Overview
Sponsor-declared trial summary
SAPHO syndrome (synovitis, acne, pustulosis, hyperostosis, osteitis) is a rare disease in which arthritis and osteitis is accompanied by skin lesions such as pustular psoriasis or acne. SAPHO syndrome significantly reduces the quality of life of patients due to the chronic occurrence of pain, limiting the daily activity of patients. About half of patients present also with symptoms of severe axial spondyloarthritis. It can also lead to local bone destruction (usually sternoclavicular joints).
The aim of the proposed clinical trial is to evaluate the efficacy and safety of etanercept therapy compared to placebo in patients with SAPHO syndrome who continue to receive standard treatment with NSAIDs and / or conventional disease-modifying antirheumatic drugs.
Key facts
- Sponsor
- Narodowy Instytut Geriatrii Reumatologii I Rehabilitacji Im Prof. Dr Hab. Med. Eleonory Reicher
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Immune System Diseases [C20]
- Trial duration
- 22 Oct 2024 → ongoing
- Decision date (initial)
- 2024-10-31
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Agencja Badań Medycznych
External identifiers
- EU CT number
- 2024-516919-25-00
- EudraCT number
- 2022-003474-23
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy, Therapy
The aim of the proposed clinical trial is to evaluate the efficacy and safety of etanercept therapy compared to placebo in patients with SAPHO syndrome who continue to receive standard treatment with NSAIDs and / or conventional disease-modifying antirheumatic drugs.
Secondary objectives 1
- Increasing the access of patients with SAPHO syndrome to modern forms of treatment, so far unavailable to them, in the form of a biological drug - etanercept.
Conditions and MedDRA coding
SAPHO syndrome (synovitis, acne, pustulosis, hyperostosis, osteitis) is a rare disease in which arthritis and osteitis is accompanied by skin lesions such as pustular psoriasis or acne. SAPHO syndrome significantly reduces the quality of life of patients due to the chronic occurrence of pain, limiting the daily activity of patients. About half of patients present also with symptoms of severe axial spondyloarthritis. It can also lead to local bone destruction (usually sternoclavicular joints).
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.0 | PT | 10051316 | SAPHO syndrome | 100000004859 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Diagnosis of SAPHO syndrome according to modified Kahn criteria from 2003.
- Age over 18.
- Patient overall disease and pain assessment on VAS both ≥ 4 cm.
- Expressing informed consent to participate in the study.
Exclusion criteria 7
- According to the Summary of Product Characteristics (SmPC) for Enbrel.
- Pregnancy, breastfeeding, inability to use effective contraception during the examination.
- Change in the dose of NSAIDs treatment in the last 4 weeks.
- Dose modification of disease-modifying antirheumatic drugs (DMARDs) over the past 12 weeks.
- Use of biological drugs / synthetic targeted drugs in the last 12 weeks.
- Use of corticosteroids (orally or local injections) in the last 4 weeks.
- Any medical condition that the investigator judges to contraindicate etanercept treatment.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Improvement 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 VAS scale by min. 50% after 12 weeks and a decrease in pain assessed by the patient on the VAS scale by min. 50% after 12 weeks
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 6
Enbrel 25 mg powder and solvent for solution for injection
PRD6538803 · Product
- Active substance
- Etanercept
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 12 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AB01 — -
- Marketing authorisation
- EU/1/99/126/003
- MA holder
- PFIZER EUROPE MA EEIG
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Enbrel 50 mg solution for injection in pre-filled syringe
PRD6538817 · Product
- Active substance
- Etanercept
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBDERMAL USE
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 36 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AB01 — -
- Marketing authorisation
- EU/1/99/126/016
- MA holder
- PFIZER EUROPE MA EEIG
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Enbrel 50 mg solution for injection in pre-filled syringe
PRD6538802 · Product
- Active substance
- Etanercept
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 36 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AB01 — -
- Marketing authorisation
- EU/1/99/126/017
- MA holder
- PFIZER EUROPE MA EEIG
