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
Chronic graft versus host diseasre (GVHD) is a serious reaction that might occur in a person (the host) who has received cells or organs (graft) from another person because the graft attacks the host's cells. Currently there are no approved therapies for chronic GVHD in the USA, and patients with chroninc GVHD are treated with immunosuppressant drugs. T-lymphocytes (a type of white blood cells) are likely to play a role in the development of chronic GVHD. Due to the capacity of ponesimod to block the traffic of T-lymphocytes, ponesimod may be a new therapeutic approach to treat chroninc GVHD.
The main objective of this study is to assess the effectiveness and safety of several doses of ponesimod in subjects with chronic GVHD who did not respond to standard available treatments.
|Condition or disease||Intervention/treatment||Phase|
|Chronic Graft Versus Host Disease||Drug: Ponesimod||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||1 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2, Open-label, Single-arm, Intra-subject Dose-escalation Study to Investigate the Biological Activity, Safety, Tolerability, and Pharmacokinetics of Ponesimod in Subjects With Symptomatic Moderate or Severe Chronic GVHD Inadequately Responding to First or Second Line Therapy|
|Actual Study Start Date :||September 29, 2016|
|Actual Primary Completion Date :||March 2, 2017|
|Actual Study Completion Date :||March 3, 2017|
Study treatment consists of 3 consecutive periods: 5 mg ponesimod treatment period (including up-titration), 10 mg treatment period (including up-titration) and a 20 mg treatment period.
Oral film-coated tablets at the doses of 2, 3, 4, 5, 6, 7, 8, 9, 10, and 20 mg. One tablet of ponesimod at any dose will be taken orally once daily.
Other Name: ACT-128800
- Change in Peripheral Absolute Lymphocyte Count From Baseline to Week 4, 8 and 12 [ Time Frame: From baseline to Week 12 ]The primary pharmacodynamic endpoint assesses intra-subject dose response during the first 12 weeks of treatment.
- Incident Rate of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: From the first study drug intake up to 30 days after last study drug intake (Week 24) ]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.
- Assessment of a Partial or Complete Overall Response at Week 24 [ Time Frame: At Week 24 ]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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02461134
|United States, Arizona|
|Virginia Piper Cancer Institute|
|Scottsdale, Arizona, United States, 85258|
|United States, California|
|Moore Cancer Center - UCSD|
|La Jolla, California, United States, 92093|
|David Geffen School of Med at UCLA|
|Los Angeles, California, United States, 90095|
|United States, Illinois|
|Chicago, Illinois, United States, 60611|
|United States, Indiana|
|Indiana University Simon Cancer Center|
|Indianapolis, Indiana, United States, 46202|
|United States, Maryland|
|National Cancer Institute|
|Bethesda, Maryland, United States, 20892-1203|
|United States, Minnesota|
|University of Minnesota - Masonic Cancer CTR CLIN TRIALS CTR|
|Minneapolis, Minnesota, United States, 55455|
|United States, Missouri|
|Washington Univ School of Med, Oncology (St.Louis)|
|Saint Louis, Missouri, United States, 63110|
|United States, New York|
|Stony Brook Univ. Medical Center|
|Stony Brook, New York, United States, 11794|
|United States, Washington|
|Fred Hutchinson Cancer Res CTR|
|Seattle, Washington, United States, 98109-1023|
|Study Chair:||Daniele D'Ambrosio, MD, PhD||Actelion|