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PTX-022

Phase 3

Pachyonychia Congenita | Small molecule | Rare Disease |Palvella Therapeutics, Inc.|Last Updated: Apr 27, 2026

Success Probability
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Market & Valuation
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials3
Total Enrollment154
FDA Designations
No designations recorded
Clinical trial landscape

PTX-022 · 6 trials · 4 indications

Phase 3 2Phase 2 4
NCT05643872A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia CongenitaPachyonychia Congenita
RECRUITING45 Analytics
NCT04520750VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia CongenitaPachyonychia Congenita
COMPLETED36 Analytics
PHASE3RECRUITING
A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
Pachyonychia Congenita
NCT0564387245
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PHASE3COMPLETED
VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita
Pachyonychia Congenita
NCT0452075036
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Study Endpoints
Primary Endpoints
Treatment emergent adverse events
6 months
Incidence of Treatment-Emergent Adverse Events
18 Months
Changes in vital sign measurements from baseline
18 months
Changes in weight measurements from baseline
18 months
Changes in clinical laboratory measurements from baseline
18 months
Overall Angiokeratoma Investigator Global Assessment (AK-IGA)
3 months
Overall Patient Global Impression of Change (PGI-C)
3 months
Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms
Month 6
Incidence of dermatological, treatment emergent adverse events after treatment with active
Month 6
Patient Global Assessment of Activities Scale
6 months
Secondary Endpoints
Pharmacokinetic parameters
Prior to dose, 12 hours and 24 hours
Patient Global Assessment of Activities Scale
18 Months
Pain at its worst as assessed by NRS
18 months
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
PTX-022EXPERIMENTAL -
Open label treatment armEXPERIMENTALPTX-022 QTORIN
Open labelEXPERIMENTAL -
Experimental: PTX-022EXPERIMENTALPTX-022 QTORIN
ActiveEXPERIMENTAL -
VehiclePLACEBO_COMPARATOR -
Open label periodEXPERIMENTAL -
Randomized period - Dosing APLACEBO_COMPARATOR -
Randomized period - Dosing BEXPERIMENTAL -
Randomized period - Dosing CEXPERIMENTAL -
Interventions
NameTypeDescription
PTX-022DRUGQTORIN rapamycin 3.9% anhydrous gel
Vehicle comparatorDRUGVehicle comparator topical gel
PlaceboDRUGPlacebo topical
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Eligibility Criteria
Age Range16 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Key Inclusion Criteria: * Completed the PALV-05 (VAPAUS) study * Agree to contraceptive use Key Exclusion Criteria: * Females who are pregnant or breastfeeding * Concomitant use of sirolimus or any sirolimus-containing medications (systemic or topical) * Any significant concurrent condition that ...

Countries:United StatesUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07552077primaryCompletionDate: changed
LOWMay 26, 2026NCT05643872primaryCompletionDate: changed
LOWMay 24, 2026NCT07552077studyFirstPostDate: changed
LOWMay 24, 2026NCT05643872studyFirstPostDate: changed
LOWMay 21, 2026NCT07552077NEW_TRIAL: changed
LOWMay 21, 2026NCT07552077NEW_TRIAL: changed