Education and Training

Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita

This study evaluates the safety and efficacy of PTX-022, topical rapamycin, in the treatment of adults with moderate to severe Pachyonychia Congenita.

This study includes four-parts, and if a participant completes all parts, the participant will have received at least 3-months of PTX-022 treatment.

Stanford is currently not accepting patients for this trial.

Stanford Investigator(s):

Intervention(s):

  • drug: PTX-022
  • drug: Placebo

Eligibility


Inclusion Criteria:

   - Adult patients, 18 years or older

   - Diagnosed Pachyonychia Congenita (PC), genetically confirmed

   - Moderate to Severe PC

   - Able and willing to comply with all protocol-required activities

   - Willing and able to provide written informed consent

Exclusion Criteria:

   - Any significant concurrent condition (including involving the inferior to the ankle)
   that could adversely affect participation.

   - Any intentional changes in the patient's daily activities (associated with standing,
   walking and keeping balance), not resulting from an improvement in the patient's
   condition due to treatment.

   - Patient's deemed by the investigator as unwilling or unable to remain compliant with
   all tests and procedures, including adherence to study drug administration and other
   protocol-required activities.

Ages Eligible for Study

18 Years - N/A

Genders Eligible for Study

All

Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Karima
650-723-0636
Not Recruiting