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

Palvella Therapeutics

Status and phase: Completed; Phase 3; Phase 2

Conditions: Pachyonychia Congenita

Treatments: Drug: Placebo; Drug: PTX-022

Study type: Interventional

Funder types: Industry

Identifiers

NCT03920228

PALV-02

Details and patient eligibility

About

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.

Enrollment: 73 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Double Blind

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73 participants in 4 patient groups, including a placebo group

Open label period

Experimental group

Treatment:

Drug: PTX-022

Randomized period - Dosing A

Placebo Comparator group

Treatment:

Drug: Placebo

Randomized period - Dosing B

Experimental group

Treatment:

Drug: PTX-022

Drug: Placebo

Randomized period - Dosing C

Experimental group

Treatment:

Drug: PTX-022