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Enbrel 25 mg powder and solvent for solution for injection
PRD6538758 · Product
- Active substance
- Etanercept
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 36 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AB01 — -
- Marketing authorisation
- EU/1/99/126/005
- MA holder
- PFIZER EUROPE MA EEIG
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Enbrel 50 mg solution for injection in pre-filled syringe
PRD6538810 · Product
- Active substance
- Etanercept
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 36 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AB01 — -
- Marketing authorisation
- EU/1/99/126/018
- MA holder
- PFIZER EUROPE MA EEIG
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Enbrel 25 mg powder and solvent for solution for injection
PRD6538813 · Product
- Active substance
- Etanercept
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 12 Week(s)
- Authorisation status
- Authorised
- ATC code
- L04AB01 — -
- Marketing authorisation
- EU/1/99/126/004
- MA holder
- PFIZER EUROPE MA EEIG
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Placebo 1
SUB12398MIG · Substance
- Active substance
- Water for Injections
- Pharmaceutical form
- SOLUTION FOR INJECTION OR INFUSION
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 2 ml millilitre(s)
- Max total dose
- 2 ml millilitre(s)
- Max treatment duration
- 12 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Narodowy Instytut Geriatrii Reumatologii I Rehabilitacji Im Prof. Dr Hab. Med. Eleonory Reicher
- Sponsor organisation
- Narodowy Instytut Geriatrii Reumatologii I Rehabilitacji Im Prof. Dr Hab. Med. Eleonory Reicher
- Address
- Ul. Spartanska 1
- City
- Warsaw
- Postcode
- 02-637
- Country
- Poland
Scientific contact point
- Organisation
- Narodowy Instytut Geriatrii Reumatologii I Rehabilitacji Im Prof. Dr Hab. Med. Eleonory Reicher
- Contact name
- Centrum Wsparcia Badań Klinicznych
Public contact point
- Organisation
- Narodowy Instytut Geriatrii Reumatologii I Rehabilitacji Im Prof. Dr Hab. Med. Eleonory Reicher
- Contact name
- Centrum Wsparcia Badań Klinicznych
Third parties 1
| Organisation | City, country | Duties |
|---|---|---|
| Jan Orłowski Orcon Pharma ORL-000018117
|
Warsaw, Poland | On site monitoring, Code 10, Code 11, Interactive response technologies (IRT), Code 5, Data management, E-data capture, Code 8 |
Locations
1 EU/EEA country · 3 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Poland | Ongoing, recruiting | 60 | 3 |
| Rest of world | — | 0 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Poland | 2024-10-22 | 2024-10-22 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 19 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_2_Trial schedule | 1.2 |
| Protocol (for publication) | D1_3_Protoko SAPHO v1-2_final_redacted | 1.2 |
| Protocol (for publication) | D1_Protocol SAPHO_for_publication | 1.1 |
| Protocol (for publication) | D1_Protocol SAPHO_not_for_publication | 1.1 |
| Recruitment arrangements (for publication) | K1_ Recruitment arrangements_SAPHO | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 1 |
| Recruitment arrangements (for publication) | K2_ Recruitment material_SAPHO | 1 |
| Recruitment arrangements (for publication) | K2_1_Study Information brochure | 1 |
| Recruitment arrangements (for publication) | K2_2_Study Information Poster PL | 1 |
| Recruitment arrangements (for publication) | K2_3_Study Information electronic brochure | 1 |
| Subject information and informed consent form (for publication) | L1_1_ICF_ z_Informacja_dla_ pacjenta_SAPHO_ver1-1_final | 1.1 |
| Subject information and informed consent form (for publication) | L1_2_Informacja dla ciezarnej Uczestniczki_SAPHO_ver1-1_final | 1.1 |
| Subject information and informed consent form (for publication) | L1_3_Informacja dla ciezarnej Partnerki_SAPHO_ver1-1_final | 1.1 |
| Subject information and informed consent form (for publication) | L1_4_Formularz Ankiety Uczestnika Badania_final | 2 |
| Subject information and informed consent form (for publication) | L1_5_Formularz Swiadomej Zgody na Biobankowanie_final | 2 |
| Subject information and informed consent form (for publication) | L2_Medication intake schedule | 1.1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_1_enbrel-epar-product-information_pl | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_1_enbrelproductinformation_pl | 2 |
| Synopsis of the protocol (for publication) | D1_1_Protocol synopsis_PL | 1.2 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-09-10 | Poland | Acceptable 2024-10-29
|
2024-10-31 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2026-03-04 | Poland | Acceptable 2026-04-13
|
2026-04-20 |