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Clinical Trials/NCT05180708
NCT05180708CompletedPhase 3

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

Palvella Therapeutics, Inc.8 sites in 2 countries87 target enrollmentStarted: November 29, 2021Last updated:
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Conditions
Interventions

Trial Snapshot

Phase
Phase 3
Status
Completed
Enrollment
87
Locations
8
Primary Endpoint
Patient Global Assessment of Activities Difficulty Scale
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

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.

Study Design

Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Eligibility Criteria

Ages
18 Years to — (Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

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

Exclusion Criteria

  • •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

Arms & Interventions

Active

Active Comparator

Intervention: QTORIN 3.9% rapamycin anhydrous gel (Drug)

Vehicle

Placebo Comparator

Intervention: Vehicle (Drug)

Outcomes

Primary Outcomes

Patient Global Assessment of Activities Difficulty Scale

Time Frame: 6 months

Secondary Outcomes

  • PROMIS physical function(6 months)
  • Clinician global impression of severity (CGI-S)(6 months)
  • Pain at its worst as assessed by numerical rating scale(6 months)
  • Clinician global impression of change (CGI-C)(6 months)
  • Patient global impression of severity (PGI-S)(6 months)
  • Patient global impression of change (PGI-C)(6 months)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (8)

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