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

SAPHO Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02544659
• Other study ID #: PUMCH-SAPHO-01
• Status: Completed
• Phase: Phase 1
• First received: August 28, 2015
• Last updated: October 12, 2016
• Start date: October 2015
• Est. completion date: October 2016

Study information

• Verified date: September 2015
• Source: Peking Union Medical College Hospital
• Contact: n/a
• Is FDA regulated: No
• Health authority: China: National Natural Science Foundation
• Study type: Interventional

Clinical Trial Summary

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

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: October 2016
• Est. primary completion date: October 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 70 Years

Eligibility

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 Design

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• SAPHO Syndrome
• Syndrome

Intervention

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)

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Peking Union Medical College Hospital

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)	No
Secondary	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)	No
Secondary	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)	No
Secondary	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)	No
Secondary	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)	No
Secondary	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)	No
Secondary	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)	No
Secondary	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)	No
Secondary	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)	No
Secondary	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)	No
Secondary	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)	No