None listed
Conditions
Brief summary
Children and teenagers with chronic neurological disease are at risk of having thin bones (osteoporosis). Throughout your life, new bone cells grow and old bone cells break down to make room for the new, stronger bone. Osteoporosis is characterised by the old bone breaking down faster than the new bone can replace it, resulting in the bones losing minerals (such as calcium). This makes bones weaker and more likely to break even after a minor injury. All the bones in your body are weaker if you have osteoporosis but not everyone who has osteoporosis gets a broken bone. Bone pain and fracture are common complications of osteoporosis. Bisphosphonates are a class of drug that slows down the thinning of bone, and are the standard treatment for osteoporosis in adults. Bisphophonates come in oral and intravenous forms. Gastrointestinal problems and compliance issues associated with oral bisphosphonates can be avoided by the use of intravenous bisphosphonates. Pamidronate, an intravenous bisphosphonate, is given every 6-8 weeks and has been shown to be effective in children. Zoledronate, a new potent intravenous bisphosphonate, is given yearly. Since intravenous bisphosphonates have to be given during a 3 day stay in hospital, zoledronate requires far fewer hospital admissions than does pamidronate.
Interventions
Calcium 600mg oral tablet daily for 6 months
Vitamin D 400 IU oral solution daily for 6 months
Calcium and Vitamin D supplementation will be started at baseline and continued throughout the study.
There is no wash out period.
At 6 months and 18 months patient will recieve intravenous Zoledronate. The dose will be 0.05mg/kg with a maximum dose of 2mg.
The calculated dose of reconstituted zoledronate will be diluted in 50ml of Normal Saline and infused over 30 minutes.
Sponsors
Princess Margaret Hospital for Children
Study design
Allocation
Non-randomised trial
Intervention model
Crossover
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
4 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
Children and adolescents eligible to participate in the study must be: 1. Diagnosed with a chronic neurological condition. 2. Aged = 4 years to < 18, inclusive 3. Have an increased risk of fractures as defined by BMD <-2.5 at one or more sites and, a history of at least one radiologically confirmed, non traumatic or low impact fracture and/or significant bone pain (as determined by appearing to be in pain after other causes of pain have been excluded) 4. Able and willing to participate in the study as evidenced by a parent/ legal guardian signing a valid written consent form. 5. All pre existing factors which could contribute to a reduction in BMD such as diet, sunlight exposure, anticonvulsant medication and weight bearing programme have been addressed 6 months prior to commencement of infusion. 6. If female, must have a negative serum pregnancy test at baseline and be using effective contraception if sexually active.
Exclusion criteria
EXCLUSION CRITERIA 1. Body weight < 10kg. 2. History of cancer within past five years. 3. Untreated rickets within one year of treatment. 4. Documented history of an abnormal or allergic reaction to bisphosphonates. 5. Pubertal delay. 6. Clinically significant abnormal laboratory finding at screening - Abnormal liver function tests – Alanine Amino Transferase (ALT) & Aspartate Amino Transferase (AST) > 2x upper limit of normal. - Abnormal thyroid stimulating hormone (TSH) and/ or parathyroid hormone (PTH). - Serum 25 (OH) vitamin D < 20 nmol/L
Outcome results
None listed