Trial Outcomes & Findings for Androgen Effect on Klinefelter Syndrome Motor Outcome (NCT NCT00348946)
NCT ID: NCT00348946
Last Updated: 2021-07-08
Results Overview
Outcome measures were tested using the following assessments: Bruininks-Osertesky Test of Motor Proficiency (BOT) subscales of (1) Visual Motor Control, (2) Upper limb Speed, and (3) Strength, Physical and Neurological Evaluation for Soft Signs (PANESS), and Hand Strength Dynamometer. BOT assess the child's motor development and includes standard scores (mean=100, SD=15) and subtest scores and is normed for sex and age (4-14.5 years). PANESS assesses the time required to press thumb to 4 fingers 20 times for the dominant and nondominant hands and includes standard scores (mean=100, SD=15) with age-specific norms (4-18 years). Hand strength dynamometer assess hand strength in the dominant and nondominant hands and includes standard scores (mean=100, SD=15). Data is expressed as standard scores with mean of 100 and SD of 15. The minimum standard score is 50; the maximum standard score is 145. Higher scorers imply better function.
COMPLETED
PHASE2
93 participants
2 years per subject
2021-07-08
Participant Flow
Participant milestones
| Measure |
Oxandrolone
androgen oxandrolone: Oxandrolone ,0.6 \>mg/kg/day, orally, for 2 years.
|
Placebo
placebo: an inactive substance
|
|---|---|---|
|
Overall Study
STARTED
|
46
|
47
|
|
Overall Study
COMPLETED
|
35
|
35
|
|
Overall Study
NOT COMPLETED
|
11
|
12
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Androgen Effect on Klinefelter Syndrome Motor Outcome
Baseline characteristics by cohort
| Measure |
Oxandrolone
n=46 Participants
androgen oxandrolone: Oxandrolone ,0.6 \>mg/kg/day, orally, for 2 years.
|
Placebo
n=47 Participants
placebo: an inactive substance
|
Total
n=93 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
46 Participants
n=5 Participants
|
47 Participants
n=7 Participants
|
93 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
6.9 years
STANDARD_DEVIATION 2.2 • n=5 Participants
|
8.5 years
STANDARD_DEVIATION 2.6 • n=7 Participants
|
7.7 years
STANDARD_DEVIATION 2.5 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
46 Participants
n=5 Participants
|
47 Participants
n=7 Participants
|
93 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Ethnicity · White
|
33 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
67 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Ethnicity · African American
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Ethnicity · Hispanic
|
5 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Ethnicity · Asian/Pacific Islander
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Ethnicity · Other
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Tanner 1 pubic hair
|
43 Participants
n=5 Participants
|
38 Participants
n=7 Participants
|
81 Participants
n=5 Participants
|
|
BMI Standard Deviation Score (SDS)
|
0.40 kg/m^2
STANDARD_DEVIATION 1.15 • n=5 Participants
|
0.58 kg/m^2
STANDARD_DEVIATION 1.17 • n=7 Participants
|
0.49 kg/m^2
STANDARD_DEVIATION 1.16 • n=5 Participants
|
|
Waist circumference percentile >75 percentile
|
17 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
39 Participants
n=5 Participants
|
|
Total cholesterol
|
167 mg/dL
STANDARD_DEVIATION 33 • n=5 Participants
|
162 mg/dL
STANDARD_DEVIATION 27 • n=7 Participants
|
164 mg/dL
STANDARD_DEVIATION 29 • n=5 Participants
|
|
LDL cholesterol
|
106 mg/dL
STANDARD_DEVIATION 31 • n=5 Participants
|
104 mg/dL
STANDARD_DEVIATION 24 • n=7 Participants
|
104.4 mg/dL
STANDARD_DEVIATION 25.4 • n=5 Participants
|
|
Triglycerides
|
75.4 mg/dL
STANDARD_DEVIATION 58.2 • n=5 Participants
|
67.7 mg/dL
STANDARD_DEVIATION 32.6 • n=7 Participants
|
70.7 mg/dL
STANDARD_DEVIATION 46 • n=5 Participants
|
|
Triglycerides > 100 mg/dL
|
9 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
HDL cholesterol
|
46 mg/dL
STANDARD_DEVIATION 10.4 • n=5 Participants
|
44.8 mg/dL
