Study of the Efficacy and Safety of Etanercept Treatment in Patients With SAPHO Syndrome (SAPHO)

A Multicenter, Randomized, Double-blind Clinical Trial Evaluating the Efficacy and Safety of Etanercept Versus Placebo in the Treatment of Patients With SAPHO Syndrome

The study includes adult patients with SAPHO syndrome (ORPHA: 793), meeting the modified classification criteria according to Kahn (2003), with the ineffectiveness of standard treatment (patient's global assessment of the disease on the VAS scale greater than or equal to 4 cm with accompanying pain on the VAS scale greater than or equal to 4 cm) treated with non-steroidal anti-inflammatory drugs in a stable dose for at least 4 weeks and/or classical disease-modifying antirheumatic drugs in stable doses for at least 12 weeks.

Study Overview

• Status: Recruiting
• Conditions: SAPHO Syndrome
• Intervention / Treatment: Drug: Etanercept; Drug: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: Jakub Wroński, PhD, MD; Phone Number: +48 22 6880632; Email: jakub.wronski@spartanska.pl

Study Locations

• Poland
  • Mazowieckie
    • Warsaw, Mazowieckie, Poland, 02-637
      • Recruiting
      • Centrum Wsparcia Badań Klinicznych
      • Contact: Agnieszka Kurowska; Phone Number: +48 691 326 114; Email: agnieszka.kurowska@spartanska.pl
      • Contact: Marta Kurek; Phone Number: +48 603 315 033; Email: marta.kurek@spartanska.pl

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Diagnosis of SAPHO syndrome according to modified Kahn criteria from 2003.
2. Age over 18.
3. Patient overall disease and pain assessment on VAS both ≥ 4 cm.
4. Expressing informed consent to participate in the study.

Exclusion Criteria:

1. According to the Summary of Product Characteristics (SmPC) for Enbrel.
2. Pregnancy, breastfeeding, inability to use effective contraception during the examination.
3. Change in the dose of NSAIDs treatment in the last 4 weeks.
4. Dose modification of disease-modifying antirheumatic drugs (DMARDs) over the past 12 weeks.
5. Use of biological drugs / synthetic targeted drugs in the last 12 weeks.
6. Use of corticosteroids (orally or local injections), bisphosphonates and/or antibiotics in the last 4 weeks.
7. Any medical condition that the investigator judges to contraindicate etanercept treatment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Etanercept; treatment with etanercept in addition to NSAID treatment and/or classic Disease Modifying Antirheumatic Drugs	Drug: Etanercept; treatment with etanercept in addition to NSAID treatment and/or classic Disease Modifying Antirheumatic Drugs
Placebo Comparator: Placebo; treatment with placeboin addition to NSAID treatment and/or classic Disease Modifying Antirheumatic Drugs	Drug: Placebo; treatment with placebo in addition to NSAID treatment and/or classic Disease Modifying Antirheumatic Drugs

