Compounds > oxandrolone
Page last updated: 2024-11-07

oxandrolone and Becker Muscular Dystrophy

oxandrolone has been researched along with Becker Muscular Dystrophy in 4 studies

Oxandrolone: A synthetic hormone with anabolic and androgenic properties.

Research Excerpts

ExcerptRelevanceReference
" No adverse reactions attributable to oxandrolone were recorded."2.70A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophy. ( Fenichel, GM; Florence, J; Griggs, RC; King, WM; Kissel, J; Kramer, TI; Mendell, JR; Moxley, RT; Pandya, S; Pestronk, A; Robison, VD; Sheng, K; Wang, H, 2001)
" This report also confirms that our regime of low dosage, intermittent prednisolone, with cycles of 10 days of treatment, either per month or alternating with 10 days off treatment, is well tolerated and can therefore be recommended for long-term use in children with Duchenne muscular dystrophy."1.31Remission of clinical signs in early duchenne muscular dystrophy on intermittent low-dosage prednisolone therapy. ( Dubowitz, V; Kinali, M; Main, M; Mercuri, E; Muntoni, F, 2002)

Research

Studies (4)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's3 (75.00)29.6817
2010's1 (25.00)24.3611
2020's0 (0.00)2.80

Authors

AuthorsStudies
McNeil, DE1
Davis, C1
Jillapalli, D1
Targum, S1
Durmowicz, A1
Coté, TR1
Dubowitz, V1
Kinali, M1
Main, M1
Mercuri, E1
Muntoni, F1
Balagopal, P1
Olney, R1
Darmaun, D1
Mougey, E1
Dokler, M1
Sieck, G1
Hammond, D1
Fenichel, GM1
Griggs, RC1
Kissel, J1
Kramer, TI1
Mendell, JR1
Moxley, RT1
Pestronk, A1
Sheng, K1
Florence, J1
King, WM1
Pandya, S1
Robison, VD1
Wang, H1

Clinical Trials (1)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen[NCT01603407]Phase 3196 participants (Actual)Interventional2013-01-31Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

6 Minute Walk Test

Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventionmeters (Least Squares Mean)
Daily Prednisone384.95
Daily Deflazacort384.17
Intermittent Prednisone346.81

Forced Vital Capacity

Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventionliters (Least Squares Mean)
Daily Prednisone1.4
Daily Deflazacort1.4
Intermittent Prednisone1.5

Fractional Shortening Percent

Measured by trans-thoracic echocardiogram and 12-lead ECG. (NCT01603407)
Timeframe: 36 months

Interventionpercentage of fractional shortening (Least Squares Mean)
Daily Prednisone33.74
Daily Deflazacort34.01
Intermittent Prednisone34.33

Heart Rate

Measured by trans-thoracic echocardiogram and 12-lead ECG. (NCT01603407)
Timeframe: 36 months

Interventionbpm (Least Squares Mean)
Daily Prednisone94.10
Daily Deflazacort93.52
Intermittent Prednisone91.65

Left Ventricular Ejection Fraction Percent

Measured by trans-thoracic echocardiogram and 12-lead ECG. (NCT01603407)
Timeframe: 36 months

Interventionpercentage of ejection fraction (Least Squares Mean)
Daily Prednisone61.88
Daily Deflazacort62.65
Intermittent Prednisone62.45

North Star Ambulatory Assessment (NSAA) Score

"The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy (DMD). It is usually used to monitor the progression of the disease and treatment effects.~The activities are graded as follows:~2 - Normal - no obvious modification of activity~1 - Modified method but achieves goal independent of physical assistance from another 0 - Unable to achieve independently This scale is ordinal with 34 as the maximum score indicating fully-independent function." (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventionscore on a scale (Least Squares Mean)
Daily Prednisone23.7
Daily Deflazacort24.0
Intermittent Prednisone20.7

Number of Participants Who Tolerated the Regimen

The number of participants who completed 36 months of follow-up on the originally assigned dosage (for weight) of study medication. (NCT01603407)
Timeframe: 3 years

