The Foundation tracks upcoming drugs/treatments of relevance to patients with SJIA, AOSD, and MAS. We track both current clinical trials in SJIA, AOSD / MAS, and also clinical trials in other related diseases, such as HLH or other autoinflammatory diseases.
(Data for below section was gathered by searching for trials on ClinicalTrials.gov and European Trial Registry. Data is current as of January 29th, 2023. Please get in touch if you see any mistakes or trials we are missing.)
Below is a summary of the main types of drugs covered in the tables.
- IL1 & IL6 inhibitors: There are current trials for canakinumab in Germany & Japan. Additionally, there are trials for sarilumab, an IL6 biosimilar in Europe and South America.
- IL18 inhibitors: There are three IL18 inhibitors coming up through the pipeline.
- Tadekinig Alfa from AB2 Bio continues to be in trial for NLRC4 & XIAP deficiency. We are also aware of an SJIA patient who earlier received the drug through compassionate use.
- AVTX-007 (camoteskimab) is now with Apollo Therapeutics (was earlier with Avalo Therapeutics which launched the clinical trial in AOSD).
- MAS-825 from Novartis is a bi-specific antibody against IL1 & IL18. Several patients with SJIA, MAS & Interstitial Lung Disease have been using the drug via compassionate use. The current trials for MAS-825 are in NLRC4 & XIAP but we hope there will be trials in SJIA & AOSD soon.
- Interferon gamma inhibitor: Emapalumab is an interferon-gamma (IFNy) blocking antibody from Sobi that is currently approved for HLH in USA, and is in clinical trials for MAS in USA and Europe. It has also been used by several patients with SJIA, MAS & Lung Disease in our patient group.
- Jak-inhibitors: Several jak-inhibitors are in trials for SJIA including tofacitinib, upadatacitinib. Additionally, ruxolitinib and itadatacitinib are in trials for HLH.
- NLRP3 inflammasome inhibitors: A new class of drugs, NLRP3 Inflammasome inhibitors, which block both IL1 & IL18 pathways are in trials for autoinflammatory diseases such as CAPS, FCAS etc.
The first table list current trials SJIA & AOSD (does not include trials that have concluded or terminated).
Table 1. Current Clinical Trials AOSD / SJIA
| Drug | Type | Indication | Patient Group targeted | Trial Number | Status / Phase | Sponsors | Last Update | Locations |
|---|---|---|---|---|---|---|---|---|
| AVTX-007 Camoteskimab | IL18 Inhibitor | AOSD | AOSD - ILAR criteria | NCT04752371 | Recruiting / Phase 1 | Apollo Therapeutics | 8/31/2022 | United States, Belgium, Poland, Ukraine |
| Canakinumab | IL1 Inhibitor | SJIA | SIJA - ILAR criteria (arthritis required) | 2018-004284-30 | Recruiting | Asklepios Klink Sankt Augustin | Germany | |
| Canakinumab | IL1 Inhibitor | AOSD | SIJA - ILAR criteria (arthritis required) | NCT04717635 | Recruiting / Phase 3 | Novartis Pharmaceuticals | 7/15/2022 | Japan |
| Sarilumab | IL6 Inhibitor (Biosimilar) | SJIA | SIJA - ILAR criteria (arthritis required) | NCT02991469 | Recruiting / Phase 2 | Sanofi | 1/10/2023 | Argentina, Bulgaria, Canada, Czechia... For full countries list, see link. |
| Baracitinib | Jak-Inhibitor | SJIA | SIJA - ILAR criteria (arthritis required) | 2017-004495-60 | Recruiting | Eli Lilly & Company | Austria, Belgium, Czechia, Denmark... For full countries list, see link. | |
| Baracitinib | Jak-Inhibitor | SJIA | SIJA - ILAR criteria (arthritis required) | NCT04088396 | Recruiting / Phase 3 | Eli Lilly & Company | 12/28/2022 | India, Israel, Japan, Austria... For full countries list, see link. |
| Tofacitinib | Jak-Inhibitor | SJIA | SIJA - ILAR criteria (arthritis required) | NCT03000439 | Recruiting / Phase 3 | Pfizer | 1/5/2023 | United States, Argentina, Belgium, Brazil... For full countries list, see link. |
| Tofacitinib | Jak-Inhibitor | SJIA | SIJA - ILAR criteria (arthritis required) | 2017-002018-29 | Recruiting | Pfizer | Belgium, Germany, Hungary, Italy, Netherlands, Poland, Slovakia | |
| Upadacitinib | Jak-Inhibitor | JIA / SJIA | SJIA with active arthritis | NCT03725007 | Recruiting / Phase 1 | AbbVie | 1/26/2023 | United States, Canada, Germany, Hungary... For full countries list, see link. |
| Upadacitinib & Tocilizumab | Jak-Inhibitor +IL6 Inhibitor | SJIA | SIJA - ILAR criteria (arthritis required) | NCT05609630 | Not yet recruiting / Phase 3 | AbbVie | 11/8/2022 | |
| Emapalumab | IFNY Inhibitor | AOSD / SJIA / MAS | SJIA / AOSD Diagnosis / MAS criteria described in trial | 2021-001577-24 | Recruiting | Sobi AG | Belgium, Czechia, France, Germany... For full countries list, see link. | |
| Emapalumab | IFNY Inhibitor | AOSD / SJIA / MAS | SJIA / AOSD Diagnosis / MAS criteria described in trial | NCT05001737 | Recruiting / Phase 3 | Sobi AG | 1/4/2023 | Canada, United States, China, Japan... For full countries list, see link. |
The second table summarizes current trials in MAS, Primary & Secondary HLH. Historically drugs first tested in HLH have been later used in MAS. As such, the foundation follows trials in Primary & Secondary HLH as well.
