Necrobiosis LipoidicaPhase 2NCT04800562
A Clinical Trial to Evaluate PCS499 in Treating Ulcerations in Patients Who Have Necrobiosis Lipoidica
A Phase 2 study testing PCS499 in people with Necrobiosis Lipoidica, run by Processa Pharmaceuticals at 4 sites in United States.
Trial summary
This is a randomized, double blind, placebo controlled study that will evaluate the efficacy and safety of PCS499 as compared to placebo for the treatment of ulcerations of patients with necrobiosis lipoidica (NL) and will inform the design of future studies. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Necrobiosis Lipoidica
- What is being tested?
- PCS499
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 4 participants
- Where
- 4 sitesUnited States
- Study dates
- 12 Apr 2021to 14 Apr 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 patients age 18 to 80 years of age, inclusive, at Screening.
- Biopsy-confirmed diagnosis of NL. Biopsies of continually active lesions performed outside of this clinical study will need to be reviewed and the diagnosis confirmed by the study pathologist. For patients with no previous history of biopsy, no biopsy within the previous 5 years, a biopsy that is not confirmed to be NL, or newly active lesion, a biopsy to confirm a diagnosis of NL will be performed at the Screening visit.
- Ulcers on a single leg ("reference leg") at Baseline should consist of at least one (1) ulcer with a minimum ulcer surface area of 1 cm2, total ulcer area of a minimum of 2 cm2, and no more than 6 ulcers. If ulcers are present on both legs, the Investigator will select the "reference leg". The ulcer(s) on the reference leg ("reference ulcers") and all other ulcers associated with the patient's NL ("other ulcers") present at the Baseline visit will be followed in the study.
- Women of childbearing potential must have a negative serum pregnancy test at the Screening Visit and a negative urine pregnancy test at Baseline before dosing. Women of childbearing potential must use one of the following acceptable methods of contraception throughout the study: oral contraceptive medication, intrauterine device (IUD), hormonal implants, injectable contraceptive medications, double-barrier methods, or tubal ligation.
- Females who are postmenopausal (age-related amenorrhea >= 12 consecutive months and increased follicle-stimulating hormone [FSH] > 40 mIU/mL. If necessary to confirm postmenopausal status, an FSH will be drawn at Screening) or who have undergone hysterectomy or bilateral oophorectomy are exempt from pregnancy testing.
- Male patients must be willing to use appropriate contraceptive measures and refrain from sexual activity with any female who is pregnant or lactating.
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- Patient must be willing and able to swallow whole tablets.
- Patient must be willing and able to comply with study procedures.
- Patient must be willing and able to provide signed, informed consent.
Who cannot join
- Current or previous (within 4 weeks of Baseline) treatment with:
- Oral corticosteroids.
- Topical drugs (including prescription and over-the counter) on the reference leg. (Topical non-medicated moisturizer treatment products can be administered);
- Systemic pentoxifylline, theophylline, or cilostazol.
- Oral retinoid.
- Other systemic immunosuppressant or immunomodulatory drugs, including but not limited to calcineurin inhibitors (e.g., tacrolimus), thalidomide, apremilast, anti-malarials (e.g., hydroxychloroquine, chloroquine), cyclosporine, mycophenolate mofetil, azathioprine, methotrexate, etc.
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- Current or previous (within 12 weeks of Baseline) treatment with any biologic therapy (e.g., adalimumab, etanercept, infliximab, anakinra, etc.).
- Phototherapy/photochemotherapy (NBUVB, UVB, PUVA) within 6 weeks prior to Baseline
- Skin grafting, or other surgical procedure (other than debridement) within 6 weeks prior to Baseline.
- History of drug allergy, including but not limited to pentoxifylline or other xanthine derivatives, or other allergy, which in the opinion of the Investigator, contraindicates participation.
- Anticipated concurrent use of a strong CYP1A2 inhibiting drug, including but not limited to cimetidine and/or fluvoxamine, during the course of the study (after Screening).
- Fever (>38°C), or chronic, persistent, or recurring infection(s) at Screening or Baseline.
- Any infection requiring oral antimicrobial therapy within 2 weeks prior to Baseline or any infection requiring parenteral antibiotics or hospitalization within 12 weeks prior to Baseline. Any treatment for such infections must have been completed and the infection cured for at least 2 weeks prior to Baseline.
- History of sarcoidosis, pyoderma gangrenosum, or any other disorder (in the judgment of the Investigator) that would interfere with the evaluation of NL or require protocol prohibited medication.
- History of any life threatening infection or sepsis within 12 months of Baseline:
- Clinically significant cardiac disease including but not limited to unstable angina, acute myocardial infarction within 6 months of Baseline, and arrhythmia requiring therapy.
