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A Phase II Open Label Clinical Trial of Etanercept for the Treatment of Hidradenitis Suppurativa

Phase 2
18 Years
Not Enrolling
Hidradenitis Suppurativa

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Trial Information

A Phase II Open Label Clinical Trial of Etanercept for the Treatment of Hidradenitis Suppurativa

Purpose: The primary objective of this study is to determine the safety and estimate the
efficacy of etanercept for the treatment of hidradenitis suppurativa. The secondary
objective of this study is to determine the impact of etanercept treatment of hidradenitis
suppurativa on skin related quality of life.


Each patient will participate in this study for a maximum of 6 months. The study consists of
a screening visit, baseline assessment visit (Day 1), a treatment period (Week 2 - Week 14),
and a one month follow-up visit (Week 18 visit). The total duration of the study will be
approximately 2 years.

Subject Recruitment and Selection:

It is planned that enrollment will be 12-21 patients.


Hidradenitis suppurativa is a physically, psychologically, and socially disabling disease
characterized by inflammatory, cystic papules and nodules affecting the underarms, groin,
perineum, and breasts. Lesions can become erosive and often develop deep abscesses and sinus
tracts and drain foul smelling pus. Left untreated, hidradenitis can result in permanent
scarring. In the most severe cases, characterized by chronic ulceration and granulation,
there may be an increased risk of aggressive squamous cell carcinoma.

Current treatment of hidradenitis consists of intra-lesional injections of steroids, topical
and/or systemic antibiotics, hormonal therapy, and isotretinoin. For many patients with
severe hidradenitis (stage II and III), these therapies are often ineffective. Patients with
stage II and III hidradenitis often require surgical excision of the affected area (a highly
morbid procedure) to control the disease. Unfortunately, for most patients with
hidradenitis, existing therapies are ineffective and there is an unmet medical need for
therapies that control this disabling and destructive disease.

The pathophysiology of hidradenitis is unknown. The leading hypothesis is that occlusion of
apocrine ducts leads to severe dilatation, apocrine gland inflammation, with ensuing
bacterial growth and neutrophilic infiltration and destruction of the duct. The importance
of the immune dysregulation in hidradenitis is further demonstrated by its association in
many individuals with inflammatory bowel disease.

The pathologic immune reaction to follicular occlusion in hidradenitis suggests a strong
rationale for the use of treatments that may neutralize this inflammatory reaction. In fact,
the existing standard treatment of hidradenitis is intra-lesional injections of steroids, in
the effort to minimize the destructive nature of the immune response. Medications that are
broadly immuno-suppressive, such as cyclosporine, have also been used to successfully treat
hidradenitis, but are limited by organ toxicity. This rationale is further supported by case
reports of dramatic and rapid (e.g. within days) improvement in hidradenitis treated with
infliximab, a monoclonal antibody that blocks TNF-alpha.

Etanercept is a TNF-alpha inhibitor currently FDA approved to treat various inflammatory
disorders including rheumatoid arthritis, psoriatic arthritis and psoriasis. By inhibiting
TNF-alpha, etanercept stops the inflammatory cascade by binding directly to circulating
TNF-alpha and inhibiting its binding to cell surface receptors.

Etanercept has been used in over 200,000 patients world wide for more than 5 years and has a
well established safety record. The most common adverse effect of etanercept is injection
site reaction which is typically mild and self-limited. Currently, laboratory monitoring for
patients being treated with etanercept is not recommended according to its label since the
drug has not been associated with a significant incidence of laboratory abnormalities.

The well established safety profile of etanercept and its potent role in suppressing
pathologic immune responses through TNF-inhibition make it a promising agent for the
treatment of hidradenitis suppurativa. In this phase II clinical trial, we will determine
preliminary evidence of safety and estimate the efficacy of etanercept in the treatment of
hidradenitis. This study will provide critical preliminary data for planning larger pivotal

Research Design:

This is a phase II, open label, two-stage clinical trial of etanercept for the treatment of
hidradenitis. This design is a widely accepted method for early investigations of safety and
efficacy of medications for new indications. Etanercept 50 mg will be administered
subcutaneously once a week for 12 weeks in an open label manner. At week 12, the etanercept
dose will be tapered to 25 mg subcutaneously once a week for 2 weeks.

This is an 18 week study. Subjects will be screened to determine eligibility. Day -95 to -3
will be a screening period which will allow washout of concurrent therapies if necessary.

