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

Phase 2

Completed

• Conditions: Hidradenitis Suppurativa
• Interventions: Drug: Placebo Oral Tablet; Drug: Apremilast

• Registration Number: NCT03049267
• Lead Sponsor: M.B.A. van Doorn

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.

Study Timeline

• Study First Submitted: 2016-10-25
• Study Start: 2017-02-02
• Study First Submitted QC: 2017-02-07
• Study First Posted: 2017-02-10
• Primary Completion: 2017-12-06
• Study Completion: 2018-06-28
• Status Verified: 2018-07
• Last Update Submitted: 2018-07-23
• Last Update Posted: 2018-07-24

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo Oral Tablet	Placebo Oral Tablet	N=5
Apremilast	Apremilast	N=15

Primary Outcome Measures

Name	Time	Method
Change of expression levels of inflammatory cytokine mRNA in HS lesional skin.	t=16 weeks	measurement by qPCR
Change of expression levels of inflammatory cytokine protein in HS lesional skin.	t=16 weeks	measurement by ELISA

Secondary Outcome Measures

Name	Time	Method
Hidradenitis Suppurativa Physician's Global Assessment (HS-PGA) score	t=0 weeks, t=4 weeks, t=16 weeks	Based on the HS lesion count
Hidradenitis Suppurativa Clinical Response (HiSCR)	t=0 weeks, t=16 weeks	Based on the AN count; The proposed definition of 50% and 30% responders to treatment (HiSCR achievers) is respectively: (i) at least a 50% and 30% reduction in ANs, (ii) no increase in the number of abscesses, and (iii) no increase in the number of draining fistulas from baseline.
Dermatology Life Quality Index (DLQI)	t=0 weeks, t=4 weeks, t=16 weeks	To assess the patient reported outcome measures (PROM) quality of life
Abscesses count	t=0 weeks, t=4 weeks, t=16 weeks	Total number of abscesses \[A\]
Nodule count	t=0 weeks, t=4 weeks, t=16 weeks	Total number of inflammatory \[N\] and non-inflammatory nodules
Numerical Rating Scale (NRS)	t=0 weeks, t=4 weeks, t=16 weeks	To assess the patient reported outcome measures (PROMs) pain, pruritus and patient disease global assessment score;
Incidence of Treatment-Emergent Adverse Events	Multiple time points between t=0 weeks and t=16 weeks	Vital signs: heart rate, temperature, blood pressure. Patient reported adverse events Safety laboratories: White blood cell count, Absolute neutrophil count, Hemoglobin, Platelets, Serum Creatinine, ALT, Alkaline phosphatase
Fistula count	t=0 weeks, t=4 weeks, t=16 weeks	Total count of draining fistulas

Trial Locations

Locations (1)

Erasmus University Medical Center; 🇳🇱 Rotterdam, Netherlands