Adult-Onset Still's DiseaseNCT07151157
Therapeutic Value of Sirolimus in Refractory Adult-Onset Still's Disease
A study testing add-on sirolimus therapy in people with Adult-Onset Still's Disease, run by Ruijin Hospital at 1 site in China.
Trial summary
This study aims to further evaluate the anti-inflammatory effects of the mTOR inhibitor sirolimus in patients with adult-onset Still's disease (AOSD), and to verify whether it can reduce inflammation and control disease activity by restoring ZFP36L2 expression and promoting the degradation of inflammatory cytokine… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Adult-Onset Still's Disease
- What is being tested?
- add-on sirolimus therapy
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteChina
- Study dates
- 30 Aug 2025to 30 Aug 2026
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age over 18 years;
- Meeting the Yamaguchi criteria, with infection and malignancy excluded;
- Meeting the definition of refractory AOSD, i.e., patients whose symptoms show no significant improvement after treatment with glucocorticoids at 1 mg/kg, or who experience relapse during tapering of glucocorticoids to 15 mg/d;
- Complete clinical information;
- Signed and dated informed consent form;
- Willingness to comply with study procedures and cooperate throughout the entire study.
Who cannot join
- Presence of other rheumatic diseases;
- History of, or planned, hematopoietic stem cell transplantation;
- Presence of severe comorbidities considered by the investigator to be unsuitable for participation in this clinical study, such as severe cardiac or pulmonary insufficiency, severe bone marrow suppression, or severe hepatic or renal dysfunction;
- Acute conditions such as bowel perforation or complete intestinal obstruction;
- Pregnant, planning pregnancy, or breastfeeding women;
- Poor compliance.
Trial locations and contacts
1 site in 1 country.
China1 site
Ruiiin Hospital Shanghai JiaoTong University School of Medicine
Shanghai, Shanghai Municipality, 200025
About this study
This study aims to further evaluate the anti-inflammatory effects of the mTOR inhibitor sirolimus in patients with adult-onset Still's disease (AOSD), and to verify whether it can reduce inflammation and control disease activity by restoring ZFP36L2 expression and promoting the degradation of inflammatory cytokine mRNAs. A total of three patients with refractory AOSD will be enrolled, defined as those showing no significant improvement after treatment with glucocorticoids at 1 mg/kg, or experiencing relapse during tapering of glucocorticoids to 15 mg/day. On the basis of their existing therapy, patients will receive oral sirolimus at a dose of two capsules per day, one in the morning and one in the evening. On the day prior to initiating sirolimus and at weeks 4, 8, and 12 of treatment, 10 ml of peripheral blood will be collected by trained medical staff, and neutrophils will be isolated within 2 hours to assess ZFP36L2 expression levels. Throughout treatment, patients' clinical manifestations, including fever, rash, and joint pain, will be monitored, and dynamic changes in multiple inflammation-related markers will be measured in peripheral blood at weeks 4, 8, and 12. These include C-reactive protein (CRP), erythrocyte sedimentation rate (ESR), serum ferritin, soluble interleukin-2 receptor (sIL-2R), and a panel of cytokines. Finally, correlations among sirolimus treatment, ZFP36L2 expression, inflammatory responses, and disease activity will be analyzed to explore its potential therapeutic mechanisms in AOSD.
- Conditions
- Adult-Onset Still's Disease
- Being tested
- add-on sirolimus therapy
- Keywords
- Adult-Onset Still's disease, sirolimus, mTOR, ZFP36L2
- Medical subject headings
- Still's Disease, Adult-Onset
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
add-on sirolimus therapyDrug
On the basis of the existing treatment, oral sirolimus will be added at a dose of two capsules per day, one in the morning and one in the evening.
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus combination therapy group | Experimental | On the basis of the existing treatment, oral sirolimus will be added at a dose of two capsules per day, one in the morning and one in the evening. Receives: Drug: add-on sirolimus therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
AOSD Disease Activity Score
Pouchot Systemic Score Scoring items (1 point each): fever; arthritis/arthralgia; rash; sore throat; hepatosplenomegaly; pericarditis/pleuritis; lymphadenopathy; abnormal liver function; leukocytosis; hyperamylasemia/thrombocytopenia; elevated ESR or CRP; other systemic manifestations (e.g., myalgia, fatigue). Total score: 0-12 points, with higher scores indicating greater disease activity.
Measured: at weeks 12
Secondary outcomes (8)
AOSD Disease Activity Score
Pouchot Systemic Score Scoring items (1 point each): fever; arthritis/arthralgia; rash; sore throat; hepatosplenomegaly; pericarditis/pleuritis; lymphadenopathy; abnormal liver function; leukocytosis; hyperamylasemia/thrombocytopenia; elevated ESR or CRP; other systemic manifestations (e.g., myalgia, fatigue). Total score: 0-12 points, with higher scores indicating greater disease activity.
Measured: Baseline (day before sirolimus) and at weeks 4, and 8
Serum C-reactive Protein (CRP) Concentration
CRP will be measured from peripheral blood samples as a marker of systemic inflammation.
Measured: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Erythrocyte Sedimentation Rate (ESR)
ESR will be assessed from peripheral blood samples to evaluate inflammatory activity.
Measured: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Concentration of Serum Ferritin
Ferritin will be measured from peripheral blood samples as an indicator of disease activity and inflammation.
Measured: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Level of Soluble Interleukin-2 Receptor (sIL-2R)
sIL-2R will be measured from peripheral blood samples as a marker of immune activation.
