Efficacy of Bisphosphonates in Patients With Synovitis, Acne, Pustulosis, Hyperostosis, and Osteitis (SAPHO) Syndrome

Study Overview
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Brief Summary
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This study is designed to evaluate long term efficacy of intravenous bisphosphonates for bone marrow edema in patients with SAPHO syndrome.

Detailed Description
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The study will evaluate the long term efficacy at 3 days, 3 and 6 months after pamidronate disodium administered. Patients will be administered pamidronate disodium 1 mg/kg, IV, QD, for 3 days, and every 3 months (up to month 6).

Official Title
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Unrandomized, Open Label Single-center Study to Assess the Long Term Efficacy of Intravenous Bisphosphonates for Bone Marrow Edema in Patients With SAPHO Syndrome

Conditions
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SAPHO Syndrome

Intervention / Treatment
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* Drug: pamidronate disodium


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female patients between the ages of 18 and 70 years Only 1 of the 4 inclusion criteria is sufficient to arrive at a diagnosis of SAPHO syndrome： Osteo-articular manifestations of acne conglobata, acne fulminans, or hidradenitis suppurativa Osteo-articular manifestations of PPP Hyperostosis (of the anterior chest wall, limbs or spine) with or without dermatosis CRMO involving the axial or peripheral skeleton with or without dermatosis Palmoplantar pustulosis(PPP); chronic recurrent multifocal osteomyelitis(CRMO) MRI shows bone marrow edema in affected site in patients Blood serum of patients show the normal white blood cell count, liver and renal function Patients who like to be followed up for 1 years Other protocol defined inclusion criteria may apply Exclusion Criteria: Women in pregnancy or lactation. Septic osteomyelitis Infectious chest wall arthritis Infections PPP Palmo-plantar keratodermia DISH except for fortuitous association Osteoarticular manifestations of retinoid therapy Other protocol defined exclusion criteria may apply

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: pamidronate disodium<br>the patients will be administered intravenous pamidronate disodium | Drug: pamidronate disodium<br>* the patients will be administered pamidronate disodium 1 mg/kg, IV, QD, for 3 days, and every 3 month (up to month 6)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| response to the treatment by the change of MRI result at screening, baseline, month 3 and month 6 |  | 3 and 6 month (up to 6 month) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| physician's global assessment of disease activity at screening, baseline, month 3 and month 6 |  | 3 days, 3 and 6 month (up to 6 month) | 
| response to the treatment by the change of Visual Analogue ale/Score (VAS) at screening, baseline, month 3 and month 6 |  | 3 days, 3 and 6 month (up to 6 month) | 
| response to the treatment by the change of bath ankylosing spondylitis disease activity index (BASDAI) at screening, baseline, month 3 and month 6 |  | 3 days, 3 and 6 month (up to 6 month) | 
| response to the treatment by the change of bath ankylosing spondylitis functional index (BASFI) at screening, baseline, month 3 and month 6 |  | 3 days, 3 and 6 month (up to 6 month) | 
| response to the treatment by the change of bath ankylosing spondylitis metroloty index (BASMI) at screening, baseline, month 3 and month 6 |  | 3 days, 3 and 6 month (up to 6 month) | 
| response to the treatment by the change of ankylosing spondylitis disease activity score (ASDAS) at screening, baseline, month 3 and month 6 |  | 3 days, 3 and 6 month (up to 6 month) | 
| response to the treatment by the change of erythrocyte sedimentation rate (ESR) at screening, baseline, month 3 and month 6 |  | 3 days, 3 and 6 month (up to 6 month) | 
| response to the treatment by the change of high sensitivity-C reaction protein (hs-CRP) at screening, baseline, month 3 and month 6 |  | 3 days, 3 and 6 month (up to 6 month) | 
| response to the treatment by the change of Osteocalcin at screening, baseline, month 3 and month 6 |  | 3 days, 3 and 6 month (up to 6 month) | 
| response to the treatment by the change of beta-Crosslaps (beta-CTx) at screening, baseline, month 3 and month 6 |  | 3 days, 3 and 6 month (up to 6 month) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Treatment, Bisphosphonates, Response rate