Trial to Determine the Immunomodulatory Activity of PTM-001 in Patients With Hidradenitis Suppurativa

Study Overview
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Brief Summary
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Study PTM-001-01 is a 12-week, randomized, placebo controlled, double blind study with a 12 week open-label extension to examine the immunomodulatory activity of PTM-001 in participants with Hidradenitis Suppurativa (HS). Participants will be randomized to receive PTM-001 (400 mg) or matching placebo every day for 12 weeks after which all participants will receive open-label PTM-001 400 mg daily for an additional 12 weeks. Randomization will be stratified by Hurley Stage.

Official Title
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A 12-Week Double-Blind, Placebo-Controlled Randomized Phase 2 Trial With a 12-Week Open-Label Extension to Determine the Immunomodulatory Activity of PTM-001 in Patients With Hidradenitis Suppurativa

Conditions
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Hidradenitis Suppurativa

Intervention / Treatment
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* Drug: PTM-001
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Had onset of symptoms consistent with HS at least 6 months prior to Screening. Has had active HS for at least 2 months. Has ≥ 5 HS abscesses or inflammatory nodules at Screening. Agrees to maintain baseline wound care and antibiotic therapy, including oral antibiotics, for the duration of the trial. Agrees to use contraception Exclusion Criteria: Has other skin disease or condition that can interfere with HS assessment. Has a positive test for TB, HIV, hepatitis B and/or hepatitis C at Screening. Has a history of retinopathy or known clinically significant cardiovascular or hematologic conditions. Has taken any biologic drug within 3 serum half-lives. A list of potential biologics and their half-lives Has started oral antibiotics within 28 days of Study Day 1. Has, within 2 weeks prior to Day 1, received a medication prohibited based on cytochrome P450 (CYP3A4) interaction Has such extensive disease that, in the opinion of the Investigator, it is difficult to discriminate between active lesions and scarring. Has more than 15 active tunnels at Screening. Is pregnant, nursing or considering becoming pregnant. Has a history of malignancy except non-melanoma skin cancer or cervical carcinoma in situ.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Patients will be randomized to receive PTM-001 (400 mg) or matching placebo every day for 12 weeks after which all patients will receive open-label PTM-001 400 mg daily for an additional 12 weeks
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PTM-001 400 mg daily for 12 weeks<br> | Drug: PTM-001<br>* PTM-001 (400 mg) every day for 12 weeks<br>|
| Placebo Comparator: Placebo daily for 12 weeks<br> | Drug: Placebo<br>* Matching placebo every day for 12 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Effect of PTM-001 on IL-1β protein levels in lesional skin biopsies |  | Baseline to Week 12 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Demonstrate a change in IL-1⍺, IL-23p19, IL-17A, IL-5, IL-6, TNF⍺, HBD-2 protein levels in lesional skin biopsies |  | Baseline to Week 12 | 
| Demonstrate a change in IL-1β, IL-1⍺, IL-23p19, IL-17A, IL-5, IL-6, TNF⍺, HBD-2 mRNA expression in lesional skin biopsies |  | Baseline to Week 12 | 
| Demonstrate a change in IL-1β, IL-1⍺, IL-23p19, IL-17A, IL-5, IL-6, TNF⍺, HBD-2 protein levels in serum |  | Baseline to Week 12 | 
| Demonstrate a change in serum amyloid A levels |  | Baseline to Week 12 | 
| Change in Pain, as measured using a 10-point numerical rating scale, 1 through 10, with 10 being the worst. |  | Baseline to Week 12 | 
| Change in Quality of Life using Dermatology Life Quality Index (DLQI) |  | Baseline to Week 12 | 
| Change in clinical status using the Hurley Staging |  | Baseline to Week 12 | 
| Change in clinical status using the International Hidradenitis Suppurative Severity Score System (IHS4) |  | Baseline to Week 12 | 
| Change in clinical status using the Hidradenitis Suppurative Clinical Response (HiSCR) |  | Baseline to Week 12 | 
| Change in clinical status using the Hidradenitis Suppurative Physician Global Assessment (HS-PGA) |  | Baseline to Week 12 | 
| Change in clinical status using the Hidradenitis Suppurative Area and Severity Index-Revised (HASI-R) |  | Baseline to Week 12 | 
| Change in abscesses and inflammatory nodules count (AN count) |  | Baseline to Week 12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hidradenitis Suppurativa, HS, Inflammatory skin disease, Humira, P2X7, IL-1β