A Clinical Trial to Evaluate PCS499 in Treating Ulcerations in Patients Who Have Necrobiosis Lipoidica

NCT04800562 | Terminated Phase 2 FDA Drug

• 1 other identifier: PCS499-NL02
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 4

Brief 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. 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

Outcome Measures

Primary Outcomes (1)

• 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

  ~ 6 months

Study Arms (2)

PCS499 900mg BID

EXPERIMENTAL

PCS499 900mg twice a day with food

Drug: PCS499

Placebo

PLACEBO COMPARATOR

similar in appearance to active study drug

Other: placebo

Interventions

PCS499 DRUG

PCS499 900mg twice a day with food

PCS499 900mg BID

placebo OTHER

placebo comparator

Placebo

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 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.
• 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.

You may not qualify if:

• 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.
• 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:

Sponsors & Collaborators

• Processa Pharmaceuticals: lead

Study Sites (4)

Processa Clinical Site

San Diego, California, 92123, United States

Location

Processa Clinical Site

Rochester, Minnesota, 55905, United States

Location

Processa Clinical Site

Charleston, South Carolina, 29407, United States

Location

Processa Clinical Site

Milwaukee, Wisconsin, 53266, United States

Location

MeSH Terms

Conditions

Necrobiosis Lipoidica

Condition Hierarchy (Ancestors)

Necrobiotic Disorders; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Metabolic; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Sian Bigora, PharmD

  Processa Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 10, 2021
• First Posted: March 16, 2021
• Study Start: April 12, 2021
• Primary Completion: February 28, 2023
• Study Completion: April 14, 2023
• Last Updated: August 3, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)