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Journal of the American Academy of Dermatology
Sous presse. Epreuves corrigées par l'auteur. Disponible en ligne depuis le mardi 4 février 2020
Doi : 10.1016/j.jaad.2019.09.014
accepted : 9 September 2019
Phase 2 trial of a neurokinin-1 receptor antagonist for the treatment of chronic itch in patients with epidermolysis bullosa: A randomized clinical trial

Albert S. Chiou, MD a, , Sara Choi, BA a, Melissa Barriga, MSc a, Yana Dutt-Singkh, MD a, Daniel C. Solis, BA b, Jaron Nazaroff, BS c, Irene Bailey-Healy, MLS a, Shufeng Li, MS a, Kim Shu, MBA d, Mark Joing, MBA d, Paul Kwon, MD d, Jean Y. Tang, MD, PhD a
a Department of Dermatology, Stanford University School of Medicine, Redwood City, California 
b Department of Internal Medicine, University of California, Riverside, California 
c University of California, Irvine School of Medicine, Irvine, California 
d Menlo Therapeutics Inc (formerly Tigercat Pharma, Inc), Redwood City, California 

Reprint requests: Albert S. Chiou, MD, Department of Dermatology, Stanford University School of Medicine, 450 Broadway St, Pavilion C, 2nd Floor, Redwood City, CA 94063.Department of DermatologyStanford University School of Medicine450 Broadway StPavilion C, 2nd FloorRedwood CityCA94063

Chronic pruritus causes major morbidity in epidermolysis bullosa (EB). The substance P-neurokinin 1 receptor (SP-NK1) pathway is a promising target for treating EB-related pruritus.


To evaluate the safety and efficacy of the oral NK1 receptor antagonist serlopitant in treating moderate-severe pruritus in EB.


The study randomized 14 patients to serlopitant or placebo for 8 weeks, followed by a 4-week washout and optional open-label extension. The primary end point was change in itch as measured by the Numeric Rating Scale. Secondary end points were change in itch during dressing changes and wound size.


We observed greater itch reduction with serlopitant, equivalent to a 0.64-point comparative reduction on the 11-point Numeric Rating Scale by week 8, although this failed to meet statistical significance (P  = .11). More serlopitant patients achieved ≥3-point reduction compared with placebo (43% vs 14%, P  = .35). In post hoc analysis excluding 1 patient with a concurrent seborrheic dermatitis flare, serlopitant achieved significantly greater median itch reduction from baseline by week 4 (−2 points vs 0, P  = .01). We observed no statistically significant differences in secondary end points. Serlopitant was well-tolerated.


Small sample size due to disease rarity.


The potential itch reduction with serlopitant observed in this trial will be pursued by a larger powered trial (NCT03836001).

The full text of this article is available in PDF format.

Key words : chronic itch, drug response, epidermolysis bullosa, neurokinin 1 receptor, NK1 receptor, NK1 receptor antagonist, pruritus, serlopitant, substance P

Abbreviations used : AE, EB, NRS, RDEB, SAE, SP-NK1R

 Albert S. Chiou, MD, and Sara Choi, BA, are cofirst authors.
 Funding sources: This investigator-initiated study was enabled by a grant from the EB Research Partnership (EBRP) and Epidermolysis Bullosa Medical Research Foundation (EBMRF). Dr Chiou was supported by a Dermatology Foundation Medical Dermatology Career Development Award.
 Conflicts of interest: Authors Shu, Joing, and Kwon are employed by Menlo Therapeutics, which provided the investigational drug. Design of the study, interpretation of the data, and drafting of this manuscript were performed independently by the Stanford-affiliated authors. Authors Chiou, Choi, Barriga, Dutt-Singkh, Solis, Nazaroff, Bailey-Healy, Li, and Tang have no conflicts of interest to declare.
 IRB approval status: Reviewed and approved by Stanford Institutional Review Board (approval #37606).

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