Trial Outcomes & Findings for Study of orBec® as Monotherapy in the Treatment of Patients With Upper GI Symptoms Caused by Chronic Graft Versus Host Disease (GVHD) (NCT NCT01925950)
NCT ID: NCT01925950
Last Updated: 2018-11-01
Results Overview
Patients who achieved a Complete Response (CR) of their GI GVHD Symptoms during the Part 1, 16 week treatment period and who remained a CR at the end of the 16 week treatment period were to be eligible to continue into Part 2 of the study. All others were to discontinue. GI GVHD was assessed using a composite score based on the symptoms of satiety, nausea/vomiting, and anorexia. Each symptom was scored on a scale of 0-3, such that the minimum score = 0 and the maximum score = 9. At entry, all subjects must have a score of ≥ 3. A CR will be defined as a composite score of 0.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
2 participants
Primary outcome timeframe
16 weeks
Results posted on
2018-11-01
Participant Flow
Participant milestones
| Measure |
Placebo
Matching placebo was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug.
Part 1: 2 mg of placebo 4 times per day for up to 16 weeks.
Part 2: Patients who achieved a Complete Response (CR) on Part 1 were to taper study drug according to the following schedule:
Weeks 17-24: 2 mg placebo 3 times per day, up to 8 weeks; Weeks 25-32: 2 mg placebo 2 times per day, up to 8 weeks; Weeks 33-48: 2 mg placebo daily, up to 16 weeks.
|
orBec
orBec, oral beclomethasone dipropionate, was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug.
Part 1: 2 mg of orBec 4 times per day for up to 16 weeks.
Part 2: Patients who achieved a Complete Response (CR) on Part 1 were to taper study drug according to the following schedule:
Weeks 17-24: 2 mg orBec 3 times per day, up to 8 weeks; Weeks 25-32: 2 mg orBec 2 times per day, up to 8 weeks; Weeks 33-48: 2 mg orBec daily, up to 16 weeks.
|
|---|---|---|
|
Part 1
STARTED
|
1
|
1
|
|
Part 1
COMPLETED
|
1
|
1
|
|
Part 1
NOT COMPLETED
|
0
|
0
|
|
Part 2
STARTED
|
1
|
0
|
|
Part 2
COMPLETED
|
0
|
0
|
|
Part 2
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Placebo
Matching placebo was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug.
Part 1: 2 mg of placebo 4 times per day for up to 16 weeks.
Part 2: Patients who achieved a Complete Response (CR) on Part 1 were to taper study drug according to the following schedule:
Weeks 17-24: 2 mg placebo 3 times per day, up to 8 weeks; Weeks 25-32: 2 mg placebo 2 times per day, up to 8 weeks; Weeks 33-48: 2 mg placebo daily, up to 16 weeks.
|
orBec
orBec, oral beclomethasone dipropionate, was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug.
Part 1: 2 mg of orBec 4 times per day for up to 16 weeks.
Part 2: Patients who achieved a Complete Response (CR) on Part 1 were to taper study drug according to the following schedule:
Weeks 17-24: 2 mg orBec 3 times per day, up to 8 weeks; Weeks 25-32: 2 mg orBec 2 times per day, up to 8 weeks; Weeks 33-48: 2 mg orBec daily, up to 16 weeks.
|
|---|---|---|
|
Part 2
Withdrawal by Subject
|
1
|
0
|
Baseline Characteristics
Study of orBec® as Monotherapy in the Treatment of Patients With Upper GI Symptoms Caused by Chronic Graft Versus Host Disease (GVHD)
Baseline characteristics by cohort
| Measure |
Placebo
n=1 Participants
Control
Placebo
|
orBec
n=1 Participants
Investigational drug
orBec
|
Total
n=2 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
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 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
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 16 weeksPatients who achieved a Complete Response (CR) of their GI GVHD Symptoms during the Part 1, 16 week treatment period and who remained a CR at the end of the 16 week treatment period were to be eligible to continue into Part 2 of the study. All others were to discontinue. GI GVHD was assessed using a composite score based on the symptoms of satiety, nausea/vomiting, and anorexia. Each symptom was scored on a scale of 0-3, such that the minimum score = 0 and the maximum score = 9. At entry, all subjects must have a score of ≥ 3. A CR will be defined as a composite score of 0.
Outcome measures
| Measure |
Placebo
n=1 Participants
Matching placebo was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug.
2 mg of placebo 4 times per day for up to 16 weeks.
|
orBec
n=1 Participants
orBec, oral beclomethasone dipropionate, was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug.
2 mg of orBec 4 times per day for up to 16 weeks.
|
|---|---|---|
|
Gastrointestinal (GI) Graft-vs-Host Disease (GVHD) Symptoms
|
1 Participants
|
0 Participants
|
Adverse Events
Placebo
Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths
orBec
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=1 participants at risk
Matching placebo was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug.
Part 1: 2 mg of placebo 4 times per day for up to 16 weeks.
Part 2: Patients who achieved a Complete Response (CR) on Part 1 were to taper study drug according to the following schedule:
Weeks 17-24: 2 mg placebo 3 times per day, up to 8 weeks; Weeks 25-32: 2 mg placebo 2 times per day, up to 8 weeks; Weeks 33-48: 2 mg placebo daily, up to 16 weeks.
|
orBec
n=1 participants at risk
orBec, oral beclomethasone dipropionate, was formulated as 1 mg immediate release (IR) and 1 mg delayed release (DR) tablets, which comprised 1 dose of study drug.
Part 1: 2 mg of orBec 4 times per day for up to 16 weeks.
Part 2: Patients who achieved a Complete Response (CR) on Part 1 were to taper study drug according to the following schedule:
Weeks 17-24: 2 mg orBec 3 times per day, up to 8 weeks; Weeks 25-32: 2 mg orBec 2 times per day, up to 8 weeks; Weeks 33-48: 2 mg orBec daily, up to 16 weeks.
|
|---|---|---|
|
Hepatobiliary disorders
biliary strictures
|
100.0%
1/1 • Number of events 1 • 48-week study period
|
0.00%
0/1 • 48-week study period
|
|
Infections and infestations
fungal pneumonia
|
100.0%
1/1 • Number of events 1 • 48-week study period
|
0.00%
0/1 • 48-week study period
|
|
Respiratory, thoracic and mediastinal disorders
shortness of breath
|
100.0%
1/1 • Number of events 1 • 48-week study period
|
0.00%
0/1 • 48-week study period
|
|
Vascular disorders
pulmonary emboli
|
100.0%
1/1 • Number of events 1 • 48-week study period
|
0.00%
0/1 • 48-week study period
|
|
General disorders
intractable pain
|
100.0%
1/1 • Number of events 1 • 48-week study period
|
0.00%
0/1 • 48-week study period
|
Other adverse events
Adverse event data not reported
Additional Information
Dr. Richard Straube
Soligenix, Inc.
Phone: 609-538-8200
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60