Trial Outcomes & Findings for Bone Health in Facioscapulohumeral Muscular Dystrophy (NCT NCT02413190)

NCT ID: NCT02413190

Last Updated: 2019-11-05

Results Overview

To determine if bone mineral density is reduced in individuals with FSHD compared to normative data of individuals of the same age and gender without FSHD.The Bone Mineral Density Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). Negative numbers indicate values lower than the reference population and positive numbers indicate values higher than the reference population

Recruitment status

COMPLETED

Target enrollment

94 participants

Primary outcome timeframe

Single visit

Results posted on

2019-11-05

Participant Flow

Participant milestones

Participant milestones
Measure
FSHD
94 subjects
Overall Study
STARTED
94
Overall Study
COMPLETED
94
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Bone Health in Facioscapulohumeral Muscular Dystrophy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
FSHD
n=94 Participants
94 subjects with genetically confirmed FSHD1
Age, Continuous
48.9 years
STANDARD_DEVIATION 51.2 • n=99 Participants
Sex: Female, Male
Female
46 Participants
n=99 Participants
Sex: Female, Male
Male
48 Participants
n=99 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
Race (NIH/OMB)
Asian
4 Participants
n=99 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
Race (NIH/OMB)
Black or African American
1 Participants
n=99 Participants
Race (NIH/OMB)
White
88 Participants
n=99 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
Race (NIH/OMB)
Unknown or Not Reported
1 Participants
n=99 Participants
Region of Enrollment
United States
48 Participants
n=99 Participants
Region of Enrollment
Australia
46 Participants
n=99 Participants

PRIMARY outcome

Timeframe: Single visit

Population: Z score: Higher numbers represent higher bone mineral density.

To determine if bone mineral density is reduced in individuals with FSHD compared to normative data of individuals of the same age and gender without FSHD.The Bone Mineral Density Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). Negative numbers indicate values lower than the reference population and positive numbers indicate values higher than the reference population

Outcome measures

Outcome measures
Measure
FSHD
n=94 Participants
94 subjects with genetically confirmed FSHD1
Bone Mineral Density in FSHD
-0.4 z-score
Interval -4.0 to 3.0

Adverse Events

FSHD

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Kathryn Wagner

Kennedy Krieger Institute

Phone: 443-923-9525

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place