NCT02461134

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

Terminated Phase 2 Results posted Last updated 4 February 2025

What this trial tests

Phase 2 trial testing Ponesimod in Chronic Graft Versus Host Disease in 1 participant. Terminated before completion.

Timeline

29 September 2016

Primary endpoint
2 March 2017

3 March 2017

Quick facts

Lead sponsor	Actelion
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	1
Start date	29 September 2016
Primary completion	2 March 2017
Estimated completion	3 March 2017

Drugs / interventions tested

• Ponesimod — full drug profile →

Conditions studied

• Chronic Graft Versus Host Disease — all drugs for Chronic Graft Versus Host Disease →

Sponsor

Actelion — full company profile →

Who can join

Adults 18 to 70, any sex, with Chronic Graft Versus Host Disease. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Incident Rate of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Secondary · 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.

Participants with AEs

Group	Value	95% CI
Ponesimod	1

Participants with SAEs

Group	Value	95% CI
Ponesimod	1

Participants discontinued prematurely study drug

Group	Value	95% CI
Ponesimod	1

Adverse events — posted to ClinicalTrials.gov

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.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Ponesimod

Serious: 1/1 (100%)

Deaths: 0/1

Serious adverse events (4 terms)

Reaction	System	Ponesimod
Gastritis	Gastrointestinal disorders	—
Virus infection	Infections and infestations	—
Haematemesis, Parainfluenza	Gastrointestinal disorders	—
Hip fracture	Injury, poisoning and procedural complications	—

Other adverse events (2 terms — click to expand)

Reaction	System	Ponesimod
Atrial flutter	Cardiac disorders	—
Hypertension	Vascular disorders	—

Most-reported serious reactions: Gastritis, Virus infection, Haematemesis, Parainfluenza, Hip fracture.

Data from ClinicalTrials.gov NCT02461134 adverse events section.

Sponsor's own description

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.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. Sphingosine 1-Phosphate Receptor Modulators and Drug Discovery.
   Park SJ, Im DS. · · 2017 · cited 103× · PMID 28035084 · DOI 10.4062/biomolther.2016.160
2. Ponesimod, a selective S1P1 receptor modulator: a potential treatment for multiple sclerosis and other immune-mediated diseases.
   D'Ambrosio D, Freedman MS, Prinz J. · · 2016 · cited 74× · PMID 26770667 · DOI 10.1177/2040622315617354
3. The S1P-S1PR Axis in Neurological Disorders-Insights into Current and Future Therapeutic Perspectives.
   Lucaciu A, Brunkhorst R, Pfeilschifter JM, Pfeilschifter W, et al · · 2020 · cited 53× · PMID 32580348 · DOI 10.3390/cells9061515
4. T cell trafficking in human chronic inflammatory diseases.
   Giovenzana A, Codazzi V, Pandolfo M, Petrelli A. · · 2024 · cited 5× · PMID 39171290 · DOI 10.1016/j.isci.2024.110528