Trial Outcomes & Findings for Clinical Study to Investigate the Biological Activity, Safety, Tolerability, and Pharmacokinetics of Ponesimod in Subjects With Symptomatic Chronic GVHD (NCT NCT02461134)
NCT ID: NCT02461134
Last Updated: 2025-02-04
Results Overview
The primary pharmacodynamic endpoint assesses intra-subject dose response during the first 12 weeks of treatment.
TERMINATED
PHASE2
1 participants
From baseline to Week 12
2025-02-04
Participant Flow
11 subjects were screened at 5 different sites. 1 subject was finally recruited into the study. The study was prematurely terminated due to poor recruitment.
The protocol-defined study population included male and female subjects aged 18 to 70 years with chronic Graft versus Host disease (GVHD). It was planned to recruit 30 subjects.
Participant milestones
| Measure |
Ponesimod
It was planned that enrolled subjects receive ponesimod in escalating doses of 5, 10 and 20 mg over the course of the treatment period of 24 weeks in total (4 weeks of 5 mg incl. up-titration, 4 weeks of 10 mg incl. up-titration and 16 weeks of 20 mg).
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|---|---|
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Overall Study
STARTED
|
1
|
|
Overall Study
COMPLETED
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0
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Ponesimod
It was planned that enrolled subjects receive ponesimod in escalating doses of 5, 10 and 20 mg over the course of the treatment period of 24 weeks in total (4 weeks of 5 mg incl. up-titration, 4 weeks of 10 mg incl. up-titration and 16 weeks of 20 mg).
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|---|---|
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Overall Study
Adverse Event
|
1
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Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Ponesimod
n=1 Participants
It was planned that enrolled subjects receive ponesimod in escalating doses of 5, 10 and 20 mg over the course of the treatment period of 24 weeks in total (4 weeks of 5 mg incl. up-titration, 4 weeks of 10 mg incl. up-titration and 16 weeks of 20 mg).
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|---|---|
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Age, Categorical
<=18 years
|
0 Participants
n=1 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=1 Participants
|
|
Age, Categorical
>=65 years
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0 Participants
n=1 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=1 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=1 Participants
|
PRIMARY outcome
Timeframe: From baseline to Week 12Population: Due to the premature termination of the study and consequent lack of meaningful data, no analyses were performed. The statistical analysis plan issued is obsolete, it was not finalized and was therefore not executed.
The primary pharmacodynamic endpoint assesses intra-subject dose response during the first 12 weeks of treatment.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From the first study drug intake up to 30 days after last study drug intake (Week 24)Population: The premature termination of the study led to a consequent lack of meaningful data. As the statistical analysis plan issued was not finalized and was not executed, no statistical analyses were performed. As there was only 1 patient enrolled in the study, the safety events reported occur with 100% frequency.
This outcome measure reports the occurrence of adverse events (AEs), and serious adverse events (SAEs) during the treatment period and the follow-up period, and AEs leading to premature discontinuation of study drug. A treatment-emergent AE is any AE temporally associated with the use of study treatment whether or not considered by the investigator as related to study treatment.
Outcome measures
| Measure |
Ponesimod
n=1 Participants
It was planned that enrolled subjects receive ponesimod in escalating doses of 5, 10 and 20 mg over the course of the treatment period of 24 weeks in total (4 weeks of 5 mg incl. up-titration, 4 weeks of 10 mg incl. up-titration and 16 weeks of 20 mg).
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|---|---|
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Incident Rate of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with AEs
|
1 Participants
|
|
Incident Rate of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants with SAEs
|
1 Participants
|
|
Incident Rate of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants discontinued prematurely study drug
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1 Participants
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OTHER_PRE_SPECIFIED outcome
Timeframe: At Week 24Population: Due to the premature termination of the study and consequent lack of meaningful data, no analyses were performed. The statistical analysis plan issued is obsolete, it was not finalized and was therefore not executed.
The exploratory efficacy endpoint is based on the 2014 NIH Consensus Development Project response criteria. A complete overall response is defined as a resolution of all reversible manifestations due to chronic GVHD in each organ as defined per NIH Consensus Development Project response criteria. A partial overall response is defined as improvement in a measure for at least one organ without progression in measures for any other organ.
