Trial Outcomes & Findings for A Study to Investigate the Safety and Effect of the Study Drug (FE 204205) in Patients With Cirrhotic Portal Hypertension (NCT NCT02929407)
NCT ID: NCT02929407
Last Updated: 2019-07-12
Results Overview
The trial was terminated early due to low recruitment. Only four subjects were enrolled into the open-label, exploratory Part 1 of the trial, and only three of these four subjects (all receiving different dosing regimens) completed the trial and the HVPG assessments prior to the early termination decision. No subjects were enrolled into the randomized, double-blind, placebo-controlled Part 2 of the trial. As only Part 2 of the trial would have been appropriately powered, and the three subjects with HVPG assessments from Part 1 all received different dosing regimens, no meaningful statistical analysis could be conducted.
TERMINATED
PHASE1
4 participants
From baseline (pre-dose) to 2 hours after start of infusion
2019-07-12
Participant Flow
The trial was terminated early due to low recruitment. Only four subjects were enrolled into the open-label, exploratory Part 1 of the trial (all receiving different dosing regimens), and no subjects were enrolled into the randomized, double-blind, placebo-controlled Part 2 of the trial prior to early termination.
A total of five subjects were screened, of which, four subjects were enrolled in Part 1 of the trial.
Participant milestones
| Measure |
FE 204205
FE 204205, given once daily as 2 hour intravenous (IV) infusion, on three consecutive days. The dose in each subject was escalated from one day to the next if all dose escalation criteria were met.
The dosing regimen for each subject is given below:
Subject 101: Day 1: 0.0325 mg; Subject 102: Day 1: 0.01 mg, Day 2: 0.026 mg, Day 3: 0.05 mg; Subject 103: Day 1: 0.01 mg, Day 2: 0.01 mg, Day 3: 0.01 mg; Subject 105: Day 1: 0.01 mg, Day 2: 0.01 mg, Day 3: 0.026 mg.
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
FE 204205
FE 204205, given once daily as 2 hour intravenous (IV) infusion, on three consecutive days. The dose in each subject was escalated from one day to the next if all dose escalation criteria were met.
The dosing regimen for each subject is given below:
Subject 101: Day 1: 0.0325 mg; Subject 102: Day 1: 0.01 mg, Day 2: 0.026 mg, Day 3: 0.05 mg; Subject 103: Day 1: 0.01 mg, Day 2: 0.01 mg, Day 3: 0.01 mg; Subject 105: Day 1: 0.01 mg, Day 2: 0.01 mg, Day 3: 0.026 mg.
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|---|---|
|
Overall Study
Adverse Event
|
1
|
Baseline Characteristics
A Study to Investigate the Safety and Effect of the Study Drug (FE 204205) in Patients With Cirrhotic Portal Hypertension
Baseline characteristics by cohort
| Measure |
FE 204205
n=4 Participants
FE 204205, given once daily as 2 hour IV infusion, on three consecutive days. The dose in each subject was escalated from one day to the next if all dose escalation criteria were met.
The dosing regimen for each subject is given below:
Subject 101: Day 1: 0.0325 mg; Subject 102: Day 1: 0.01 mg, Day 2: 0.026 mg, Day 3: 0.05 mg; Subject 103: Day 1: 0.01 mg, Day 2: 0.01 mg, Day 3: 0.01 mg; Subject 105: Day 1: 0.01 mg, Day 2: 0.01 mg, Day 3: 0.026 mg.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From baseline (pre-dose) to 2 hours after start of infusionPopulation: There were too few subjects for statistical analysis (see measure description).
The trial was terminated early due to low recruitment. Only four subjects were enrolled into the open-label, exploratory Part 1 of the trial, and only three of these four subjects (all receiving different dosing regimens) completed the trial and the HVPG assessments prior to the early termination decision. No subjects were enrolled into the randomized, double-blind, placebo-controlled Part 2 of the trial. As only Part 2 of the trial would have been appropriately powered, and the three subjects with HVPG assessments from Part 1 all received different dosing regimens, no meaningful statistical analysis could be conducted.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Up to Day 14Population: There were too few subjects for statistical analysis (see measure description).
The trial was terminated early due to low recruitment. Only four subjects (all received different dosing regimens) were enrolled into open-label, exploratory Part 1 of the trial, prior to the early termination. No subjects were enrolled in the randomized, double-blind, placebo-controlled Part 2 of the trial. Thus no meaningful statistical analysis could be conducted. Due to the low number of subjects and individual dosing regimens, AEs were pooled for all dose levels.
