Pyoderma GangrenosumPhase 2NCT03072953
Efficacy and Safety of APD334 in Patients With Pyoderma Gangrenosum
A Phase 2 study testing APD334 in people with Pyoderma Gangrenosum, run by Arena Pharmaceuticals at 6 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Pyoderma Gangrenosum
- What is being tested?
- APD334
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 7 Jun 2017to 22 May 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- Liver function tests (alanine aminotransferase/aspartate aminotransferase, bilirubin and alkaline phosphatase) < 2x the upper limit of normal.
- 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.
- No clinical abnormalities noted in the12-lead electrocardiogram in the opinion of the investigator (Refer also to exclusion criterion 13).
- 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.
Who cannot join
- 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.
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- 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.
- History of retinal macular edema.
- History of or signs and symptoms of progressive multifocal leukoencephalopathy (PML) as assessed by the PML checklist at screening.
- History of more than one episode of herpes zoster or any episode of disseminated zoster.
- 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.
- Receipt of live vaccine within 6 weeks prior to Day 1.
- History of lymphoproliferative disorder, lymphoma, leukemia, myeloproliferative disorder, or multiple myeloma.
- 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.
- History of severe allergic or anaphylactic reactions requiring medical attention.
- Leukopenia or lymphopenia at screening.
- Current or recent history (within 1 year prior to Day 1) of alcohol dependence or illicit drug use.
- Active psychiatric problems that, in the investigator's opinion, may interfere with compliance with the study procedures.
- History of any other clinically significant medical condition that, in the investigator's opinion, would preclude participant from safe participation in the study.
- Inability to attend all the study visits or comply with study procedures.
- Prior exposure to etrasimod (APD334) or prior participation in any study of etrasimod (APD334).
Trial locations and contacts
6 sites in 2 countries.
6 sites in 2 countries
Australia5 sites
Eastern Clinical Research Unit
Box Hill, Victoria, 3128
Fremantle Dermatology
Fremantle, Western Australia, 6160
Royal Melbourne Hospital
Parkville, Victoria, 3050
Westmead Hospital
Westmead, New South Wales, 2145
Veracity Clinical Research
Woolloongabba, Queensland, 4102
About this study
Official title: A Phase 2a, Open-label, Proof of Concept Study to Determine the Efficacy and Safety of Etrasimod (APD334) in Patients With Pyoderma Gangrenosum
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
- Being tested
- APD334
- Medical subject headings
- Pyoderma Gangrenosum
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
APD334Drug
Also called Etrasimod
APD334 active treatment
| Study group | Role | What they receive |
|---|---|---|
| APD334 | Other | APD334 active treatment for 12 weeks. Receives: Drug: APD334 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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.
Measured: 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.
Measured: Week 12
Exploratory Endpoint - Change From Baseline in C-reactive Protein Levels
Measured: Week 12
Exploratory Endpoint - Assessments of Target Lesions
Changes in surface area
Measured: Week 12
Exploratory Endpoint - Assessment of Punch Biopsies
Changes in histology.
Measured: Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2017
- Study start7 Jun 2017
- Primary completion22 May 2018
- Study completion22 May 2018
Why it stopped: Terminated \[Sponsor decision to terminate the study\]
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pyoderma GangrenosumStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open label
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arena Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Arena CT.gov Administrator, Study director, Arena Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03072953
- Sponsor's protocol ID
- APD334-011
- Status last verified
- May 2021
- First submitted
- 17 Feb 2017
- Last updated
- 11 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Open-label, Proof of Concept Study to Determine the Efficacy and Safety of Etrasimod (APD334) in Patients With Pyoderma Gangrenosum
- ClinicalTrials.gov ID
- NCT03072953
- Acronym / sponsor ID
- APD334-011
- Conditions
- Pyoderma Gangrenosum
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated \[Sponsor decision to terminate the study\]
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Arena PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 7 Jun 2017 (actual)
- Primary completion
- 22 May 2018 (actual)
- Study completion
- 22 May 2018 (actual)
- First posted
- 8 Mar 2017 (actual)
- Results
- Posted 11 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open label
| Arm | Type | Receives |
|---|---|---|
| APD334 | Other | Drug: APD334APD334 active treatment for 12 weeks. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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:
- 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.
Key exclusion criteria (29 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| APD334Also: EtrasimodAPD334 active treatment | Drug | APD334 |
Primary outcome
Exploratory Endpoint - Change From Baseline in Physician Global Assessments for Active Skin Manifestations
Time frame: Week 12
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).
Exploratory Endpoint - Change From Baseline in Patient Global Assessments for Active Skin Manifestations
Time frame: Week 12
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.
Exploratory Endpoint - Change From Baseline in Dermatology Life Quality Index (DLQI) Score
Time frame: Week 12
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.
Exploratory Endpoint - Change From Baseline in C-reactive Protein Levels
Time frame: Week 12
Exploratory Endpoint - Assessments of Target Lesions
Time frame: Week 12
Changes in surface area
Exploratory Endpoint - Assessment of Punch Biopsies
Time frame: Week 12
Changes in histology.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Arena CT.gov Administrator study director
Arena Pharmaceuticals
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing APD334 in people with Pyoderma Gangrenosum, run by Arena Pharmaceuticals at 6 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 11 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Australia, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Jun 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Exploratory Endpoint - Change From Baseline in Physician Global Assessments for Active Skin Manifestations (time frame: Week 12). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2021.