Trial Outcomes & Findings for Rituximab for the Primary Treatment of Denovo Extensive Chronic Graft Versus Host Disease (GVHD) (NCT NCT01161628)
NCT ID: NCT01161628
Last Updated: 2016-04-20
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
2 years
Results posted on
2016-04-20
Participant Flow
Participant milestones
| Measure |
Rituxan
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Overall Study
STARTED
|
25
|
|
Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
Rituxan
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Overall Study
Death
|
6
|
|
Overall Study
Lack of Efficacy
|
2
|
Baseline Characteristics
Rituximab for the Primary Treatment of Denovo Extensive Chronic Graft Versus Host Disease (GVHD)
Baseline characteristics by cohort
| Measure |
Rituxan
n=25 Participants
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
19 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
21 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black/African American
|
4 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Rituxan
n=22 Participants
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Rate of Complete Response of cGVHD to Treatment.
|
84 percentage of patients
|
PRIMARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Rituxan
n=22 Participants
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Rate of Overall Response of cGVHD to Treatment
|
88 percentage of patients
|
PRIMARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Rituxan
n=22 Participants
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Rate of Partial Response of cGVHD to Treatment
|
5 percentage of patients
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: 20 out of the 25 patients enrolled received no corticosteroids at all during the course of treatment for cGVHD
Outcome measures
| Measure |
Rituxan
n=25 Participants
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Requirement for Systemic Corticosteroid Use
|
20 participants
|
SECONDARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Rituxan
n=17 Participants
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Time to Immunosuppression Withdrawal
|
300 days
Interval 138.0 to 488.0
|
SECONDARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Rituxan
n=25 Participants
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Incidence of Overall Survival
|
82 percentage of patients
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Only 2 patients out of the 22 evaluable patients enrolled received steroids during the course of treatment for cGVHD. A total of 25 patients were enrolled; 3 patients were excluded from this analysis due to treatment failure.
Outcome measures
| Measure |
Rituxan
n=2 Participants
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Duration of Systemic Corticosteroid Use
|
15 days
Interval 13.0 to 18.0
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Of the surviving patients at 2 years (82% of initial enrolled population)
Outcome measures
| Measure |
Rituxan
n=20 Participants
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Incidence of Disease-free Survival
|
79 percentage of patients
|
SECONDARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Rituxan
n=25 Participants
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Incidence of Non-relapse Mortality
|
21 percentage of patients
|
Adverse Events
Rituxan
Serious events: 17 serious events
Other events: 25 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Rituxan
n=25 participants at risk
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
General disorders
Failure to thrive
|
4.0%
1/25 • Number of events 1 • adverse event data was collected for 2 years.
|
|
Cardiac disorders
Ischemic cardiomyopathy
|
4.0%
1/25 • Number of events 1 • adverse event data was collected for 2 years.
|
|
Renal and urinary disorders
Acute renal failure
|
4.0%
1/25 • Number of events 1 • adverse event data was collected for 2 years.
|
|
Infections and infestations
Pneumonia
|
16.0%
4/25 • Number of events 4 • adverse event data was collected for 2 years.
|
|
Hepatobiliary disorders
Liver dysfunction
|
4.0%
1/25 • Number of events 1 • adverse event data was collected for 2 years.
|
|
General disorders
Weakness
|
4.0%
1/25 • Number of events 1 • adverse event data was collected for 2 years.
|
|
Vascular disorders
Deep Vein Thrombosis
|
4.0%
1/25 • Number of events 1 • adverse event data was collected for 2 years.
|
|
Infections and infestations
Sepsis
|
4.0%
1/25 • Number of events 1 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
respiratory failure
|
4.0%
1/25 • Number of events 1 • adverse event data was collected for 2 years.
|
|
Psychiatric disorders
altered mental status
|
4.0%
1/25 • Number of events 1 • adverse event data was collected for 2 years.
|
|
Musculoskeletal and connective tissue disorders
Hip Fracture
|
4.0%
1/25 • Number of events 1 • adverse event data was collected for 2 years.
|
|
Investigations
Fever
|
52.0%
13/25 • Number of events 13 • adverse event data was collected for 2 years.
