Idiopathic Thrombocytopenic Purpura
Conditions
Brief summary
This study will compare the efficacy, safety, and pharmacokinetics of standard treatment versus standard treatment plus MabThera in patients with ITP. The anticipated time on study treatment is \<3 months, and the target sample size is 100-500 individuals.
Interventions
DRUGrituximab
DRUGDexamethasone
Sponsors
Hoffmann-La Roche
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* adult patients \>=18 years of age; * untreated ITP.
Exclusion criteria
* ITP with relapse; * positive test result for HIV or hepatitis B or C; * active infection requiring systemic therapy; * malignancy within 3 years before study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With a Sustained Response | Week 24 | Sustained response defined as a platelet count of greater than or equal to (≥) 50x10\^9/L at 6 months (Week 24) after the initial treatment. Participants failing therapy before Month 6 (Week 24) and treated in other ways were considered failures. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With an Initial Response | Week 4 | Initial response was defined as an increase in platelet count of ≥50x10\^9/L by Day 30 (Week 4) after the start of treatment in either treatment arm. |
| Percentage of Participants With an Initial Complete Response | Week 4 | Initial complete response was defined as an increase in platelet count of ≥100x10\^9/L by Day 30 (Week 4) after the initiation of treatment in either treatment arm. |
Countries
Italy
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Arm A: Dexamethasone Participants received 40 mg dexamethasone, orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4). Participants in this treatment arm who failed to achieve a sustained response and had a platelet count of ≤20 x 10\^9 platelets per liter (L; from Day 30 up to end of 6 months) were treated with salvage treatment of dexamethasone 40 mg, orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4) and rituximab 375 mg/m\^2, IV, with premedication of oral acetaminophen 500 mg and chlorpheniramine 10 mg IV on Days 7, 14, 21, and 28. | 52 |
| Arm B: Dexamethasone + Rituximab Participants received dexamethasone 40 mg, orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4) and rituximab 375 mg per square meter (mg/m\^2), IV, with premedication of oral acetaminophen 500 mg and chlorpheniramine 10 mg IV on Days 7, 14, 21, and 28. Nonresponsive participants with platelets \<20 x10\^9/L or with active bleeding could have also received an additional treatment course of dexamethasone 40 mg, orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4) and rituximab 375 mg/m\^2, IV on Days 7, 14, 21, and 28 administered with IgG IV (at investigator discretion) and/or low/medium dose steroids (at investigator discretion) on Days 7, 14, 21, and 28. | 49 |
| Total | 101 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Did not take treatment | 1 | 1 |
Baseline characteristics
| Characteristic | Arm A: Dexamethasone | Arm B: Dexamethasone + Rituximab | Total |
|---|---|---|---|
| Age, Continuous | 47 years STANDARD_DEVIATION 19 | 49 years STANDARD_DEVIATION 16 | 48.41 years STANDARD_DEVIATION 18.03 |
| Sex: Female, Male Female | 33 Participants | 27 Participants | 60 Participants |
| Sex: Female, Male Male | 19 Participants | 22 Participants | 41 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 25 / 52 | 37 / 49 | 18 / 27 |
| serious Total, serious adverse events | 1 / 52 | 3 / 49 | 3 / 27 |
Outcome results
Primary
Percentage of Participants With a Sustained Response
Sustained response defined as a platelet count of greater than or equal to (≥) 50x10\^9/L at 6 months (Week 24) after the initial treatment. Participants failing therapy before Month 6 (Week 24) and treated in other ways were considered failures.
Time frame: Week 24
Population: The Intent-to-Treat (ITT) population includes all participants who were randomized, who received at least (\<) 1 dose of study medication, and who had at least 1 follow-up contact.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Arm A: Dexamethasone | Percentage of Participants With a Sustained Response | 36 percentage of participants |
| Arm B: Dexamethasone + Rituximab | Percentage of Participants With a Sustained Response | 63 percentage of participants |
p-value: 0.004Fisher Exact
Secondary
Percentage of Participants With an Initial Complete Response
Initial complete response was defined as an increase in platelet count of ≥100x10\^9/L by Day 30 (Week 4) after the initiation of treatment in either treatment arm.
Time frame: Week 4
Population: ITT population; excluding participants who received additional steroid therapy or IV Ig course during the first month of therapy.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Arm A: Dexamethasone | Percentage of Participants With an Initial Complete Response | 23 percentage of participants |
| Arm B: Dexamethasone + Rituximab | Percentage of Participants With an Initial Complete Response | 48 percentage of participants |
p-value: 0.015Fisher Exact
Secondary
Percentage of Participants With an Initial Response
Initial response was defined as an increase in platelet count of ≥50x10\^9/L by Day 30 (Week 4) after the start of treatment in either treatment arm.
Time frame: Week 4
Population: ITT population; excluding participants who received additional steroid therapy or IV Ig course during the first month of therapy.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Arm A: Dexamethasone | Percentage of Participants With an Initial Response | 27 percentage of participants |
| Arm B: Dexamethasone + Rituximab | Percentage of Participants With an Initial Response | 68 percentage of participants |
p-value: 0.001Fisher Exact