An open-label continuation trial of sirolimus for tocilizumab-refractory idiopathic multicentric Castleman's disease: Study protocol for an investigator-initiated, multicenter, open-label trial (SPIRIT Compliant)

JRCT ID: jRCT2051200050

Registered date:31/08/2020

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: idiopathic multicentric Castleman's disease
• Date of first enrollment: 09/10/2020
• Target sample size: 20
• Study type: Interventional
• Intervention(s): After the evaluation and testing at 16 weeks in the preceding trial, the patient is immediately transferred to this trial and started on the investigational drug (open-label, single-arm).

Outcome(s)

• Primary Outcome: The primary endpoint is the incidence of adverse events.

Secondary Outcome

(1) The safety evaluation indices of this clinical trial are as follows: adverse events (adverse event incidence rate, serious adverse event incidence rate, and side effect incidence rate), clinical examination (hematological examination, blood biochemical examination, and urinalysis), all medically important indicators (physical findings, vital signs, electrocardiogram results, echocardiographic findings, etc.).
(2) CHAP score (presence or absence of decrease): presence or absence of a score decrease of 1 or more from baseline at 2, 4, 8, 12, and 24 weeks, every 12 weeks thereafter, and at the time of the study completion or drug discontinuation.
(3) CHAP score: change from baseline at 2, 4, 8, 12, and 24 weeks, every 12 weeks thereafter, and at the time of the study completion or drug discontinuation.
(4) CHAP score minus CRP score: change from baseline at 2, 4, 8, 12, and 24 weeks, every 12 weeks thereafter, and at the time of the study completion or drug discontinuation.
(5) Hb (g/dL): change from baseline at 2, 4, 8, 12, and 24 weeks, every 12 weeks thereafter, and at the time of the study completion or drug discontinuation.
(6) Alb (g/dL): change from baseline at 2, 4, 8, 12, and 24 weeks, every 12 weeks thereafter, and at the time of the study completion or drug discontinuation.
(7) CRP (mg/dL): change from baseline at 2, 4, 8, 12, and 24 weeks, every 12 weeks thereafter, and at the time of the study completion or drug discontinuation.
(8) Physician global assessment (disease activity assessment, 100 mm visual analog scale [VAS]): change from baseline at 4, 8, 12, and 24 weeks, every 12 weeks thereafter, and at the time of the study completion or drug discontinuation.
(9) Patient global assessment (disease activity assessment, 100 mm VAS): change from baseline at 4, 8, 12, and 24 weeks, every 12 weeks thereafter, and at the time of the study completion or drug discontinuation.
(10) Lymph node changes in subjects with lymphadenopathy: change from baseline at 24 weeks, every 24 weeks thereafter, and at the time of the study completion or drug discontinuation, as well as changes in the number of lymph nodes >10 mm in diameter from baseline at 24 weeks, every 24 weeks thereafter, and at the time of the study completion or drug discontinuation.

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

Patients may be included if all of the following criteria are met:
(1) Patients who have completed 16-week treatment with investigational drug treatment in the preceding trial
(2) Patients who have received a thorough explanation of the contents of explanatory documents and other matters concerning clinical trials understood the contents thereof and provided written consent based on their free will to participate in this trial. In case the patient is under 20 years of age at the time of obtaining the consent, the patient has obtained written consent to participate in the clinical trial from the proxy and written consent to participate in the clinical trial from the patient himself/herself.

Exclude criteria

Patients with any one of the following will be excluded
(1) Patients with an Eastern Cooperative Oncology Group Performance Status of 4.
(2) Patients (males and females of childbearing potential) who are unable to use an appropriate method of contraception during the period of study drug administration and 12 weeks after the last dose of study drug
(3) Female patients who are breastfeeding or pregnant
(4) Patients with complications of serious diseases that are deemed unsuitable for the clinical trial by the investigator or sub-investigator
(5) Patients whose condition in a prior study is deemed unsuitable for continued treatment by the investigator or subinvestigator
(6) Other patients who deemed inappropriate by the investigator or sub-investigator

Related Information

• Primary Sponsor: Kawakami Atsushi
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development