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

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ClinicalTrials.gov Identifier: NCT03920228
Recruitment Status : Recruiting
First Posted : April 18, 2019
Last Update Posted : September 25, 2019
Sponsor:
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)
Estimated Enrollment : 60 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 : April 2020
Estimated Study Completion Date : June 2020


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

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

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

Drug: Placebo
Placebo topical

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




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 ]


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

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 ClinicalTrials.gov identifier (NCT number): NCT03920228


Contacts
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Contact: Emily Cook 484-253-1463 emily.cook@palvellatx.com

Locations
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United States, Arizona
Arizona Research Center Recruiting
Phoenix, Arizona, United States, 85053
Contact: Principal Investigator         
United States, California
Stanford University Recruiting
Palo Alto, California, United States, 94304
Contact: Principal Investigator         
United States, Connecticut
Yale University Recruiting
New Haven, Connecticut, United States, 06519
Contact: Principal Investigator         
United States, Florida
International Dermatology Research Recruiting
Miami, Florida, United States, 33144
Contact: Principal Investigator         
United States, Illinois
Northwestern University Recruiting
Chicago, Illinois, United States, 60611
Contact: Principal Investigator         
United States, Minnesota
Minnesota Clinical Study Center Recruiting
Fridley, Minnesota, United States, 55432
Contact: Principal Investigator         
United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Principal Investigator         
United States, Pennsylvania
Paddington Testing Co. Recruiting
Philadelphia, Pennsylvania, United States, 19103
Contact: Principal Investigator         
United States, Utah
University of Utah Recruiting
Murray, Utah, United States, 84107
Contact: Principal Investigator         
Sponsors and Collaborators
Palvella Therapeutics, Inc.
Investigators
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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.
ClinicalTrials.gov Identifier: NCT03920228     History of Changes
Other Study ID Numbers: PALV-02
First Posted: April 18, 2019    Key Record Dates
Last Update Posted: September 25, 2019
Last Verified: September 2019
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.:
PC
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