Trial Outcomes & Findings for TCP Regimen in Newly Diagnosed MCD:a Prospective, Single-center, Single-arm, Phase-II Pilot Trial (NCT NCT03043105)

NCT ID: NCT03043105

Last Updated: 2020-04-16

Results Overview

Durable tumor and symptomatic response is complete response (CR) + partial response (PR). CR: complete disappearance of all measurable and evaluable disease (eg, pleural effusion) and resolution of baseline symptoms attributed to multicentric Castleman's disease, sustained for at least 18 weeks. PR: \>=50 percent decrease in sum of the product of the diameters of indicator lesion(s), with at least stable disease in all other evaluable disease in the absence of treatment failure sustained for at least 6 months.

Recruitment status

UNKNOWN

Study phase

PHASE2

Target enrollment

25 participants

Primary outcome timeframe

From baseline to the time point when a patient achieves treatment response for 24 weeks.

Results posted on

2020-04-16

Participant Flow

Participant milestones

Participant milestones
Measure
TCP Treatment Group
The newly-diagnosed symptomatic MCD patients received thalidomide, cyclophosphamide and prednisone (TCP ) treatment. The accurate dose of TCP regimen is listed as follows. * Thalidomide: 100mg QN for 1 year; And maintained with 100mg QN for the second year; * Cyclophosphamide: 300mg/m2 on Day 1, 8, 15, 22 every month for 1 year; * Prednisone: 1mg/kg on Day 1-2, 8-9, 15-16, 22-23 every month for 1 year.
Overall Study
STARTED
25
Overall Study
COMPLETED
25
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

TCP Regimen in Newly Diagnosed MCD:a Prospective, Single-center, Single-arm, Phase-II Pilot Trial

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
TCP Treatment Group
n=25 Participants
The newly-diagnosed symptomatic MCD patients received thalidomide, cyclophosphamide and prednisone (TCP ) treatment. The accurate dose of TCP regimen is listed as follows. * Thalidomide: 100mg QN for 1 year; And maintained with 100mg QN for the second year; * Cyclophosphamide: 300mg/m2 on Day 1, 8, 15, 22 every month for 1 year; * Prednisone: 1mg/kg on Day 1-2, 8-9, 15-16, 22-23 every month for 1 year.
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
25 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Age, Continuous
40 years
n=5 Participants
Sex: Female, Male
Female
8 Participants
n=5 Participants
Sex: Female, Male
Male
17 Participants
n=5 Participants
Race/Ethnicity, Customized
East Asian
25 Participants
n=5 Participants
Region of Enrollment
China
25 participants
n=5 Participants

PRIMARY outcome

Timeframe: From baseline to the time point when a patient achieves treatment response for 24 weeks.

Durable tumor and symptomatic response is complete response (CR) + partial response (PR). CR: complete disappearance of all measurable and evaluable disease (eg, pleural effusion) and resolution of baseline symptoms attributed to multicentric Castleman's disease, sustained for at least 18 weeks. PR: \>=50 percent decrease in sum of the product of the diameters of indicator lesion(s), with at least stable disease in all other evaluable disease in the absence of treatment failure sustained for at least 6 months.

Outcome measures

Outcome measures
Measure
TCP Treatment Group
n=25 Participants
The newly-diagnosed symptomatic MCD patients received thalidomide, cyclophosphamide and prednisone (TCP ) treatment. The accurate dose of TCP regimen is listed as follows. * Thalidomide: 100mg QN for 1 year; And maintained with 100mg QN for the second year; * Cyclophosphamide: 300mg/m2 on Day 1, 8, 15, 22 every month for 1 year; * Prednisone: 1mg/kg on Day 1-2, 8-9, 15-16, 22-23 every month for 1 year.
Number of Patients With Durable Tumor and Symptomatic Response
12 Participants

SECONDARY outcome

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

Progression-free survival (PFS) is defined as the time to death or treatment failure. Treatment failure is defined as: sustained increase in grade ≥2 disease-related symptoms persisting ≥12 weeks; new disease-related grade ≥3 symptoms; sustained \>1 point increase in ECOG-PS persisting for ≥12 weeks; radiological progression; or initiation of another treatment for MCD.

Outcome measures

Outcome measures
Measure
TCP Treatment Group
n=25 Participants
The newly-diagnosed symptomatic MCD patients received thalidomide, cyclophosphamide and prednisone (TCP ) treatment. The accurate dose of TCP regimen is listed as follows. * Thalidomide: 100mg QN for 1 year; And maintained with 100mg QN for the second year; * Cyclophosphamide: 300mg/m2 on Day 1, 8, 15, 22 every month for 1 year; * Prednisone: 1mg/kg on Day 1-2, 8-9, 15-16, 22-23 every month for 1 year.
Progression-free Survival
23.32 months
Interval 17.22 to 29.42

SECONDARY outcome

Timeframe: From date of randomization until the date of death from any cause, assessed up to 36 months.

Overall survival, defined as the time to patients' death, is measured.

Outcome measures

Outcome measures
Measure
TCP Treatment Group
n=25 Participants
The newly-diagnosed symptomatic MCD patients received thalidomide, cyclophosphamide and prednisone (TCP ) treatment. The accurate dose of TCP regimen is listed as follows. * Thalidomide: 100mg QN for 1 year; And maintained with 100mg QN for the second year; * Cyclophosphamide: 300mg/m2 on Day 1, 8, 15, 22 every month for 1 year; * Prednisone: 1mg/kg on Day 1-2, 8-9, 15-16, 22-23 every month for 1 year.
Overall Survival
32.16 months
Interval 28.08 to 36.24

SECONDARY outcome

Timeframe: From baseline to 24 weeks after treatment.

