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About the Valo Study

The VALO study is sponsored by Palvella Therapeutics in collaboration with the Pachyonychia Congenita (PC) Project. VALO is a multi-center, four-part, Phase 2/3 study evaluating the safety and effectiveness of PTX-022 (experimental topical rapamycin) in approximately 60 adults with moderate-to-severe PC. The VALO study will determine if PTX-022 is safe and effective in helping improve the overall condition of adults with moderate-to-severe PC, measured by their difficulty in carrying out daily activities that involve being on their feet.

About Pachyonychia Congenita

Pachyonychia congenita (PC) is a rare, autosomal-dominant disease caused by a mutation in one or more of the genes responsible for the production of keratins. The production of mutated keratin filaments is the primary driver of pachyonychia congenita. Ultimately this genetic defect leads to the exquisite plantar pain derived from blistering, the presence of neurovascular structures, dermal inflammation and atypical painful callous formations on the feet. Furthermore, the stress from simple activities such as walking or standing causes trauma to these weakened cells, which triggers the production of mutated keratins, which in turn exacerbate these manifestations and contribute to increased pain and debilitation. There are currently no FDA- or EMA-approved treatments for PC.

About PTX-022 (QTORIN 3.9% rapamycin anhydrous gel)

PTX-022 is a topical formulation of rapamycin being developed to treat PC. Rapamycin has been shown to inhibit the production of a kinase (a type of protein responsible for transcription) called mTOR, which is involved in the production of certain keratin proteins. In disorders such as PC that are associated with overproduction of aberrant keratin proteins, the inhibition of mTOR may decrease the translation of mutant keratin-expressing genes and potentially block signaling pathways linked to skin fragility, keratoderma, pain and ambulatory impairment.

Interested in Enrolling?

The trial will require up to 6 clinic visits and will last up to 36 weeks. To qualify for VALO, participants must meet certain criteria, including genetically confirmed diagnosis of KRT6A, KRT6B or KRT16 mutation.

Deciding whether to participate in a clinical trial is a voluntary and personal decision. We encourage you to talk with your family about whether enrolling in any clinical trial is the right decision for you. To learn more or to get started, please complete the form below.

Travel for Trial Locations

Travel assistance will be provided for participants in VALO and up to one caretaker to accompany them. Transportation (airfare, trains, car services) and housing (hotels) will be booked for you through a travel service that specializes in helping participants in clinical trials. The travel service specializes in assisting participants enrolled in trials for rare diseases and understands the challenges associated with traveling with PC.

Get Started
Trial Requirements
  • – Moderate-to severe PC
  • – Adults, age 18 or older
  • -Able and willing to comply with all protocol-requiring activities

Find a Trial Location

Clear Search
Arizona Research Center

15601 N 28th Ave, Phoenix, AZ 85053, USA

Stanford University

700 Welch Rd, Palo Alto, CA 94305, USA

Yale University

HRT (333) Cedar Street, 604c, New Haven, CT 06519

International Dermatology Research

8739 West Flagler Street, Suite 200 Miami, FL 33144

Northwestern University

676 N. St. Clair Street, Suite 1600 Chicago, IL 60611

Minnesota Clinical Study Center

7205 University Avenue N.E. Fridley, MN 55432

Oregon Health and Science University

3303 SW Bond Avenue, Portland, OR 97239

Paddington Testing Co.

1845 Walnut Street, Suite 1650, Philadelphia, PA 19103

University of Utah

243 East 6100 South Salt Lake City, UT 84107, USA

Arizona Research Center

Travel Assistance Available

Stanford University

Travel Assistance Available

Yale University

Travel Assistance Available

International Dermatology Research

Travel Assistance Available

Northwestern University

Travel Assistance Available

Minnesota Clinical Study Center

Travel Assistance Available

Oregon Health and Science University

Travel Assistance Available

Paddington Testing Co.

Travel Assistance Available

University of Utah

Travel Assistance Available