Efficacy and Safety of APD334 in Patients With Pyoderma Gangrenosum

NCT03072953 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: APD334-011
• Study Type: interventional
• Target: 2
• Locations: 2 countries
• Sites: 6

Brief Summary

The purpose of this Phase 2a, open label, proof-of-concept clinical study is to assess the efficacy and safety of etrasimod (APD334) in patients with Pyoderma Gangrenosum.

Conditions

Pyoderma Gangrenosum

Outcome Measures

Primary Outcomes (6)

• Exploratory Endpoint - Change From Baseline in Physician Global Assessments for Active Skin Manifestations

  The physician's global assessment for active skin manifestations recorded the number of ulcers, target lesion noted for endpoint evaluation, diameters of each target lesion and score of evaluation at each visit. The scores ranged from 0 (total resolution) to 4 (no evidence of healing).

  Week 12

• Exploratory Endpoint - Change From Baseline in Patient Global Assessments for Active Skin Manifestations

  The patient global assessment for active skin manifestation recorded the disease and pain severity using a visual analogue to mark the participant's score. Participants were asked to rate their disease severity from "not severe" to "extremely severe" and pain levels from "no pain at all' to "worst pain imaginable" in the past one week.

  Week 12

• Exploratory Endpoint - Change From Baseline in Dermatology Life Quality Index (DLQI) Score

  The DLQI questionnaire assessed how much a participant's life is affected through their skin problem in the last week, and includes the following parameters: symptoms and feelings, daily activities, leisure and sport activities, work or school activities, personal relationships and treatment- related feelings. Participants responded to the 10 questions on a scale from 0 (not at all) to 3 (very much) with a total score ranging from 0 to 30. Higher scores indicated that the skin problem had an extremely large effect on the participant's life whereas lower scores indicated that the disease has minimal to no effect at all.

  Week 12

• Exploratory Endpoint - Change From Baseline in C-reactive Protein Levels

  Week 12

• Exploratory Endpoint - Assessments of Target Lesions

  Changes in surface area

  Week 12

• Exploratory Endpoint - Assessment of Punch Biopsies

  Changes in histology.

  Week 12

Other Outcomes (1)

• Number of Participants With Adverse Events and Clinically Significant (CS) Safety Measurements

  Up to approximately 12 weeks

Study Arms (1)

APD334

OTHER

APD334 active treatment for 12 weeks.

Drug: APD334

Interventions

APD334 DRUG

APD334 active treatment

Also known as: Etrasimod

APD334

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female (18-80 years).
• Able to provide a signed informed consent prior to any study related procedure being conducted.
• Diagnosis of PG with active, non-healing ulcer.
• Considered to be in stable health in the opinion of the investigator as determined by:
• A screening physical examination with no clinically significant abnormalities unrelated to PG.
• Vital signs at screening: pulse rate ≥ 55 bpm, systolic blood pressure ≥ 90 mmHg, and diastolic blood pressure ≥ 55 mmHg.
• Liver function tests (alanine aminotransferase/aspartate aminotransferase, bilirubin and alkaline phosphatase) \< 2x the upper limit of normal.
• No evidence of macular edema in an ophthalmology evaluation (performed by an ophthalmologist), supported with optical coherence tomography, where available (dependent on site capability) at screening.
• Eligible male and female participants must agree not to participate in a conception process (i.e. active attempt to let female partner to become pregnant or to impregnate, sperm donation, oocyte donation, in vitro fertilization) for at least 30 days after the last dose of study drug.
• Non-sterile participants who are sexually active must take adequate contraception measures.

You may not qualify if:

• Clinically significant infection as judged by the investigator with an end date less than 6-weeks prior to treatment start (Day 1). In case of infection requiring hospitalization or intravenous antimicrobial therapy, or opportunistic infection, this infection must have ended at least 8 weeks prior to Day 1.
• Infection with hepatitis C virus anytime in the past; confirmed active infection with hepatitis B virus at screening.
• History of severe renal or severe hepatic impairment.
• Current active or latent tuberculosis (TB).
• A positive diagnostic TB test at screening.
• Exposure to B-cell or T-cell targeted therapies (such as natalizumab, rituximab, abatacept) within 5 half-lives prior to Day 1.
• Exposure to other immunosuppressive, immunomodulating or antineoplastic agents.
• Receipt of any investigational agent within 30 days or 5 half lives (whichever is longer), prior to Day 1.
• Use of moderate to strong inhibitors of CYP2C9.
• Abnormal forced expiratory volume (FEV1) or forced vital capacity (FVC).
• Any known history of congenital or acquired immuno-deficiency.
• Recent history (within 6 months of screening visit) of cardio- or cerebrovascular disease, acute coronary syndrome, myocardial infarction, unstable angina, cerebro-vascular accident, including transient ischemic attack.
• History or presence of cardiac arrhythmia, conduction system disease, or use of Class Ia or Class III anti arrhythmic agents, or baseline QTc ≥ 500 msec.
• Congestive heart failure (NYHA III or NYHA IV)
• Any surgical procedure requiring general anesthesia within 30 days prior to Day 1 or plans to undergo major surgery during the study period.

Sponsors & Collaborators

• Arena Pharmaceuticals: lead

Study Sites (6)

Westmead Hospital

Westmead, New South Wales, 2145, Australia

Location

Veracity Clinical Research

Woolloongabba, Queensland, 4102, Australia

Location

Eastern Clinical Research Unit

Box Hill, Victoria, 3128, Australia

Location

Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia

Location

Fremantle Dermatology

Fremantle, Western Australia, 6160, Australia

Location

Braemar Day Hospital

Hamilton, New Zealand

Location

MeSH Terms

Conditions

Pyoderma Gangrenosum

Interventions

etrasimod

Condition Hierarchy (Ancestors)

Pyoderma; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vascular; Skin Ulcer

Limitations and Caveats

Due to limited enrollment (N=2), this clinical trial was terminated by the Sponsor and no efficacy analyses were conducted. To protect the participant's privacy, demography and efficacy data are not reported.

Results Point of Contact

• Title: Arena CT.gov Administrator
• Organization: Arena Pharmaceuticals, Inc.

ct.gov@arenapharm.com

+1 855-218-9153

Study Officials

• Arena CT.gov Administrator

  Arena Pharmaceuticals

  STUDY DIRECTOR

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

Model Details: Open label

Study Record Dates

• First Submitted: February 17, 2017
• First Posted: March 8, 2017
• Study Start: June 7, 2017
• Primary Completion: May 22, 2018
• Study Completion: May 22, 2018
• Last Updated: June 11, 2021
• Results First Posted: June 11, 2021

Record last verified: 2021-05

Data Sharing

• IPD Sharing: Will not share

Locations

AU (5); NZ (1)