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

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

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

Study Overview

• Status: Completed
• Conditions: SAPHO Syndrome
• Intervention / Treatment: Drug: pamidronate disodium

Detailed Description

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).

• Study Type: Interventional
• Enrollment (Actual): 30
• Phase: Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 68 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Male and female patients between the ages of 18 and 70 years

2. 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)
3. MRI shows bone marrow edema in affected site in patients
4. Blood serum of patients show the normal white blood cell count, liver and renal function
5. Patients who like to be followed up for 1 years
6. Other protocol defined inclusion criteria may apply

Exclusion Criteria:

1. Women in pregnancy or lactation.
2. Septic osteomyelitis
3. Infectious chest wall arthritis
4. Infections PPP
5. Palmo-plantar keratodermia
6. DISH except for fortuitous association
7. Osteoarticular manifestations of retinoid therapy
8. Other protocol defined exclusion criteria may apply

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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)

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital
• Investigators: Principal Investigator: Chen Li, Master, Department of Traditional Chinese Medicine, Peking Union Medical College Hospital

Study record dates

Study Major Dates

• Study Start: October 1, 2015
• Primary Completion (Actual): October 1, 2016
• Study Completion (Actual): October 1, 2016

Study Registration Dates

• First Submitted: August 28, 2015
• First Submitted That Met QC Criteria: September 8, 2015
• First Posted (Estimate): September 9, 2015

Study Record Updates

• Last Update Posted (Estimate): October 13, 2016
• Last Update Submitted That Met QC Criteria: October 12, 2016
• Last Verified: September 1, 2015

More Information

Terms related to this study

• Keywords: Treatment; Bisphosphonates; Response rate
• Additional Relevant MeSH Terms: Pathologic Processes; Disease; Musculoskeletal Diseases; Bone Diseases; Bone Diseases, Developmental; Osteochondrodysplasias; Syndrome; Acquired Hyperostosis Syndrome; Physiological Effects of Drugs; Bone Density Conservation Agents; Pamidronate
• Other Study ID Numbers: PUMCH-SAPHO-01