Measured: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Concentration of Inflammatory Cytokines (e.g., IL-6, TNF-α)
Multiple inflammatory cytokines will be measured from peripheral blood samples to evaluate systemic inflammatory status.
Measured: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Number of participants with Clinical symptoms such as rash and arthralgia
Changes in clinical symptoms such as rash and joint pain will be observed and recorded through physical examination.
Measured: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Expression level of ZFP36L2 in neutrophils
Collect 10 mL of peripheral blood from patients and isolate neutrophils within 2 hours to measure the expression level of ZFP36L2.
Measured: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Sep 2025
- Study start30 Aug 2025
- Primary completion1 Apr 2026
- Study completion30 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Adult-Onset Still's DiseaseStudies of the same condition, open and under way.
- More trials run by Ruijin HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ruijin Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07151157
- Sponsor's protocol ID
- 2025-495
- Status last verified
- Aug 2025
- First submitted
- 24 Aug 2025
- Last updated
- 18 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Therapeutic Value of Sirolimus in Refractory Adult-Onset Still's Disease
- ClinicalTrials.gov ID
- NCT07151157
- Acronym / sponsor ID
- 2025-495
- Conditions
- Adult-Onset Still's Disease
- Keywords
- Adult-Onset Still's disease, sirolimus, mTOR, ZFP36L2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- Ruijin HospitalUniversity, hospital or other
- Study start
- 30 Aug 2025 (actual)
- Primary completion
- 1 Apr 2026 (actual)
- Study completion
- 30 Aug 2026 (estimated)
- First posted
- 3 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study aims to further evaluate the anti-inflammatory effects of the mTOR inhibitor sirolimus in patients with adult-onset Still's disease (AOSD), and to verify whether it can reduce inflammation and control disease activity by restoring ZFP36L2 expression and promoting the degradation of inflammatory cytokine mRNAs. A total of three patients with refractory AOSD will be enrolled, defined as those showing no significant improvement after treatment with glucocorticoids at 1 mg/kg, or experiencing relapse during tapering of glucocorticoids to 15 mg/day. On the basis of their existing therapy, patients will receive oral sirolimus at a dose of two capsules per day, one in the morning and one in the evening. On the day prior to initiating sirolimus and at weeks 4, 8, and 12 of treatment, 10 ml of peripheral blood will be collected by trained medical staff, and neutrophils will be isolated within 2 hours to assess ZFP36L2 expression levels. Throughout treatment, patients' clinical manifestations, including fever, rash, and joint pain, will be monitored, and dynamic changes in multiple inflammation-related markers will be measured in peripheral blood at weeks 4, 8, and 12. These include C-reactive protein (CRP), erythrocyte sedimentation rate (ESR), serum ferritin, soluble interleukin-2 receptor (sIL-2R), and a panel of cytokines. Finally, correlations among sirolimus treatment, ZFP36L2 expression, inflammatory responses, and disease activity will be analyzed to explore its potential therapeutic mechanisms in AOSD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sirolimus combination therapy group | Experimental | Drug: add-on sirolimus therapyOn the basis of the existing treatment, oral sirolimus will be added at a dose of two capsules per day, one in the morning and one in the evening. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age over 18 years;
- Meeting the Yamaguchi criteria, with infection and malignancy excluded;
- Meeting the definition of refractory AOSD, i.e., patients whose symptoms show no significant improvement after treatment with glucocorticoids at 1 mg/kg, or who experience relapse during tapering of glucocorticoids to 15 mg/d;
- Complete clinical information;
- Signed and dated informed consent form;
Key exclusion criteria (6 listed)
- Presence of other rheumatic diseases;
- History of, or planned, hematopoietic stem cell transplantation;
- Presence of severe comorbidities considered by the investigator to be unsuitable for participation in this clinical study, such as severe cardiac or pulmonary insufficiency, severe bone marrow suppression, or severe hepatic or renal dysfunction;
- Acute conditions such as bowel perforation or complete intestinal obstruction;
- Pregnant, planning pregnancy, or breastfeeding women;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| add-on sirolimus therapyOn the basis of the existing treatment, oral sirolimus will be added at a dose of two capsules per day, one in the morning and one in the evening. | Drug | Sirolimus combination therapy group |
Primary outcome
AOSD Disease Activity Score
Time frame: at weeks 12
Pouchot Systemic Score Scoring items (1 point each): fever; arthritis/arthralgia; rash; sore throat; hepatosplenomegaly; pericarditis/pleuritis; lymphadenopathy; abnormal liver function; leukocytosis; hyperamylasemia/thrombocytopenia; elevated ESR or CRP; other systemic manifestations (e.g., myalgia, fatigue). Total score: 0-12 points, with higher scores indicating greater disease activity.
8 secondary outcomes
AOSD Disease Activity Score
Time frame: Baseline (day before sirolimus) and at weeks 4, and 8
Serum C-reactive Protein (CRP) Concentration
Time frame: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Erythrocyte Sedimentation Rate (ESR)
Time frame: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Concentration of Serum Ferritin
Time frame: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Level of Soluble Interleukin-2 Receptor (sIL-2R)
Time frame: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Concentration of Inflammatory Cytokines (e.g., IL-6, TNF-α)
Time frame: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Number of participants with Clinical symptoms such as rash and arthralgia
Time frame: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Expression level of ZFP36L2 in neutrophils
Time frame: Baseline (day before sirolimus) and at weeks 4, 8, and 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing add-on sirolimus therapy in people with Adult-Onset Still's Disease, run by Ruijin Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 18 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
AOSD Disease Activity Score (time frame: at weeks 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Aug 2026.