Trial Outcomes & Findings for Imatinib Mesylate to Treat Skin Changes in Patients With Chronic Graft-Versus-Host Disease (NCT NCT00702689)
NCT ID: NCT00702689
Last Updated: 2020-03-30
Results Overview
A change in ROM is 25% or greater from baseline. A partial response required improvement in 25% or more in ROM. Progression required 25% or greater loss of ROM.Patients with negative values in the Table are those who lost ROM. Percent improvement in ROM for 1-3 target joints. For patients with \>1 target joint, the average ROM improvement was calculated. The average percentage change in ROM deficit from baseline to 6 months was obtained based on the number of degrees of ROM change (6 months)/total ROM deficit (baseline) at each joint.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
6 months
Results posted on
2020-03-30
Participant Flow
Participant milestones
| Measure |
Imatinib Mesylate in Patients With cGVHD
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
14
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
Imatinib Mesylate in Patients With cGVHD
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
4
|
|
Overall Study
Recurrent malignancy
|
1
|
|
Overall Study
Adverse Event
|
1
|
Baseline Characteristics
Imatinib Mesylate to Treat Skin Changes in Patients With Chronic Graft-Versus-Host Disease
Baseline characteristics by cohort
| Measure |
Imatinib Mesylate in Patients With cGVHD
n=20 Participants
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
18 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
42.59 years
STANDARD_DEVIATION 17.49 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
17 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
20 Participants
n=5 Participants
|
|
Chronic Graft Versus Host Disease (cGVHD) category
Overlap
|
5 percentage of participants
n=5 Participants
|
|
Chronic Graft Versus Host Disease (cGVHD) category
Classic
|
95 percentage of participants
n=5 Participants
|
|
Chronic Graft Versus Host Disease (cGVHD) category
Late acute
|
0 percentage of participants
n=5 Participants
|
|
Chronic Graft Versus Host Disease presentation
De novo
|
30 Percentage of participants
n=5 Participants
|
|
Chronic Graft Versus Host Disease presentation
Quiescent
|
20 Percentage of participants
n=5 Participants
|
|
Chronic Graft Versus Host Disease presentation
Progressive
|
50 Percentage of participants
n=5 Participants
|
|
Global National Institutes of Health Graft Versus Host Disease (GVHD) score
Mild (1)
|
0 Percentage of participants
n=5 Participants
|
|
Global National Institutes of Health Graft Versus Host Disease (GVHD) score
Moderate (2)
|
0 Percentage of participants
n=5 Participants
|
|
Global National Institutes of Health Graft Versus Host Disease (GVHD) score
Severe (3)
|
100 Percentage of participants
n=5 Participants
|
|
Donor Source
Bone marrow
|
10 Percentage of participants
n=5 Participants
|
|
Donor Source
Peripheral blood
|
90 Percentage of participants
n=5 Participants
|
|
Donor Source
Cord
|
0 Percentage of participants
n=5 Participants
|
|
Donor Match (6/6)
|
90 Percentage of participants
n=5 Participants
|
|
Months from Chronic Graft Versus Host Disease (cGVHD) diagnosis
|
39.85 Months
n=5 Participants
|
|
Months from transplant
|
55.4 Months
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
Pred/tacro
|
2 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
Pred
|
1 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
MPred/MTX
|
1 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
Pred/siro
|
2 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
Pred/siro/tacro
|
1 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
MPred/tacro/siro/MMF
|
1 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
Pred/siro/MMF
|
2 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
Tacro
|
1 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
Pred/tacro/siro
|
1 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
Tacro/MMF
|
3 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
Pred/MMF
|
1 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
Siro/MMF
|
1 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
MPred/tacro/MMF
|
1 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
Siro
|
1 Participants
n=5 Participants
|
|
Concomitant Immunosuppressive Medication (ISM)
None
|
1 Participants
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 1
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 2
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 3
|
2 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 4
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 5
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 6
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 7
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 8
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 9
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 10
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 11
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 12
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
*Patient 13
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 14
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 15
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 16
|
1 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 17
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 18
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 19
|
3 joints
n=5 Participants
|
|
Assessable Joints at Baseline
Patient 20
|
3 joints
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 1
|
1.28 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 2
|
1.14 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 3
|
.75 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 4
|
1.0 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 5
|
1.48 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 6
|
1.86 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 7
|
1.90 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 8
|
1.57 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 9
|
1.71 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 10
|
1.43 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 11
|
1.75 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 12
|
1.43 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 13
|
1.38 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 14
