Pachyonychia CongenitaPhase 1NCT02152007
Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
A Phase 1 study testing 1% sirolimus cream (TD201 1%) in people with Pachyonychia Congenita, run by TransDerm, Inc. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Pachyonychia Congenita
- What is being tested?
- 1% sirolimus cream (TD201 1%)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2014to Nov 2015
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
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- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase 1b Clinical Trial Using Topical Sirolimus for the Treatment of Pachyonychia Congenita
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
- Being tested
- 1% sirolimus cream (TD201 1%)
- Keywords
- pachyonychia congenita
- Medical subject headings
- Pachyonychia Congenita
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
1% sirolimus cream (TD201 1%)Drug
1% sirolimus cream (TD201 1%)
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: 1% sirolimus cream (TD201 1%) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Patient-reported weekly assessment in the PC Quality of Life Index
Measured: Weekly for 39 weeks
Daily Assessments Recording in the PC Measurement Diary
Measured: Weekly for 39 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2014 (estimated)
- Study startJan 2014
- Primary completionJul 2015
- Study completionNov 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pachyonychia CongenitaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- TransDerm, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Pachyonychia Congenita Project
- Responsible party
- Sponsor
- Investigators
- Roger L Kaspar, PhD, Study chair, TransDerm, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02152007
- Sponsor's protocol ID
- TD201 - CS01
- Other IDs
- FD-R-05116
- Status last verified
- Oct 2016
- First submitted
- 5 Feb 2014
- Last updated
- 7 Oct 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1b Clinical Trial Using Topical Sirolimus for the Treatment of Pachyonychia Congenita
- ClinicalTrials.gov ID
- NCT02152007
- Acronym / sponsor ID
- TD201 - CS01
- Conditions
- Pachyonychia Congenita
- Keywords
- pachyonychia congenita
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- TransDerm, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Pachyonychia Congenita Project
- Study start
- Jan 2014
- Primary completion
- Jul 2015 (actual)
- Study completion
- Nov 2015 (actual)
- First posted
- 2 Jun 2014 (estimated)
- Results
- Posted 7 Oct 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Split-body 1% sirolimus cream (TD201 1%) | Experimental | Drug: 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. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (9 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 1% sirolimus cream (TD201 1%)1% sirolimus cream (TD201 1%) | Drug | Split-body 1% sirolimus cream (TD201 1%) |
Primary outcome
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.
2 secondary outcomes
Weekly Assessments Recorded in the PC Quality of Life Index
Time frame: Weekly for 39 weeks
Daily Assessments Recording in the PC Measurement Diary
Time frame: Weekly for 39 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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Epidermal Growth Factor Receptor Inhibition for Keratinopathies
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Roger L Kaspar, PhD study chair
TransDerm, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 1% sirolimus cream (TD201 1%) in people with Pachyonychia Congenita, run by TransDerm, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Oct 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Oct 2016.