A Study to Evaluate the Safety and Tolerability of PCS499 for the Treatment of Necrobiosis Lipoidica

NCT03698864 | Completed Phase 2 Results FDA Drug

• 1 other identifier: PCS499-NL01
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

Brief Summary

This is an open-label study that will evaluate the safety of PCS499 for the treatment of necrobiosis lipoidica (NL) and will inform the design of future studies. Approximately 12 NL patients (6-9 patients without ulceration and 3-6 patients with ulceration) who also meet other inclusion/exclusion criteria will be enrolled in the study. The primary objective of this study is to evaluate the safety and tolerability profile of PCS499 in patients with Necrobiosis Lipoidica.

Conditions

Necrobiosis Lipoidica

Outcome Measures

Primary Outcomes (1)

• Evaluation of Patients With Adverse Events

  Evaluation of Adverse Events/Serious Adverse Events (by type, severity, and relatedness)

  12 months

Study Arms (1)

PCS499 900mg twice a day

EXPERIMENTAL

Drug: PCS499

Interventions

PCS499 DRUG

PCS499 900mg twice a day with food

PCS499 900mg twice a day

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.
• Reference NL lesion with a score of 2 or greater on the Investigator Global Assessment: Necrobiosis Lipoidica (IGA:NL) Activity scale AND surface area with minimum size of 10 cm2. If more than one lesion is present, the reference lesion area is the lesion with the highest disease severity.
• 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, topical, or intralesional corticosteroids;
• Systemic pentoxifylline, theophylline, or cilostazol
• Oral or topical retinoid;
• Other systemic or topical immunosuppressant 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:
• Clinically significant cardiac disease including but not limited to unstable angina, acute myocardial infarction within 6 months of Baseline, and arrhythmia requiring therapy.

Sponsors & Collaborators

• Processa Pharmaceuticals: lead

Study Sites (2)

Processa Investigational Site

Philadelphia, Pennsylvania, 19104, United States

Location

Processa Investigational Site

Pittsburgh, Pennsylvania, 15213, 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

Limitations and Caveats

open label study

Results Point of Contact

• Title: Mary G. Nyberg, VP Clinical Operations
• Organization: Processa Pharmaceuticals

mnyberg@processapharmaceuticals.com

443-776-3133

Study Officials

• Maya Das, MD

  Processa Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 1, 2018
• First Posted: October 9, 2018
• Study Start: November 7, 2018
• Primary Completion: February 25, 2020
• Study Completion: February 25, 2020
• Last Updated: October 3, 2022
• Results First Posted: October 3, 2022

Record last verified: 2022-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)