|
1.29 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 15
|
1.43 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 16
|
.86 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 17
|
1.13 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 18
|
1.57 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 19
|
1.14 scores on a scale
n=5 Participants
|
|
Average National Institutes of Health Chronic Graft Versus Host Disease (cGVHD) Score at Baseline
Patient 20
|
2.00 scores on a scale
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 1
|
5 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 2
|
4 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 3
|
3 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 4
|
4 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 5
|
6 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 6
|
7 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 7
|
7 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 8
|
6 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 9
|
7 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 10
|
5 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 11
|
7 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 12
|
6 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 13
|
6 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 14
|
5 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 15
|
6 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 16
|
2 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 17
|
4 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 18
|
4 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 19
|
4 affected organs
n=5 Participants
|
|
Measure of Number of Organs with Graft Versus Host Disease (GVHD)
Patient 20
|
7 affected organs
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 1
|
57.15 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 2
|
2.7 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 3
|
6.3 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 4
|
0.18 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 5
|
8.1 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 6
|
59.4 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 7
|
29.7 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 8
|
15.3 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 9
|
3.33 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 10
|
0 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 11
|
82.08 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 12
|
12.6 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 13
|
1.8 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 14
|
5.4 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 15
|
49.77 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 16
|
0 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 17
|
84.24 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 18
|
15.84 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 19
|
9.45 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Moveable Sclerosis at Baseline
Patient 20
|
0 Percent BSA moveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 1
|
7.56 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 2
|
56.7 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 3
|
36.9 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 4
|
19.98 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 5
|
77.94 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 6
|
0 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 7
|
32.4 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 8
|
36.9 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 9
|
9 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 10
|
8.28 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 11
|
0 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 12
|
8.64 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 13
|
71.1 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 14
|
31.5 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 15
|
10.8 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 16
|
9.54 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 17
|
0 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 18
|
4.5 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 19
|
11.7 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Percent Body Surface Area (BSA) Nonmoveable Sclerosis at Baseline
Patient 20
|
23.4 Percent BSA nonmoveable sclerosis
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 1
|
37 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 2
|
56 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 3
|
73 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 4
|
7 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 5
|
34 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 6
|
47 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 7
|
61 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 8
|
35 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 9
|
42 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 10
|
-7 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 11
|
56 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 12
|
37 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 13
|
27 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 14
|
32 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 15
|
22 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 16
|
71 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 17
|
64 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 18
|
54 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 19
|
33 ROM Percent of Predicted
n=5 Participants
|
|
Baseline Range of Motion (ROM) Percent (of Predicted)
Patient 20
|
-5 ROM Percent of Predicted
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsA change in ROM is 25% or greater from baseline. A partial response required improvement in 25% or more in ROM. Progression required 25% or greater loss of ROM.Patients with negative values in the Table are those who lost ROM. Percent improvement in ROM for 1-3 target joints. For patients with \>1 target joint, the average ROM improvement was calculated. The average percentage change in ROM deficit from baseline to 6 months was obtained based on the number of degrees of ROM change (6 months)/total ROM deficit (baseline) at each joint.
