Acute Gastrointestinal Graft vs Host Disease
Conditions
Keywords
BDP, orBec, GVHD, beclomethasone dipropionate, bone marrow transplant, hematopoietic cell transplant, HCT, stem cell transplant, marrow transplant, SCT, beclomethasone 17,21-dipropionate
Brief summary
Use of an oral topically-active glucocorticoid with limited side effects will control the gastrointestinal inflammatory process of GVHD and minimize glucocorticoid exposure.
Interventions
DRUGoral beclomethasone 17,21-dipropionate
Two tablets QID for 50 days
DRUGPlacebo
Two tablets QID for 50 days
Sponsors
Soligenix
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Receipt of allogeneic hematopoietic cell transplant * Diagnosis of GI graft vs. host disease (GVHD) * No GI infection * Must be able to swallow tablets * Must be able to read and understand informed consent * Adequate birth control methods for the duration of the study
Exclusion criteria
* Significant Skin GVHD * Liver GVHD * Persistent vomiting * HIV positive * Pregnancy/lactation
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Proportion of Subjects With GVHD Treatment Failure | Day 80 | The primary endpoint is the occurrence (yes, no) during the 80-day study period of GVHD treatment failure defined as use of prednisone or equivalent IV corticosteroids at doses higher than stated in the protocol, or use of any additional other glucocorticoid (including unblinded BDP) or addition of other immunosuppressant medications, in response to uncontrolled signs or symptoms of GVHD |
Secondary
| Measure | Time frame |
|---|---|
| Cumulative Exposure to Prednisone | Day 80 |
| Survival Status | Day 200 |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Active oral beclomethasone 17,21-dipropionate (BDP) | 70 |
| Placebo | 70 |
| Total | 140 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 1 | 1 |
| Overall Study | Death | 4 | 4 |
| Overall Study | Lost to Follow-up | 1 | 1 |
| Overall Study | Physician Decision | 1 | 2 |
| Overall Study | Protocol Violation | 1 | 0 |
| Overall Study | Withdrawal by Subject | 8 | 9 |
Baseline characteristics
| Characteristic | Active | Placebo | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 8 Participants | 4 Participants | 12 Participants |
| Age, Categorical Between 18 and 65 years | 62 Participants | 66 Participants | 128 Participants |
| Sex: Female, Male Female | 34 Participants | 36 Participants | 70 Participants |
| Sex: Female, Male Male | 36 Participants | 34 Participants | 70 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 70 / 70 | 70 / 70 |
| serious Total, serious adverse events | 32 / 70 | 28 / 70 |
Outcome results
Primary
The Proportion of Subjects With GVHD Treatment Failure
The primary endpoint is the occurrence (yes, no) during the 80-day study period of GVHD treatment failure defined as use of prednisone or equivalent IV corticosteroids at doses higher than stated in the protocol, or use of any additional other glucocorticoid (including unblinded BDP) or addition of other immunosuppressant medications, in response to uncontrolled signs or symptoms of GVHD
Time frame: Day 80
Population: Data was not analyzed as the study was terminated due to futility
Secondary
Cumulative Exposure to Prednisone
Time frame: Day 80
Secondary
Survival Status
Time frame: Day 200