BCCs in Gorlin Syndrome PatientsPhase 2NCT04893486
CODY: A Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel For The Prevention Of Basal Cell Carcinomas (BCCs) In Patients With Gorlin Syndrome
A Phase 2 study testing PTX-022 in people with BCCs in Gorlin Syndrome Patients, run by Palvella Therapeutics, Inc. at 16 sites in 2 countries.
Trial summary
A Phase 2b study looks at the safety and efficacy of a treatment that is being investigated for people with certain diseases. This study will be conducted at multiple centers in the United States where participants with Gorlin Syndrome, also known as basal cell nevus syndrome, will be randomly placed into two groups… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- BCCs in Gorlin Syndrome Patients
- What is being tested?
- PTX-022
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 73 participants
- Where
- 16 sitesin 2 countries
- Study dates
- 6 May 2021to 13 Mar 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults, 18 years or older
- Meet diagnostic criteria for Gorlin Syndrome
- Willing to avoid application of a non-study topical medication to the face (prescription or over the counter) during the study.
- Willing to forego treatment of BCCs with anything other than the study medication except when the Investigator believes that delay of treatment of a BCC potentially might compromise the health of the subject. During the trial, the only allowed form of BCC treatment is surgical.
- Participant will be encouraged to use their preferred sunscreen with a sun protector factor (SPF) of at least 30 daily
Who cannot join
- Recently participated in a clinical trial evaluating an investigational product for the treatment of BCCs or Gorlin Syndrome within 3 months prior to to starting study medication. Participant can start the study screening period prior to completing the 3 month washout.
- Recently used topical or systemic (oral) treatment that might interfere with the evaluation of the study medication.
Trial locations and contacts
16 sites in 2 countries.
16 sites in 2 countries
United States14 sites
DermResearch Inc
Austin, Texas, 78759
Duke University
Durham, North Carolina, 27705
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033
Burke Pharmaceuticals Research
Hot Springs, Arkansas, 71913
University of Utah
Murray, Utah, 84107
Clinical Coordinating Center (Minnesota)
New Brighton, Minnesota, 55112
Virginia Clinical Research, Inc.
Norfolk, Virginia, 23502
Park Avenue Dermatology
Orange Park, Florida, 32073
Ameriderm Research
Ormond Beach, Florida, 32174
Stanford Univeristy
Palo Alto, California, 94304
Mayo Clinic - Minnesota
Rochester, Minnesota, 55905
Mayo Clinic
Scottsdale, Arizona, 85259
Gwinett Clinical Research Center
Snellville, Georgia, 30078
St. Louis University
St Louis, Missouri, 63110
United Kingdom2 sites
St. Pancras Clinical Research
London
Salford Foundation Trust
Salford, M6 8HD
About this study
Official title: A Multicenter, Phase 2B, Randomized, Double-Blind, Stratified, Vehicle-Controlled Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel In Prevention Of BCCs In Patients With Gorlin Syndrome
A Phase 2b study looks at the safety and efficacy of a treatment that is being investigated for people with certain diseases. This study will be conducted at multiple centers in the United States where participants with Gorlin Syndrome, also known as basal cell nevus syndrome, will be randomly placed into two groups; one group will receive the active topical gel, the other a topical vehicle gel, also know as placebo. Participants will apply this topical product to their face once a day for 6 months. The study will be looking at the number of new BCCs that develop on the faces of all the participants during this time.
- Conditions
- BCCs in Gorlin Syndrome Patients
- Being tested
- PTX-022, Vehicle comparator
- Keywords
- Gorlin Syndrome, Basal Cell Carcinoma, Nevoid basal cell carcinoma syndrome, Basal cell nevus syndrome BCNS, Gorlin Syndrome Alliance, Prevention, Topical, Sirolimus
- Medical subject headings
- Basal Cell Nevus Syndrome, Carcinoma, Basal Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PTX-022Drug
QTORIN 3.9 % Sirolimus Topical Gel
Vehicle comparatorDrug
Vehicle comparator topical gel
| Study group | Role | What they receive |
|---|---|---|
| Active | Experimental | Not described Receives: Drug: PTX-022 |
| Vehicle | Placebo comparator | Not described Receives: Drug: Vehicle comparator |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms
Measured: Month 6
Incidence of dermatological, treatment emergent adverse events after treatment with active
Measured: Month 6
Secondary outcomes (2)
Percentage of participants developing 2 or more new biopsy confirmed BCCs on the face.
