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

NCT02461134 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: AC-058C202
• Study Type: interventional
• Target: 1
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Chronic Graft Versus Host Disease

Keywords

GVHD; ponesimod

Outcome Measures

Primary Outcomes (1)

• Change in Peripheral Absolute Lymphocyte Count From Baseline to Week 4, 8 and 12

  The primary pharmacodynamic endpoint assesses intra-subject dose response during the first 12 weeks of treatment.

  From baseline to Week 12

Secondary Outcomes (1)

• Incident Rate of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

  From the first study drug intake up to 30 days after last study drug intake (Week 24)

Other Outcomes (1)

• Assessment of a Partial or Complete Overall Response at Week 24

  At Week 24

Study Arms (1)

Ponesimod

EXPERIMENTAL

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

Interventions

Ponesimod DRUG

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.

Also known as: ACT-128800

Ponesimod

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Actelion: lead

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Interventions

ponesimod

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Limitations and Caveats

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.

Results Point of Contact

• Title: Clinical Trial Disclosure Desk
• Organization: Actelion Pharmaceuticals Ltd

clinical-trials-disclosure@its.jnj.com

+41 61 565 6565

Study Officials

• Daniele D'Ambrosio, MD, PhD

  Actelion

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 28, 2015
• First Posted: June 3, 2015
• Study Start: September 29, 2016
• Primary Completion: March 2, 2017
• Study Completion: March 3, 2017
• Last Updated: February 4, 2025
• Results First Posted: May 9, 2018

Record last verified: 2025-01