Outcome measures
| Measure |
Imatinib Mesylate in Patients With cGVHD
n=14 Participants
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt 2 6mo response% change in deficit from baseline
|
94 Percent change from baseline
|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt 7 6mo response% change in deficit from baseline
|
35 Percent change from baseline
|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt 8 6mo response% change in deficit from baseline
|
16 Percent change from baseline
|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt10 6mo response% change in deficit from baseline
|
21 Percent change from baseline
|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt12 6mo response% change in deficit from baseline
|
16 Percent change from baseline
|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt13 6mo response% change in deficit from baseline
|
61 Percent change from baseline
|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt14 6mo response% change in deficit from baseline
|
27 Percent change from baseline
|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt15 6mo response% change in deficit from baseline
|
22 Percent change from baseline
|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt16 6mo response% change in deficit from baseline
|
3 Percent change from baseline
|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt17 6mo response% change in deficit from baseline
|
-25 Percent change from baseline
|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt18 6mo response% change in deficit from baseline
|
-2 Percent change from baseline
|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt19 6mo response% change in deficit from baseline
|
31 Percent change from baseline
|
|
Percent Change in Absolute Range of Motion (ROM) From Baseline to 6 Months
Pt20 6mo response% change in deficit from baseline
|
15 Percent change from baseline
|
PRIMARY outcome
Timeframe: 6 monthsProgressive disease is defined as joint ROM: decrease of \>25% in composite ROM score on 2 consecutive evaluations at least 2 weeks apart, but not greater than 4 weeks apart or steroid pulse: \>1 steroid pulse per 3 month period if administered for sclerotic-type chronic graft versus host disease (ScGVHD). Response is joint ROM: increase of \>25% in composite ROM score. Maximal response is a response with no further improvement over 2 sequential 3-month evaluations. Stable disease does not meet the criteria for progression, response, or maximal response.
Outcome measures
| Measure |
Imatinib Mesylate in Patients With cGVHD
n=14 Participants
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Primary Range of Motion (ROM) Response
Partial Response
|
5 participants
|
|
Primary Range of Motion (ROM) Response
Stable Disease
|
7 participants
|
|
Primary Range of Motion (ROM) Response
Progressive Disease
|
2 participants
|
SECONDARY outcome
Timeframe: Date treatment consent signed to date off study, approximately, 41 months, 27 daysHere is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.
Outcome measures
| Measure |
Imatinib Mesylate in Patients With cGVHD
n=20 Participants
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Number of Participants With Adverse Events
|
20 Participants
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: This outcome is the average percentage change in ROM deficit among 13 evaluable patients based on each patients baseline range of motion deficit compared to his/her ROM deficit at 6 months.
One or more joints were assessed for ROM deficit by a physiatrist with expertise in graft versus host disease and joint ROM.
Outcome measures
| Measure |
Imatinib Mesylate in Patients With cGVHD
n=13 Participants
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Average Percentage Change in Range of Motion (ROM) Deficit
|
24.2 Percent change
Interval 15.5 to 30.5
|
SECONDARY outcome
Timeframe: Baseline and 6 MonthsTotal skin score was graded by the National Institutes of Health Consensus Criteria. Skin score was calculated by dividing the total score by seven domains (skin, eye, oral, joint, gastrointestinal, hepatic, pulmonary) in men and 8 domains in women (previous domains noted plus gynecologic). Total skin score is a percentage of body surface area (BSA) involvement (range 0-100%). It was calculated from the sum of moveable body surface BSA and non-moveable BSA. Higher numbers = greater body surface area affected.
Outcome measures
| Measure |
Imatinib Mesylate in Patients With cGVHD
n=14 Participants
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Total Skin Score at Baseline and 6 Months
Patient # 2 - Baseline
|
66.6 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 2 - 6 months
|
54 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 3 - Baseline
|
43.38 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 3 - 6 months
|
NA units on a scale
This patient had progressive disease and was taken off study prior to 6 month endpoint. Hence, no data points at 6 months.
|
|
Total Skin Score at Baseline and 6 Months
Patient # 7 - Baseline
|
66.24 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 7 - 6 months
|
55.53 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 8 - Baseline
|
53.1 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 8 - 6 months
|
55.26 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 10 - Baseline
|
10.8 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 10 - 6 months
|
6.12 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 12 - Baseline
|
21.24 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 12 - 6 months
|
30.06 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 13 - Baseline
|
79.2 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 13 - 6 months
|
62.28 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 14 - Baseline
|
39.96 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 14 - 6 months
|
40.5 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 15 - Baseline
|
71.46 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 15 - 6 months
|
61.83 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 16 - Baseline
|
9.54 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 16 - 6 months
|
8.1 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 17 - Baseline
|
84.96 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 17 - 6 months
|
85.11 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 18 - Baseline
|
26.64 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 18 - 6 months
|
24.3 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 19 - Baseline
|
21.15 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 19 - 6 months
|
37.8 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 20 - Baseline
|
23.4 units on a scale
|
|
Total Skin Score at Baseline and 6 Months
Patient # 20 - 6 months
|
25.2 units on a scale
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsThe provider global rating is a physician impression of severity of cGVHD symptoms from a scale of zero (no symptoms) to 10 (most severe GVHD symptoms possible).
