Study of the Effect of Fostamatinib Upon Cutaneous Inflammation in the Setting of Hidradenitis Suppurativa

An Exploratory, Proof-of-Concept Study of the Effect of Fostamatinib Upon Cutaneous Inflammation in the Setting of Hidradenitis Suppurativa

A 16 week exploratory, Proof of Concept study to evaluate the effect of Fostamatinib (12 weeks of treatment) upon cutaneous inflammation in Hidradenitis Suppurativa.

Study Overview

• Status: Completed
• Conditions: Hidradenitis Suppurativa
• Intervention / Treatment: Drug: Fostamatinib

Detailed Description

Sample size: 20 patients Study duration: 16 weeks

Primary Endpoints:

Alterations in Gene Expression Profiling, cell counts (CD3+, CD11c+, Neutrophil Elastase+, CD20+, CD138+) at Week 4 compared to Baseline.

Alterations in Gene Expression Profiling, cell counts (CD3+, CD11c+, Neutrophil Elastase+, CD20+, CD138+) at Week 12 compared to Baseline.

Secondary Endpoints:

Safety and Tolerability (Grade 2/3 Adverse Effects); Changes in Disease Activity (Measures by AN count and IHS4 scoring).

Study procedures Clinical Examination, Patient Questionnaires, Skin Biopsy, Blood Draw, medical photography

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Trina Vincent, R.N.; Phone Number: +61280381044; Email: trina.vincent@holdsworthhouse.com.au
• Study Contact Backup: Name: John Frew, MBBS; Phone Number: 02 93317228; Email: john.frew@holdsworthhouse.com.au

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2010
      • Holdsworth House Medical Practice

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Moderate to Severe (Hurley Stage 2 or 3) Hidradenitis Suppurativa as determined by the PI

Exclusion Criteria:

• Uncontrolled hypertension (systolic blood pressure [BP] ≥135 mmHg and/or diastolic BP ≥85 mmHg), unstable angina, congestive heart failure of New York Heart Association classification III or IV, serious cardiac arrhythmia requiring treatment (exceptions: atrial fibrillation, paroxysmal supraventricular tachycardia) at screening.
• History of myocardial infarction within 3 months prior to screening.
• Shock or hypotension requiring vasoactive peptides, such as dopamine, norepinephrine, epinephrine, or dobutamine at screening or at randomization.
• Renal function impairment with creatinine clearance <30 mL/min at screening (estimated glomerular filtration rate will be calculated using the modification of diet in renal disease equation).
• Liver function impairment with aspartate aminotransferase/alanine aminotransferase >=3x the upper limit of normal or bilirubin >2X the upper limit of normal at screening.
• Neutrophil count <1000/µL at screening.
• History of an allergic reaction or hypersensitivity to the study treatment or any component of the study treatment formulation.
• Has documented HIV infection or documented, active hepatitis B or hepatitis C infection.
• Women lactating, pregnant or of childbearing potential who are not willing to avoid becoming pregnant during the study.
• Individuals with concurrent use of systemic antibiotics/ oral retinoids/ systemic immunosuppressants (those with prior use of these medications must have conducted a washout period of 4 weeks or 5 half-lives of the drug, whichever is longer).
• Any medical, psychological or social condition that, in the opinion of the Investigator, would jeopardize the health or well-being of the participant during any study procedures or the integrity of the data.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Open Label Fostamatinib; Open label Fostamatinib 100mg dose adjusted by the Principal Investigator after week 1	Drug: Fostamatinib; Open label Fostamatinib; Other Names: Tavalisse; R935788

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Week 4 evaluation	Alterations in Gene Expression Profiling, cell counts (CD3+, CD11c+, Neutrophil Elastase+, CD20+, CD138+) at Week 4 compared to	4 weeks
Week 12 evaluation	Alterations in Gene Expression Profiling, cell counts (CD3+, CD11c+, Neutrophil Elastase+, CD20+, CD138+) at Week 12 compared to Baseline	12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Grade 2/3 Adverse Events	Number of Grade 2/3 Adverse Effects of Fostamatinib over the 12 Week course of treatment.	12 weeks
Abscess and Nodule Count Week 4	Changes in Abscess and Nodule count at Week 4 compared to baseline	4 weeks
International Hidradenitis Suppurativa Severity Score (IHS4) Week 4	Changes in IHS4 score at Week 4 compared to baseline	4 weeks
Abscess and Nodule count week 12	Changes in Abscess and Nodule count at Week 12 compared to baseline	12 weeks
International Hidradenitis Suppurativa Severity Score (IHS4) Week 12	Changes in IHS4 score at Week 12 compared to baseline	12 weeks
Physician Rated Overall Disease Severity	Changes in physician rated overall disease severity by visual analogue scale (0-10) at week 12 compared to baseline	12 weeks
Dermatology Life Quality Index (DLQI)	Changes in the DLQI (0-30) at week 12 compared to baseline	12 weeks

Collaborators and Investigators

• Sponsor: Holdsworth House Medical Practice
• Collaborators: Rigel Pharmaceuticals
• Investigators: Principal Investigator: John Frew, MBBS, Holdsworth House Medical Practice

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2021
• Primary Completion (Actual): January 13, 2023
• Study Completion (Actual): January 27, 2023

Study Registration Dates

• First Submitted: August 30, 2021
• First Submitted That Met QC Criteria: September 3, 2021
• First Posted (Actual): September 10, 2021

Study Record Updates

• Last Update Posted (Actual): March 1, 2023
• Last Update Submitted That Met QC Criteria: February 27, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Keywords: fostamatinib; HS; Tavalisse
• Additional Relevant MeSH Terms: Pathologic Processes; Sweat Gland Diseases; Skin Diseases; Infections; Bacterial Infections; Bacterial Infections and Mycoses; Skin Diseases, Infectious; Suppuration; Skin Diseases, Bacterial; Inflammation; Hidradenitis Suppurativa; Hidradenitis
• Other Study ID Numbers: JFR-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes