Clinical Trial Details
— Status: Not yet recruiting
Administrative data
NCT number |
NCT05746689 |
Other study ID # |
Sirolimus for IgG4-RD |
Secondary ID |
|
Status |
Not yet recruiting |
Phase |
N/A
|
First received |
|
Last updated |
|
Start date |
March 1, 2023 |
Est. completion date |
December 31, 2028 |
Study information
Verified date |
February 2023 |
Source |
Peking University International Hospital |
Contact |
Yuying WANG, Master |
Phone |
8615210976309 |
Email |
wangyuying028[@]126.com |
Is FDA regulated |
No |
Health authority |
|
Study type |
Interventional
|
Clinical Trial Summary
gG4-related disease (IgG4-RD) is a newly recognized systemic autoimmune disease that can
involve the pan-creatobiliary tract, retroperitoneum/aorta, head and neck region, and
salivary glands, et al. Glucocorticoids are the first-line agents for the treatment of
IgG4-RD, however, in order to maintain long-term disease stability and avoid disease relapse,
glucocorticoids maintenance therapy should last for a long period, which may induce various
glucocorticoid-associated adverse reactions. Sirolimus plays dual roles in inhibiting
lymphocyte activation and fibroblast proliferation. It is inferred from its mechanism that
sirolimus is a good potential treatment option for IgG4-RD. Therefore, we conducted this
single-arm clinical trial on patients with IgG4-RD to determine the efficacy and safety of
sirolimus.
Description:
IgG4-related disease (IgG4-RD) is a newly recognized systemic autoimmune disease that can
involve the pan- creatobiliary tract, retroperitoneum/aorta, head and neck region, and
salivary glands, et al. IgG4-RD is characterized by elevated serum IgG4 levels, tumefactive
lesions with a dense lymphoplasmacytic infiltration rich in IgG4 positive plasma cells and
storiform fibrosis of related organs.
Glucocorticoids are the first-line agents for the treatment of IgG4-RD, however, in order to
maintain long-term disease stability and avoid disease relapse, glucocorticoids maintenance
therapy should last for a long period, which may induce various glucocorticoid-associated
adverse reactions. For some mild IgG4-RD patients without internal organ damage, long-term
glucocorticoids therapy may have a low benefit/risk ratio. Further, a substantial proportion
of patients cannot tolerate glucocorticoids.
Sirolimus, also known as rapamycin, is a macrolide compound that inhibits its mechanistic
target (mTOR), which regulates cell growth and metabolism in response to environmental cues.
mTOR is also essential in driving abnormal lineage specification within the immune system in
various rheumatic diseases. We discovered that mTOR was highly activated in IgG4RD tissues,
and its inhibitor sirolimus appeared as a good treatment candidate.