Outcome measures
| Measure |
Imatinib Mesylate in Patients With cGVHD
n=14 Participants
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #2 at Baseline
|
5 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #2 at 6 Months
|
4 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #3 at Baseline
|
3 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #3 at 6 Months
|
NA Provider Global Rating Score
This patient had progressive disease and was taken off study prior to 6 month endpoint. Hence, no data points at 6 months.
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #7 at Baseline
|
6 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #7 at 6 Months
|
8 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #8 at Baseline
|
6 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #8 at 6 Months
|
7 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #10 at Baseline
|
5 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #10 at 6 Months
|
4 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #12 at Baseline
|
6 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #12 at 6 Months
|
7 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #13 at 6 Months
|
4 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #14 at Baseline
|
7 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #14 at 6 Months
|
6 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient # 15 at Baseline
|
7 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #15 at 6 Months
|
6 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #16 at Baseline
|
5 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #16 at 6 Months
|
4 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #17 at Baseline
|
6 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #17 at 6 months
|
8 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #18 at Baseline
|
8 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #18 at 6 Months
|
8 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #19 at Baseline
|
8 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #19 at 6 Months
|
5 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #20 at Baseline
|
8 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #20 at 6 Months
|
8 Provider Global Rating Score
|
|
Total Chronic Graft Versus Host Disease (cGVHD) Provider Global Rating Score at Baseline and 6 Months
Patient #13 at Baseline
|
6 Provider Global Rating Score
|
SECONDARY outcome
Timeframe: Baseline and 6 MonthsLung function was graded by the National Institutes of Health Chronic Graft Versus Host Disease organ response criteria. The Lung function score = forced expiratory volume 1 (FEV1) score + carbon monoxide diffusing capacity (DLCO) score, with a possible range of 2 (better outcome)-12 (worst outcome). The percent predicted FEV1 and DLCO (adjusted for hematocrit but not alveolar volume) should be converted to a numeric score as follows: \>80% =1; 70-79% = 2; 60-69% = 3; 50-59% = 4; 40-49% = 5; \<40% = 6.
Outcome measures
| Measure |
Imatinib Mesylate in Patients With cGVHD
n=14 Participants
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Lung Function Score at Baseline and 6 Months
Patient #2 at Baseline
|
2 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #2 at 6 Months
|
2 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #3 at Baseline
|
9 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #3 at 6 Months
|
NA units on a scale
This patient had progressive disease and was taken off study prior to 6 month endpoint. Hence, no data points at 6 months.
|
|
Lung Function Score at Baseline and 6 Months
Patient #7 at Baseline
|
3 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #7 at 6 Months
|
8 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #8 at Baseline
|
5 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #8 at 6 Months
|
6 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #10 at Baseline
|
3 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #10 at 6 Months
|
3 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #12 at Baseline
|
4 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #12 at 6 Months
|
5 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #13 at Baseline
|
4 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #13 at 6 Months
|
4 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #14 at Baseline
|
5 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #14 at 6 Months
|
5 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient # 15 at Baseline
|
7 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #15 at 6 Months
|
6 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #16 at Baseline
|
3 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #16 at 6 Months
|
2 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #17 at Baseline
|
5 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #17 at 6 months
|
6 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #18 at Baseline
|
8 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #18 at 6 Months
|
8 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #19 at Baseline
|
9 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #19 at 6 Months
|
9 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #20 at Baseline
|
2 units on a scale
|
|
Lung Function Score at Baseline and 6 Months
Patient #20 at 6 Months
|
2 units on a scale
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: Pred:prednisone; tacro:tacrolimus; MPred:methylprednisolone; siro:sirolimus; and MMF:mycophenolate mofetil
Change in immunosuppression was defined by an increase or decrease in steroid use form baseline.