Outcome measures
| Measure |
FE 204205
n=4 Participants
FE 204205, given once daily as 2 hour IV infusion, on three consecutive days. The dose in each subject was escalated from one day to the next if all dose escalation criteria were met.
The dosing regimen for each subject is given below:
Subject 101: Day 1: 0.0325 mg; Subject 102: Day 1: 0.01 mg, Day 2: 0.026 mg, Day 3: 0.05 mg; Subject 103: Day 1: 0.01 mg, Day 2: 0.01 mg, Day 3: 0.01 mg; Subject 105: Day 1: 0.01 mg, Day 2: 0.01 mg, Day 3: 0.026 mg.
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|---|---|
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Type, Frequency and Intensity of Adverse Events (AEs)
Frequency of AEs
|
3 Participants
|
|
Type, Frequency and Intensity of Adverse Events (AEs)
Intensity of AEs (Mild)
|
1 Participants
|
|
Type, Frequency and Intensity of Adverse Events (AEs)
Intensity of AEs (Moderate)
|
1 Participants
|
|
Type, Frequency and Intensity of Adverse Events (AEs)
Intensity of AEs (Severe)
|
1 Participants
|
|
Type, Frequency and Intensity of Adverse Events (AEs)
Type of AEs (Serious)
|
2 Participants
|
|
Type, Frequency and Intensity of Adverse Events (AEs)
Type of AEs (Non-serious)
|
2 Participants
|
PRIMARY outcome
Timeframe: From baseline (pre-dose) up to Day 14Population: There were too few subjects for statistical analysis (see measure description).
The trial was terminated early due to low recruitment. Only four subjects (all received different dosing regimens) were enrolled into open-label, exploratory Part 1 of the trial, prior to the early termination. No subjects were enrolled in the randomized, double-blind, placebo-controlled Part 2 of the trial. Thus no meaningful statistical analysis could be conducted.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: From baseline (pre-dose) to 3 hours after start of infusionPopulation: There were too few subjects for statistical analysis (see measure description).
The trial was terminated early due to low recruitment. Only four subjects (all received different dosing regimens) were enrolled into open-label, exploratory Part 1 of the trial, prior to the early termination. No subjects were enrolled in the randomized, double-blind, placebo-controlled Part 2 of the trial. Thus no meaningful statistical analysis could be conducted.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Pre-dose, 0.5, 1, 2, 3, and 4 hours after start of infusionPopulation: There were too few subjects for statistical analysis (see measure description).
The trial was terminated early due to low recruitment. Only four subjects (all received different dosing regimens) were enrolled into open-label, exploratory Part 1 of the trial, prior to the early termination. No subjets were enrolled in the randomized, double-blind, placebo-controlled Part 2 of the trial. Thus no meaningful statistical analysis could be conducted.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Pre-dose, 0.5, 1, 2, 3, and 4 hours after start of infusionPopulation: There were too few subjects for statistical analysis (see measure description).
The trial was terminated early due to low recruitment. Only four subjects were enrolled (all received different dosing regimens) into open-label, exploratory Part 1 of the trial, prior to the early termination. No subjects were enrolled in the randomized, double-blind, placebo-controlled Part 2 of the trial. Thus no meaningful statistical analysis could be conducted.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Pre-dose, 0.5, 1, 2, 3, and 4 hours after start of infusionPopulation: There were too few subjects for statistical analysis (see measure description).
The trial was terminated early due to low recruitment. Only four subjects (all received different dosing regimens) were enrolled into open-label, exploratory Part 1 of the trial, prior to the early termination. No subjects were enrolled in the randomized, double-blind, placebo-controlled Part 2 of the trial. Thus no meaningful statistical analysis could be conducted.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Pre-dose, 0.5, 1, 2, 3, and 4 hours after start of infusionPopulation: There were too few subjects for statistical analysis (see measure description).
The trial was terminated early due to low recruitment. Only four subjects were enrolled (all received different dosing regimens) into open-label, exploratory Part 1 of the trial, prior to the early termination. No subjects were enrolled in the randomized, double-blind, placebo-controlled Part 2 of the trial. Thus no meaningful statistical analysis could be conducted.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Pre-dose, 0.5, 1, 2, 3, and 4 hours after start of infusionPopulation: There were too few subjects for statistical analysis (see measure description).
The trial was terminated early due to low recruitment. Only four subjects were enrolled (all received different dosing regimens) into open-label, exploratory Part 1 of the trial, prior to the early termination. No subjects were enrolled in the randomized, double-blind, placebo-controlled Part 2 of the trial. Thus no meaningful statistical analysis could be conducted.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: From baseline (pre-dose) up to Day 14Population: There were too few subjects for statistical analysis (see measure description).
