Efficacy and Safety of APD334 in Patients With Pyoderma Gangrenosum

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03072953
• Date of registration: 17/02/2017
• Prospective Registration: Yes
• Primary sponsor: Arena Pharmaceuticals
• Public title: Efficacy and Safety of APD334 in Patients With Pyoderma Gangrenosum
• Scientific title: A Phase 2a, Open-label, Proof of Concept Study to Determine the Efficacy and Safety of Etrasimod (APD334) in Patients With Pyoderma Gangrenosum
• Date of first enrolment: June 7, 2017
• Target sample size: 2
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03072953
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia, New Zealand

Contacts

• Name: Arena CT.gov Administrator
• Affiliation: Arena Pharmaceuticals

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male or female (18-80 years).

2. Able to provide a signed informed consent prior to any study related procedure being
conducted.

3. Diagnosis of PG with active, non-healing ulcer.

4. Considered to be in stable health in the opinion of the investigator as determined by:

1. A screening physical examination with no clinically significant abnormalities
unrelated to PG.

2. Vital signs at screening: pulse rate = 55 bpm, systolic blood pressure = 90 mmHg,
and diastolic blood pressure = 55 mmHg.

3. Liver function tests (alanine aminotransferase/aspartate aminotransferase,
bilirubin and alkaline phosphatase) < 2x the upper limit of normal.

4. All other pre-study clinical laboratory findings within normal range, or if
outside of the normal range are not deemed clinically significant in the opinion
of the investigator with exemption to leucopenia and lymphopenia - please refer
to exclusion criterion 24.

5. No clinical abnormalities noted in the12-lead electrocardiogram in the opinion of
the investigator (Refer also to exclusion criterion 13).

6. 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.

5. 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.

Exclusion Criteria:

1. 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.

2. Infection with hepatitis C virus anytime in the past; confirmed active infection with
hepatitis B virus at screening.

3. History of severe renal or severe hepatic impairment.

4. Current active or latent tuberculosis (TB).

5. A positive diagnostic TB test at screening.

6. Exposure to B-cell or T-cell targeted therapies (such as natalizumab, rituximab,
abatacept) within 5 half-lives prior to Day 1.

7. Exposure to other immunosuppressive, immunomodulating or antineoplastic agents.

8. Receipt of any investigational agent within 30 days or 5 half lives (whichever is
longer), prior to Day 1.

9. Use of moderate to strong inhibitors of CYP2C9.

10. Abnormal forced expiratory volume (FEV1) or forced vital capacity (FVC).

11. Any known history of congenital or acquired immuno-deficiency.

12. 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.

13. 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.

14. Congestive heart failure (NYHA III or NYHA IV)

15. Any surgical procedure requiring general anesthesia within 30 days prior to Day 1 or
plans to undergo major surgery during the study period.

16. History of retinal macular edema.

17. History of or signs and symptoms of progressive multifocal leukoencephalopathy (PML)
as assessed by the PML checklist at screening.

18. History of more than one episode of herpes zoster or any episode of disseminated
zoster.

19. Participants without documented positive varicella zoster virus (VZV) IgG antibody
status or participants who have not completed VZV vaccination within 6 weeks prior to
Day 1.

20. Receipt of live vaccine within 6 weeks prior to Day 1.

21. History of lymphoproliferative disorder, lymphoma, leukemia, myeloproliferative
disorder, or multiple myeloma.

22. History of malignancy except for adequately treated basal cell skin cancer and in situ
carcinoma of the cervix of the uterus that have been completely excised with
documented, clear margins.

23. History of severe allergic or anaphylactic reactions requiring medical attention.

24. Leukopenia or lymphopenia at screening.

25. Current or recent history (within 1 year prior to Day 1) of alcohol dependence or
illicit drug use.

26. Active psychiatric problems that, in the investigator's opinion, may interfere with
compliance with the study procedures.

27. History of any other clinically significant medical condition that, in the
investigator's opinion, would preclude participant from safe participation in the
study.

28. Inability to attend all the study visits or comply with study procedures.

29. Prior exposure to etrasimod (APD334) or prior participation in any study of etrasimod
(APD334).



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pyoderma Gangrenosum

Intervention(s)

Drug: APD334

Primary Outcome(s)

Exploratory endpoint - Change from baseline in C-reactive protein levels. [Time Frame: Week 12]

Exploratory endpoint - Change from baseline in Dermatology Life Quality Index (to measure how much a patient's life is affected through their skin problems). [Time Frame: Week 12]

Exploratory endpoint - Assessment of punch biopsies. [Time Frame: Week 12]

Exploratory endpoint - Assessments of target lesions. [Time Frame: Week 12]

Exploratory endpoint - Change from baseline in Patient Global Assessments for active skin manifestations: visual analog scale for assessment of severity of the disease and severity of pain by patient. [Time Frame: Week 12]

Exploratory endpoint - Change from baseline in: Physician Global Assessments for active skin manifestations. [Time Frame: Week 12]

Secondary ID(s)

APD334-011

Source(s) of Monetary Support

Please refer to primary and secondary sponsors