Trial Outcomes & Findings for Low Dose Rituximab in Thrombotic Thrombocytopenic Purpura (NCT NCT01554514)
NCT ID: NCT01554514
Last Updated: 2021-08-17
Results Overview
Exacerbation is recurring TTP ≤30 days after a Treatment Response (normal platelet count for 2 days) and discontinuation of plasma exchange. Refractory TTP is failure to achieve a Treatment Response by day 28, or failure to achieve a Durable Treatment Response (lasting at least 30 days) by day 60.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
19 participants
Primary outcome timeframe
60 days
Results posted on
2021-08-17
Participant Flow
Participant milestones
| Measure |
Low Dose Rituximab
this is a single-arm trial
rituximab: rituximab intravenously 100 mg every week for four doses
|
|---|---|
|
Overall Study
STARTED
|
19
|
|
Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Low Dose Rituximab
this is a single-arm trial
rituximab: rituximab intravenously 100 mg every week for four doses
|
|---|---|
|
Overall Study
1 patient ineligible with congenital TTP
|
1
|
|
Overall Study
Physician Decision
|
1
|
Baseline Characteristics
Low Dose Rituximab in Thrombotic Thrombocytopenic Purpura
Baseline characteristics by cohort
| Measure |
Low Dose Rituximab
n=19 Participants
this is a single-arm trial
rituximab: rituximab intravenously 100 mg every week for four doses
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
18 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
|
Age, Continuous
|
46.7 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
13 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
19 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 60 daysPopulation: Completed adjuvant riituximab therapy
Exacerbation is recurring TTP ≤30 days after a Treatment Response (normal platelet count for 2 days) and discontinuation of plasma exchange. Refractory TTP is failure to achieve a Treatment Response by day 28, or failure to achieve a Durable Treatment Response (lasting at least 30 days) by day 60.
Outcome measures
| Measure |
Low Dose Rituximab
n=17 Participants
this is a single-arm trial
rituximab: rituximab intravenously 100 mg every week for four doses
|
|---|---|
|
Incidence of the Composite Primary Outcome of Exacerbation or Refractory TTP
Number with exacerbation
|
2 Participants
|
|
Incidence of the Composite Primary Outcome of Exacerbation or Refractory TTP
Number with refractory TTP
|
0 Participants
|
SECONDARY outcome
Timeframe: 60 daysPopulation: Completed adjuvant rituximab therapy
Treatment Response is 2 consecutive days with platelet count ≥150, 000/µL Durable Treatment Response is a Treatment Response that persists for ≥30 days after discontinuation of plasma exchange and includes those with exacerbations
Outcome measures
| Measure |
Low Dose Rituximab
n=17 Participants
this is a single-arm trial
rituximab: rituximab intravenously 100 mg every week for four doses
|
|---|---|
|
Incidence of Durable Treatment Response
|
17 Participants
|
SECONDARY outcome
Timeframe: 60 daysPopulation: Completed adjuvant rituximab therapy
Median time to treatment response
Outcome measures
| Measure |
Low Dose Rituximab
n=17 Participants
this is a single-arm trial
rituximab: rituximab intravenously 100 mg every week for four doses
|
|---|---|
|
Number of Days to Durable Treatment Response
|
5 days
Interval 1.0 to 18.0
|
SECONDARY outcome
Timeframe: Between 30 days and 2 yearsRelapse is recurring TTP \>30 days after Treatment Response
Outcome measures
| Measure |
Low Dose Rituximab
n=17 Participants
this is a single-arm trial
rituximab: rituximab intravenously 100 mg every week for four doses
|
|---|---|
|
Incidence of Relapse
|
5 Participants
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Completed adjuvant rituximab
Mean months to relapse
Outcome measures
| Measure |
Low Dose Rituximab
n=17 Participants
this is a single-arm trial
rituximab: rituximab intravenously 100 mg every week for four doses
|
|---|---|
|
Months to Relapse
|
17.3 months
Interval 11.0 to 22.0
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Completed adjuvant rituximab
Incidence of death will be assessed at 4 weeks, 1 year and 2 years
Outcome measures
| Measure |
Low Dose Rituximab
n=17 Participants
this is a single-arm trial
rituximab: rituximab intravenously 100 mg every week for four doses
|
|---|---|
|
Incidence of Death
|
1 Participants
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Completed adjuvant rituximab therapy
Incidence, type and severity of treatment-related adverse events will be assessed. Patient reports, lab values, and physical exam were used to identify treatment-related adverse events.
