SarcoidosisJAK-SARCNCT04751279
Expression and Role of the JAK/STAT Pathway in Sarcoidosis Granuloma Cells
An observational study of Sarcoidosis and JAK-STAT Pathway Deregulation, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Recent studies suggest that the JAK/STAT signaling pathway constitutes a new step in the clinical and therapeutic progress of sarcoidosis. Further investigations are necessary to identify the most suitable patients to receive treatment targeting this pathway, in particular in cases of severe sarcoidosis refractory to… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Sarcoidosis, JAK-STAT Pathway Deregulation
- What is being tested?
- Blood sample
- How many people
- 80 participants
- Where
- 1 siteFrance
- Study dates
- 9 Mar 2021to 2 Jan 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with mediastino-pulmonary sarcoidosis receiving or not receiving treatment for their sarcoidosis. Subjects treated for their sarcoidosis (corticosteroids and/or immunosuppressants) are not excluded because of two recent cases reported of multi-treated patients in which an involvement of the JAK/STAT pathway was demonstrated.
Who may be able to join
- Patients with a diagnosis of mediastinopulmonary sarcoidosis made according to ATS / ERS / WASOG criteria
- Or patient suspected of having mediastino-pulmonary sarcoidosis, without any other probable causal factor identified on the usual standard examination at the time of the sample with the need for diagnostic confirmation at the end of the study according to the criteria of the ATS / ERS / WASOG
- Sarcoidosis with stage 1 to 4 pulmonary involvement
- Patients who had a chest CT scan in the 6 months preceding the sample. Examination carried out as part of routine care
Who cannot join
- Pregnancy.
- Opposition expressed to participation in the study.
- Patients on State Medical Aid.
Trial locations and contacts
1 site in 1 country.
Valerie BESNARD, PhD contact
France1 site, 1 recruiting
- Recruiting
Service de pneumologie Hôpital Avicenne
Bobigny, 93000
Florence JENY, DR, 01 48 95 74 35, florence.jeny@aphp.fr
Zahia BEN ABDESSELAM, Dr, 01 48 95 74 35, zahia.ben-abdesselam@aphp.fr
About this study
Recent studies suggest that the JAK/STAT signaling pathway constitutes a new step in the clinical and therapeutic progress of sarcoidosis. Further investigations are necessary to identify the most suitable patients to receive treatment targeting this pathway, in particular in cases of severe sarcoidosis refractory to the various therapeutic lines.
Read the detailed description
The investigators hypothesize that the JAK/STAT signaling pathway constitutes a new step in the clinical and therapeutic progress of sarcoidosis, in particular in cases of severe sarcoidosis refractory to the various therapeutic lines. The main objective of this proposal is to evaluate the presence and stage of activation of the JAK/STAT pathway in PBMCs from inactive and active sarcoidosis patients. The second objective will be to determine the role of the JAK/STAT pathway in the formation and maintenance of granulomas and their association with the severity of sarcoidosis and fibrogenesis.
- Conditions
- Sarcoidosis, JAK-STAT Pathway Deregulation
- Being tested
- Blood sample
- Keywords
- Sarcoidosis, JAK/STAT
- Medical subject headings
- Sarcoidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood sampleOther
Blood sample collection of 5 blood sample at the inclusion in the study the samle consite of 5 tubesEthylene Diamine Tetra Acetate (ADTA) of 7 ml
| Study group | Role | What they receive |
|---|---|---|
| Patients with pulmonary sarcoidosis without signs of chest activity and recent diagnosis (<5 years) | Patients with pulmonary sarcoidosis without signs of chest activity (Benamore score <2) and recent diagnosis (<5 years) Receives: Other: Blood sample | |
| Patients with pulmonary sarcoidosis with signs of chest activity and recent diagnosis (<5 years | Patients with pulmonary sarcoidosis with signs of chest activity (Benamore score ≥2) and recent diagnosis (<5 years) Receives: Other: Blood sample | |
| Patients with pulmonary sarcoidosis without signs of activity , persistent form (>5 years) | Patients with pulmonary sarcoidosis without signs of activity (Benamore score <2), persistent form (>5 years) Receives: Other: Blood sample | |
| Patients with pulmonary sarcoidosis with signs of chest activity , persistent form (>5 years) | Patients with pulmonary sarcoidosis with signs of chest activity (Benamore score ≥2), persistent form (>5 years) Receives: Other: Blood sample |
What is being measured
The primary outcome is the main question the study is designed to answer.
JAK/STAT familly numbers protein expression in PBMCs
Evaluation will be carried out by:
- studying the expression of members of the JAK/STAT pathway, the expression of cytokines and chemokines phagocytosis and macrophage differentiation the expression of cytokines and chemokines
Measured: 1 year after inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2021
- Study start9 Mar 2021
- Primary completion1 Jan 2024 (estimated)
- Study completion2 Jan 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for SarcoidosisStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Florence JENY, MD, Principal investigator, Assistance Publique - Hôpitaux Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04751279
- Sponsor's protocol ID
- APHP201205
- Other IDs
- 2020-A02330-39
- Acronym
- JAK-SARC
- Status last verified
- Nov 2023
- First submitted
- 29 Jan 2021
- Last updated
- 18 Nov 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Rotenberg C, Besnard V, Brillet PY, Giraudier S, Nunes H, Valeyre D. Dramatic response of refractory sarcoidosis under ruxolitinib in a patient with associated JAK2-mutated polycythemia. Eur Respir J. 2018 Dec 20;52(6):1801482. doi: 10.1183/13993003.01482-2018. Print 2018 Dec. No abstract available.
