Dettagli per singolo studio

DEC-NET Serial number GB388

Published online	11/04/2005 16.09.00
Last updated	10/04/2006 10.19.40


This trial has been approved by an ethics committee
Current trial status	Open (actively recruiting new participants)
First subject enrolment Target N. of subjects	09/2005 60
Major Disease (ICD9 class)	OSTEOGENESIS IMPERFECTA
Experimental drug
Risedronate Treatment regimen (dosage and duration) 1mg/kg/week
Gender	Both
Age (range)	children under 16
Eligibility criteria
Inclusion criteria
Any OI child at high risk for fractures as defined by a history of at least 2 non-traumatic or low impact fractures within 2 years of enrollment and at least one of the following factors related to disease severity Musculoskeletal pain Poor mobility as a result of fractures and/or pain History of a surgical procedure related to bone fractures or bone deformities Must be able to remain upright without food for 30 minutes after taking medication
Exclusion criteria
History of intolerance of or allergic reaction risedronate Protocol violations leading to withdrawal from the original study
Trial design/methodology
Phase	4
Kind of study	Efficacy
Design	Randomised Blinded Single blind
Purpose of study
An open label extension study to further investigate the therapeutic efficacy and dose response of risedronate treatment in children with moderate to severe osteogenesis imperfecta
Primary outcomes
Change in lumbar spine bone mineral density z-score
Secondary outcomes
Total body bone mass, lumbar spine bone area, fracture frequency, vertebral height, pain, mobility
Summary of study design, objectives, and ongoing research findings
We would plan to offer a period of extended treatment using risedronate at a dose of 1mg/kg/week to children participating in our current study. The target recruitment for the study was 60 children randomised to receive 0.2, 1 or 2mg/kg/week of risedronate for 2 years (double-blind part of the study), followed by 1mg/kg/week for 1 year (open label part of the study). The primary outcome for the study was number of fractures. Assessments are conducted at baseline 3,6, 12, 18, 24, 30 and 36 months. The assessments comprised bone density, bone turnover markers and clinical assessment and fracture recording at each visit. Bone biopsy was undertaken at baseline and after 2 years and skeletal surveys at annual intervals. Families report fractures as they occur and record pain and diet in a diary. We propose to extend the open label administration of risedronate by a further 2 years to a total of 5 years, similar to the duration of bisphosphonates therapy in adults. We would undertake assessment of bone turnover markers and bone density at 6 month intervals. Bone turnover is assessed on a 5ml sample of blood and a urine sample. Bone density is assessed using a dual energy x-ray absorptiometer. The diary system for recording fractures and bone pain would also continue. We would x-ray the spine annually and repeat the bone biopsy at the end of the 5 year period. The bone biopsy will provide information on the changes occurring in response to treatment at the tissue level. This is a routine procedure in our hospital prior to starting children on bone-active therapy and also for monitoring purposes.