Outcome measures
Outcome data not reported
Adverse Events
Ponesimod
Serious adverse events
| Measure |
Ponesimod
n=1 participants at risk
It was planned that enrolled subjects receive ponesimod in escalating doses of 5, 10 and 20 mg over the course of the treatment period of 24 weeks in total (4 weeks of 5 mg incl. up-titration, 4 weeks of 10 mg incl. up-titration and 16 weeks of 20 mg).
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|---|---|
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Gastrointestinal disorders
Gastritis
|
100.0%
1/1 • Number of events 1 • Study treatment was terminated on Day 81 and the subject was exposed to ponesimod for a total of 81 days (i.e., Day 1 to Day 81). None of the reported adverse events (AEs) were considered by the investigator to be related to the study drug. As there was only 1 patient enrolled in the study, all (S)AEs reported occur with 100% frequency.
All AEs were coded using MedDRA version 19.0. No AEs were reported during the screening period.
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|
Infections and infestations
Virus infection
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100.0%
1/1 • Number of events 1 • Study treatment was terminated on Day 81 and the subject was exposed to ponesimod for a total of 81 days (i.e., Day 1 to Day 81). None of the reported adverse events (AEs) were considered by the investigator to be related to the study drug. As there was only 1 patient enrolled in the study, all (S)AEs reported occur with 100% frequency.
All AEs were coded using MedDRA version 19.0. No AEs were reported during the screening period.
|
|
Gastrointestinal disorders
Haematemesis, Parainfluenza
|
100.0%
1/1 • Number of events 1 • Study treatment was terminated on Day 81 and the subject was exposed to ponesimod for a total of 81 days (i.e., Day 1 to Day 81). None of the reported adverse events (AEs) were considered by the investigator to be related to the study drug. As there was only 1 patient enrolled in the study, all (S)AEs reported occur with 100% frequency.
All AEs were coded using MedDRA version 19.0. No AEs were reported during the screening period.
|
|
Injury, poisoning and procedural complications
Hip fracture
|
100.0%
1/1 • Number of events 1 • Study treatment was terminated on Day 81 and the subject was exposed to ponesimod for a total of 81 days (i.e., Day 1 to Day 81). None of the reported adverse events (AEs) were considered by the investigator to be related to the study drug. As there was only 1 patient enrolled in the study, all (S)AEs reported occur with 100% frequency.
All AEs were coded using MedDRA version 19.0. No AEs were reported during the screening period.
|
Other adverse events
| Measure |
Ponesimod
n=1 participants at risk
It was planned that enrolled subjects receive ponesimod in escalating doses of 5, 10 and 20 mg over the course of the treatment period of 24 weeks in total (4 weeks of 5 mg incl. up-titration, 4 weeks of 10 mg incl. up-titration and 16 weeks of 20 mg).
|
|---|---|
|
Cardiac disorders
Atrial flutter
|
100.0%
1/1 • Number of events 1 • Study treatment was terminated on Day 81 and the subject was exposed to ponesimod for a total of 81 days (i.e., Day 1 to Day 81). None of the reported adverse events (AEs) were considered by the investigator to be related to the study drug. As there was only 1 patient enrolled in the study, all (S)AEs reported occur with 100% frequency.
All AEs were coded using MedDRA version 19.0. No AEs were reported during the screening period.
|
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Vascular disorders
Hypertension
|
100.0%
1/1 • Number of events 1 • Study treatment was terminated on Day 81 and the subject was exposed to ponesimod for a total of 81 days (i.e., Day 1 to Day 81). None of the reported adverse events (AEs) were considered by the investigator to be related to the study drug. As there was only 1 patient enrolled in the study, all (S)AEs reported occur with 100% frequency.
All AEs were coded using MedDRA version 19.0. No AEs were reported during the screening period.
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Additional Information
Clinical Trial Disclosure Desk
Actelion Pharmaceuticals Ltd
Results disclosure agreements
- Principal investigator is a sponsor employee Any study-related publication written independently by investigators must be submitted to Actelion for review at least 30 days prior to submission for publication or presentation. Upon review, Actelion may provide comments, and may also request alterations and/or deletions for the sole purpose of protecting its confidential information and/or patent rights.
- Publication restrictions are in place
Restriction type: OTHER