Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

NCT ID: NCT02152007

Last Updated: 2016-10-07

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-01-31
• Study Completion Date: 2015-11-30

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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

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.

Group Type: EXPERIMENTAL

1% sirolimus cream (TD201 1%)

Intervention Type: DRUG

1% sirolimus cream (TD201 1%)

Interventions

1% sirolimus cream (TD201 1%)

1% sirolimus cream (TD201 1%)

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

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

Exclusion Criteria

A Subject with any of the following criteria is not eligible for inclusion in this study:

• 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

• Minimum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pachyonychia Congenita Project

OTHER

Sponsor Role: collaborator

TransDerm, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Roger L Kaspar, PhD

Role: STUDY_CHAIR

TransDerm, Inc.

Locations

Stanford University

Stanford, California, United States

Countries

United States

Other Identifiers

FD-R-05116

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

TD201 - CS01

Identifier Type: -

Identifier Source: org_study_id

NCT02057614

Identifier Type: -

Identifier Source: nct_alias