STANDARD_DEVIATION 10.1 • n=7 Participants
|
45.7 mg/dL
STANDARD_DEVIATION 10 • n=5 Participants
|
|
HDL cholesterol < 50 mg/dL
|
31 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
62 Participants
n=5 Participants
|
|
Fasting blood glucose, mg/dL
|
84.8 mg/dL
STANDARD_DEVIATION 8.4 • n=5 Participants
|
85.2 mg/dL
STANDARD_DEVIATION 6.7 • n=7 Participants
|
85.2 mg/dL
STANDARD_DEVIATION 7.6 • n=5 Participants
|
|
Fasting blood glucose > 110 mg/dL
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Systolic blood pressure
|
87.2 mm Hg
STANDARD_DEVIATION 9.7 • n=5 Participants
|
90.9 mm Hg
STANDARD_DEVIATION 9.4 • n=7 Participants
|
88.7 mm Hg
STANDARD_DEVIATION 9.6 • n=5 Participants
|
|
Systolic blood pressure > 90th percentile for age and height
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Diastolic blood pressure
|
52.3 mm Hg
STANDARD_DEVIATION 6.9 • n=5 Participants
|
56.9 mm Hg
STANDARD_DEVIATION 7.3 • n=7 Participants
|
55 mm Hg
STANDARD_DEVIATION 8.5 • n=5 Participants
|
|
Diastolic blood pressure > 90th percentile for age and height
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 years per subjectPopulation: Some participants were not able to complete all test and/or it was determined by the investigator that their results do reflect their ability and they were removed from the analysis.
Outcome measures were tested using the following assessments: Bruininks-Osertesky Test of Motor Proficiency (BOT) subscales of (1) Visual Motor Control, (2) Upper limb Speed, and (3) Strength, Physical and Neurological Evaluation for Soft Signs (PANESS), and Hand Strength Dynamometer. BOT assess the child's motor development and includes standard scores (mean=100, SD=15) and subtest scores and is normed for sex and age (4-14.5 years). PANESS assesses the time required to press thumb to 4 fingers 20 times for the dominant and nondominant hands and includes standard scores (mean=100, SD=15) with age-specific norms (4-18 years). Hand strength dynamometer assess hand strength in the dominant and nondominant hands and includes standard scores (mean=100, SD=15). Data is expressed as standard scores with mean of 100 and SD of 15. The minimum standard score is 50; the maximum standard score is 145. Higher scorers imply better function.
Outcome measures
| Measure |
Oxandrolone
n=35 Participants
androgen oxandrolone: Oxandrolone ,06 \>mg/kg/day, orally, for 2 years
|
Placebo
n=36 Participants
placebo: an inactive substance
|
|---|---|---|
|
Motor Function/Strength
PANESS SS- dominant hand
|
91 score on a scale
Standard Deviation 3
|
91 score on a scale
Standard Deviation 3
|
|
Motor Function/Strength
BOT Visual-motor control SS
|
86 score on a scale
Standard Deviation 1
|
81 score on a scale
Standard Deviation 1
|
|
Motor Function/Strength
BOT Upper limb speed SS
|
92 score on a scale
Standard Deviation 1
|
89 score on a scale
Standard Deviation 2
|
|
Motor Function/Strength
BOT Strength SS
|
88 score on a scale
Standard Deviation 1
|
85 score on a scale
Standard Deviation 1
|
|
Motor Function/Strength
Hand Dynamometer SS- dominant hand
|
123 score on a scale
Standard Deviation 2
|
118 score on a scale
Standard Deviation 2
|
SECONDARY outcome
Timeframe: 2 years per subjectOutcome measures were tested using the Differential Ability Scales - 2nd edition (DAS-II). DAS-II provides an age- and sex-standardized assessment of intellectual functioning (General Concept Ability subscale similar to IQ) in children ages 2-17 years of age (mean=100, SD=15). The Verbal Cluster measures the child's ability to define words and perform verbal reasoning tasks. The Nonverbal Cluster measures the child's inductive and sequential reasoning abilities. The Spatial Cluster measures visuospatial construction ability, spatial memory, and spatial reasoning. Data is expressed as standard scores with mean of 100 and SD of 15. The minimum standard score is 50; the maximum standard score is 145. Higher scores imply better function.