|
Other adverse events
| Measure |
Rituxan
n=25 participants at risk
all patients will receive Rituxan for the treatment of newly diagnosed chronic GVHD
Rituximab: Rituximab 375 mg/m2/dose x 4 weekly doses on days 1, 8, 15 and 22 and then at 3, 6, 9 and 12 months.
|
|---|---|
|
Gastrointestinal disorders
abdominal discomfort
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
abdominal distention
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
abdominal pain
|
24.0%
6/25 • adverse event data was collected for 2 years.
|
|
Musculoskeletal and connective tissue disorders
muscle aches
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Renal and urinary disorders
acute renal failure
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Hepatobiliary disorders
hypoalbuminemia
|
24.0%
6/25 • adverse event data was collected for 2 years.
|
|
Hepatobiliary disorders
increased alkaline phosphatase
|
20.0%
5/25 • adverse event data was collected for 2 years.
|
|
Hepatobiliary disorders
increased ALT
|
40.0%
10/25 • adverse event data was collected for 2 years.
|
|
Nervous system disorders
altered mental status
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Blood and lymphatic system disorders
neutropenia
|
80.0%
20/25 • adverse event data was collected for 2 years.
|
|
Blood and lymphatic system disorders
anemia
|
56.0%
14/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
anorexia
|
52.0%
13/25 • adverse event data was collected for 2 years.
|
|
Psychiatric disorders
anxiety
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Hepatobiliary disorders
increased AST
|
40.0%
10/25 • adverse event data was collected for 2 years.
|
|
Musculoskeletal and connective tissue disorders
back pain
|
20.0%
5/25 • adverse event data was collected for 2 years.
|
|
Musculoskeletal and connective tissue disorders
shoulder pain
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Infections and infestations
bacteremia
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Renal and urinary disorders
BKV cystitis
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Eye disorders
blurry vision
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Blood and lymphatic system disorders
bruising
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Cardiac disorders
cardiomyopathy
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
chest congestion
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Cardiac disorders
chest pain
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Investigations
chills
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Eye disorders
conjunctivitis
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
constipation
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
44.0%
11/25 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
crackles at lung bases
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Renal and urinary disorders
hypercreatinemia
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Investigations
deconditioned
|
24.0%
6/25 • adverse event data was collected for 2 years.
|
|
Endocrine disorders
dehydration
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Psychiatric disorders
depression
|
20.0%
5/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
diarrhea
|
52.0%
13/25 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
diminished breath sounds
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Nervous system disorders
dizziness
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Nervous system disorders
drowsiness
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Eye disorders
dry eyes
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Investigations
dry lips
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
dry mouth
|
40.0%
10/25 • adverse event data was collected for 2 years.
|
|
Skin and subcutaneous tissue disorders
dry skin
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Renal and urinary disorders
dysuria
|
28.0%
7/25 • adverse event data was collected for 2 years.
|
|
Ear and labyrinth disorders
ear pain
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Cardiac disorders
edema
|
32.0%
8/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
buccal mucosa erythema
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Skin and subcutaneous tissue disorders
erythematous lesion
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Musculoskeletal and connective tissue disorders
extremity pain
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Eye disorders
eye drainage
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Eye disorders
eye irritation
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Investigations
fatigue
|
68.0%
17/25 • adverse event data was collected for 2 years.
|
|
Investigations
fever
|
24.0%
6/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
food intolerance
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
gastritis
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
GVHD - gut
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
GVHD - oral
|
20.0%
5/25 • adverse event data was collected for 2 years.
|
|
Skin and subcutaneous tissue disorders
GVHD - skin
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Renal and urinary disorders
hematuria
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
hemorrhoids
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Metabolism and nutrition disorders
hypercalcemia
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Metabolism and nutrition disorders
hyperglycemia
|
52.0%
13/25 • adverse event data was collected for 2 years.
|
|
Metabolism and nutrition disorders
hyperkalemia
|
32.0%
8/25 • adverse event data was collected for 2 years.
|
|
Metabolism and nutrition disorders
hypernatremia
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Skin and subcutaneous tissue disorders
hyperpigmentation
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Cardiac disorders
hypertension
|
24.0%
6/25 • adverse event data was collected for 2 years.
|
|
Metabolism and nutrition disorders
hypocalcemia
|
24.0%
6/25 • adverse event data was collected for 2 years.
|
|
Metabolism and nutrition disorders
hypokalemia
|
28.0%
7/25 • adverse event data was collected for 2 years.
|
|
Metabolism and nutrition disorders
hypomagnesemia
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Metabolism and nutrition disorders
hyponatremia
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Cardiac disorders
hypotension
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
hypoxia
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Nervous system disorders
insomnia
|
24.0%
6/25 • adverse event data was collected for 2 years.
|
|
Musculoskeletal and connective tissue disorders
joint pain
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Musculoskeletal and connective tissue disorders
joint stiffness
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Investigations
lethargy
|
20.0%
5/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
lichenoid changes in mouth
|
20.0%
5/25 • adverse event data was collected for 2 years.
|
|
Investigations
lip lesion
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Nervous system disorders
loss of coordination
|
40.0%
10/25 • adverse event data was collected for 2 years.
|
|
Cardiac disorders
lower extremity edema
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Blood and lymphatic system disorders
lymphopenia
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Ear and labyrinth disorders
mastoiditis
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
mouth pain
|
24.0%
6/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
mouth sensitivity
|
40.0%
10/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
mouth sores
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
mucositis
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Musculoskeletal and connective tissue disorders
muscle pain
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Musculoskeletal and connective tissue disorders
myalgia
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
nausea
|
40.0%
10/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
oral lesions
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Investigations
catheter site pain
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Infections and infestations
parainfluenza
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Musculoskeletal and connective tissue disorders
pelvic pain
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Nervous system disorders
peripheral neuropathy
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
pleural effusions
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Infections and infestations
pneumonia
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Infections and infestations
pneumonitis
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Investigations
pruritus
|
40.0%
10/25 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
pulmonary infiltrates
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Infections and infestations
respiratory syncytial virus
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Skin and subcutaneous tissue disorders
rash
|
44.0%
11/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
rectal pain
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
respiratory distress
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
respiratory failure
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
respiratory insufficiency
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Infections and infestations
rhinovirus
|
20.0%
5/25 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
rhonchi
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Investigations
sepsis
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Respiratory, thoracic and mediastinal disorders
shortness of breath
|
36.0%
9/25 • adverse event data was collected for 2 years.
|
|
Investigations
sinus congestion
|
20.0%
5/25 • adverse event data was collected for 2 years.
|
|
Skin and subcutaneous tissue disorders
skin lesion
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
sore throat
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Infections and infestations
staph epi bacteremia
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Metabolism and nutrition disorders
steroid-induced diabetes
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
stool urgency
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Cardiac disorders
tachycardia
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Blood and lymphatic system disorders
thrombocytopenia
|
36.0%
9/25 • adverse event data was collected for 2 years.
|
|
Nervous system disorders
tremors
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Infections and infestations
upper respiratory infection
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Renal and urinary disorders
urinary frequency
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Renal and urinary disorders
urinary incontinence
|
12.0%
3/25 • adverse event data was collected for 2 years.
|
|
Infections and infestations
urinary tract infection
|
16.0%
4/25 • adverse event data was collected for 2 years.
|
|
Renal and urinary disorders
urinary urgency
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Reproductive system and breast disorders
vaginal irritation
|
8.0%
2/25 • adverse event data was collected for 2 years.
|
|
Gastrointestinal disorders
vomiting
|
32.0%
8/25 • adverse event data was collected for 2 years.
|
|
Blood and lymphatic system disorders
leukopenia
|
52.0%
13/25 • adverse event data was collected for 2 years.
|
|
Investigations
weakness
|
52.0%
13/25 • adverse event data was collected for 2 years.
|
|
Metabolism and nutrition disorders
weight loss
|
52.0%
13/25 • adverse event data was collected for 2 years.
|
Additional Information
Scott R. Solomon, MD (Principal Investigator)
Blood and Marrow Transplant Group of Georgia
Phone: 404-255-1930
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60