PTX-022 for Pachyonychia Congenita

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Pachyonychia CongenitaPTX-022 - Drug
Eligibility
Any Age
All Sexes
What conditions do you have?
Select

Study Summary

PALV-08 is a multicenter, open-label treatment (OLT) study enrolling adults with Pachyonychia Congenita (PC) with genotyped keratin mutations KRT6A, KRT6B, KRT6C or KRT16 who were previously enrolled in the PALV-05 (VAPAUS) trial. The purpose of this OLT study is to investigate the safety of long term exposure and pharmacokinetics (PK) of QTORIN rapamycin 3.9% anhydrous gel or "PTX-022".

Eligible Conditions
  • Pachyonychia Congenita

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

1 Primary · 1 Secondary · Reporting Duration: 6 months

6 months
Treatment emergent adverse events
Hour 24
Pharmacokinetic parameters

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Trial Design

1 Treatment Group

PTX-022
1 of 1

Experimental Treatment

45 Total Participants · 1 Treatment Group

Primary Treatment: PTX-022 · No Placebo Group · Phase 3

PTX-022
Drug
Experimental Group · 1 Intervention: PTX-022 · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 6 months

Who is running the clinical trial?

Palvella Therapeutics, Inc.Lead Sponsor
5 Previous Clinical Trials
264 Total Patients Enrolled
3 Trials studying Pachyonychia Congenita
189 Patients Enrolled for Pachyonychia Congenita

Eligibility Criteria

Age Any Age · All Participants · 3 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have completed the PALV-05 (VAPAUS) study.
You must agree to use reliable contraceptive methods in order to participate.