Outcome measures
| Measure |
Oxandrolone
n=35 Participants
androgen oxandrolone: Oxandrolone ,06 \>mg/kg/day, orally, for 2 years
|
Placebo
n=36 Participants
placebo: an inactive substance
|
|---|---|---|
|
Cognitive Function and Language
DAS: General Concept Ability
|
96 score on a scale
Standard Deviation 2
|
94 score on a scale
Standard Deviation 2
|
|
Cognitive Function and Language
DAS: Verbal Cluster
|
92 score on a scale
Standard Deviation 2
|
90 score on a scale
Standard Deviation 2
|
|
Cognitive Function and Language
DAS: Nonverbal Cluster
|
102 score on a scale
Standard Deviation 2
|
101 score on a scale
Standard Deviation 2
|
|
Cognitive Function and Language
DAS: Spatial Cluster
|
96 score on a scale
Standard Deviation 2
|
94 score on a scale
Standard Deviation 2
|
SECONDARY outcome
Timeframe: 2 years per participantPopulation: Some participants were not able to complete all assessments and/or it was determined by the investigator that their results do reflect their ability and they were removed from the analysis .
Outcome measures were tested using the following cognitive assessments: Digit Span Backward, A Neuropsychological Assessment (NEPSY) subscales of (1) Phonemic Fluency and (2) Semantic Fluency, and Connors' Continuous Performance Test (CPT-II) subscales (1) Omissions, (2) Commissions, (3) Hit Reaction, (4) Variability, and (5) Preservations. Digit Span Backward tests working memory and is normed for children ages 5-16 years. Phonemic Fluency measures the number of words that the child can name beginning with the letters F and S (ages 6-12). Semantic Fluency measures the number of words the child can name in the categories food and drink (ages 4-12). CPT-II measures the ability to maintain attention over an extended period of time. All scores are reported as standard scores with a mean of 100 and SD of 15. The minimum standard score is 50; the maximum standard score is 145. Higher scores imply better function.
Outcome measures
| Measure |
Oxandrolone
n=32 Participants
androgen oxandrolone: Oxandrolone ,06 \>mg/kg/day, orally, for 2 years
|
Placebo
n=33 Participants
placebo: an inactive substance
|
|---|---|---|
|
Working Memory/Attention
Digit span backward
|
96 score on a scale
Standard Deviation 2
|
92 score on a scale
Standard Deviation 2
|
|
Working Memory/Attention
Phonemic fluency
|
95 score on a scale
Standard Deviation 3
|
91 score on a scale
Standard Deviation 2
|
|
Working Memory/Attention
Semantic fluency
|
99 score on a scale
Standard Deviation 4
|
94 score on a scale
Standard Deviation 3
|
|
Working Memory/Attention
CPT: hit reaction time
|
84 score on a scale
Standard Deviation 3
|
80 score on a scale
Standard Deviation 3
|
|
Working Memory/Attention
CPT: omissions
|
88 score on a scale
Standard Deviation 4
|
83 score on a scale
Standard Deviation 4
|
|
Working Memory/Attention
CPT: commissions
|
101 score on a scale
Standard Deviation 2
|
100 score on a scale
Standard Deviation 2
|
|
Working Memory/Attention
CPT variability
|
86 score on a scale
Standard Deviation 2
|
86 score on a scale
Standard Deviation 2
|
|
Working Memory/Attention
CPT: preservations
|
76 score on a scale
Standard Deviation 5
|
74 score on a scale
Standard Deviation 5
|
SECONDARY outcome
Timeframe: 2 years per participantsPopulation: Some participants were not able to complete all assessments and/or it was determined by the investigator that their results do reflect their ability and they were removed from the analysis.
Outcome measures were tested using the following social assessments: The Child Behavior Checklist (CBCL) and The Children's Depression Inventory (CDI). The CBCL is standardized measure of behavior problems and social competency normed for children ages 4-16. Higher scores indicate more problems, with the cutoff for the clinical range at a t score greater than or equal to 67. The CDI assess cognitive, affective and behavioral signs of depression in children ages 6-17. The CDI total score reflects the presence of overall depressive symptoms. All scores are expressed as t-scores with a mean of 50 and SD of 10. Lower scores imply better function and higher scores indicate more problem behaviors.
