
Patients will receive Burosumab monthly at visits 1,2 and 3 subcutaneously at a dose of 1.0 mg/kg. Dose may be adjusted as needed.
Burosumab Injection \[Crysvita\]: Burosumab/Crysvita SC injection monthly
|
|---|---|
|
Skeletal Muscle Adenosine Triphosphate (ATP) Synthesis Rate
|
9.41 umol/g/min
Interval 8.7827 to 10.0333
|
SECONDARY outcome
Timeframe: 2.5 monthsmeasured in mg/dl
Outcome measures
| Measure |
Patients With XLH
n=10 Participants
Patients will receive Burosumab monthly at visits 1,2 and 3 subcutaneously at a dose of 1.0 mg/kg. Dose may be adjusted as needed.
Burosumab Injection \[Crysvita\]: Burosumab/Crysvita SC injection monthly
|
|---|---|
|
Serum Phosphate
|
2.85 mg/dL
Interval 2.4231 to 3.2769
|
SECONDARY outcome
Timeframe: 2.5 monthsIntracellular phosphorus concentration in skeletal muscle (umol/g muscle)
Outcome measures
| Measure |
Patients With XLH
n=10 Participants
Patients will receive Burosumab monthly at visits 1,2 and 3 subcutaneously at a dose of 1.0 mg/kg. Dose may be adjusted as needed.
Burosumab Injection \[Crysvita\]: Burosumab/Crysvita SC injection monthly
|
|---|---|
|
Intracellular Phosphate Concentration in Umol/g Muscle
|
2.31 umol/g muscle
Interval 1.9654 to 2.6446
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 2.5 monthsfunctional testing outcome measured in meters
Outcome measures
| Measure |
Patients With XLH
n=10 Participants
Patients will receive Burosumab monthly at visits 1,2 and 3 subcutaneously at a dose of 1.0 mg/kg. Dose may be adjusted as needed.
Burosumab Injection \[Crysvita\]: Burosumab/Crysvita SC injection monthly
|
|---|---|
|
Six-minute Walk Test
|
440.4 meter
Interval 388.06 to 492.74
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 2.5 monthsfunctional testing outcome measured in the number completed in 30 seconds
Outcome measures
| Measure |
Patients With XLH
n=10 Participants
Patients will receive Burosumab monthly at visits 1,2 and 3 subcutaneously at a dose of 1.0 mg/kg. Dose may be adjusted as needed.
Burosumab Injection \[Crysvita\]: Burosumab/Crysvita SC injection monthly
|
|---|---|
|
Sit to Stand
|
14.2 repetitions
Interval 10.8312 to 17.5688
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 2.5 monthsfunctional testing outcome measured in seconds
Outcome measures
| Measure |
Patients With XLH
n=10 Participants
Patients will receive Burosumab monthly at visits 1,2 and 3 subcutaneously at a dose of 1.0 mg/kg. Dose may be adjusted as needed.
Burosumab Injection \[Crysvita\]: Burosumab/Crysvita SC injection monthly
|
|---|---|
|
Timed up and go Test
|
7.47 second
Interval 6.4473 to 8.4967
|
Adverse Events
Patients With XLH
Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Patients With XLH
n=10 participants at risk
Patients will receive Burosumab monthly at visits 1,2 and 3 subcutaneously at a dose of 1.0 mg/kg. Dose may be adjusted as needed.
Burosumab Injection \[Crysvita\]: Burosumab/Crysvita SC injection monthly
|
|---|---|
|
Renal and urinary disorders
Partial ureteropelvic junction obstruction by staghorn calculus
|
10.0%
1/10 • Number of events 1 • 3 months (time of consent to completion of visit 5)
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place