Effect of Risedronate on Bone Morbidity in Fibrous Dysplasia of Bone

Study Overview
=================
Brief Summary
-----------------
This trial is intended to test the efficacy of an oral bisphosphonate (risedronate) to decrease bone pain and improve radiological aspect in fibrous dysplasia of bone.

Detailed Description
-----------------
In open pilot studies, it has been suggested that bisphosphonates may alleviate bone pain and help decrease the surface of osteolytic lesion in patients with fibrous dysplasia of bone (FD). So, in this randomized placebo controlled trial, we test the hypothesis that the bisphosphonate risedronate reduces bone pain in patients with FD (study I, one year duration) and decrease osteolytic lesions (study II, three years duration). Patients will take risedronate during 2 months courses, every 6 months or a matching placebo. Dosage will be : 30mg tablet/day for adults and 5mg tablet x 2,4 according to the age and weight of the child. All participants will receive calcium and vitamin D. All patients with renal phosphate wasting will receive an oral phosphate supplement.

Official Title
-----------------
Effect of Risedronate on Bone Morbidity in Fibrous Dysplasia of Bone

Conditions
-----------------
Fibrous Dysplasia of Bone

Intervention / Treatment
-----------------
* Drug: risedronate
* Drug: placebo
* Drug: risedronate
* Drug: placebo


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Study I: patients with FD, with bone pain intensity above 3 on visual analogical scale from 0 to 10 Study II: patients with FD with at least one osteolytic lesion and no current bone pain Exclusion Criteria: patients < 8 years old other diseases affecting bone metabolism patients with malignant diseases or other conditions likely to reduce their life expectancy to less than 3 years patients with history of significant upper gastrointestinal disorders renal failure (creatinine clearance < 25 ml/mn) severe liver disease history of iritis or uveitis rickets or osteomalacia allergy to bisphosphonates pregnancy or lactation prior treatment with a bisphosphonate laboratory abnormalities that may be considered as clinically significant by trial physicians

Ages Eligible for Study
-----------------
Minimum Age: 8 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>treatment duration: 1 year | Drug: risedronate<br>* During two months courses, every 6 months : 30mg tablet/day for adults and 10mg/day or 20mg/day for children, according to the age and weight of the child.<br>|
| Placebo Comparator: 2<br>treatment duration: 1 year | Drug: placebo<br>* placebo and risedronate have exactly the same aspect. During two months courses, every 6 months : 30mg tablet/day for adults and 10mg/day or 20mg/day for children, according to the age and weight of the child.<br>|
| Experimental: 3<br>duration treatment: 3 years | Drug: risedronate<br>* During two months courses, every 6 months : 30mg tablet/day for adults and 10mg/day or 20mg/day for children, according to the age and weight of the child.<br>|
| Placebo Comparator: 4<br>treatment duration: 3 years | Drug: placebo<br>* placebo and risedronate have exactly the same aspect. During two months courses, every 6 months : 30mg tablet/day for adults and 10mg/day or 20mg/day for children, according to the age and weight of the child.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Intensity of bone pain, assessed by visual analogical scale ranging from 0 to 10, on the most painful site. |  | one year | 
| Surface of osteolytic lesions at three years. Radiological improvement. |  | Three years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Variation of biochemical markers of bone turnover at three years |  | three years | 
| Number of painful sites |  | one year | 
| Improvement in quality of life |  | one to three years | 
| Variation in bone mineral density of the femoral neck at three years |  | three years | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
fibrous dysplasia of bone, Mac Cune Albright syndrome, bisphosphonates, risedronate