- Patient has QTc interval ≥ 480 milliseconds on Screening Electrocardiogram (ECG); a second Screening ECG may be done at investigator's discretion but the average of the two QTc screening intervals must not be ≥ 480 milliseconds.
- History of cerebral hemorrhage, cerebrovascular accident, transient ischemic attack, gastrointestinal bleeding, or retinal hemorrhage within 6 months of Baseline.
- Patient has active or history of neoplastic disease (except for adequately treated non-invasive basal cell and/or squamous cell carcinoma or carcinoma in situ of the cervix) within the past 5 years prior to Baseline.
- Presence of clinically significant medical condition(s) including but not limited to: renal, hepatic, cardiovascular, hematological, gastrointestinal, endocrine, pulmonary, neurological, psychiatric, substance abuse, and/or any other clinically significant disease or disorder, which in the opinion of the Investigator (by its nature or by being inadequately controlled), may put the patient at risk due to participation in the study, influence the results of the study, and/or affect the patient's ability to complete the study.
- History of or current diagnosis of active tuberculosis (TB); undergoing treatment for latent TB infection (LTBI); untreated LTBI (as determined by documented results within 3 months of the Screening Visit of a positive TB skin test with purified protein derivative with induration >= 5 millimeter (mm), or a positive QuantiFERON-TB test or positive or borderline T-Spot [Elispot] test); or positive TB test at Screening. Subjects with documented completion of appropriate LTBI treatment would not be excluded and are not required to be tested.
- Vaccination with live or live-attenuated virus vaccine within 1 month prior to Baseline.
- The results of the following laboratory tests performed at the central laboratory at Screening meet any of the criteria below:
- Hemoglobin < 8.0 g/dL (International System of Units (SI): < 80 g/L);
- White blood cells < 3.0 x 10^3 cells/mm^3 (SI: < 3.0 x 10^9 cells/L);
- Neutrophils < 1.0 x 10^3 cells/mm^3 (SI: < 1.0 x 10^9 cells/L);
- Lymphocytes < 0.5 x 10^3 cells/mm^3 (SI: < 0.5 x 10^9 cells/L);
- Platelets < 100 x 10^3 cells/mm^3 (SI: < 100 x 10^9 cells/L)
- Alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or alkaline phosphatase (ALP) ≥ 2 x upper limit of normal (ULN);
- Total bilirubin level ≥ 2 x ULN unless the individual has been diagnosed with Gilbert's disease and this is clearly documented;
- Estimated glomerular filtration rate < 40 mL/min/1.73 m^2 based on the Modification of Diet in Renal Disease (MDRD) formula.
- HbA1c > 10%
- Positive HIV serology
- Evidence of active Hepatitis B Virus (HBV) infection
- Evidence of active Hepatitis C Virus (HCV) infection
- Women who are pregnant or breastfeeding.
- Patient unwilling or unable to swallow tablets whole.
- Any other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the study.
- Use of any investigational product within 30 days prior to Baseline or currently enrolled in another study that involves clinical investigations.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Processa Clinical Site
Charleston, South Carolina, 29407
Processa Clinical Site
Milwaukee, Wisconsin, 53266
Processa Clinical Site
Rochester, Minnesota, 55905
Processa Clinical Site
San Diego, California, 92123
About this study
Official title: A Randomized, Double-blind, Placebo-controlled Clinical Trial to Evaluate the Efficacy and Safety of PCS499 in Treating Ulcerations in Patients Who Have Necrobiosis Lipoidica
This is a randomized, double blind, placebo controlled study that will evaluate the efficacy and safety of PCS499 as compared to placebo for the treatment of ulcerations of patients with necrobiosis lipoidica (NL) and will inform the design of future studies. Approximately 20 ulcerated NL patients who also meet other inclusion/exclusion criteria will be enrolled in the study. The primary objective of this study is to evaluate the efficacy of PCS499 as compared with placebo in ulcerated patients with Necrobiosis Lipoidica.
