Study of Sirolimus in CTD-TP in China (SSCI)
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|ClinicalTrials.gov Identifier: NCT03688191|
Recruitment Status : Active, not recruiting
First Posted : September 28, 2018
Last Update Posted : April 17, 2019
|Condition or disease||Intervention/treatment||Phase|
|Connective Tissue Diseases Thrombocytopenia||Drug: Sirolimus||Phase 4|
Connective tissue disease related thrombocytopenia (CTD-TP) is one of the common visceral complications of connective tissue diseases (CTD) with a poor response to traditional therapy. To date, some successful cases were reported in sirolimus treating refractory CTD-TP as a newly therapeutic method. However, there is still a lack of prospective clinical trials. This study aims to evaluate the efficacy of sirolimus in the treatment of refractory CTD-TP. To investigate this issue, investigators have designed a single-arm, open-label clinical trial of refractory CTD-TP patients in Peking Union Medical College Hospital (PUMCH). In this study, the researchers will enroll 20 patients who have a poor response to at least 6 months' traditional treatment. The participants will receive oral sirolimus treatment for 12 months. The primary end point is the change of platelet count in CBC. Secondary end points include the change of CTD activity, the time to disease complete remission and all-cause serious adverse events (SAE).
Does sirolimus improve the prognosis in patients with connective tissue disease related thrombocytopenia (CTD-TP) in Chinese patients?
- To determine the efficacy of sirolimus for the treatment of CTD-TP in Chinese patients
- To evaluate the safety of sirolimus for the treatment of CTD-TP in Chinese patients
- To analyze the clinical characteristics of CTD-TP in the sirolimus effective patients
This is a prospective open-label trial in a period of 12 months. Thrombocytopenia is defined that the count of platelet is less than 100×109/L. CTDs include SLE, pSS, RA, scleroderma, RA, dermatomyositis and MCTD. The newly diagnosed CTD-TP patients who have received no steroid or IS treatment will be ruled out the trial. All the participants should fulfill one of the criteria of SLE, pSS, RA, scleroderma, RA, dermatomyositis and MCTD and suffer from refractory TP, or be diagnosed CTD-associated TP by two experimental rheumatologists. The current standard treatment for CTD-TP is glucocorticoid plus one kind of immunosuppressant. The patients who still have TP after more than 3 months' standard treatment will be included into the trial. After signing informed consent forms, participants will receive oral sirolimus at a starting dose of 2 mg per day for 3 days and a sequential dose of 1mg per day, with dose adjusted according to tolerance and to maintain a therapeutic range of 6-15 ng/mL. Baseline data will be collected at enrollment. All participants will finish their 5 follow-up assessments (1st mon, 2nd mon, 4th mon, 6th mon and 12th mon) in 12 months.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Sirolimus in Patients With Connective Tissue Disease Related Thrombocytopenia (CTD-TP): a Single-arm, Open-label Clinical Trial in China|
|Actual Study Start Date :||September 21, 2017|
|Actual Primary Completion Date :||December 31, 2018|
|Estimated Study Completion Date :||September 30, 2019|
Experimental: Study Group
participants who received sirolimus treatment
Patients will receive oral sirolimus at a starting dose of 2 mg per day for 3 days and a sequential dose of 1mg per day, with dose adjusted according to tolerance and to maintain a therapeutic range of 6-15 ng/mL.
- Number of participants with complete remission of platelet count [ Time Frame: 6 months ]Complete remission of platelet count means the platelet count over 100X10^9/L. every participants will take CBC as their screening times.
- Number of participants with partial remission of platelet count [ Time Frame: 6 months ]Partial remission of ITP means that the platelet count was less than 100X10^9/L, but it was more than two times higher than that before treatment.
- Bleeding Events with treatment-related adverse events [ Time Frame: 6 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03688191
|Peking Union Medical College Hospital|
|Beijing, Beijing, China, 100005|
|Study Chair:||Xiaofeng Peng, Professor||Peking Union Medical College Hospital|
|Principal Investigator:||Chanyuan WU, MD||Peking Union Medical College Hospital|
|Study Director:||Mengtao LI, Professor||Peking Union Medical College Hospital|