Outcome measures
| Measure |
Low Dose Rituximab
n=17 Participants
this is a single-arm trial
rituximab: rituximab intravenously 100 mg every week for four doses
|
|---|---|
|
Treatment-related Adverse Events
|
13 treatment-related adverse events
|
Adverse Events
Low Dose Rituximab
Serious events: 10 serious events
Other events: 17 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Low Dose Rituximab
n=19 participants at risk；n=17 participants at risk
this is a single-arm trial
rituximab: rituximab intravenously 100 mg every week for four doses
|
|---|---|
|
General disorders
multi-system organ failure
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Gastrointestinal disorders
Lower Gastrointestinal Bleed
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Cardiac disorders
heart failure
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Infections and infestations
sepsis, gram-negative bacterial infection
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Infections and infestations
fever, central line complication, bacteremia
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Investigations
thrombocytopenia
|
5.9%
1/17 • Number of events 2 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
shock, acute respiratory failure
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Cardiac disorders
Atrial Fibrillation with Rapid Ventricular Response
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
General disorders
neck pain, abdominal distention
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Infections and infestations
cellulitis
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Cardiac disorders
chest pain, palpitations, shortness of breath
|
5.9%
1/17 • Number of events 2 • 2 years
|
|
Blood and lymphatic system disorders
Relapsed Thrombotic Thrombocytopenia Purpura
|
11.8%
2/17 • Number of events 4 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Hyper-somnolence with hypoxia and hypotension
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Infections and infestations
Pancreatitis (presenting symptom abdominal pain)
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
General disorders
fever
|
11.8%
2/17 • Number of events 2 • 2 years
|
Other adverse events
| Measure |
Low Dose Rituximab
n=19 participants at risk；n=17 participants at risk
this is a single-arm trial
rituximab: rituximab intravenously 100 mg every week for four doses
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
rhinorrhea
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
General disorders
fatigue
|
17.6%
3/17 • Number of events 3 • 2 years
|
|
Metabolism and nutrition disorders
decreased appetite
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
23.5%
4/17 • Number of events 4 • 2 years
|
|
Nervous system disorders
dizziness
|
11.8%
2/17 • Number of events 3 • 2 years
|
|
Nervous system disorders
headache
|
23.5%
4/17 • Number of events 5 • 2 years
|
|
Renal and urinary disorders
hematuria
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Blood and lymphatic system disorders
anemia
|
41.2%
7/17 • Number of events 12 • 2 years
|
|
Investigations
thrombocytopenia
|
23.5%
4/17 • Number of events 6 • 2 years
|
|
Blood and lymphatic system disorders
cervical lymphadenopathy
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Nervous system disorders
left hand numbness
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Gastrointestinal disorders
dyspepsia
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Skin and subcutaneous tissue disorders
ulcer to right nostril
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
nasal congestion
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Psychiatric disorders
sleep disturbances/insomnia
|
11.8%
2/17 • Number of events 2 • 2 years
|
|
Vascular disorders
hypertension
|
17.6%
3/17 • Number of events 3 • 2 years
|
|
Skin and subcutaneous tissue disorders
acne
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Investigations
weight gain
|
17.6%
3/17 • Number of events 3 • 2 years
|
|
General disorders
swelling
|
11.8%
2/17 • Number of events 2 • 2 years
|
|
Psychiatric disorders
depression
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Psychiatric disorders
delirium
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Investigations
elevated creatinine
|
11.8%
2/17 • Number of events 2 • 2 years
|
|
Skin and subcutaneous tissue disorders
pruritis
|
17.6%
3/17 • Number of events 4 • 2 years
|
|
Skin and subcutaneous tissue disorders
hives
|
5.9%
1/17 • Number of events 7 • 2 years
|
|
General disorders
fever
|
11.8%
2/17 • Number of events 2 • 2 years
|
|
Gastrointestinal disorders
constipation
|
17.6%
3/17 • Number of events 3 • 2 years
|
|
Gastrointestinal disorders
diarrhea
|
17.6%
3/17 • Number of events 3 • 2 years
|
|
Metabolism and nutrition disorders
hypomagnesemia
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Psychiatric disorders
anxiety
|
5.9%
1/17 • Number of events 3 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
pneumothorax
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
pneumonia
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Cardiac disorders
heart palpitations
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
General disorders
pain - lips
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
shortness of breath
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Metabolism and nutrition disorders
hyperglycemia
|
11.8%
2/17 • Number of events 4 • 2 years
|
|
Investigations
Lymphocyte decrease
|
11.8%
2/17 • Number of events 5 • 2 years
|
|
General disorders
Edema to face
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Metabolism and nutrition disorders
hypocalcemia
|
5.9%
1/17 • Number of events 1 • 2 years
|
|
Investigations
Elevated Total Bilirubin
|
5.3%
1/19 • Number of events 3 • 2 years
|
|
General disorders
flu-like symptoms
|
10.5%
2/19 • Number of events 2 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
dyspnea
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
General disorders
non-cardiac chest pain
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Gastrointestinal disorders
hemorrhoids
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Gastrointestinal disorders
hemorrhoidal hemorrhage
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Gastrointestinal disorders
gingival bleeding
|
5.3%
1/19 • Number of events 2 • 2 years
|
|
Metabolism and nutrition disorders
hypernatremia
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Injury, poisoning and procedural complications
Fall
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Eye disorders
blurred vision
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Eye disorders
double vision
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Eye disorders
floaters
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Investigations
ALT Elevation
|
10.5%
2/19 • Number of events 5 • 2 years
|
|
Investigations
AST elevation
|
5.3%
1/19 • Number of events 3 • 2 years
|
|
Gastrointestinal disorders
GI Disorder
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Nervous system disorders
neuropathy
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Gastrointestinal disorders
nausea
|
5.3%
1/19 • Number of events 4 • 2 years
|
|
Immune system disorders
anaphylaxis
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Musculoskeletal and connective tissue disorders
muscle pain/myalgia
|
10.5%
2/19 • Number of events 2 • 2 years
|
|
Gastrointestinal disorders
vomiting
|
5.3%
1/19 • Number of events 2 • 2 years
|
|
Gastrointestinal disorders
gastro-esophageal reflux disease
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Nervous system disorders
paresthesia - hands
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Social circumstances
menopause
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Infections and infestations
uterine infection
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Infections and infestations
vaginal discharge
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Infections and infestations
endometritis
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Psychiatric disorders
hallucinations
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Cardiac disorders
tachycardia
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Social circumstances
hot flashes
|
5.3%
1/19 • Number of events 1 • 2 years
|
|
Social circumstances
night sweats
|
5.3%
1/19 • Number of events 1 • 2 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place