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Clinical Study to Investigate the Biological Activity, Safety, Tolerability, and Pharmacokinetics of Ponesimod in Subjects With Symptomatic Chronic GVHD

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ClinicalTrials.gov Identifier: NCT02461134
Recruitment Status : Terminated (Low recruitment)
First Posted : June 3, 2015
Results First Posted : May 9, 2018
Last Update Posted : May 9, 2018
Sponsor:
Information provided by (Responsible Party):
Actelion

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Chronic Graft Versus Host Disease
Intervention Drug: Ponesimod
Enrollment 1
Recruitment Details 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.
Pre-assignment Details 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.
Arm/Group Title Ponesimod
Hide Arm/Group Description 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).
Period Title: Overall Study
Started 1
Completed 0
Not Completed 1
Reason Not Completed
Adverse Event             1
Arm/Group Title Ponesimod
Hide Arm/Group Description 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).
Overall Number of Baseline Participants 1
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
<=18 years
0
   0.0%
Between 18 and 65 years
1
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
Female
1
 100.0%
Male
0
   0.0%
Race and Ethnicity Not Collected   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants
[1]
Measure Analysis Population Description: Race and Ethnicity were not collected from any participant.
1.Primary Outcome
Title Change in Peripheral Absolute Lymphocyte Count From Baseline to Week 4, 8 and 12
Hide Description The primary pharmacodynamic endpoint assesses intra-subject dose response during the first 12 weeks of treatment.
Time Frame From baseline to Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Ponesimod
Hide Arm/Group Description:
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).
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
2.Secondary Outcome
Title Incident Rate of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Hide Description 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.
Time Frame From the first study drug intake up to 30 days after last study drug intake (Week 24)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Ponesimod
Hide Arm/Group Description:
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).
Overall Number of Participants Analyzed 1
Measure Type: Count of Participants
Unit of Measure: Participants
Participants with AEs
1
 100.0%
Participants with SAEs
1
 100.0%
Participants discontinued prematurely study drug
1
 100.0%
3.Other Pre-specified Outcome
Title Assessment of a Partial or Complete Overall Response at Week 24
Hide Description 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.
Time Frame At Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Ponesimod
Hide Arm/Group Description:
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).
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame 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.
Adverse Event Reporting Description All AEs were coded using MedDRA version 19.0. No AEs were reported during the screening period.
 
Arm/Group Title Ponesimod
Hide Arm/Group Description 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).
All-Cause Mortality
Ponesimod
Affected / at Risk (%)
Total   0/1 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Ponesimod
Affected / at Risk (%) # Events
Total   1/1 (100.00%)    
Gastrointestinal disorders   
Gastritis  1  1/1 (100.00%)  1
Haematemesis, Parainfluenza  1  1/1 (100.00%)  1
Infections and infestations   
Virus infection  1  1/1 (100.00%)  1
Injury, poisoning and procedural complications   
Hip fracture  1  1/1 (100.00%)  1
1
Term from vocabulary, MedDRA
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Ponesimod
Affected / at Risk (%) # Events
Total   1/1 (100.00%)    
Cardiac disorders   
Atrial flutter  1  1/1 (100.00%)  1
Vascular disorders   
Hypertension  1  1/1 (100.00%)  1
1
Term from vocabulary, MedDRA
Indicates events were collected by systematic assessment
The study was terminated early due to poor recruitment with 1 subject enrolled. Due to consequent lack of meaningful data, no conclusive analyses could be performed. The statistical analysis plan is obsolete and therefore not applicable.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Clinical Trial Disclosure Desk
Organization: Actelion Pharmaceuticals Ltd
Phone: +41 61 565 6565
EMail: clinical-trials-disclosure@its.jnj.com
Layout table for additonal information
Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT02461134     History of Changes
Other Study ID Numbers: AC-058C202
First Submitted: April 28, 2015
First Posted: June 3, 2015
Results First Submitted: March 2, 2018
Results First Posted: May 9, 2018
Last Update Posted: May 9, 2018