The trial was terminated early due to low recruitment. Only four subjects were enrolled (all received different dosing regimens) into open-label, exploratory Part 1 of the trial, prior to the early termination. No subjects were enrolled in the randomized, double-blind, placebo-controlled Part 2 of the trial. Thus no meaningful statistical analysis could be conducted.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: From baseline (pre-dose) to 3 hours after start of infusionPopulation: There were too few subjects for statistical analysis (see measure description).
The trial was terminated early due to low recruitment. Only four subjects were enrolled (all received different dosing regimens) into open-label, exploratory Part 1 of the trial, prior to the early termination. No subjects were enrolled in the randomized, double-blind, placebo-controlled Part 2 of the trial. Thus no meaningful statistical analysis could be conducted.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: From baseline (pre-dose) to 3 hours after start of infusionPopulation: There were too few subjects for statistical analysis (see measure description).
The trial was terminated early due to low recruitment. Only four subjects (all received different dosing regimens) were enrolled into open-label, exploratory Part 1 of the trial, prior to the early termination. No subjects were enrolled in the randomized, double-blind, placebo-controlled Part 2 of the trial. Thus no meaningful statistical analysis could be conducted.
Outcome measures
Outcome data not reported
Adverse Events
FE 204205
Serious adverse events
| Measure |
FE 204205
n=4 participants at risk
FE 204205, given once daily as 2 hour IV infusion, on three consecutive days. The dose in each subject was escalated from one day to the next if all dose escalation criteria were met.
The dosing regimen for each subject is given below:
Subject 101: Day 1: 0.0325 mg; Subject 102: Day 1: 0.01 mg, Day 2: 0.026 mg, Day 3: 0.05 mg; Subject 103: Day 1: 0.01 mg, Day 2: 0.01 mg, Day 3: 0.01 mg; Subject 105: Day 1: 0.01 mg, Day 2: 0.01 mg, Day 3: 0.026 mg.
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|---|---|
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Vascular disorders
Arterial Hypertension
|
25.0%
1/4 • Number of events 1 • AEs were recorded from obtaining the informed consent to the follow-up visit. In part 1 of the trial, AEs were recorded from obtaining the informed consent (up to 21 days before first administration of FE 204205) to the end-of-trial visit (between 7 and 14 days after first administration of FE 204205). Part 2 of the trial was never initiated.
An AE was defined as any untoward medical occurrence in a subject participating in a clinical trial. An AE could be any unfavourable and unintended sign, symptom or disease temporally associated with the use of the investigational medicinal product (IMP), whether or not considered to be caused by the IMP. Due to the low number of subjects and individual dosing regimens, AEs were pooled for all dose levels.
|
|
Gastrointestinal disorders
Diarrhoea
|
25.0%
1/4 • Number of events 1 • AEs were recorded from obtaining the informed consent to the follow-up visit. In part 1 of the trial, AEs were recorded from obtaining the informed consent (up to 21 days before first administration of FE 204205) to the end-of-trial visit (between 7 and 14 days after first administration of FE 204205). Part 2 of the trial was never initiated.
An AE was defined as any untoward medical occurrence in a subject participating in a clinical trial. An AE could be any unfavourable and unintended sign, symptom or disease temporally associated with the use of the investigational medicinal product (IMP), whether or not considered to be caused by the IMP. Due to the low number of subjects and individual dosing regimens, AEs were pooled for all dose levels.
|
|
Nervous system disorders
Hepatic Encephalopathy
|
25.0%
1/4 • Number of events 1 • AEs were recorded from obtaining the informed consent to the follow-up visit. In part 1 of the trial, AEs were recorded from obtaining the informed consent (up to 21 days before first administration of FE 204205) to the end-of-trial visit (between 7 and 14 days after first administration of FE 204205). Part 2 of the trial was never initiated.
An AE was defined as any untoward medical occurrence in a subject participating in a clinical trial. An AE could be any unfavourable and unintended sign, symptom or disease temporally associated with the use of the investigational medicinal product (IMP), whether or not considered to be caused by the IMP. Due to the low number of subjects and individual dosing regimens, AEs were pooled for all dose levels.
|
Other adverse events
| Measure |
FE 204205
n=4 participants at risk
FE 204205, given once daily as 2 hour IV infusion, on three consecutive days. The dose in each subject was escalated from one day to the next if all dose escalation criteria were met.
The dosing regimen for each subject is given below:
Subject 101: Day 1: 0.0325 mg; Subject 102: Day 1: 0.01 mg, Day 2: 0.026 mg, Day 3: 0.05 mg; Subject 103: Day 1: 0.01 mg, Day 2: 0.01 mg, Day 3: 0.01 mg; Subject 105: Day 1: 0.01 mg, Day 2: 0.01 mg, Day 3: 0.026 mg.
