Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
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|ClinicalTrials.gov Identifier: NCT02152007|
Recruitment Status : Completed
First Posted : June 2, 2014
Results First Posted : October 7, 2016
Last Update Posted : October 7, 2016
|Condition or disease||Intervention/treatment||Phase|
|Pachyonychia Congenita||Drug: 1% sirolimus cream (TD201 1%)||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Phase 1b Clinical Trial Using Topical Sirolimus for the Treatment of Pachyonychia Congenita|
|Study Start Date :||January 2014|
|Actual Primary Completion Date :||July 2015|
|Actual Study Completion Date :||November 2015|
Experimental: 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.
Drug: 1% sirolimus cream (TD201 1%)
1% sirolimus cream (TD201 1%)
- Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels [ Time Frame: Two weeks and every 1-2 months for 24 weeks or within 2 weeks after the last dose of study drug ]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.
- Weekly Assessments Recorded in the PC Quality of Life Index [ Time Frame: Weekly for 39 weeks ]Patient-reported weekly assessment in the PC Quality of Life Index
- Daily Assessments Recording in the PC Measurement Diary [ Time Frame: Weekly for 39 weeks ]
- Investigator Assessment of Local Tolerability [ Time Frame: Prior to application of study drug and within 15-45 minutes after application of study drug at each visit for 39 weeks ]Investigator assessment of local tolerability at the application sites on the plantar surfaces will be evaluated by the Investigator according to a 4-point scale (0, 1, 2, or 3; none to severe) with regard to: erythema, pruritis, stinging/burning, and crusting/erosion
- Standardized Photographs [ Time Frame: Each study visit over 39 weeks ]An expert in the disease who is blinded to the study treatment will read the photographs of the callus area taken at each study visit. The reader will assess changes to the calluses based on criteria such as blisters, cracks, small/large size, and red or bloody spots on the callus. Change in calluses will be reported for both the right and left foot.
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): NCT02152007
|United States, California|
|Stanford, California, United States, 94305|
|Study Chair:||Roger L Kaspar, PhD||TransDerm, Inc.|