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in the scope of disease activity as assessed by the patient- a decrease in the overall disease activity on the Visual Analogue Scale by min. 50 percent and a decrease in pain assessed by the patient on the Visual Analogue Scale by min. 50 percent	Change in the scope of disease activity as assessed by the patient - a decrease in the overall disease activity as assessed by the patient on the Visual Analogue Scale by min. 50 percent after 12 weeks from randomization day and a decrease in pain assessed by the patient on the Visual Analogue Scale by min. 50 percent after 12 weeks from randomization day. The minimum value is - 0, and the maximum value is - 100 mm. The higher scores mean a worse outcome. A decrease by a minimum of 50 percent means a better outcome.	12 weeks (day 85)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of remission	Occurrence of remission - complete resolution of osteoarticular and skin complaints in the patient's assessment - after 4, 8 and 12 weeks from randomization day	after 4, 8 and 12 weeks
Occurrence of partial remission	Occurrence of partial remission - complete resolution of osteoarticular or skin symptoms in the patient's assessment - after 4, 8 and 12 weeks from randomization day	after 4, 8 and 12 weeks
Change in patient-assessed disease activity	Change in patient-assessed disease activity - a minimum 50 percent decrease in patient-assessed Visual Analogue Scale overall disease activity from the randomization score at weeks 4 and 8 and a minimum 50 percent decrease in patient-assessed Visual Analogue Scale pain from the score on daily basis randomization after 4 and 8 weeks. The minimum value is - 0, and the maximum is - 100 mm. The higher scores mean a worse outcome. A decrease by a minimum of 50 percent means a better outcome.	after 4 and 8 weeks
Occurrence of the patient acceptable symptom state (PASS score)	Occurrence of the patient acceptable symptom state (PASS score) after 4, 8 and 12 weeks from randomization day. Possible answer- "yes" or "no", with "yes" means a better outcome.	after 4, 8 and 12 weeks
Change in physician-assessed disease activity	Improvement in physician-assessed disease activity - a minimum 50 percent decrease in physician-assessed overall disease activity on the Visual Analogue Scale from the randomization score at 4, 8 and 12 weeks. The minimum value is - 0, and the maximum is - 100 mm. The higher scores mean a worse outcome. A decrease by a minimum of 50 percent means a better outcome.	at 4, 8 and 12 week
Change in the C-reactive Protein from Randomization Day Score	Change in C-reactive Protein from Randomization Day Score at Weeks 4, 8 and 12. A decrease in C-reactive Protein means improvement.	at Weeks 4, 8 and 12
Change in the Erythrocyte Sedimentation Rate from Randomization Day Score	Change in the Erythrocyte Sedimentation Rate Score from Randomization Day Score at Weeks 4, 8 and 12. A decrease in the Erythrocyte Sedimentation Rate Score means improvement.	at Weeks 4, 8 and 12
Change in quality of life on the Short Form-36 health survey	Change in quality of life on the Short Form -36 (SF-36) health survey from the score on the day of randomization at 4, 8 and 12 weeks. The SF-36 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability.	at 4, 8 and 12 weeks
Change in the Work Productivity and Activity Impairments (WPAI) from the Randomization Day Score	Change in the Work Productivity and Activity Impairments (WPAI) from the Randomization Day Score after 4, 8 and 12 weeks. WPAI contains four domains. For each domain, the minimum value is 0 percent, and the maximum is 100 percent. Decreased Work Productivity and Activity Impairments (WPAI) Score means improvement.	after 4, 8 and 12 weeks
decrease in Ankylosing Spondylitis Disease Activity Score (ASDAS- C-reactive protein) ≥1.1	In patients with axial involvement, a decrease in Ankylosing Spondylitis Disease Activity Score (ASDAS- C-reactive protein) ≥1.1 from the Randomization Day Score at weeks 4, 8, and 12. The minimum value is 0, the maximum value is infinity. A decrease means a better outcome.	after 4, 8 and 12 weeks
a decrease in the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) Score of at least 50 percent	a decrease in the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) Score of at least 50 percent from the Randomization Day Score at weeks 4, 8, and 12 was achieved (applies to patients with axial involvement). The minimum value is 0, the maximum value is 10. A decrease by a minimum of 50 percent means a better outcome.	after 4, 8 and 12 weeks
status of remission- Ankylosing Spondylitis Disease Activity Score (ASDAS- C-reactive protein) below 1.3	status of remission - Ankylosing Spondylitis Disease Activity Score (ASDAS- C-reactive protein) below 1.3 - after 4, 8 and 12 weeks from randomization day (applies to patients with axial involvement). A score below 1.3 means a better outcome.	after 4, 8 and 12 weeks
decrease in the Bath Ankylosing Spondylitis Functional Index (BASFI) by a minimum of 50 percent	a decrease in the Bath Ankylosing Spondylitis Functional Index (BASFI) by a minimum of 50 percent compared to the result on the day of randomization after 4, 8 and 12 weeks (applies to patients with axial involvement). The minimum value is 0, the maximum value is 10. A decrease by a minimum of 50 percent means a better outcome.	after 4, 8 and 12 weeks
decrease in the Dermatology Life Quality Index (DLQI) by at least 50 percent from the result on the day of randomization	In patients with severe acne - a decrease in the Dermatology Life Quality Index (DLQI) by at least 50 percent from the result on the day of randomization after 4, 8 and 12 weeks. The minimum value is 0, the maximum value is 30. The decrease by a minimum of 50 percent means a better outcome.	after 4, 8 and 12 weeks
decrease in Body Surface Area (BSA) index by a minimum of 50 percent from the score on the day of randomization after 4, 8 and 12 weeks	For patients with psoriasis: decrease in Body Surface Area (BSA) index by a minimum of 50 percent from the score on the day of randomization after 4, 8 and 12 weeks. The minimum value is 0 percent, and the maximum value is 100 percent. The decrease by a minimum of 50 percent means a better outcome.	after 4, 8 and 12 weeks
decrease in the Dermatology Life Quality Index (DLQI) by a minimum of 50 percent	For patients with psoriasis: decrease in the Dermatology Life Quality Index (DLQI) by a minimum of 50 percent from the score on the day of randomization after 4, 8 and 12 weeks. The minimum value is 0, the maximum value is 30. The decrease by a minimum of 50 percent means a better outcome.	after 4, 8 and 12 weeks

Collaborators and Investigators

• Sponsor: National Institute of Geriatrics, Rheumatology and Rehabilitation, Poland
• Collaborators: Medical Research Agency, Poland
• Investigators: Principal Investigator: Jakub Wroński, PhD, MD, National Institute of Geriatrics, Rheumatology and Rehabilitation

Study record dates

Study Major Dates

• Study Start (Estimated): December 13, 2023
• Primary Completion (Estimated): October 18, 2028
• Study Completion (Estimated): October 18, 2028

Study Registration Dates

• First Submitted: July 28, 2023
• First Submitted That Met QC Criteria: August 21, 2023
• First Posted (Actual): August 25, 2023

Study Record Updates

• Last Update Posted (Actual): November 14, 2023
• Last Update Submitted That Met QC Criteria: November 13, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Disease; Musculoskeletal Diseases; Bone Diseases; Bone Diseases, Developmental; Osteochondrodysplasias; Syndrome; Acquired Hyperostosis Syndrome; Physiological Effects of Drugs; Peripheral Nervous System Agents; Analgesics; Sensory System Agents; Anti-Inflammatory Agents, Non-Steroidal; Analgesics, Non-Narcotic; Anti-Inflammatory Agents; Antirheumatic Agents; Immunosuppressive Agents; Immunologic Factors; Gastrointestinal Agents; Etanercept
• Other Study ID Numbers: NIGRIR_003SAPHO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No