The effects of pubertal induction on bone health in children with neuromuscular conditions

Phase 3

Recruiting

• Conditions: cerebral palsy; Spinal muscular atrophy; Muscular dystrophies; Neurological - Other neurological disorders; Musculoskeletal - Other muscular and skeletal disorders

• Registration Number: ACTRN12619001372178
• Lead Sponsor: Murdoch Childrens Research Institute

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-10-08
• Last Update Posted: 14 October 2019

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 58

Inclusion Criteria

Participants will include girls 13 years and over, and boys 14.5 years and older, who have not reached puberty as defined by onset of breast development in girls, testicular enlargement in boys >4ml , with associated and continuing changes in feminization or virilisation over at least 6 months and who have one of the following conditions

1)Spinal muscular atrophy Types 2 & 3
2)Cerebral palsy with a level 4 or 5 on the Gross Motor Function Classification System (GMFCS)
3)Muscular dystrophies other than Duchenne (eg Becker) and congenital myopathies
(eg. Nemaline )

Exclusion Criteria

Children who
•Have Duchenne Muscular Dystrophy (because of corticosteroid use interferes with pubertal onset and progress)
•Are currently participating in other trials because altered pubertal status is likely to affect other care aspects in many areas
•Are using or have prior use of bisphosphonates or other bone modifying drugs
•Have used corticosteroids for the last 12 months
•Has active or completed puberty
•Have a contraindication to Androgens (Testogel®) or oestrogens (Climara®) use due to a severe pro-coagulopathic disorder or medication allergies
•Have vertebral fractures greater than Genant 3 found on back x-ray at screening or 6 months prior as this will require bisphosphonate treatment
•Previous incidences of deep vein thrombosis, clots or stroke
•Older than 18 years of age

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Primary outcome measure will be bone mass as assessed by Bone mineral density (BMD) using Dual energy X-ray Absorptiometry (DXA) scan for trabecular bone and Peripheral Quantitative Computerised Tomography (pQCT) [Baseline, 12 and 24 months.]

Secondary Outcome Measures

Name	Time	Method
fracture incidence as detected by review of Xrays[baseline, 12 and 24 months];Quality of life (QoL) scores from WHOQOL-BREF[baseline, 12 and 24 months];change in physical activity using International Physical Activity Questionnaire (IPAQ)[baseline, 12 and 24 months];change in mobility via 6 minute walk test[baseline, 12 and 24 months];change in Parathyroid levels in blood[baseline, 12 and 24 months];change in strength via grip strength test using hand dynamometer[baseline, 12 and 24 months];change in alkalaine phosphatase levels in blood [baseline, 12 and 24 months];change in vitamin d levels in blood[baseline, 12 and 24 months]