Short-term Safety, Efficacy and Mode of Action of Apremilast in Moderate Suppurative Hidradenitis

Short-term Safety, Efficacy and Mode of Action of Apremilast in Moderate Suppurative Hidradenitis: A Randomised Double-blind Placebo Controlled Trial

Sponsors

Lead Sponsor: M.B.A. van Doorn

Collaborator: Celgene

Source Erasmus Medical Center
Brief Summary

Study design: A double-blind randomised placebo-controlled trial

Intervention: Randomized placebo controlled treatment of 20 HS patients of which fifteen patients will be randomized to apremilast and five patients to placebo. The total duration of the treatment period per subject is 16 weeks.

Primary objectives: To evaluate the expression profile of inflammatory cytokines in HS lesional skin at week four (t=4) and week sixteen (t=16):

- of patients receiving apremilast compared to placebo;

- within both groups relative to baseline (t=0).

Secondary objectives:

- To prospectively evaluate the clinical efficacy of apremilast.

- To assess the effect of apremilast on patient reported outcomes measures.

- To assess the short-term safety and tolerability of apremilast in patients with hidradenitis suppurativa.

Detailed Description

Rationale:

Hidradenitis suppurativa (HS) is a chronic, inflammatory, recurrent, debilitating skin disease. It is characterized by painful, deep-seated, inflamed boils in the inverse areas of the body, most commonly the axillae, inguinal and anogenital regions.

Systemic therapy with immunosuppressive agents (systemic corticosteroids, dapsone, cyclosporin) has been investigated in the past decades and has shown limited efficacy. The use of the selective immunosuppressant apremilast has not yet been evaluated in HS. The investigators hypothesize a beneficial effect of apremilast in HS patients, similar to the efficacy of apremilast in psoriasis patients. Namely, it has been shown that the immune dysregulation in the pathogenesis of HS shows many similarities with that of psoriasis. Moreover, the TNF-α blocker adalimumab was registered for HS after approval for the treatment in patients with psoriasis.

Overall Status Completed
Start Date February 2, 2017
Completion Date June 28, 2018
Primary Completion Date December 6, 2017
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Change of expression levels of inflammatory cytokine mRNA in HS lesional skin. t=16 weeks
Change of expression levels of inflammatory cytokine protein in HS lesional skin. t=16 weeks
Secondary Outcome
Measure Time Frame
Abscesses count t=0 weeks, t=4 weeks, t=16 weeks
Nodule count t=0 weeks, t=4 weeks, t=16 weeks
Fistula count t=0 weeks, t=4 weeks, t=16 weeks
Hidradenitis Suppurativa Physician's Global Assessment (HS-PGA) score t=0 weeks, t=4 weeks, t=16 weeks
Hidradenitis Suppurativa Clinical Response (HiSCR) t=0 weeks, t=16 weeks
Numerical Rating Scale (NRS) t=0 weeks, t=4 weeks, t=16 weeks
Dermatology Life Quality Index (DLQI) t=0 weeks, t=4 weeks, t=16 weeks
Incidence of Treatment-Emergent Adverse Events Multiple time points between t=0 weeks and t=16 weeks
Enrollment 20
Condition
Intervention

Intervention Type: Drug

Intervention Name: Apremilast

Description: Fifteen patients will be supplied of apremilast for daily oral use; 16 weeks.

Arm Group Label: Apremilast

Other Name: Otezla, CC-10004

Intervention Type: Drug

Intervention Name: Placebo Oral Tablet

Description: Five patients will be supplied with placebo tablets with identical labeling as apremilast for daily use; 16 weeks.

Arm Group Label: Placebo Oral Tablet

Other Name: Placebo comparator

Eligibility

Criteria:

Key inclusion criteria:

- Adult (≥ 18 years of age) male or female patients with moderate HS according to a PGA of 3 on the 5-point HS-Physician Global Assessment (HS-PGA);

- HS of more than 6 months duration; have lesions in at least two anatomical locations.

Key exclusion criteria:

- Contra-indication for apremilast; previous use of apremilast; have any current and/or recurrent clinically significant skin condition in the treatment area other than HS;

- Presence of other uncontrolled major disease;

- Pregnant or lactating women.

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Errol Prens Principal Investigator Erasmus Medical Center
Location
Facility: Erasmus University Medical Center
Location Countries

Netherlands

Verification Date

July 2018

Responsible Party

Type: Sponsor-Investigator

Investigator Affiliation: Erasmus Medical Center

Investigator Full Name: M.B.A. van Doorn

Investigator Title: MD, PhD

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Apremilast

Type: Experimental

Description: N=15

Label: Placebo Oral Tablet

Type: Placebo Comparator

Description: N=5

Acronym SMASH
Patient Data No
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Double (Participant, Investigator)

Source: ClinicalTrials.gov