Clinical trial NCT03043105: TCP Regimen in Newly Diagnosed MCD:a Prospective, Single-center, Single-arm, Phase-II Pilot Trial
Official title: Thalidomide, Cyclophosphamide and Prednisone in Newly Diagnosed Multicentric Castleman's Disease: a Prospective, Single-center, Single-arm, Phase-II Pilot Trial
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Multicentric Castleman Disease
Keywords: Multicentric Castleman's Disease
Thalidomide, cyclophosphamide and prednisone (TCP regimen)
Efficacy
Safety
Lead sponsor: Peking Union Medical College Hospital
Sponsor class: OTHER
First posted: 2017-02-03
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: This will be a single center, single arm, phase-II pilot study.
Primary purpose: TREATMENT

Brief summary: To explore the effectiveness and safety of thalidomide, cyclophosphamide and prednisone (TCP regimen) in newly diagnosed Multicentric Castleman's disease (MCD) patients.

Detailed description: This is a single center, open-labeled , single arm, phase-II pilot study which aims to evaluate the efficacy and safety of thalidomide, cyclophosphamide and prednisone (TCP regimen) in newly diagnosed Multicentric Castleman's disease (MCD) patients.There would be two phases of the study. The treatment and the response evaluation phase will last from the time of enrollment up to 24 months (evaluation will be carried out every 3 months). The follow-up phase to assess for progression of disease will last from 24 months (2 years) to 4 years after enrollment (evaluation will be carried out every 12 months).The total study duration will be 4 years after the last patient starts study medication.

Eligibility criteria: * Inclusion Criteria:

  * ≥18 years, all race/ethnic groups in China；
  * Newly diagnosed and previously untreated (patients are allowed to have received oral prednisone for up to 1 week before enrollment) symptomatic MCD patients (symptomatic disease is defined by the presence of clinical symptoms with the NCI-CTCAE grading ≥1 that are attributable to the disease, and for which treatment is indicated);
  * Eastern Cooperative Oncology Group (ECOG) Performance Status ≤2;
  * Clinical laboratory values meeting these criteria at screening: absolute neutrophil count ≥ 1•0 x 109/L, Platelets ≥ 50 x 109/L, Alanine aminotransferase (ALT) within 2•5 x upper limit of normal (ULN); total bilirubin within 2•5 x ULN; estimated glomerular filtration rate (according to MDRD formula) \<15ml/min;
  * Women of childbearing potential must agree to use birth control measures during the study and for at least 3 months after receiving the last dose of study agent, and must have a negative pregnancy test at screening period. Men must agree to use birth control measures during the study and for at least 3 months after receiving the last dose of study agent;
  * Informed consent must be signed.
* Exclusion Criteria:

  * age under 18 years;
  * ECOG (eastern cooperative oncology group) status above 2;
  * Immunosuppressive or anti-neoplastic drugs within the last 3 months;
  * serious diseases including malignancy;
  * Plan to have babies within 1 year after enrollment (for women and men), or pregnancy / breast-feeding (for women);
  * Known hypersensitivity to study agents;
  * Active infection requiring systemic treatment;
  * Other severe concurrent disease (eg. uncontrolled diabetes, symptomatic coronary heart disease) that is likely to interfere with study procedures or results, or that in the opinion of the investigator would constitute a hazard for participating in this study;
  * Unwilling or unable to provide informed consent;
  * Unwilling to return for follow-up at PUMCH.

Primary outcomes:
measure: Number of Patients With Durable Tumor and Symptomatic Response
timeFrame: From baseline to the time point when a patient achieves treatment response for 24 weeks.
Secondary outcomes:
measure: Progression-free Survival
timeFrame: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 36 months
measure: Overall Survival
timeFrame: From date of randomization until the date of death from any cause, assessed up to 36 months.
measure: Change in SF-36 Score
timeFrame: From baseline to 24 weeks after treatment.
measure: Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0 ( ≥1 Grade)
timeFrame: From initiation of TCP regimen to 3 months after the end of treatment or to time point of the initiation of second line therapy.
measure: Number of Participants With Treatment-related Serious Adverse Events as Assessed by CTCAE v4.0 ( ≥3 Grade)
timeFrame: From initiation of TCP regimen to 3 months after the end of treatment or to time point of the initiation of second line therapy.
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
Study officials:
References:
citation: Zhang L, Zhao AL, Duan MH, Li ZY, Cao XX, Feng J, Zhou DB, Zhong DR, Fajgenbaum DC, Li J. Phase 2 study using oral thalidomide-cyclophosphamide-prednisone for idiopathic multicentric Castleman disease. Blood. 2019 Apr 18;133(16):1720-1728. doi: 10.1182/blood-2018-11-884577. Epub 2019 Feb 13.