Potential Risks:

Etanercept was generally well tolerated in patients with rheumatoid arthritis, psoriatic
arthritis, and ankylosing spondylitis. Adverse events that were reported in at least 3% of
all patients with higher incidence in patients treated with etanercept than placebo are:

- Injection site reaction;

- Infection;

- Headache;

- Nausea;

- Rhinitis;

- Dizziness;

- Pharyngitis;

- Cough;

- Asthenia;

- Abdominal pain;

- Rash;

- Peripheral edema;

- Respiratory disorder;

- Dyspepsia;

- Sinusitis;

- Vomiting;

- Mouth ulcer;

- Alopecia

Potential Benefits:

No direct benefits from participation in the study can be guaranteed. The study medication
will be provided by the Financial Sponsor at no charge.

Inclusion Criteria:

- Subjects must be able to give informed consent.

- Severe hidradenitis suppurativa clinically confirmed by the investigator and defined
as recurrent abscesses, with 4 or more lesions (e.g. nodules or abscesses) with sinus
track and scar formation (e.g. Stage II or III disease) that has not responded to
previous standard therapies such as topical or oral antibiotics, or intralesional
injections of steroids.

- Age 18 or older.

- Willingness to use at least one form of effective contraception during the study
period and for one month after discontinuation of etanercept if female and of child
bearing capacity or if male. If the patient elects to use a hormonal form of
contraception then the patient must be on the same form of hormonal contraception for
90 days prior to the start of Etanercept and must plan to continue using the same
form of hormonal contraception for the duration of the study (e.g. until week 18).

Exclusion Criteria:

- Use of oral or topical antibiotics, isotretinoin, or intralesional steroids within 30
days prior to day 0 or at any time during the study treatment period.

- Use of systemic immunosuppressants within 90 days prior to day 0 of this study.

- Use of an investigational medication 90 days prior to day 0 of this study.

- Use of a live vaccine 90 days prior to day 0 of this study.

- Any previous use of TNF- α inhibitors.

- If using a hormonal form of contraception, the patient will be excluded if they have
not used the same form of hormonal contraception for 90 days prior to the start of
the etanercept (e.g. day 0) or are not willing to continue the use of the same form
of hormonal contraception for the duration of the study.

- Active infection within 30 days of day 0 of the study that is moderate (discomfort
sufficient to reduce or affect normal daily activity) or severe (incapacitating with
inability to work or perform normal daily activities) or requires treatment with

- History of tuberculosis or other mycobacterial disease or positive screening visit
PPD (≥ 5 mm).

- Known history of an immuno-suppressing disease (e.g. HIV)

- Clinically significant abnormality in liver function, renal function, chemistry panel
or CBC (AST or ALT ≥ 2 times the laboratory's upper limit of normal, hemoglobin <
10.0 g/dL, platelet count <125,000/cm3, white blood count <3,500 cells/cm3 or >
15,000 cells/cm3, or serum creatinine ≥ 2.0 mg/dL) or severe co-morbidities defined
as diabetes mellitus requiring insulin, congestive heart failure, history of
myocardial infarction, unstable angina, uncontrolled hypertension (systolic blood
pressure > 180 mmHg or diastolic blood pressure >110 mmHg), severe pulmonary disease
(requiring oxygen therapy), history of cancer within 5 years (other than resected
basal cell or squamous cell carcinoma and in situ cervical cancer), known history of
active hepatitis B or C or HIV infection, history of demyelinating diseases or lupus.

- Pregnancy or lactation

- History of alcohol or drug abuse within 12 months of screening visit

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Physician's Global Assessment Score - Response Rate (Percentage)

Outcome Description:

Efficacy was measured using the Physician Global Assessment (PGA). Responders were classified as those achieving at least a 50% reduction on the Physician Global Assessment score at week 12 compared with baseline. A response rate was calculated as the percentage of patients that were classified as responders at 12-weeks. PGA was scored at baseline and at 12 weeks on a 100-mm visual analog scale, with 0 indicating no disease and 100-mm indicating severe disease.

Outcome Time Frame:

12 weeks

Safety Issue:


Principal Investigator

Joel Gelfand, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of Pennsylvania


United States: Institutional Review Board

Study ID:




Start Date:

April 2005

Completion Date:

November 2008

Related Keywords:

  • Hidradenitis Suppurativa
  • clinical trial; efficacy; etanercept; hidradenitis suppurativa; quality of life; safety; tnf
  • Hidradenitis
  • Hidradenitis Suppurativa