A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita

NCT05180708 | Completed Phase 3 FDA Drug

• 1 other identifier: PALV-05
• Study Type: interventional
• Target: 87
• Locations: 2 countries
• Sites: 8

Brief Summary

This study evaluates the safety and efficacy of QTORIN 3.9% rapamycin anhydrous gel in the treatment of adults with Pachyonychia Congenita. This study includes a screening period, baseline period and 6-month treatment period.

Conditions

Pachyonychia Congenita

Outcome Measures

Primary Outcomes (1)

• Patient Global Assessment of Activities Difficulty Scale

  6 months

Secondary Outcomes (6)

• Pain at its worst as assessed by numerical rating scale

  6 months

• PROMIS physical function

  6 months

• Patient global impression of severity (PGI-S)

  6 months

• Patient global impression of change (PGI-C)

  6 months

• Clinician global impression of severity (CGI-S)

  6 months

Study Arms (2)

Active

ACTIVE COMPARATOR

Drug: QTORIN 3.9% rapamycin anhydrous gel

Vehicle

PLACEBO COMPARATOR

Drug: Vehicle

Interventions

QTORIN 3.9% rapamycin anhydrous gel DRUG

QTORIN 3.9% rapamycin anhydrous gel

Active

Vehicle DRUG

Matching vehicle (QTORIN anyhydrous gel)

Vehicle

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult patients, 18 years or older (16 years or older in the UK)
• Diagnosed with Pachyonychia Congenita (PC), genetically confirmed

You may not qualify if:

• Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications.
• Any significant concurrent condition (including conditions involving or inferior to the ankle) that could adversely affect the patient's participation and/or the assessment of the safety and efficacy in the study.
• Participation in any other interventional clinical research trial within 30 days prior to the screening visit or during the patient's participation in this study

Sponsors & Collaborators

• Palvella Therapeutics, Inc.: lead

Study Sites (8)

Stanford University

Palo Alto, California, 94304, United States

Location

Yale University

New Haven, Connecticut, 06519, United States

Location

Park Avenue Dermatology

Orange Park, Florida, 32073, United States

Location

Minnesota Clinical Study Center

Fridley, Minnesota, 55432, United States

Location

Oregon Health and Sciences University

Portland, Oregon, 97239, United States

Location

DermResearch, Inc.

Austin, Texas, 78759, United States

Location

University of Utah

Murray, Utah, 84107, United States

Location

Royal London Hospital

London, Whitechapel, E11FR, United Kingdom

Location

Related Links

• PC Project
• Palvella Therapeutics, Inc.

MeSH Terms

Conditions

Pachyonychia Congenita

Condition Hierarchy (Ancestors)

Ectodermal Dysplasia; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Nail Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• Amy Paller, MD

  Northwestern University

  STUDY CHAIR

• Joyce Teng, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

• David Hansen, MD

  University of Utah

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 20, 2021
• First Posted: January 6, 2022
• Study Start: November 29, 2021
• Primary Completion: June 30, 2023
• Study Completion: June 30, 2023
• Last Updated: August 29, 2024

Record last verified: 2024-08

Locations

US (7); GB (1)