- Conditions
- Necrobiosis Lipoidica
- Being tested
- PCS499, placebo
- Medical subject headings
- Necrobiosis Lipoidica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PCS499Drug
PCS499 900mg twice a day with food
placeboOther
placebo comparator
| Study group | Role | What they receive |
|---|---|---|
| PCS499 900mg BID | Experimental | PCS499 900mg twice a day with food Receives: Drug: PCS499 |
| Placebo | Placebo comparator | similar in appearance to active study drug Receives: Other: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of ulcer closure as assessed by skin exam in patients treated with PCS499 as compared to placebo
Evaluation of proportion of patients with wound closure based on skin evaluation in patients with NL as compared with placebo
Measured: ~ 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2021
- Study start12 Apr 2021
- Primary completion28 Feb 2023
- Study completion14 Apr 2023
Why it stopped: lack of enrollment of this ultra rare disease
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Necrobiosis LipoidicaStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Processa Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sian Bigora, PharmD, Study director, Processa Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04800562
- Sponsor's protocol ID
- PCS499-NL02
- Status last verified
- Aug 2023
- First submitted
- 10 Mar 2021
- Last updated
- 3 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- 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 3 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled Clinical Trial to Evaluate the Efficacy and Safety of PCS499 in Treating Ulcerations in Patients Who Have Necrobiosis Lipoidica
- ClinicalTrials.gov ID
- NCT04800562
- Acronym / sponsor ID
- PCS499-NL02
- Conditions
- Necrobiosis Lipoidica
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: lack of enrollment of this ultra rare disease
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Processa PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 12 Apr 2021 (actual)
- Primary completion
- 28 Feb 2023 (actual)
- Study completion
- 14 Apr 2023 (actual)
- First posted
- 16 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a randomized, double blind, placebo controlled study that will evaluate the efficacy and safety of PCS499 as compared to placebo for the treatment of ulcerations of patients with necrobiosis lipoidica (NL) and will inform the design of future studies. Approximately 20 ulcerated NL patients who also meet other inclusion/exclusion criteria will be enrolled in the study. The primary objective of this study is to evaluate the efficacy of PCS499 as compared with placebo in ulcerated patients with Necrobiosis Lipoidica.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PCS499 900mg BID | Experimental | Drug: PCS499PCS499 900mg twice a day with food |
| Placebo | Placebo comparator | Other: placebosimilar in appearance to active study drug |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Male or female patients age 18 to 80 years of age, inclusive, at Screening.
- Biopsy-confirmed diagnosis of NL. Biopsies of continually active lesions performed outside of this clinical study will need to be reviewed and the diagnosis confirmed by the study pathologist. For patients with no previous history of biopsy, no biopsy within the previous 5 years, a biopsy that is not confirmed to be NL, or newly active lesion, a biopsy to confirm a diagnosis of NL will be performed at the Screening visit.
- Ulcers on a single leg ("reference leg") at Baseline should consist of at least one (1) ulcer with a minimum ulcer surface area of 1 cm2, total ulcer area of a minimum of 2 cm2, and no more than 6 ulcers. If ulcers are present on both legs, the Investigator will select the "reference leg". The ulcer(s) on the reference leg ("reference ulcers") and all other ulcers associated with the patient's NL ("other ulcers") present at the Baseline visit will be followed in the study.
- Women of childbearing potential must have a negative serum pregnancy test at the Screening Visit and a negative urine pregnancy test at Baseline before dosing. Women of childbearing potential must use one of the following acceptable methods of contraception throughout the study: oral contraceptive medication, intrauterine device (IUD), hormonal implants, injectable contraceptive medications, double-barrier methods, or tubal ligation.
- Females who are postmenopausal (age-related amenorrhea >= 12 consecutive months and increased follicle-stimulating hormone [FSH] > 40 mIU/mL. If necessary to confirm postmenopausal status, an FSH will be drawn at Screening) or who have undergone hysterectomy or bilateral oophorectomy are exempt from pregnancy testing.
Key exclusion criteria (39 listed)
- Current or previous (within 4 weeks of Baseline) treatment with:
- Current or previous (within 12 weeks of Baseline) treatment with any biologic therapy (e.g., adalimumab, etanercept, infliximab, anakinra, etc.).
- Phototherapy/photochemotherapy (NBUVB, UVB, PUVA) within 6 weeks prior to Baseline
- Skin grafting, or other surgical procedure (other than debridement) within 6 weeks prior to Baseline.
- History of drug allergy, including but not limited to pentoxifylline or other xanthine derivatives, or other allergy, which in the opinion of the Investigator, contraindicates participation.
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 |
|---|---|---|
| PCS499PCS499 900mg twice a day with food | Drug | PCS499 900mg BID |
| placeboplacebo comparator | Other | Placebo |
Primary outcome
Incidence of ulcer closure as assessed by skin exam in patients treated with PCS499 as compared to placebo
Time frame: ~ 6 months
Evaluation of proportion of patients with wound closure based on skin evaluation in patients with NL as compared with placebo
Results and safety
The study was stopped early (reason given: lack of enrollment of this ultra rare disease). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2023.
- Individual participant dataSharing planned: No
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.
Sian Bigora, PharmD study director
Processa Pharmaceuticals
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PCS499 in people with Necrobiosis Lipoidica, run by Processa Pharmaceuticals at 4 sites in United States.
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 9 inclusion and 39 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 3 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. 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. Some participants in this study may receive a placebo. 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?
The study was stopped early (reason given: lack of enrollment of this ultra rare disease). Results are not available in the source record.
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?
Incidence of ulcer closure as assessed by skin exam in patients treated with PCS499 as compared to placebo (time frame: ~ 6 months). 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 3 Aug 2023.