Trial Outcomes & Findings for Higher Dose of Rituxan Versus Standard Doses of Rituxan With Cyclophosphamide, Vincristine, and Prednisone in Subjects With Chronic ITP (NCT NCT00774202)

Results Overview

Outcome measure was determined by comparing the study participants' historical responses to their initial treatment of rituximab at standard dose/regimen without "enhancement" (based on duration of response and type of response) to the participants response to their study treatment responses. Thus each patient was his or her own control although all study treatments included standard dose rituximab treatments (one at double the dose and one with additional treatments).

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

17 participants

Primary outcome timeframe

2 years

Results posted on

2019-01-09

Participant Flow

Participant milestones

Participant milestones
Measure	Standard Dose of Rituxan Plus CVP (R-CVP) 'Rituxan + Cyclophosphamide, Vincristine and Prednisone (R-CVP). Rituximab administered as an IV infusion at the standard dose of 375 mg/m2 for 4 doses. First Rituxan infusion will be given 5 days (± 3 days) prior to the first CVP, and the following 3 infusions will be given on the same day as the 3 cycles of CVP at week 2, week 5, and week 8.	Double Dose of Rituximab Rituximab administered at a dose of 750 mg/m2 once a week x 4 consecutive weeks (4 infusions in total).
Overall Study STARTED	9	8
Overall Study COMPLETED	9	8
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Higher Dose of Rituxan Versus Standard Doses of Rituxan With Cyclophosphamide, Vincristine, and Prednisone in Subjects With Chronic ITP

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Standard Dose of Rituxan Plus CVP (R-CVP) n=9 Participants 'Rituxan + Cyclophosphamide, Vincristine and Prednisone (R-CVP). Rituximab administered as an IV infusion at the standard dose of 375 mg/m2 for 4 doses. First Rituxan infusion will be given 5 days (± 3 days) prior to the first CVP, and the following 3 infusions will be given on the same day as the 3 cycles of CVP at week 2, week 5, and week 8.	Double Dose of Rituximab n=8 Participants Rituximab administered at a dose of 750 mg/m2 once a week x 4 consecutive weeks (4 infusions in total).	Total n=17 Participants Total of all reporting groups
Age, Customized age continuous	36 years STANDARD_DEVIATION 17 • n=5 Participants	44 years STANDARD_DEVIATION 19 • n=7 Participants	40 years STANDARD_DEVIATION 10 • n=5 Participants
Sex/Gender, Customized Males entered at Baseline	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Sex/Gender, Customized Females entered at baseline	5 Participants n=5 Participants	4 Participants n=7 Participants	9 Participants n=5 Participants
Initial response to treatment with standard dose rituximab Complete Response (CR)	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Initial response to treatment with standard dose rituximab Partial Response (PR)	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Initial response to treatment with standard dose rituximab No Response (NR)	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Population: the number of patients in each arm that increase their platelet count consistently above 100,000/uL after treatment

Outcome measure was determined by comparing the study participants' historical responses to their initial treatment of rituximab at standard dose/regimen without "enhancement" (based on duration of response and type of response) to the participants response to their study treatment responses. Thus each patient was his or her own control although all study treatments included standard dose rituximab treatments (one at double the dose and one with additional treatments).

Outcome measures

Outcome measures
Measure	Standard Dose of Rituxan Plus CVP (R-CVP) n=9 Participants 'Rituxan + Cyclophosphamide, Vincristine and Prednisone (R-CVP). Rituximab administered as an IV infusion at the standard dose of 375 mg/m2 for 4 doses. First Rituxan infusion will be given 5 days (± 3 days) prior to the first CVP, and the following 3 infusions will be given on the same day as the 3 cycles of CVP at week 2, week 5, and week 8.	Double Dose of Rituximab n=8 Participants Rituximab administered at a dose of 750 mg/m2 once a week x 4 consecutive weeks (4 infusions in total).
Efficacy of Higher Double Doses of Rituxan and of Standard Dose of Rituxan + Cyclophosphamide, Vincristine, Prednisone Worse Response	1 Participants	1 Participants
Efficacy of Higher Double Doses of Rituxan and of Standard Dose of Rituxan + Cyclophosphamide, Vincristine, Prednisone Better Response	0 Participants	0 Participants
Efficacy of Higher Double Doses of Rituxan and of Standard Dose of Rituxan + Cyclophosphamide, Vincristine, Prednisone same response as historical rituximab	8 Participants	7 Participants

SECONDARY outcome

Timeframe: 2 years

How many participants had SAEs among those receiving R-CVP or among those receiving double dose rituximab and did participants in one arm have substantially more SAEs than those in the other arm

Outcome measures

Outcome measures
Measure	Standard Dose of Rituxan Plus CVP (R-CVP) n=9 Participants 'Rituxan + Cyclophosphamide, Vincristine and Prednisone (R-CVP). Rituximab administered as an IV infusion at the standard dose of 375 mg/m2 for 4 doses. First Rituxan infusion will be given 5 days (± 3 days) prior to the first CVP, and the following 3 infusions will be given on the same day as the 3 cycles of CVP at week 2, week 5, and week 8.	Double Dose of Rituximab n=8 Participants Rituximab administered at a dose of 750 mg/m2 once a week x 4 consecutive weeks (4 infusions in total).
Number of Participants With SAEs	0 number of patients with SAEs	1 number of patients with SAEs

SECONDARY outcome

Timeframe: 2 years

The goal is to see if there are major differences between the two arms for each group in term of efficacy and of toxicity both overall and in comparison to the previous responses to rituximab alone. The comparisons are for level of response eg CR (\>100k) vs PR (230-100k) vs NR (\<30k) and for duration of response-----duration of response is controlled by comparison to duration of response from initial rituximab infusions

Outcome measures

Outcome measures
Measure	Standard Dose of Rituxan Plus CVP (R-CVP) n=9 Participants 'Rituxan + Cyclophosphamide, Vincristine and Prednisone (R-CVP). Rituximab administered as an IV infusion at the standard dose of 375 mg/m2 for 4 doses. First Rituxan infusion will be given 5 days (± 3 days) prior to the first CVP, and the following 3 infusions will be given on the same day as the 3 cycles of CVP at week 2, week 5, and week 8.	Double Dose of Rituximab n=8 Participants Rituximab administered at a dose of 750 mg/m2 once a week x 4 consecutive weeks (4 infusions in total).
Relative Efficacy of the 2 Groups CR	4 number of responders	4 number of responders
Relative Efficacy of the 2 Groups PR	1 number of responders	0 number of responders
Relative Efficacy of the 2 Groups response longer than previous response	0 number of responders	0 number of responders

Adverse Events

Rituximab + C, V, P

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Double Dose Rituximab

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Rituximab + C, V, P n=9 participants at risk	Double Dose Rituximab n=8 participants at risk
Blood and lymphatic system disorders Serum Sickness	11.1% 1/9 • Number of events 1 • collected over 2 years based on adverse event reporting	0.00% 0/8 • collected over 2 years based on adverse event reporting

Other adverse events

Other adverse events
Measure	Rituximab + C, V, P n=9 participants at risk	Double Dose Rituximab n=8 participants at risk
General disorders non-serious	44.4% 4/9 • Number of events 4 • collected over 2 years based on adverse event reporting	25.0% 2/8 • Number of events 2 • collected over 2 years based on adverse event reporting

Additional Information

James B. Bussel, M.D. Professor of Pediatrics

Weill Cornell Medical College

Phone: 212-746-3474

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place