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.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

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.

United States1 site

Stanford University
Stanford, California, 94305

Other trials in United States

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 groups in NCT02152007
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.

Jan 2014Nov 2015

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)

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

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Not yet recruitingPhase 2
Safety, Tolerability, and Efficacy of Oral KM-023 in Adult Patients With Pachyonychia Congenita (pEDD-PC)
Pachyonychia Congenita
Not yet recruitingPhase 1/2
Epidermal Growth Factor Receptor Inhibition for Keratinopathies
Epidermolytic Ichthyosis, Palmoplantar Keratoderma
RecruitingPhase 3
A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
Pachyonychia Congenita
RecruitingObservational study
International Pachyonychia Congenita Research Registry
Pachyonychia Congenita

All Pachyonychia Congenita trials

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.

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

Results posted to the registry on 7 Oct 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Split-body 1% Sirolimus Cream (TD201 1%)	15	14

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. Number. Unit: participants

Measure	Split-body 1% Sirolimus Cream (TD201 1%) n = 15
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

Population analysed: Starting at week 13, visit 4, there were only 14 participants with available data.

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: Duration of the study, up to 39 weeks. Other adverse events reported at 3% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Split-body 1% Sirolimus Cream (TD201 1%)	0 of 15 (0%)	6 of 15 (40%)	Not reported

Limitations and caveats (from the sponsor): This study was primarily concerned with safety endpoints. Efficacy endpoints need to be improved for follow on studies.

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

Related trials for Pachyonychia Congenita

Not yet recruitingPhase 2
Safety, Tolerability, and Efficacy of Oral KM-023 in Adult Patients With Pachyonychia Congenita (pEDD-PC)
Pachyonychia Congenita
Not yet recruitingPhase 1/2
Epidermal Growth Factor Receptor Inhibition for Keratinopathies
Epidermolytic Ichthyosis, Palmoplantar Keratoderma
RecruitingPhase 3
A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
Pachyonychia Congenita
RecruitingObservational study
International Pachyonychia Congenita Research Registry
Pachyonychia Congenita

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?

On the ClinicalTrials.gov record for NCT02152007.

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.