Table 2. Current Clinical Trials in MAS / Primary & Secondary HLH
| Drug | Type | Indication | Patient Group targeted | Trial Number | Status / Phase | Sponsors | Last Update | Locations |
|---|---|---|---|---|---|---|---|---|
| MAS-825 | IL1 + IL18 Inhibitor | NLRC4-GOF | NLRC4 mutation | NCT04641442 | Recruiting / Phase 2 | Novartis Pharmaceuticals | 11/1/2022 | Canada, United States, Japan, Turkey, ... For full countries' list, see link. |
| Tadekinig alfa (IL-18BP) | IL18 Inhibitor | NLRC4 / XIAP deficiency | Genetic diagnosis of NLRC4 mutation or XIAP deficiency | NCT03113760 | Not recruiting / Phase 3 | AB2 Bio | 7/27/2022 | United States, Canada, Germany |
| Emapalumab | IFNY inhibitor | AOSD / SJIA / MAS | SJIA / AOSD Diagnosis / MAS criteria described in trial | 2021-001577-24 | Recruiting | Sobi AG | Belgium, Czechia, France, Germany, ... For full countries' list, see link. | |
| Emapalumab | IFNY inhibitor | AOSD/ SJIA / MAS / SLE | SJIA / AOSD Diagnosis / MAS criteria described in trial | NCT05001737 | Recruiting / Phase 3 | Sobi AG | 1/4/2023 | Canada, United States, China, Japan, ... For full countries' list, see link. |
| Itacitinib | Jak-Inhibitor | HLH | Not clear - rashmi | NCT05063110 | Recruiting / Phase 2 | Hôpitaux de Paris / Incyte Corporation | 8/23/2022 | France, Bobigny |
| Ruxolitinib, Dexamethasone, Etoposide | Jak-Inhibitor / Corticosteroid / Chemotherapy Drug | HLH | Active HLH (diagnosis criteria described in trial) - rashmi | NCT04551131 | Recruiting / Phase 1 (ruxo), Phase 2 (dexa) | St. Jude Children's Research Hospital | 12/6/2022 | United States |
| ELA026 | Anti-SIRP monoclonal antibody | HLH | HLH (criteria HLH-2004) | NCT05416307 | Recruiting / Phase 1 | Electra Therapeutics | 10/25/2022 | Austria, Italy, Netherlands, Spain, United Kingdom |
| Gemtuzumab Ozogamicin | Monoclonal anti-CD33 antibody | HLH / MAS | HLH or MAS (primary, secondary or refractory) | 2020-002428-36 | Recruiting / Phase 2 | University of Birmingham | United Kingdom |
The third table summarizes trials for Inflammasome inhibitors currently being tested in different Autoinflammatory Diseases such as CAPS, FCAS.
Table 3. Current Clinical Trials for Inflammasome Inhibitors in Autoinflammatory Disease
| Drug | Type | Indication | Trial Number | Status / Phase | Sponsors | Last Update | Locations |
|---|---|---|---|---|---|---|---|
| ZYIL1 | Inflammasome Inhibitor | CAPS | NCT05186051 | Completed / Phase 2 | Zydus Lifesciences Limited | 07/11/2022 | Australia |
| ATI-450 | Inflammasome Inhibitor | CAPS | NCT04524858 | Terminated / Phase 2 | Aclaris Therapeutics | 04/01/2021 | United States |
| ATI-450 | Inflammasome Inhibitor | Sepsis | NCT04635878 | Recruiting | University Hospital, Toulouse | 06/09/2022 | France |
| ATI-450 | Inflammasome Inhibitor | CAPS | NCT05292768 | Not yet recruiting | Hôpitaux de Paris,Institut Imagine | 03/23/2022 | France |
| DFV890 | Inflammasome Inhibitor | FCAS | NCT04868968 | Recruiting / Phase 2 | Novartis Pharmaceuticals | 12/15/2022 | United States, France, Germany, Italy |
| Dapansutrile | Inflammasome Inhibitor | COVID 19 | NCT04540120 | Recruiting / Phase 2 | Olatec Therapeutics | 09/19/2022 | United States, Netherland, Switzerland |
| Depansutrile | Inflammasome Inhibitor | Schnitzler Syndrome | NCT03595371 | Recruiting / Phase 2 | Olatec Therapeutics LLC / Radboud University Medical Center | 07/06/2021 | Netherlands |
| Isunakinra & PD-(L)1 Inhibitor | Insunakinra: IL1 Inhibitor / PD-(L)1: targeting PD-1 or PD-L1 | Solid Tumor, Adult | NCT04121442 | Recruiting / Isunakinra: Phase 1 & PD-(L)1 Inhibitor: Phase 2 | Buzzard Pharmaceuticals / Baylor Research Institute | 07/21/2021 | United States, Texas |
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