Outcome measures
| Measure |
Oxandrolone
n=35 Participants
androgen oxandrolone: Oxandrolone ,06 \>mg/kg/day, orally, for 2 years
|
Placebo
n=36 Participants
placebo: an inactive substance
|
|---|---|---|
|
Psychosocial and Behavior Domain
CBCL: internalizing total t-score
|
54 score on a scale
Standard Deviation 1
|
57 score on a scale
Standard Deviation 1
|
|
Psychosocial and Behavior Domain
CBCL: externalizing total t-score
|
51 score on a scale
Standard Deviation 2
|
54 score on a scale
Standard Deviation 1
|
|
Psychosocial and Behavior Domain
CDI: total t-score
|
45 score on a scale
Standard Deviation 2
|
47 score on a scale
Standard Deviation 2
|
|
Psychosocial and Behavior Domain
CBCL: behavior total t-score
|
56 score on a scale
Standard Deviation 1
|
59 score on a scale
Standard Deviation 1
|
SECONDARY outcome
Timeframe: 2 years per subjectOutcome measures were tested using The Piers-Harris Self Concept Scale. Scoring provides a total standard score and scores on six subscales: physical appearance and attributes, freedom from anxiety, intellectual and school status, behavioral adjustment, happiness and satisfaction, and popularity. Subscales are summed and standardized to provide the total standard score with a mean of 100 and SD of 15. The minimum standard score is 50; the maximum standard score is 145. Higher scores imply better function.
Outcome measures
| Measure |
Oxandrolone
n=16 Participants
androgen oxandrolone: Oxandrolone ,06 \>mg/kg/day, orally, for 2 years
|
Placebo
n=20 Participants
placebo: an inactive substance
|
|---|---|---|
|
Psychosocial and Behavior Domain
|
105 units on a scale
Standard Deviation 3
|
100 units on a scale
Standard Deviation 2
|
Adverse Events
Oxandrolone
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Oxandrolone
n=46 participants at risk
androgen oxandrolone: Oxandrolone ,0.6 \>mg/kg/day, orally, for 2 years
|
Placebo
n=47 participants at risk
placebo: an inactive substance
|
|---|---|---|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal complaints
|
63.0%
29/46 • Number of events 54 • Two years
|
61.7%
29/47 • Number of events 53 • Two years
|
|
Infections and infestations
Infections (any)
|
78.3%
36/46 • Number of events 77 • Two years
|
80.9%
38/47 • Number of events 77 • Two years
|
|
Nervous system disorders
Headache
|
45.7%
21/46 • Number of events 56 • Two years
|
46.8%
22/47 • Number of events 42 • Two years
|
|
Nervous system disorders
Seizure
|
2.2%
1/46 • Number of events 1 • Two years
|
0.00%
0/47 • Two years
|
|
Musculoskeletal and connective tissue disorders
Emergency room visits - Musculoskeletal
|
10.9%
5/46 • Number of events 5 • Two years
|
4.3%
2/47 • Number of events 2 • Two years
|
|
Immune system disorders
Emergency room visits - Asthma/allergy
|
6.5%
3/46 • Number of events 4 • Two years
|
0.00%
0/47 • Two years
|
|
Gastrointestinal disorders
Emergency room visits - Gastrointestinal
|
4.3%
2/46 • Number of events 2 • Two years
|
2.1%
1/47 • Number of events 1 • Two years
|
|
Nervous system disorders
Emergency room visits
|
4.3%
2/46 • Number of events 2 • Two years
|
2.1%
1/47 • Number of events 1 • Two years
|
|
Infections and infestations
Emergency room visits
|
6.5%
3/46 • Number of events 4 • Two years
|
6.4%
3/47 • Number of events 3 • Two years
|
|
General disorders
Emergency room visits - other
|
2.2%
1/46 • Number of events 1 • Two years
|
2.1%
1/47 • Number of events 1 • Two years
|
|
General disorders
Hospitalizations - Medical
|
2.2%
1/46 • Number of events 1 • Two years
|
2.1%
1/47 • Number of events 1 • Two years
|
|
Psychiatric disorders
Hospitalizations - Psychiatric
|
2.2%
1/46 • Number of events 1 • Two years
|
2.1%
1/47 • Number of events 1 • Two years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place