Outcome measures
| Measure |
Imatinib Mesylate in Patients With cGVHD
n=14 Participants
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Change in Immunosuppression
↓ Pred 20 mg everyday (qd )to 5 mg every other day
|
1 participants
|
|
Change in Immunosuppression
↓ MPred 16 mg every other day(qod) to 4 mg qod
|
1 participants
|
|
Change in Immunosuppression
↓ Pred:24mg every day(qd) to 20mg qd
|
1 participants
|
|
Change in Immunosuppression
No change
|
5 participants
|
|
Change in Immunosuppression
↓ Tacro 2mg every am 1.5mg every pm to .5mg bid
|
1 participants
|
|
Change in Immunosuppression
Pred↓ 25mg qd to 15mg qd;Tacro↓ 2mg bid to 1mg bid
|
1 participants
|
|
Change in Immunosuppression
Pred↓ 2.5mg qd to 2.5mg every other day
|
1 participants
|
|
Change in Immunosuppression
↓ Siro:2mg qd to 1 mg qd
|
1 participants
|
|
Change in Immunosuppression
Pred wean then ↑ 10 12.5mg bid;Tacro↑1.0 to 1.5bid
|
1 participants
|
|
Change in Immunosuppression
MMF↓ 1g/bid to discontinued
|
1 participants
|
Adverse Events
Imatinib Mesylate in Patients With cGVHD
Serious events: 5 serious events
Other events: 20 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Imatinib Mesylate in Patients With cGVHD
n=20 participants at risk
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
10.0%
2/20 • Number of events 3 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
General disorders
Edema::head and neck
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
General disorders
Edema: limb
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
General disorders
Edema:: trunk/genital
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Vascular disorders
Hypertension
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Infections and infestations
Infection with unknown ANC::Lung (pneumonia)
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness, generalized or specific area (not due to neuropathy)::Whole body/generalized
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Gastrointestinal disorders
Nausea
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Nervous system disorders
Pain::Head/headache
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion (non-malignant)
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper Respiratory - Other (Specify, Pulmonary edema)
|
10.0%
2/20 • Number of events 3 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Gastrointestinal disorders
Vomiting
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
Other adverse events
| Measure |
Imatinib Mesylate in Patients With cGVHD
n=20 participants at risk
Cohort 1 - Pts 1-8:Adults: 400mg imatinib mesylate daily; Children: 260mg/m\^2 daily (400mg maximum), followed by dose de-escalation for adverse events.
Cohort 2 - Pts 9-20:Adults - 100 mg oral dose daily (increase to 200 mg daily after 28 days if well tolerated). Children - 65 mg/m\^2 oral dose daily (increase to 130 mg/m\^2 daily after 28 days if well tolerated)
|
|---|---|
|
Investigations
ALT, SGPT (serum glutamic pyruvic transaminase)
|
20.0%
4/20 • Number of events 8 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Investigations
AST, SGOT(serum glutamic oxaloacetic transaminase)
|
25.0%
5/20 • Number of events 10 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Metabolism and nutrition disorders
Albumin, serum-low (hypoalbuminemia)
|
20.0%
4/20 • Number of events 6 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Investigations
Alkaline phosphatase
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Immune system disorders
Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip)
|
15.0%
3/20 • Number of events 3 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Metabolism and nutrition disorders
Anorexia
|
5.0%
1/20 • Number of events 4 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Injury, poisoning and procedural complications
Bruising (in absence of Grade 3 or 4 thrombocytopenia)
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Investigations
CPK (creatine phosphokinase)
|
25.0%
5/20 • Number of events 11 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Metabolism and nutrition disorders
Calcium, serum-high (hypercalcemia)
|
15.0%
3/20 • Number of events 5 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Gastrointestinal disorders
Constipation
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
General disorders
Constitutional Symptoms-Other (Specify, cold intolerance; sleep walking)
|
10.0%
2/20 • Number of events 2 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
4/20 • Number of events 5 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Investigations
Creatinine
|
10.0%
2/20 • Number of events 8 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Skin and subcutaneous tissue disorders
Dermatology/Skin-Other (Specify, intermt. bilateral hands & L. hip)
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Gastrointestinal disorders
Diarrhea
|
30.0%
6/20 • Number of events 19 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Nervous system disorders
Dizziness
|
10.0%
