NCT05643872

Brief 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".

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
45

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Nov 2022

Shorter than P25 for phase_3

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 8, 2022

Completed
7 days until next milestone

Study Start

First participant enrolled

November 15, 2022

Completed
24 days until next milestone

First Posted

Study publicly available on registry

December 9, 2022

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2023

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2023

Completed
Last Updated

December 9, 2022

Status Verified

December 1, 2022

Enrollment Period

11 months

First QC Date

November 8, 2022

Last Update Submit

December 1, 2022

Conditions

Pachyonychia Congenita

Outcome Measures

Primary Outcomes (1)

  • Treatment emergent adverse events

    6 months

Secondary Outcomes (1)

  • Pharmacokinetic parameters

    Prior to dose, 12 hours and 24 hours

Study Arms (1)

PTX-022

EXPERIMENTAL
Drug: PTX-022

Interventions

PTX-022DRUG

QTORIN rapamycin 3.9% anhydrous gel

PTX-022

Eligibility Criteria

Age16 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Completed the PALV-05 (VAPAUS) study
  • Agree to contraceptive use

You may not qualify if:

  • Females who are pregnant or breastfeeding
  • Concomitant use of sirolimus or any sirolimus-containing medications (systemic or topical)
  • Any significant concurrent condition that could adversely affect participation and/or the assessment of the safety and efficacy in the study
  • Prior or planned treatment, including surgery or other medically necessary intervention deemed by the investigator that could adversely affect participation and/or the assessment of the safety and efficacy in the study

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Minnesota Clinical Study Center

New Brighton, Minnesota, 55112, United States

RECRUITING

MeSH Terms

Conditions

Pachyonychia Congenita

Condition Hierarchy (Ancestors)

Ectodermal DysplasiaAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin AbnormalitiesSkin Diseases, GeneticGenetic Diseases, InbornNail DiseasesSkin DiseasesSkin and Connective Tissue Diseases

Central Study Contacts

Emily Cook

CONTACT

267-738-6366emily.cook@palvellatx.com

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 8, 2022

First Posted

December 9, 2022

Study Start

November 15, 2022

Primary Completion

October 1, 2023

Study Completion

November 1, 2023

Last Updated

December 9, 2022

Record last verified: 2022-12

Data Sharing

IPD Sharing
Will not share

Locations

US(1)