InterventionParticipants (Count of Participants)
Daily Prednisone36
Daily Deflazacort36
Intermittent Prednisone37

Participant Body Mass Index

(NCT01603407)
Timeframe: 36 months

Interventionkilograms/square meter (Least Squares Mean)
Daily Prednisone18.9
Daily Deflazacort18.3
Intermittent Prednisone18.1

Participant Height

(NCT01603407)
Timeframe: 36 months

Interventioncentimeters (Least Squares Mean)
Daily Prednisone116.8
Daily Deflazacort115.3
Intermittent Prednisone119.9

Participant Weight

(NCT01603407)
Timeframe: 36 months

Interventionkilograms (Least Squares Mean)
Daily Prednisone26.3
Daily Deflazacort24.9
Intermittent Prednisone26.3

PR Interval

Measured by trans-thoracic echocardiogram and 12-lead ECG. (NCT01603407)
Timeframe: 36 months

Interventionmilliseconds (Least Squares Mean)
Daily Prednisone115.59
Daily Deflazacort116.87
Intermittent Prednisone117.90

Quality of Life - Parent

Quality of life was measured by parent/guardian self-report for all children utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life for the child. (NCT01603407)
Timeframe: Average of Months 12, 24, and 36 visits

Interventionscore on a scale (Least Squares Mean)
Daily Prednisone64.88
Daily Deflazacort63.71
Intermittent Prednisone61.33

Quality of Life- Child

Quality of life was measured by child self-report in children age 5 and older utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life. (NCT01603407)
Timeframe: Average of Months 12, 24, and 36 visits

Interventionscore on a scale (Least Squares Mean)
Daily Prednisone67.39
Daily Deflazacort64.96
Intermittent Prednisone65.07

Range of Motion (Goniometry) of Left Ankle

Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventiondegrees (Mean)
Daily Prednisone4.39
Daily Deflazacort3.29
Intermittent Prednisone2.67

Range of Motion (Goniometry) of Right Ankle

Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventiondegrees (Mean)
Daily Prednisone4.05
Daily Deflazacort2.81
Intermittent Prednisone2.29

Rise From the Floor Velocity

Reciprocal of time to rise from the floor (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventionrise/sec (Least Squares Mean)
Daily Prednisone0.24
Daily Deflazacort0.24
Intermittent Prednisone0.18

Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score

The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventionscore on a scale (Least Squares Mean)
Daily Prednisone71.2
Daily Deflazacort67.8
Intermittent Prednisone65.1

Trials

1 trial available for oxandrolone and Becker Muscular Dystrophy

ArticleYear
A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophy.
    Neurology, 2001, Apr-24, Volume: 56, Issue:8

    Topics: Anabolic Agents; Child; Child, Preschool; Double-Blind Method; Humans; Male; Muscle Tonus; Muscular

2001

Other Studies

3 other studies available for oxandrolone and Becker Muscular Dystrophy

ArticleYear
Duchenne muscular dystrophy: Drug development and regulatory considerations.
    Muscle & nerve, 2010, Volume: 41, Issue:6

    Topics: Androgens; Animals; Clinical Trials as Topic; Drug Therapy; Gentamicins; Humans; Legislation, Drug;

2010
Remission of clinical signs in early duchenne muscular dystrophy on intermittent low-dosage prednisolone therapy.
    European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society, 2002, Volume: 6, Issue:3

    Topics: Anabolic Agents; Antineoplastic Agents, Hormonal; Child, Preschool; Dose-Response Relationship, Drug

2002
Oxandrolone enhances skeletal muscle myosin synthesis and alters global gene expression profile in Duchenne muscular dystrophy.
    American journal of physiology. Endocrinology and metabolism, 2006, Volume: 290, Issue:3

    Topics: Adolescent; Albumins; Androgens; Child; Gene Expression Profiling; Gene Expression Regulation; Human

2006