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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
Sponsor:
Collaborator:
Pachyonychia Congenita Project
Information provided by (Responsible Party):
TransDerm, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Single Group Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Pachyonychia Congenita
Intervention Drug: 1% sirolimus cream (TD201 1%)
Enrollment 15
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Split-body 1% Sirolimus Cream (TD201 1%)
Hide Arm/Group Description

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.

1% sirolimus cream (TD201 1%): 1% sirolimus cream (TD201 1%)

Period Title: Overall Study
Started 15
Completed 14
Not Completed 1
Reason Not Completed
Withdrawal by Subject             1
Arm/Group Title Split-body 1% Sirolimus Cream (TD201 1%)
Hide Arm/Group Description

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.

1% sirolimus cream (TD201 1%): 1% sirolimus cream (TD201 1%)

Overall Number of Baseline Participants 15
Hide Baseline Analysis Population Description
This is a split-body design. After a 2-week run in phase in which both feet were treated with placebo, feet were randomized and one foot received 1% sirolimus and the other placebo ("randomized phase") for 8 weeks. Following the randomized phase, patients applied 1% sirolimus to both feet ("open label phase") for an additional 8 weeks.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants
<=18 years
0
   0.0%
Between 18 and 65 years
15
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants
Female
9
  60.0%
Male
6
  40.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants
American Indian or Alaska Native
0
   0.0%
Asian
2
  13.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
13
  86.7%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 15 participants
15
1.Primary Outcome
Title Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels
Hide Description 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.
Time Frame Two weeks and every 1-2 months for 24 weeks or within 2 weeks after the last dose of study drug
Hide Outcome Measure Data
Hide Analysis Population Description
Starting at week 13, visit 4, there were only 14 participants with available data.
Arm/Group Title Split-body 1% Sirolimus Cream (TD201 1%)
Hide Arm/Group Description:

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.

1% sirolimus cream (TD201 1%): 1% sirolimus cream (TD201 1%)

Overall Number of Participants Analyzed 15
Measure Type: Number
Unit of Measure: participants
Visit 1, Baseline, <2.0 ng/mL 15
Visit 2, Week 3, <2 ng/mL 15
Visit 3, Week 7, <2 ng/mL 15
Visit 4, Week 13, <2 ng/mL 14
Visit 5, Week 17, <2 ng/mL 14
Visit 6, Week 25, <2 ng/mL 14
Visit 7, Week 37, <2 ng/mL 14
2.Secondary Outcome
Title Weekly Assessments Recorded in the PC Quality of Life Index
Hide Description Patient-reported weekly assessment in the PC Quality of Life Index
Time Frame Weekly for 39 weeks
Outcome Measure Data Not Reported
3.Secondary Outcome
Title Daily Assessments Recording in the PC Measurement Diary
Hide Description [Not Specified]
Time Frame Weekly for 39 weeks
Outcome Measure Data Not Reported
4.Other Pre-specified Outcome
Title Investigator Assessment of Local Tolerability
Hide Description 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
Time Frame Prior to application of study drug and within 15-45 minutes after application of study drug at each visit for 39 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Split-body 1% Sirolimus Cream (TD201 1%) Placebo Cream (Vehicle Control)
Hide Arm/Group Description:

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.

1% sirolimus cream (TD201 1%): 1% sirolimus cream (TD201 1%)