2/20 • Number of events 2 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
10.0%
2/20 • Number of events 3 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
General disorders
Edema::head and neck
|
15.0%
3/20 • Number of events 6 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
General disorders
Edema: limb
|
15.0%
3/20 • Number of events 11 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
General disorders
Fatigue (asthenia, lethargy, malaise)
|
45.0%
9/20 • Number of events 22 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
General disorders
Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)
|
10.0%
2/20 • Number of events 2 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
General disorders
Flu-like symptoms
|
10.0%
2/20 • Number of events 2 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Vascular disorders
Flushing
|
5.0%
1/20 • Number of events 2 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Metabolism and nutrition disorders
Glucose, serum-low (hyperglycemia)
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Gastrointestinal disorders
Heartburn/dyspepsia
|
5.0%
1/20 • Number of events 2 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Investigations
Hemoglobin
|
30.0%
6/20 • Number of events 13 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Vascular disorders
Hypertension
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils::otitis externa
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Psychiatric disorders
Insomnia
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Investigations
Leukocytes (total WBC)
|
5.0%
1/20 • Number of events 2 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Nervous system disorders
leukoencephalopathy (radiographic findings)
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Metabolism and nutrition disorders
Magnesium, serum-high (hypermagnesemia)
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Metabolism and nutrition disorders
Magnesium, serum-low (hypomagnesemia)
|
5.0%
1/20 • Number of events 3 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Psychiatric disorders
Mood alteration::Depression
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness, generalized or specific area (not due to neuropathy)::Whole body/generalized
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal/Soft tissue - Other (Specify, hand & jaw cramps; hand & jaw cramps, intermittent
|
5.0%
1/20 • Number of events 2 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal cavity/paranasal sinus reactions
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Gastrointestinal disorders
Nausea
|
50.0%
10/20 • Number of events 30 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Investigations
Neutrophils/granulocytes (ANC/AGC)
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Musculoskeletal and connective tissue disorders
Pain::Bone
|
5.0%
1/20 • Number of events 2 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Reproductive system and breast disorders
Pain::Breast
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Ear and labyrinth disorders
Pain::External ear
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Nervous system disorders
Pain::Head/headache
|
25.0%
5/20 • Number of events 11 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Musculoskeletal and connective tissue disorders
Pain::Joint
|
5.0%
1/20 • Number of events 3 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Musculoskeletal and connective tissue disorders
Pain::Muscle
|
30.0%
6/20 • Number of events 14 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Skin and subcutaneous tissue disorders
Pain::Skin
|
5.0%
1/20 • Number of events 1 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Metabolism and nutrition disorders
Phosphate, serum-low (hypophosphatemia)
|
55.0%
11/20 • Number of events 26 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Investigations
Platelets
|
5.0%
1/20 • Number of events 2 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Metabolism and nutrition disorders
Potassium, serum-high (hyperkalemia)
|
15.0%
3/20 • Number of events 3 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Metabolism and nutrition disorders
Potassium, serum-low (hypokalemia)
|
5.0%
1/20 • Number of events 2 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Skin and subcutaneous tissue disorders
Rash/desquamation
|
15.0%
3/20 • Number of events 5 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Metabolism and nutrition disorders
Sodium, serum-low (hyponatremia)
|
10.0%
2/20 • Number of events 4 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Ear and labyrinth disorders
Tinnitus
|
20.0%
4/20 • Number of events 6 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
|
Gastrointestinal disorders
Vomiting
|
20.0%
4/20 • Number of events 5 • Date treatment consent signed to date off study, approximately, 41 months, 27 days.
|
Additional Information
Edward Cowen, M.D.
National Institute of Arthritis and Musculoskeletal and Skin Diseases
Phone: 301-827-2328
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place