Hidradenitis Suppurativa
Conditions
Keywords
fostamatinib, HS, Tavalisse
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.
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
Interventions
DRUGFostamatinib
Open label Fostamatinib
Sponsors
Rigel Pharmaceuticals
Holdsworth House Medical Practice
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| Grade 2/3 Adverse Events | 12 weeks | Number of Grade 2/3 Adverse Effects of Fostamatinib over the 12 Week course of treatment. |
| 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 |
| International Hidradenitis Suppurativa Severity Score (IHS4) Week 12 | 12 weeks | Changes in IHS4 score at Week 12 compared to baseline |
| Abscess and Nodule Count Week 4 | 4 weeks | Changes in Abscess and Nodule count at Week 4 compared to baseline |
Countries
Australia
Outcome results
None listed