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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03920228
Recruitment Status : Active, not recruiting
First Posted : April 18, 2019
Last Update Posted : August 19, 2020
Information provided by (Responsible Party):
Palvella Therapeutics, Inc.

Brief Summary:

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.

Condition or disease Intervention/treatment Phase
Pachyonychia Congenita Drug: PTX-022 Drug: Placebo Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 73 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Intervention Model Description:

This study consists of 2 treatment periods:

  • Open-Label
  • Randomized, Double-Blind, Placebo controlled
Masking: Double (Participant, Investigator)
Masking Description: During the open-label period, there is no masking. During the randomized, double-blind, placebo-controlled period the participant and Investigator are masked.
Primary Purpose: Treatment
Official Title: A Multicenter, Four-Part, Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in the Treatment of Adults With Moderate to Severe Pachyonychia Congenita
Actual Study Start Date : April 1, 2019
Estimated Primary Completion Date : August 2020
Estimated Study Completion Date : October 2020

Arm Intervention/treatment
Experimental: Open label period Drug: PTX-022

Placebo Comparator: Randomized period - Dosing A Drug: Placebo
Placebo topical

Experimental: Randomized period - Dosing B Drug: PTX-022

Drug: Placebo
Placebo topical

Experimental: Randomized period - Dosing C Drug: PTX-022

Primary Outcome Measures :
  1. Patient Global Assessment of Activities Scale [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. Clinician Global Impression of Change Scale [ Time Frame: 6 months ]
  2. Pain at its worst as assessed by numerical rating scale [ Time Frame: 6 months ]
  3. Number of steps taken as assessed by activity monitor [ Time Frame: 6 months ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03920228

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United States, Arizona
Arizona Research Center
Phoenix, Arizona, United States, 85053
United States, California
Stanford University
Palo Alto, California, United States, 94304
United States, Connecticut
Yale University
New Haven, Connecticut, United States, 06519
United States, Florida
International Dermatology Research
Miami, Florida, United States, 33144
United States, Illinois
Northwestern University
Chicago, Illinois, United States, 60611
United States, Minnesota
Minnesota Clinical Study Center
Fridley, Minnesota, United States, 55432
United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Paddington Testing Co.
Philadelphia, Pennsylvania, United States, 19103
United States, Utah
University of Utah
Murray, Utah, United States, 84107
Sponsors and Collaborators
Palvella Therapeutics, Inc.
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Principal Investigator: David Hansen, MD University of Utah
Principal Investigator: Joyce Teng, MD Stanford University
Additional Information:
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Responsible Party: Palvella Therapeutics, Inc. Identifier: NCT03920228    
Other Study ID Numbers: PALV-02
First Posted: April 18, 2019    Key Record Dates
Last Update Posted: August 19, 2020
Last Verified: August 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Palvella Therapeutics, Inc.:
Additional relevant MeSH terms:
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Pachyonychia Congenita
Nails, Malformed
Pathological Conditions, Anatomical
Ectodermal Dysplasia
Abnormalities, Multiple
Congenital Abnormalities
Skin Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Nail Diseases
Skin Diseases