Funding

This study was funded by Krystal Biotech (ClinicalTrials.gov Identifier: NCT04917874). The study sponsors participated in the study design; the collection, analysis, and interpretation of the data; the writing of the report; and the decision to submit the article for publication. M. Peter Marinkovich also received funding from the Office of Research and Development at the Palo Alto Veterans Affairs Medical Center.

Conflicts of Interest

M. Peter Marinkovich has undertaken paid consultancy for Amryt Pharmaceuticals DAC, is a consultant and speaker for Krystal Biotech, and is an investigator for Abeona, Castle Creek Biosciences, and Krystal Biotech, through funding administered by Stanford University Office of Sponsored Research. Amy S. Paller has been an investigator for AbbVie, Arcutis, Dermavant, Eli Lilly, Incyte, Janssen, Krystal Biotech, and UCB; a consultant for BioCryst, Boehringer-Ingelheim, Bristol Myers Squibb, Dermavant, Johnson & Johnson, Krystal Biotech, LEO Pharma, Mitsubishi Tanabe Pharma, Novartis, Primus Pharmaceuticals, Procter and Gamble, Regeneron, Sanofi/Genzyme, TWI Biotechnology, and UCB; and is on the data safety monitoring board for AbbVie, Abeona, Catawba, and Galderma. Amy S. Paller is an Editorial Board member of the American Journal of Clinical Dermatology; she was not involved in the selection of peer reviewers for the manuscript nor any of the subsequent editorial decisions. Shireen V. Guide has been an investigator for Arcutis, Boehringer-Ingelheim, Castle Biosciences, Dermavant, Incyte, Jeune, Krystal Biotech, Novartis, Pfizer, and Sanofi, and a consultant and speaker for Krystal Biotech. Mercedes E. Gonzalez has been an investigator for AbbVie, Arcutis, Amgen, Anterogen, Dermavant, Eli Lilly, Incyte, Krystal Biotech, Neilsen Biosciences, Novartis, Regeneron, and Sanofi; a consultant with honorarium for Abeona, Apogee, Arcutis, Alphyn, Dermavant, Eli Lilly, Incyte, Krystal Biotech, and Verrica Pharmaceuticals, and on the speaker’s bureau for Verrica Pharmaceuticals, Regeneron, Sanofi, Genzyme, Eli Lilly, Abbvie, Pfizer, and Krystal Biotech. Anne W. Lucky has been an investigator for Krystal Biotech and Phoenicis. Işın Sinem Bağcı has been an investigator for Castle Creek Biosciences and Krystal Biotech. Brittani Agostini, Kolleen Fitzgerald, Meghan M. Conner, and Suma M. Krishnan are employees and shareholders of Krystal Biotech. Shijie Chen and Hubert Chen were employees and shareholders of Krystal Biotech at the time of the analysis.

Ethics Approval

This study was performed in accordance with the Declaration of Helsinki, the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use Good Clinical Practice guidelines, and other applicable laws and regulations. Approval was obtained from the Stanford University Administrative Panel on Human Subjects in Medical Research (Site 1), Advarra (Sites 2 and 3), the Cincinnati Children’s Institutional Review Board (Site 4), and the Ann & Robert H. Lurie Children’s Hospital of Chicago Institutional Review Board (Site 5).

Consent to Participate

All participants gave written informed consent/assent to participate in the research. At least one parent or guardian for each adolescent patient provided written informed consent.

Consent for Publication

Patients signed an informed consent regarding publishing their data and photographs.

Availability of Data and Material

All requests for data will be reviewed by the clinical sites and the study sponsor, Krystal Biotech, to verify whether the request is subject to any intellectual property or confidentiality obligations. Requests for access to the patient-level data from this study can be submitted via e-mail to medinfo@krystalbio.com with detailed proposals for approval. A signed data access agreement with the sponsor is required before accessing shared data.

Code Availability

Not applicable.

Authors’ Contributions

All authors meet the International Committee of Medical Journal Editors criteria for authorship of this article. Study conception and design: SMK and HC; acquisition, analysis, and interpretation of data: MPM, ASP, SVG, MEG, AWL, and ISB; analysis of data: SC; supervision: SMK and BA; technical support: KF; formal analysis and writing (original draft preparation): MMC. All authors contributed to the critical revision of the manuscript for important intellectual content and approved the final version.