Population: Each patient is assessed by SF-36 scoring system, which contains 8 dimensions, including physical functioning, role physical, role emotional, vitality, mental health, social functioning, bodily pain, and general health.

SF-36 score is a self-administered scoring system which reflects a patient's general health status. SF-36 contains 8 dimensions, including physical functioning, role physical, role emotional, vitality, mental health, social functioning, bodily pain, and general health. Each dimension ranges from 0 to 100. Higher scores mean better outcome. SF-36 score at baseline was compared with SF-36 score at 24 weeks.

Outcome measures

Outcome measures
Measure
TCP Treatment Group
n=25 Participants
The newly-diagnosed symptomatic MCD patients received thalidomide, cyclophosphamide and prednisone (TCP ) treatment. The accurate dose of TCP regimen is listed as follows. * Thalidomide: 100mg QN for 1 year; And maintained with 100mg QN for the second year; * Cyclophosphamide: 300mg/m2 on Day 1, 8, 15, 22 every month for 1 year; * Prednisone: 1mg/kg on Day 1-2, 8-9, 15-16, 22-23 every month for 1 year.
Change in SF-36 Score
role emotional
19.6 score on a scale
Standard Deviation 10.18
Change in SF-36 Score
physical functioning
15.1 score on a scale
Standard Deviation 6.78
Change in SF-36 Score
role physical
26.7 score on a scale
Standard Deviation 10.53
Change in SF-36 Score
vitality
11.1 score on a scale
Standard Deviation 5.75
Change in SF-36 Score
mental health
8.5 score on a scale
Standard Deviation 5.50
Change in SF-36 Score
social functioning
13.6 score on a scale
Standard Deviation 6.70
Change in SF-36 Score
bodily pain
15.8 score on a scale
Standard Deviation 6.19
Change in SF-36 Score
general health
9.1 score on a scale
Standard Deviation 4.78

SECONDARY outcome

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.

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 (patients with grades ≥1 would be included)

Outcome measures

Outcome measures
Measure
TCP Treatment Group
n=25 Participants
The newly-diagnosed symptomatic MCD patients received thalidomide, cyclophosphamide and prednisone (TCP ) treatment. The accurate dose of TCP regimen is listed as follows. * Thalidomide: 100mg QN for 1 year; And maintained with 100mg QN for the second year; * Cyclophosphamide: 300mg/m2 on Day 1, 8, 15, 22 every month for 1 year; * Prednisone: 1mg/kg on Day 1-2, 8-9, 15-16, 22-23 every month for 1 year.
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0 ( ≥1 Grade)
10 Participants

SECONDARY outcome

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.

Number of participants with treatment-related serious adverse events as assessed by CTCAE v4.0 (patients with grades ≥3 would be included)

Outcome measures

Outcome measures
Measure
TCP Treatment Group
n=25 Participants
The newly-diagnosed symptomatic MCD patients received thalidomide, cyclophosphamide and prednisone (TCP ) treatment. The accurate dose of TCP regimen is listed as follows. * Thalidomide: 100mg QN for 1 year; And maintained with 100mg QN for the second year; * Cyclophosphamide: 300mg/m2 on Day 1, 8, 15, 22 every month for 1 year; * Prednisone: 1mg/kg on Day 1-2, 8-9, 15-16, 22-23 every month for 1 year.
Number of Participants With Treatment-related Serious Adverse Events as Assessed by CTCAE v4.0 ( ≥3 Grade)
2 Participants

Adverse Events

TCP Treatment Group

Serious events: 2 serious events
Other events: 10 other events
Deaths: 3 deaths

Serious adverse events

Serious adverse events
Measure
TCP Treatment Group
n=25 participants at risk
The newly-diagnosed symptomatic MCD patients received thalidomide, cyclophosphamide and prednisone (TCP ) treatment. The accurate dose of TCP regimen is listed as follows. * Thalidomide: 100mg QN for 1 year; And maintained with 100mg QN for the second year; * Cyclophosphamide: 300mg/m2 on Day 1, 8, 15, 22 every month for 1 year; * Prednisone: 1mg/kg on Day 1-2, 8-9, 15-16, 22-23 every month for 1 year.
Respiratory, thoracic and mediastinal disorders
Pulmonary infection
4.0%
1/25 • Number of events 1 • Safety data were collected until 30 days after the last dose of study drugs, except for secondary primary malignancies (which were assessed throughout the duration of follow-up, up to 4 years). Secondary primary malignancies were assessed throughout the duration of follow-up, up to 3 years.
Skin and subcutaneous tissue disorders
Skin rash
4.0%
1/25 • Number of events 1 • Safety data were collected until 30 days after the last dose of study drugs, except for secondary primary malignancies (which were assessed throughout the duration of follow-up, up to 4 years). Secondary primary malignancies were assessed throughout the duration of follow-up, up to 3 years.

Other adverse events

Other adverse events
Measure
TCP Treatment Group
n=25 participants at risk
The newly-diagnosed symptomatic MCD patients received thalidomide, cyclophosphamide and prednisone (TCP ) treatment. The accurate dose of TCP regimen is listed as follows. * Thalidomide: 100mg QN for 1 year; And maintained with 100mg QN for the second year; * Cyclophosphamide: 300mg/m2 on Day 1, 8, 15, 22 every month for 1 year; * Prednisone: 1mg/kg on Day 1-2, 8-9, 15-16, 22-23 every month for 1 year.
Gastrointestinal disorders
constipation
40.0%
10/25 • Number of events 10 • Safety data were collected until 30 days after the last dose of study drugs, except for secondary primary malignancies (which were assessed throughout the duration of follow-up, up to 4 years). Secondary primary malignancies were assessed throughout the duration of follow-up, up to 3 years.

Additional Information

Dr. Jian Li

Peking Union Medical College Hospital

Phone: 86-010-69155027

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place