PubMed 30361243
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expression and Role of the JAK/STAT Pathway in Sarcoidosis Granuloma Cells
- ClinicalTrials.gov ID
- NCT04751279
- Acronym / sponsor ID
- JAK-SARC / APHP201205
- Conditions
- Sarcoidosis, JAK-STAT Pathway Deregulation
- Keywords
- Sarcoidosis, JAK/STAT
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 9 Mar 2021 (actual)
- Primary completion
- 1 Jan 2024 (estimated)
- Study completion
- 2 Jan 2024 (estimated)
- First posted
- 12 Feb 2021 (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.
Recent studies suggest that the JAK/STAT signaling pathway constitutes a new step in the clinical and therapeutic progress of sarcoidosis. Further investigations are necessary to identify the most suitable patients to receive treatment targeting this pathway, in particular in cases of severe sarcoidosis refractory to the various therapeutic lines.
Detailed description
The investigators hypothesize that the JAK/STAT signaling pathway constitutes a new step in the clinical and therapeutic progress of sarcoidosis, in particular in cases of severe sarcoidosis refractory to the various therapeutic lines. The main objective of this proposal is to evaluate the presence and stage of activation of the JAK/STAT pathway in PBMCs from inactive and active sarcoidosis patients. The second objective will be to determine the role of the JAK/STAT pathway in the formation and maintenance of granulomas and their association with the severity of sarcoidosis and fibrogenesis.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Patients with pulmonary sarcoidosis without signs of chest activity and recent diagnosis (<5 years) | – | Other: Blood samplePatients with pulmonary sarcoidosis without signs of chest activity (Benamore score <2) and recent diagnosis (<5 years) |
| Patients with pulmonary sarcoidosis with signs of chest activity and recent diagnosis (<5 years | – | Other: Blood samplePatients with pulmonary sarcoidosis with signs of chest activity (Benamore score ≥2) and recent diagnosis (<5 years) |
| Patients with pulmonary sarcoidosis without signs of activity , persistent form (>5 years) | – | Other: Blood samplePatients with pulmonary sarcoidosis without signs of activity (Benamore score <2), persistent form (>5 years) |
| Patients with pulmonary sarcoidosis with signs of chest activity , persistent form (>5 years) | – | Other: Blood samplePatients with pulmonary sarcoidosis with signs of chest activity (Benamore score ≥2), persistent form (>5 years) |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with mediastino-pulmonary sarcoidosis receiving or not receiving treatment for their sarcoidosis. Subjects treated for their sarcoidosis (corticosteroids and/or immunosuppressants) are not excluded because of two recent cases reported of multi-treated patients in which an involvement of the JAK/STAT pathway was demonstrated.
Key inclusion criteria (4 listed)
- Patients with a diagnosis of mediastinopulmonary sarcoidosis made according to ATS / ERS / WASOG criteria
- Or patient suspected of having mediastino-pulmonary sarcoidosis, without any other probable causal factor identified on the usual standard examination at the time of the sample with the need for diagnostic confirmation at the end of the study according to the criteria of the ATS / ERS / WASOG
- Sarcoidosis with stage 1 to 4 pulmonary involvement
- Patients who had a chest CT scan in the 6 months preceding the sample. Examination carried out as part of routine care
Key exclusion criteria (3 listed)
- Pregnancy.
- Opposition expressed to participation in the study.
- Patients on State Medical Aid.
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 |
|---|---|---|
| Blood sampleBlood sample collection of 5 blood sample at the inclusion in the study the samle consite of 5 tubesEthylene Diamine Tetra Acetate (ADTA) of 7 ml | Other | Patients with pulmonary sarcoidosis with signs of chest activity , persistent form (>5 years), Patients with pulmonary sarcoidosis with signs of chest activity and recent diagnosis (<5 years, Patients with pulmonary sarcoidosis without signs of activity , persistent form (>5 years), Patients with pulmonary sarcoidosis without signs of chest activity and recent diagnosis (<5 years) |
Primary outcome
JAK/STAT familly numbers protein expression in PBMCs
Time frame: 1 year after inclusion
Evaluation will be carried out by: - studying the expression of members of the JAK/STAT pathway, the expression of cytokines and chemokines phagocytosis and macrophage differentiation the expression of cytokines and chemokines
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2023.
- Background: PubMed 30361243Rotenberg C, Besnard V, Brillet PY, Giraudier S, Nunes H, Valeyre D. Dramatic response of refractory sarcoidosis under ruxolitinib in a patient with associated JAK2-mutated polycythemia. Eur Respir J. 2018 Dec 20;52(6):1801482. doi: 10.1183/13993003.01482-2018. Print 2018 Dec. No abstract available.
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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.
Valerie BESNARD, PhD contact
Florence JENY, MD principal investigator
Assistance Publique - Hôpitaux Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sarcoidosis and JAK-STAT Pathway Deregulation, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 4 inclusion and 3 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: status not confirmed, as of the sponsor's last update on 18 Nov 2023. 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 France. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Florence JENY, MD (0148955129, florence.jeny@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
JAK/STAT familly numbers protein expression in PBMCs (time frame: 1 year after inclusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04751279, which also lists 1 linked publication.
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 Nov 2023.