Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

NCT02152007 | Completed Phase 1 Results

• 2 other identifiers: TD201 - CS01FD-R-05116
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

A study to evaluate safety and efficacy of topical sirolimus to treat plantar keratoderma in adults with PC. Subjects may receive either placebo or treatment with at least 1 foot receiving topical sirolimus at some time. For certain phases of the study treatment assignment to the right and left foot will be randomized in a double blind fashion. Blood levels will test systemic absorption of sirolimus. Other safety and efficacy measures will be taken through the 39-week study duration. Funding Source - FDA OOPD

Conditions

Pachyonychia Congenita

Keywords

pachyonychia congenita

Outcome Measures

Primary Outcomes (1)

• Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels

  The primary outcome measure for this Phase 1b safety study is evaluation of system absorption through measurement of serum sirolimus trough levels. The limit of detection of the assay was 2.0 ng/mL.

  Two weeks and every 1-2 months for 24 weeks or within 2 weeks after the last dose of study drug

Secondary Outcomes (2)

• Weekly Assessments Recorded in the PC Quality of Life Index

  Weekly for 39 weeks

• Daily Assessments Recording in the PC Measurement Diary

  Weekly for 39 weeks

Other Outcomes (2)

• Investigator Assessment of Local Tolerability

  Prior to application of study drug and within 15-45 minutes after application of study drug at each visit for 39 weeks

• Standardized Photographs

  Each study visit over 39 weeks

Study Arms (1)

Split-body 1% sirolimus cream (TD201 1%)

EXPERIMENTAL

This is a split-body design. Subjects will self-administer 1% topical sirolimus cream or placebo cream (no drug, vehicle control) on the plantar surface of each foot. At least one foot will be treated with topical sirolimus at some time during the study. Application will be one time daily for a total of 26 weeks. There will be an additional follow-up visit 3 months after the last application of study drug. The total duration of the study is 39 weeks.

Drug: 1% sirolimus cream (TD201 1%)

Interventions

1% sirolimus cream (TD201 1%) DRUG

1% sirolimus cream (TD201 1%)

Split-body 1% sirolimus cream (TD201 1%)

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects must:
• Be capable of understanding the purpose and risks of the study and sign a written Informed Consent Form (ICF)
• Be male or female ≥ 16 years of age at the time of the screening visit
• Have a confirmed diagnosis of PC by genotyping (e.g., familial) and clinically correlated painful keratoderma.
• Have roughly symmetrical calluses of similar severity on the plantar surface of both feet
• Women of childbearing potential must have a negative serum pregnancy test
• Subjects, whether male or female, with reproductive potential and who are sexually active must agree to use double-barrier contraception methods

You may not qualify if:

• Use of other investigational drugs within 30 days of the screening visit and/or has not recovered from any side effects of prior investigational drugs or procedure in the affected area (e.g., a biopsy)
• Significant condition in the dermatologic treatment area such as trauma, or nonhealing chronic wound
• Pregnant or nursing (lactating) female, or a positive serum pregnancy test
• Active infection either systemic or local near the site of treatment requiring chronic or prolonged use of antimicrobial agents
• Known immunodeficiency including: Hepatitis A; Hepatitis B; Hepatitis C; Human Immunodeficiency Virus (HIV)
• Prior and Current Treatment
• Unable to be discontinued from drugs known to either be inducers or inhibitors of CYP3A4/5 enzymes
• Unable to be discontinued from drugs known to be P-glycoprotein inhibitors

Sponsors & Collaborators

• TransDerm, Inc.: lead
• Pachyonychia Congenita Project: collaborator

Study Sites (1)

Stanford University

Stanford, California, 94305, United States

Location

MeSH Terms

Conditions

Pachyonychia Congenita

Condition Hierarchy (Ancestors)

Ectodermal Dysplasia; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Nail Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Limitations and Caveats

This study was primarily concerned with safety endpoints. Efficacy endpoints need to be improved for follow on studies.

Results Point of Contact

• Title: Roger Kaspar, PhD
• Organization: TransDerm, Inc.

roger.kaspar@transderminc.com

831-420-1684

Study Officials

• Roger L Kaspar, PhD

  TransDerm, Inc.

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 5, 2014
• First Posted: June 2, 2014
• Study Start: January 1, 2014
• Primary Completion: July 1, 2015
• Study Completion: November 1, 2015
• Last Updated: October 7, 2016
• Results First Posted: October 7, 2016

Record last verified: 2016-10

Locations

US (1)