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

• ClinicalTrials.gov Identifier: NCT06011889
• Recruitment Status: Recruiting
• First Posted: August 25, 2023
• Last Update Posted: November 14, 2023
• Sponsor: National Institute of Geriatrics, Rheumatology and Rehabilitation, Poland
• Collaborator: Medical Research Agency, Poland
• Information provided by (Responsible Party): National Institute of Geriatrics, Rheumatology and Rehabilitation, Poland

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
SAPHO Syndrome	Drug: Etanercept; Drug: Placebo	Phase 2; Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 60 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: A Multicenter, Randomized, Double-blind Clinical Trial Evaluating the Efficacy and Safety of Etanercept Versus Placebo in the Treatment of Patients With SAPHO Syndrome
• Estimated Study Start Date: December 13, 2023
• Estimated Primary Completion Date: October 18, 2028
• Estimated Study Completion Date: October 18, 2028

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. 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 [ Time Frame: 12 weeks (day 85) ]
   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.

Secondary Outcome Measures :

1. Change in patient-assessed disease activity [ Time Frame: after 4 and 8 weeks ]
   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.
2. Occurrence of remission [ Time Frame: after 4, 8 and 12 weeks ]
   Occurrence of remission - complete resolution of osteoarticular and skin complaints in the patient's assessment - after 4, 8 and 12 weeks from randomization day
3. Occurrence of partial remission [ Time Frame: after 4, 8 and 12 weeks ]
   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
4. Occurrence of the patient acceptable symptom state (PASS score) [ Time Frame: after 4, 8 and 12 weeks ]
   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.
5. Change in physician-assessed disease activity [ Time Frame: at 4, 8 and 12 week ]
   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.
6. Change in the C-reactive Protein from Randomization Day Score [ Time Frame: at Weeks 4, 8 and 12 ]
   Change in C-reactive Protein from Randomization Day Score at Weeks 4, 8 and 12. A decrease in C-reactive Protein means improvement.
7. Change in the Erythrocyte Sedimentation Rate from Randomization Day Score [ Time Frame: at Weeks 4, 8 and 12 ]
   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.
8. Change in quality of life on the Short Form-36 health survey [ Time Frame: at 4, 8 and 12 weeks ]
   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.
9. Change in the Work Productivity and Activity Impairments (WPAI) from the Randomization Day Score [ Time Frame: after 4, 8 and 12 weeks ]
   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.
10. decrease in Ankylosing Spondylitis Disease Activity Score (ASDAS- C-reactive protein) ≥1.1 [ Time Frame: after 4, 8 and 12 weeks ]
    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.
11. a decrease in the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) Score of at least 50 percent [ Time Frame: after 4, 8 and 12 weeks ]
    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.
12. status of remission- Ankylosing Spondylitis Disease Activity Score (ASDAS- C-reactive protein) below 1.3 [ Time Frame: after 4, 8 and 12 weeks ]
    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.
13. decrease in the Bath Ankylosing Spondylitis Functional Index (BASFI) by a minimum of 50 percent [ Time Frame: after 4, 8 and 12 weeks ]
    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.
14. decrease in the Dermatology Life Quality Index (DLQI) by at least 50 percent from the result on the day of randomization [ Time Frame: after 4, 8 and 12 weeks ]
    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.
15. 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 [ Time Frame: 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.
16. decrease in the Dermatology Life Quality Index (DLQI) by a minimum of 50 percent [ Time Frame: after 4, 8 and 12 weeks ]
    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.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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.

Contacts and Locations

Contacts

Contact: Jakub Wroński, PhD, MD; 22 6880632 ext +48; jakub.wronski@spartanska.pl

Locations

Poland

Centrum Wsparcia Badań Klinicznych; Recruiting

Warsaw, Mazowieckie, Poland, 02-637

Contact: Marta Kurek 603 315 033 ext +48 marta.kurek@spartanska.pl

Contact: Agnieszka Kurowska 691 326 114 ext +48 agnieszka.kurowska@spartanska.pl

Sponsors and Collaborators

National Institute of Geriatrics, Rheumatology and Rehabilitation, Poland

Medical Research Agency, Poland

Investigators

• Principal Investigator: Jakub Wroński, PhD, MD; National Institute of Geriatrics, Rheumatology and Rehabilitation

More Information

• Responsible Party: National Institute of Geriatrics, Rheumatology and Rehabilitation, Poland
• ClinicalTrials.gov Identifier: NCT06011889
• Other Study ID Numbers: NIGRIR_003SAPHO
• First Posted: August 25, 2023
• Last Update Posted: November 14, 2023
• Last Verified: August 2023

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Acquired Hyperostosis Syndrome; Syndrome; Disease; Pathologic Processes; Osteochondrodysplasias; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Etanercept; Anti-Inflammatory Agents, Non-Steroidal; Analgesics, Non-Narcotic; Analgesics; Sensory System Agents; Peripheral Nervous System Agents; Physiological Effects of Drugs; Anti-Inflammatory Agents; Antirheumatic Agents; Gastrointestinal Agents; Immunosuppressive Agents; Immunologic Factors