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Study of Sirolimus in CTD-TP in China (SSCI)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03688191
Recruitment Status : Active, not recruiting
First Posted : September 28, 2018
Last Update Posted : April 17, 2019
Sponsor:
Information provided by (Responsible Party):
Peking Union Medical College Hospital

Brief Summary:
We have designed a single-arm, open-label study of sirolimus in Chinese patients with refractory CTD-TP unresponsive to, or intolerant of, conventional medications at Peking Union Medical College Hospital (PUMCH). Patients received 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. Patients were treated with sirolimus for more than 6 months. Safety outcomes and the efficacy endpoints were assessed in all patients who received at least one dose of treatment.

Condition or disease Intervention/treatment Phase
Connective Tissue Diseases Thrombocytopenia Drug: Sirolimus Phase 4

Detailed Description:

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).

Research Question:

Does sirolimus improve the prognosis in patients with connective tissue disease related thrombocytopenia (CTD-TP) in Chinese patients?

Specific aims:

  1. To determine the efficacy of sirolimus for the treatment of CTD-TP in Chinese patients
  2. To evaluate the safety of sirolimus for the treatment of CTD-TP in Chinese patients
  3. To analyze the clinical characteristics of CTD-TP in the sirolimus effective patients

Methods:

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

Drug Information available for: Sirolimus

Arm Intervention/treatment
Experimental: Study Group
participants who received sirolimus treatment
Drug: Sirolimus
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.




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.


Other Outcome Measures:
  1. Bleeding Events with treatment-related adverse events [ Time Frame: 6 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 80 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients with refractory CTD-TP unresponsive to, or intolerant of, conventional medications at Peking Union Medical College Hospital.

Exclusion Criteria:

  • new diagnosed CTD-IT patients

Information from the National Library of Medicine

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


Locations
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China, Beijing
Peking Union Medical College Hospital
Beijing, Beijing, China, 100005
Sponsors and Collaborators
Peking Union Medical College Hospital
Investigators
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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

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Responsible Party: Peking Union Medical College Hospital
ClinicalTrials.gov Identifier: NCT03688191    
Other Study ID Numbers: SirolimusforCTD-IP
First Posted: September 28, 2018    Key Record Dates
Last Update Posted: April 17, 2019
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: Study of Sirolimus in CTD-IP in China

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Peking Union Medical College Hospital:
Sirolimus
connective tissue disease
thrombocytopenia
Additional relevant MeSH terms:
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Thrombocytopenia
Connective Tissue Diseases
Blood Platelet Disorders
Hematologic Diseases
Sirolimus
Everolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs