Clinical trial NCT03301285: Efficacy of Zoledronic Acid in Osteoporosis of Children With Multiple Disabilities
Official title: Study of Efficacy of Zoledronic Acid in the Management of Osteoporosis in Children With Multiple Disabilities
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Multiple Disability
Osteoporosis
Lead sponsor: Central Hospital, Nancy, France
Sponsor class: OTHER
First posted: 2017-10-04
Enrollment: 60
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_CROSSOVER
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All children followed for osteoporosis associated to multiple disabilities, treated with zoledronic acid, followed at pediatric department of Nancy hospital, between 01/01/2012 and 11/01/2016.

Brief summary: The main purpose of this study is the evaluation of the efficacy of zoledronic acid on bone density in children with osteoporosis caused by multiple disabilities.

Secondary purposes are:

1. Description of child population with osteoporosis in the context of motor impairment in Lorraine region
2. Description of osteoporosis stage (level of bone mineralization and clinical consequences) in children with multiple disabilities
3. Description of current osteoporosis preventive care
4. Description of risk factors associated to bone status (drugs)
5. Evaluation of zoledronic acid treatment on fracture numbers
6. Evaluation of zoledronic acid on phosphocalcic profile
7. Description of side effects of zoledronic acid in this indication
8. Description of treatment effects in the sub-population of children with Rett syndrome.


Eligibility criteria: Inclusion Criteria:

* Non refusal of parents of participation of their child to the study
* Patients followed for multiple disabilities
* Osteoporosis: lumbar osteodensitometry z-score \<-2.5 SD associated or not to pathologic fracture

Exclusion Criteria:

* Bone pathology due to other genetic reasons (rickets, osteogenesis imperfecta)
* Absence of multiple disabilities

Primary outcomes:
measure: Change from baseline (before start of zoledronic acid treatment) lumbar bone density
timeFrame: through study completion, 4 years
Secondary outcomes:
measure: Age
timeFrame: Baseline
measure: Sex
timeFrame: Baseline
measure: Height
timeFrame: Baseline
measure: Weight
timeFrame: Baseline
measure: Occurrence of bone fractures
timeFrame: Until baseline
measure: Long-term administration of vitamin/calcium supplement or not
timeFrame: baseline
measure: Administration of drugs or not
timeFrame: baseline
measure: Number of fractures after the start of zoledronic acid treatment
timeFrame: through study completion, 4 years
measure: Change from baseline phosphocalcic profile evaluation
timeFrame: through study completion, 4 years
measure: Frequency of side effects
timeFrame: through study completion, 4 years
measure: Change from baseline lumbar bone density in sub-population of children with Rett syndrome after 1 year of zoledronic acid treatment
timeFrame: 1 year from baseline
Other outcomes:
Locations:
city: Vandoeuvre les nancy
country: France
facility: Children's Hospital - CHRU de Nancy
Study officials:
References: