Trial Outcomes & Findings for Discontinuation or Continuation of Immunosuppressive Therapy in Participants With Chronic Graft Versus Host Disease (NCT NCT03483675)
NCT ID: NCT03483675
Last Updated: 2021-06-23
Results Overview
Descriptive summary of number of patients enrolled on the study (signed consent)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
22.9 months
Results posted on
2021-06-23
Participant Flow
4 participants were not randomized. 1 could not be reached and 3 had a GVHD flare after enrollment.
Participant milestones
| Measure |
Arm I (Discontinued IST)
Participants have their IST tapered and discontinued per the plan.
|
Arm II (Continued IST)
Participants continue to receive a fixed dose IST for an additional 9 months with no taper.
|
|---|---|---|
|
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
Discontinuation or Continuation of Immunosuppressive Therapy in Participants With Chronic Graft Versus Host Disease
Baseline characteristics by cohort
| Measure |
Arm I (Discontinued IST)
n=9 Participants
Participants have their IST tapered and discontinued per the plan.
|
Arm II (Continued IST)
n=8 Participants
Participants continue to receive a fixed dose IST for an additional 9 months with no taper.
|
Total
n=17 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=5 Participants
|
8 participants
n=7 Participants
|
17 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 22.9 monthsDescriptive summary of number of patients enrolled on the study (signed consent)
Outcome measures
| Measure |
Total Number of Participants
n=21 Participants
Total number of participants who signed consent
|
Arm II (Continued IST)
Participants continue to receive a fixed dose IST for an additional 9 months with no taper.
|
|---|---|---|
|
Feasibility of Enrolling Patients
|
21 Participants
|
—
|
SECONDARY outcome
Timeframe: 22.9 monthsDescriptive summary of percentage of patients randomized.
Outcome measures
| Measure |
Total Number of Participants
n=21 Participants
Total number of participants who signed consent
|
Arm II (Continued IST)
Participants continue to receive a fixed dose IST for an additional 9 months with no taper.
|
|---|---|---|
|
Feasibility of Randomizing Patients
|
17 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 12 months after randomizationRate of patients following study immunosuppressive therapy management based on study arm (standard taper or continuation of low dose).
Outcome measures
| Measure |
Total Number of Participants
n=9 Participants
Total number of participants who signed consent
|
Arm II (Continued IST)
n=8 Participants
Participants continue to receive a fixed dose IST for an additional 9 months with no taper.
|
|---|---|---|
|
Compliance With Treatment
|
7 Participants
|
8 Participants
|
SECONDARY outcome
Timeframe: Up to 12 months after randomizationCount of surveys completed by physicians and patients
Outcome measures
| Measure |
Total Number of Participants
n=9 Participants
Total number of participants who signed consent
|
Arm II (Continued IST)
n=8 Participants
Participants continue to receive a fixed dose IST for an additional 9 months with no taper.
|
|---|---|---|
|
Compliance With Data Collection
Total surveys collected
|
48 surveys
|
39 surveys
|
|
Compliance With Data Collection
Patient surveys missed
|
3 surveys
|
4 surveys
|
|
Compliance With Data Collection
Physician surveys missed
|
5 surveys
|
6 surveys
|
SECONDARY outcome
Timeframe: Up to 12 months after randomizationNew chronic graft versus host disease (GVHD) manifestations and/or worsening of existing manifestations
Outcome measures
| Measure |
Total Number of Participants
n=9 Participants
Total number of participants who signed consent
|
Arm II (Continued IST)
n=8 Participants
Participants continue to receive a fixed dose IST for an additional 9 months with no taper.
|
|---|---|---|
|
Graft Versus Host Disease Manifestations
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 12 months after randomizationIncidence of relapse of primary disease
Outcome measures
| Measure |
Total Number of Participants
n=9 Participants
Total number of participants who signed consent
|
Arm II (Continued IST)
n=8 Participants
Participants continue to receive a fixed dose IST for an additional 9 months with no taper.
|
|---|---|---|
|
Recurrent Malignancy
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 12 months after randomizationIncidence of grade \>= 3 infections
Outcome measures
| Measure |
Total Number of Participants
n=9 Participants
Total number of participants who signed consent
|
Arm II (Continued IST)
n=8 Participants
Participants continue to receive a fixed dose IST for an additional 9 months with no taper.
|
|---|---|---|
|
Incidence of Grade >= 3 Infections
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 12 months after randomizationIncidence of grade \>= 3 organ toxicity
Outcome measures
| Measure |
Total Number of Participants
n=9 Participants
Total number of participants who signed consent
|
Arm II (Continued IST)
n=8 Participants
Participants continue to receive a fixed dose IST for an additional 9 months with no taper.
|
|---|---|---|
|
Incidence of Grade >= 3 Organ Toxicity
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 12 months after randomizationPopulation: All participants were local, no data were collected for this outcome measure.
Enrollment rate of participants who are not local. Participants who are local defined as those who had all or some study visits completed at the cancer center.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 12 months after randomizationPopulation: All participants were local, no data were collected for this outcome measure.
Participants who are local defined as those who had all or some study visits completed at the cancer center.
Outcome measures
Outcome data not reported
Adverse Events
Arm I (Discontinued IST)
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Arm II (Continued IST)
Serious events: 2 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Arm I (Discontinued IST)
n=9 participants at risk
Participants have their IST tapered and discontinued per the plan.
Immunosuppressive Therapy: Discontinued IST
Survey Administration: Ancillary studies
|
Arm II (Continued IST)
n=8 participants at risk
Participants continue to receive a fixed dose IST for an additional 9 months with no taper.
Immunosuppressive Therapy: Continued IST
Survey Administration: Ancillary studies
|
|---|---|---|
|
Infections and infestations
Gangrene
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Cardiac disorders
heart failure
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Cardiac disorders
myocardial infarction
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Infections and infestations
osteomyelitis
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Vascular disorders
peripheral ischemia
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Eye disorders
retinal detachment
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Cardiac disorders
stent placement
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Infections and infestations
wound complications
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
Other adverse events
| Measure |
Arm I (Discontinued IST)
n=9 participants at risk
Participants have their IST tapered and discontinued per the plan.
Immunosuppressive Therapy: Discontinued IST
Survey Administration: Ancillary studies
|
Arm II (Continued IST)
n=8 participants at risk
Participants continue to receive a fixed dose IST for an additional 9 months with no taper.
Immunosuppressive Therapy: Continued IST
Survey Administration: Ancillary studies
|
|---|---|---|
|
Renal and urinary disorders
acute kidney injury
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
allergic rhinitis
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
anal fissures
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Musculoskeletal and connective tissue disorders
arthralgia
|
22.2%
2/9 • Number of events 2 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
|
Eye disorders
corneal ulcer
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Psychiatric disorders
depression
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
diarrhea
|
11.1%
1/9 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
General disorders
dizziness
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
dyspnea
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Gastrointestinal disorders
dysphagia
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
eczema
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Renal and urinary disorders
elevated creatinine
|
11.1%
1/9 • Number of events 2 • 1 year
|
0.00%
0/8 • 1 year
|
|
Nervous system disorders
essential tremor
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
General disorders
fall
|
22.2%
2/9 • Number of events 2 • 1 year
|
0.00%
0/8 • 1 year
|
|
General disorders
fatigue
|
100.0%
2/2 • Number of events 2 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
fecal incontinence
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
General disorders
fever
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
flatulence
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Eye disorders
floaters, eye
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Musculoskeletal and connective tissue disorders
fracture, clavicle
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Gastrointestinal disorders
gastroenteritis
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Gastrointestinal disorders
GI upset, drug induced
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 2 • 1 year
|
|
General disorders
headache
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Gastrointestinal disorders
hernia, umbilical
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Gastrointestinal disorders
hypertriglyceridemia
|
11.1%
1/9 • Number of events 2 • 1 year
|
0.00%
0/8 • 1 year
|
|
General disorders
insomnia
|
0.00%
0/9 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
|
Blood and lymphatic system disorders
iron deficiency anemia
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Injury, poisoning and procedural complications
IV contrast reaction
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Immune system disorders
leukopenia
|
22.2%
2/9 • Number of events 2 • 1 year
|
0.00%
0/8 • 1 year
|
|
General disorders
lightheadedness
|
11.1%
1/9 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Infections and infestations
Mucous cyst (toe)
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Gastrointestinal disorders
nausea
|
33.3%
3/9 • Number of events 4 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Nervous system disorders
neuropathy
|
11.1%
1/9 • Number of events 1 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
|
Endocrine disorders
prostatitis, presumed
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Skin and subcutaneous tissue disorders
skin, ulcer or lesion
|
11.1%
1/9 • Number of events 1 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
|
Skin and subcutaneous tissue disorders
skin, erythematous bumps, macules
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 2 • 1 year
|
|
Skin and subcutaneous tissue disorders
skin, dermatitis, pruritis
|
11.1%
1/9 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
squamous cell carcinoma
|
22.2%
2/9 • Number of events 2 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
|
Musculoskeletal and connective tissue disorders
stiffness, muscular
|
22.2%
2/9 • Number of events 2 • 1 year
|
0.00%
0/8 • 1 year
|
|
Renal and urinary disorders
urination, difficult
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
General disorders
weight gain or loss
|
11.1%
1/9 • Number of events 1 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
constipation
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
lower respiratory infection
|
44.4%
4/9 • Number of events 4 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Musculoskeletal and connective tissue disorders
myalgia
|
22.2%
2/9 • Number of events 2 • 1 year
|
37.5%
3/8 • Number of events 3 • 1 year
|
|
Reproductive system and breast disorders
phimosis
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Nervous system disorders
seizure
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Blood and lymphatic system disorders
thrombocytosis
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
|
Infections and infestations
thrush
|
0.00%
0/9 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
upper respiratory infection
|
44.4%
4/9 • Number of events 8 • 1 year
|
50.0%
4/8 • Number of events 7 • 1 year
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place