Trial Outcomes & Findings for Cystic Fibrosis Related Bone Disease: the Role of CFTR (NCT NCT01549314)
NCT ID: NCT01549314
Last Updated: 2020-04-01
Results Overview
Change in cortical volumetric bone mineral density at the radius
Recruitment status
COMPLETED
Target enrollment
79 participants
Primary outcome timeframe
Baseline and 24 months
Results posted on
2020-04-01
Participant Flow
27 subjects with CF taking ivacaftor were enrolled; however, 1 subject dropped out immediately after signing the consent form prior to any study procedures. No baseline data were obtained in this subject. Therefore, 26 subjects with CF taking ivacaftor were followed prospectively and were matched to the other 2 cohorts.
Participant milestones
| Measure |
Subjects With CF Taking Ivacaftor
Subjects with CF ages 6 to 75 years old who will be or have started taking ivacaftor within the previous 6 months
|
Subjects With CF Not Taking Ivacaftor
Subjects with CF ages 6 to 75 years old who will not be taking ivacaftor, matched for age, race, and gender with cohort 1
|
Healthy Subjects
Healthy subjects with no medical conditions known to affect bone between the ages of 6 to 75 years old, matched for age, race, and gender with cohort 2.
|
|---|---|---|---|
|
Overall Study
STARTED
|
27
|
26
|
26
|
|
Overall Study
COMPLETED
|
23
|
21
|
24
|
|
Overall Study
NOT COMPLETED
|
4
|
5
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Cystic Fibrosis Related Bone Disease: the Role of CFTR
Baseline characteristics by cohort
| Measure |
Subjects With CF Taking Ivacaftor
n=26 Participants
Subjects with CF ages 6 to 75 years old who will be or have started taking ivacaftor within the previous 6 months
|
Subjects With CF Not Taking Ivacaftor
n=26 Participants
Subjects with CF ages 6 to 75 years old who will not be taking ivacaftor, matched for age, race, and gender with cohort 1
|
Healthy Subjects
n=26 Participants
Healthy subjects with no medical conditions known to affect bone between the ages of 6 to 75 years old, matched for age, race, and gender with cohort 2.
|
Total
n=78 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
11 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
33 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
15 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
45 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Continuous
|
23.1 years
STANDARD_DEVIATION 13.1 • n=5 Participants
|
22.8 years
STANDARD_DEVIATION 13.1 • n=7 Participants
|
23.8 years
STANDARD_DEVIATION 13.2 • n=5 Participants
|
23.3 years
STANDARD_DEVIATION 13.0 • n=4 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
45 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
33 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
26 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
78 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
26 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
78 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
26 participants
n=5 Participants
|
26 participants
n=7 Participants
|
26 participants
n=5 Participants
|
78 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Baseline and 24 monthsPopulation: Subjects who were lost to follow up prior to the 24 month visit were not included in the analyses
Change in cortical volumetric bone mineral density at the radius
Outcome measures
| Measure |
Subjects With CF Taking Ivacaftor
n=23 Participants
Subjects with CF ages 6 to 75 years old who will be or have started taking ivacaftor within the previous 6 months
|
Subjects With CF Not Taking Ivacaftor
n=21 Participants
Subjects with CF ages 6 to 75 years old who will not be taking ivacaftor, matched for age, race, and gender with cohort 1
|
Healthy Subjects
n=24 Participants
Healthy subjects with no medical conditions known to affect bone between the ages of 6 to 75 years old, matched for age, race, and gender with cohort 2.
|
|---|---|---|---|
|
Bone Microarchitecture and Strength Measures of the Radius and Tibia
|
44.6 mgHA/cm3
Standard Deviation 53.9
|
40.1 mgHA/cm3
Standard Deviation 44.0
|
43.7 mgHA/cm3
Standard Deviation 53.8
|
SECONDARY outcome
Timeframe: Baseline and 24 monthsPopulation: Subjects who were lost to follow up prior to the 24 month visit were not included in the analyses
Change in PA spine bone mineral density
Outcome measures
| Measure |
Subjects With CF Taking Ivacaftor
n=23 Participants
Subjects with CF ages 6 to 75 years old who will be or have started taking ivacaftor within the previous 6 months
|
Subjects With CF Not Taking Ivacaftor
n=21 Participants
Subjects with CF ages 6 to 75 years old who will not be taking ivacaftor, matched for age, race, and gender with cohort 1
|
Healthy Subjects
n=24 Participants
Healthy subjects with no medical conditions known to affect bone between the ages of 6 to 75 years old, matched for age, race, and gender with cohort 2.
|
|---|---|---|---|
|
Areal Bone Mineral Density as Measured by DXA
|
0.032 g/cm2
Standard Deviation 0.058
|
0.024 g/cm2
Standard Deviation 0.043
|
0.029 g/cm2
Standard Deviation 0.043
|
SECONDARY outcome
Timeframe: Baseline and 24 monthsPopulation: Subjects who were lost to follow up prior to the 24 month visit were not included in the analyses
Change in osteocalcin
Outcome measures
| Measure |
Subjects With CF Taking Ivacaftor
n=23 Participants
Subjects with CF ages 6 to 75 years old who will be or have started taking ivacaftor within the previous 6 months
|
Subjects With CF Not Taking Ivacaftor
n=21 Participants
Subjects with CF ages 6 to 75 years old who will not be taking ivacaftor, matched for age, race, and gender with cohort 1
|
Healthy Subjects
n=24 Participants
Healthy subjects with no medical conditions known to affect bone between the ages of 6 to 75 years old, matched for age, race, and gender with cohort 2.
|
|---|---|---|---|
|
Bone Turnover Markers
|
-5.36 ng/ml
Standard Deviation 12.89
|
-4.51 ng/ml
Standard Deviation 16.27
|
-4.5 ng/ml
Standard Deviation 14.55
|
Adverse Events
Subjects With CF Taking Ivacaftor
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Subjects With CF Not Taking Ivacaftor
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Healthy Subjects
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Subjects With CF Taking Ivacaftor
n=26 participants at risk
Subjects with CF ages 6 to 75 years old who will be or have started taking ivacaftor within the previous 6 months
|
Subjects With CF Not Taking Ivacaftor
n=26 participants at risk
Subjects with CF ages 6 to 75 years old who will not be taking ivacaftor, matched for age, race, and gender with cohort 1
|
Healthy Subjects
n=26 participants at risk
Healthy subjects with no medical conditions known to affect bone between the ages of 6 to 75 years old, matched for age, race, and gender with cohort 2.
|
|---|---|---|---|
|
Investigations
Minor
|
0.00%
0/26 • 2 years
This was an observational study following subjects prospectively over time without an intervention. Any medications, including ivacaftor, were managed by subjects' physician. Therefore, adverse events associated with this study were related to study procedures only. One subject felt lightheaded after venipuncture, improved with food and water, and this was the only adverse event reported.
|
3.8%
1/26 • Number of events 1 • 2 years
This was an observational study following subjects prospectively over time without an intervention. Any medications, including ivacaftor, were managed by subjects' physician. Therefore, adverse events associated with this study were related to study procedures only. One subject felt lightheaded after venipuncture, improved with food and water, and this was the only adverse event reported.
|
0.00%
0/26 • 2 years
This was an observational study following subjects prospectively over time without an intervention. Any medications, including ivacaftor, were managed by subjects' physician. Therefore, adverse events associated with this study were related to study procedures only. One subject felt lightheaded after venipuncture, improved with food and water, and this was the only adverse event reported.
|
Additional Information
Dr. Melissa Putman
Massachusetts General Hospital Endocrine Unit
Phone: 857-218-5017
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place