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|---|---|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
25.0%
1/4 • Number of events 1 • AEs were recorded from obtaining the informed consent to the follow-up visit. In part 1 of the trial, AEs were recorded from obtaining the informed consent (up to 21 days before first administration of FE 204205) to the end-of-trial visit (between 7 and 14 days after first administration of FE 204205). Part 2 of the trial was never initiated.
An AE was defined as any untoward medical occurrence in a subject participating in a clinical trial. An AE could be any unfavourable and unintended sign, symptom or disease temporally associated with the use of the investigational medicinal product (IMP), whether or not considered to be caused by the IMP. Due to the low number of subjects and individual dosing regimens, AEs were pooled for all dose levels.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
25.0%
1/4 • Number of events 1 • AEs were recorded from obtaining the informed consent to the follow-up visit. In part 1 of the trial, AEs were recorded from obtaining the informed consent (up to 21 days before first administration of FE 204205) to the end-of-trial visit (between 7 and 14 days after first administration of FE 204205). Part 2 of the trial was never initiated.
An AE was defined as any untoward medical occurrence in a subject participating in a clinical trial. An AE could be any unfavourable and unintended sign, symptom or disease temporally associated with the use of the investigational medicinal product (IMP), whether or not considered to be caused by the IMP. Due to the low number of subjects and individual dosing regimens, AEs were pooled for all dose levels.
|
|
Musculoskeletal and connective tissue disorders
Limb discomfort
|
25.0%
1/4 • Number of events 1 • AEs were recorded from obtaining the informed consent to the follow-up visit. In part 1 of the trial, AEs were recorded from obtaining the informed consent (up to 21 days before first administration of FE 204205) to the end-of-trial visit (between 7 and 14 days after first administration of FE 204205). Part 2 of the trial was never initiated.
An AE was defined as any untoward medical occurrence in a subject participating in a clinical trial. An AE could be any unfavourable and unintended sign, symptom or disease temporally associated with the use of the investigational medicinal product (IMP), whether or not considered to be caused by the IMP. Due to the low number of subjects and individual dosing regimens, AEs were pooled for all dose levels.
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
25.0%
1/4 • Number of events 1 • AEs were recorded from obtaining the informed consent to the follow-up visit. In part 1 of the trial, AEs were recorded from obtaining the informed consent (up to 21 days before first administration of FE 204205) to the end-of-trial visit (between 7 and 14 days after first administration of FE 204205). Part 2 of the trial was never initiated.
An AE was defined as any untoward medical occurrence in a subject participating in a clinical trial. An AE could be any unfavourable and unintended sign, symptom or disease temporally associated with the use of the investigational medicinal product (IMP), whether or not considered to be caused by the IMP. Due to the low number of subjects and individual dosing regimens, AEs were pooled for all dose levels.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
25.0%
1/4 • Number of events 1 • AEs were recorded from obtaining the informed consent to the follow-up visit. In part 1 of the trial, AEs were recorded from obtaining the informed consent (up to 21 days before first administration of FE 204205) to the end-of-trial visit (between 7 and 14 days after first administration of FE 204205). Part 2 of the trial was never initiated.
An AE was defined as any untoward medical occurrence in a subject participating in a clinical trial. An AE could be any unfavourable and unintended sign, symptom or disease temporally associated with the use of the investigational medicinal product (IMP), whether or not considered to be caused by the IMP. Due to the low number of subjects and individual dosing regimens, AEs were pooled for all dose levels.
|
|
Nervous system disorders
Asterixis
|
25.0%
1/4 • Number of events 1 • AEs were recorded from obtaining the informed consent to the follow-up visit. In part 1 of the trial, AEs were recorded from obtaining the informed consent (up to 21 days before first administration of FE 204205) to the end-of-trial visit (between 7 and 14 days after first administration of FE 204205). Part 2 of the trial was never initiated.
An AE was defined as any untoward medical occurrence in a subject participating in a clinical trial. An AE could be any unfavourable and unintended sign, symptom or disease temporally associated with the use of the investigational medicinal product (IMP), whether or not considered to be caused by the IMP. Due to the low number of subjects and individual dosing regimens, AEs were pooled for all dose levels.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review the draft manuscript prior to publication and can request delay of publication where any contents are deemed patentable by the sponsor or confidential to the sponsor. Comments will be given within four weeks from receipt of the draft manuscript. Additional time may be required to allow Ferring to seek patent protection of the invention.
- Publication restrictions are in place
Restriction type: OTHER