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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

Brief Summary:

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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


Arm Intervention/treatment
Experimental: Ponesimod
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.
Drug: Ponesimod
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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.


Other Outcome Measures:
  1. 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.



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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Signed informed consent
  • Symptomatic moderate or severe chronic GVHD patients in need of a change of systemic immunosuppressant (IS) therapy
  • Women of child bearing potential must have a negative pregnancy test and use reliable methods of contraception

Exclusion Criteria:

  • Clinically significant medical conditions including active or uncontrolled infections, new or recurrent malignancy, serious cardiac, pulmonary, or renal disease, and uncontrolled diabetes.
  • Karnofsky Performance Score < 60.
  • Immunosuppressant therapies other than allowed background therapy
  • Anti-arrhythmic and heart rate lowering drugs.
  • Any other circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol

Information from the National Library of Medicine

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


Locations
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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
Northwestern University
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
Sponsors and Collaborators
Actelion
Investigators
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Study Chair: Daniele D'Ambrosio, MD, PhD Actelion
  Study Documents (Full-Text)

Documents provided by Actelion:

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Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT02461134     History of Changes
Other Study ID Numbers: AC-058C202
First Posted: June 3, 2015    Key Record Dates
Results First Posted: May 9, 2018
Last Update Posted: May 9, 2018
Last Verified: April 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Actelion:
GVHD
ponesimod
Additional relevant MeSH terms:
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Graft vs Host Disease
Immune System Diseases