Measured: Month 6
Percentage of participants developing 1 or more new biopsy confirmed BCCs on the face.
Measured: Month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2021
- Study start6 May 2021
- Primary completion13 Mar 2023
- Study completion13 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for BCCs in Gorlin Syndrome PatientsStudies 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 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 73 participants (actual)
Sponsor and investigators
- Lead sponsor
- Palvella Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David Bickers, MD, Study chair, Columbia UniversityJoyce Teng, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04893486
- Sponsor's protocol ID
- PALV-04
- Status last verified
- Aug 2024
- First submitted
- 14 May 2021
- Last updated
- 29 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Phase 2B, Randomized, Double-Blind, Stratified, Vehicle-Controlled Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel In Prevention Of BCCs In Patients With Gorlin Syndrome
- ClinicalTrials.gov ID
- NCT04893486
- Acronym / sponsor ID
- PALV-04
- Conditions
- BCCs in Gorlin Syndrome Patients
- Keywords
- Gorlin Syndrome, Basal Cell Carcinoma, Nevoid basal cell carcinoma syndrome, Basal cell nevus syndrome BCNS, Gorlin Syndrome Alliance, Prevention, Topical, Sirolimus
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 73 participants (actual)
- Lead sponsor
- Palvella Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 6 May 2021 (actual)
- Primary completion
- 13 Mar 2023 (actual)
- Study completion
- 13 Mar 2023 (actual)
- First posted
- 19 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Phase 2b study looks at the safety and efficacy of a treatment that is being investigated for people with certain diseases. This study will be conducted at multiple centers in the United States where participants with Gorlin Syndrome, also known as basal cell nevus syndrome, will be randomly placed into two groups; one group will receive the active topical gel, the other a topical vehicle gel, also know as placebo. Participants will apply this topical product to their face once a day for 6 months. The study will be looking at the number of new BCCs that develop on the faces of all the participants during this time.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active | Experimental | Drug: PTX-022 |
| Vehicle | Placebo comparator | Drug: Vehicle comparator |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Adults, 18 years or older
- Meet diagnostic criteria for Gorlin Syndrome
- Willing to avoid application of a non-study topical medication to the face (prescription or over the counter) during the study.
- Willing to forego treatment of BCCs with anything other than the study medication except when the Investigator believes that delay of treatment of a BCC potentially might compromise the health of the subject. During the trial, the only allowed form of BCC treatment is surgical.
- Participant will be encouraged to use their preferred sunscreen with a sun protector factor (SPF) of at least 30 daily
Key exclusion criteria (2 listed)
- Recently participated in a clinical trial evaluating an investigational product for the treatment of BCCs or Gorlin Syndrome within 3 months prior to to starting study medication. Participant can start the study screening period prior to completing the 3 month washout.
- Recently used topical or systemic (oral) treatment that might interfere with the evaluation of the study medication.
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 |
|---|---|---|
| PTX-022QTORIN 3.9 % Sirolimus Topical Gel | Drug | Active |
| Vehicle comparatorVehicle comparator topical gel | Drug | Vehicle |
Primary outcome
Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms
Time frame: Month 6
Incidence of dermatological, treatment emergent adverse events after treatment with active
Time frame: Month 6
2 secondary outcomes
Percentage of participants developing 2 or more new biopsy confirmed BCCs on the face.
Time frame: Month 6
Percentage of participants developing 1 or more new biopsy confirmed BCCs on the face.
Time frame: Month 6
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Aug 2024.
- Sign-up to be contacted about participation in the clinical trialLink provided by the sponsor.
- Gorlin Syndrome AllianceLink provided by the sponsor.
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.
David Bickers, MD study chair
Columbia University
Joyce Teng, MD principal investigator
Stanford University
16 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PTX-022 in people with BCCs in Gorlin Syndrome Patients, run by Palvella Therapeutics, Inc. at 16 sites in 2 countries.
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 5 inclusion and 2 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 29 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, United Kingdom. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms (time frame: Month 6). The study lists 2 primary outcomes in all. 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 29 Aug 2024.