Placebo Cream (Vehicle Control)
Overall Number of Participants Analyzed 15 15
Mean (Standard Deviation)
Unit of Measure: units on a scale
Visit 1 Pre, Erythema Score (0-3) 0.2  (0.41) 0.2  (0.41)
Visit 1 Pre, Pruritus Score (0-3) 0.1  (0.26) 0  (0)
Visit 1 Pre, Stinging/Burning (0-3) Score 0  (0) 0.1  (0.26)
Visit 1 Pre, Crusting/Erosion Score (0-3) 0.1  (0.26) 0.1  (0.26)
Visit 1 Post, Erythema Score (0-3) 0.2  (0.41) 0.2  (0.41)
Visit 1 Post, Pruritus Score (0-3) 0  (0) 0  (0)
Visit 1 Post, Stinging/Burning Score (0-3) 0  (0) 0.1  (0.26)
Visit 1 Post, Crusting/Erosion Score (0-3) 0.1  (0.26) 0.1  (0.26)
Visit 4 Pre, Erythema Score (0-3) 0  (0) 0  (0)
Visit 4 Pre, Pruritus Score (0-3) 0.1  (0.28) 0.1  (0.28)
Visit 4 Pre, Stinging/Burning Score (0-3) 0.1  (0.28) 0  (0)
Visit 4 Pre, Crusting/Erosion Score (0-3) 0.1  (0.28) 0.2  (0.38)
Visit 4 Post, Erythema Score (0-3) 0  (0) 0  (0)
Visit 4 Post, Pruritus Score (0-3) 0  (0) 0  (0)
Visit 4 Post, Stinging/Burning Score (0-3) 0.1  (0.28) 0  (0)
Visit 4 Post, Crusting/Erosion Score (0-3) 0.1  (0.28) 0.2  (0.38)
Visit 7 Pre, Erythema Score (0-3) 0.2  (0.58) 0.2  (0.58)
Visit 7 Pre, Pruritus Score (0-3) 0.2  (0.58) 0.2  (0.58)
Visit 7 Pre, Stinging/Burning Score (0-3) 0.3  (0.61) 0.3  (0.61)
Visit 7 Pre, Crusting/Erosion Score (0-3) 0.4  (0.65) 0.3  (0.61)
5.Other Pre-specified Outcome
Title Standardized Photographs
Hide Description 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.
Time Frame Each study visit over 39 weeks
Outcome Measure Data Not Reported
Time Frame Duration of the study, up to 39 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Split-body 1% Sirolimus Cream (TD201 1%)
Hide Arm/Group Description

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.

1% sirolimus cream (TD201 1%): 1% sirolimus cream (TD201 1%)

All-Cause Mortality
Split-body 1% Sirolimus Cream (TD201 1%)
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Split-body 1% Sirolimus Cream (TD201 1%)
Affected / at Risk (%) # Events
Total   0/15 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 3%
Split-body 1% Sirolimus Cream (TD201 1%)
Affected / at Risk (%) # Events
Total   6/15 (40.00%)    
Gastrointestinal disorders   
Nausea   1/15 (6.67%)  1
General disorders   
Pyrexia   1/15 (6.67%)  1
Infections and infestations   
Upper respiratory tract infection   1/15 (6.67%)  1
Musculoskeletal and connective tissue disorders   
Musculoskeletal pain   1/15 (6.67%)  1
Nervous system disorders   
Headache   1/15 (6.67%)  1
Respiratory, thoracic and mediastinal disorders   
Rhinorrhea   1/15 (6.67%)  1
Skin and subcutaneous tissue disorders   
Dermatitis contact   1/15 (6.67%)  1
Skin ulcer   1/15 (6.67%)  1
Urticaria   1/15 (6.67%)  1
Indicates events were collected by systematic assessment
This study was primarily concerned with safety endpoints. Efficacy endpoints need to be improved for follow on studies.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Roger Kaspar, PhD
Organization: TransDerm, Inc.
Phone: 831-420-1684
EMail: roger.kaspar@transderminc.com
Layout table for additonal information
Responsible Party: TransDerm, Inc.
ClinicalTrials.gov Identifier: NCT02152007     History of Changes
Obsolete Identifiers: NCT02057614
Other Study ID Numbers: TD201 - CS01
FD-R-05116 ( Other Grant/Funding Number: Orphan Products Development (OPD) )
First Submitted: February 5, 2014
First Posted: June 2, 2014
Results First Submitted: July 29, 2016
Results First Posted: October 7, 2016
Last Update Posted: October 7, 2016