Study of the Effect of Fostamatinib Upon Cutaneous Inflammation in the Setting of Hidradenitis Suppurativa
Study Details
Study Description
Brief Summary
A 16 week exploratory, Proof of Concept study to evaluate the effect of Fostamatinib (12 weeks of treatment) upon cutaneous inflammation in Hidradenitis Suppurativa.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2 |
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 Design
Arms and Interventions
| 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:
|
Outcome Measures
Primary Outcome Measures
- Week 4 evaluation [4 weeks]
Alterations in Gene Expression Profiling, cell counts (CD3+, CD11c+, Neutrophil Elastase+, CD20+, CD138+) at Week 4 compared to
- Week 12 evaluation [12 weeks]
Alterations in Gene Expression Profiling, cell counts (CD3+, CD11c+, Neutrophil Elastase+, CD20+, CD138+) at Week 12 compared to Baseline
Secondary Outcome Measures
- Grade 2/3 Adverse Events [12 weeks]
Number of Grade 2/3 Adverse Effects of Fostamatinib over the 12 Week course of treatment.
- Abscess and Nodule Count Week 4 [4 weeks]
Changes in Abscess and Nodule count at Week 4 compared to baseline
- International Hidradenitis Suppurativa Severity Score (IHS4) Week 4 [4 weeks]
Changes in IHS4 score at Week 4 compared to baseline
- Abscess and Nodule count week 12 [12 weeks]
Changes in Abscess and Nodule count at Week 12 compared to baseline
- International Hidradenitis Suppurativa Severity Score (IHS4) Week 12 [12 weeks]
Changes in IHS4 score at Week 12 compared to baseline
- Physician Rated Overall Disease Severity [12 weeks]
Changes in physician rated overall disease severity by visual analogue scale (0-10) at week 12 compared to baseline
- Dermatology Life Quality Index (DLQI) [12 weeks]
Changes in the DLQI (0-30) at week 12 compared to baseline
Eligibility Criteria
Criteria
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.
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Has documented HIV infection or documented, active hepatitis B or hepatitis C infection.
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Women lactating, pregnant or of childbearing potential who are not willing to avoid becoming pregnant during the study.
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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).
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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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Holdsworth House Medical Practice | Sydney | New South Wales | Australia | 2010 |
Sponsors and Collaborators
- Holdsworth House Medical Practice
- Rigel Pharmaceuticals
Investigators
- Principal Investigator: John Frew, MBBS, Holdsworth House Medical Practice
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- JFR-001