Pachyonychia Congenita
Conditions
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.
Interventions
QTORIN 3.9% rapamycin anhydrous gel
DRUGVehicle
Matching vehicle (QTORIN anyhydrous gel)
Sponsors
Palvella Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Adult patients, 18 years or older (16 years or older in the UK) * Diagnosed with Pachyonychia Congenita (PC), genetically confirmed Key
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Patient Global Assessment of Activities Difficulty Scale | 6 months |
Secondary
| Measure | Time frame |
|---|---|
| PROMIS physical function | 6 months |
| Patient global impression of severity (PGI-S) | 6 months |
| Pain at its worst as assessed by numerical rating scale | 6 months |
| Clinician global impression of severity (CGI-S) | 6 months |
| Clinician global impression of change (CGI-C) | 6 months |
| Patient global impression of change (PGI-C) | 6 months |
Countries
United Kingdom, United States
Outcome results
None listed