Basal Cell Nevus SyndromePhase 3NCT04308395

Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)

A Phase 3 study testing Patidegib Topical Gel, 2% in people with Basal Cell Nevus Syndrome, run by Sol-Gel Technologies, Ltd. at 37 sites in 9 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

This is a multicenter, open label extension study evaluating the safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult subjects with Gorlin syndrome. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Basal Cell Nevus Syndrome
What is being tested?: Patidegib Topical Gel, 2%
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 108 participants
Where: 37 sitesin 9 countries
Study dates: 3 Jun 2020to 14 Jul 2021
Phase: Phase 3
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 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

The subject must have completed PellePharm Study Pelle-926-201 or Pelle-926-301.
Study Pelle-926-301 subjects must have completed the End of Treatment Visit in Study 301, prior to the Screening Visit in this study. They must also complete all Study 301 related procedures prior to the Baseline Visit of this study.
The subject must be willing to abstain from application of a non-study topical medication (prescription or over the counter) to facial skin for the duration of the trial except as prescribed by the Investigator. Moisturizers and emollients are allowed. Subjects will be encouraged to use their preferred sunscreen with a sunscreen protection factor (SPF) of at least 30 daily on all exposed skin sites.
Female subjects must have a negative pregnancy test. For Study 301 subjects a negative serum pregnancy test result from Study 301 is acceptable if the test was done within 7 days of the Screening Visit of this study.
If the subject is a woman of child bearing potential (WOCBP), she must be willing to use birth control methods which may be considered highly effective. Hormonal contraception must be supplemented with a barrier method (preferably condom). Birth control must start prior to Baseline, continue through the duration of the study, and for 30 days after last application of investigational product (IP).
If the subject is a male with a female sex partner who is a WOCBP, the subject must be willing to use condoms, even after a vasectomy, starting prior to Baseline, through the duration of the study, and for at least 3 months after the last application of IP.
The subject is willing for all facial BCCs to be evaluated and follow treatment recommendations made only by the Investigator.
The subject is willing to forego treatment of facial BCCs with anything other than the study IP except when the Investigator believes that delay of treatment of a BCC potentially might compromise the health of the subject. In such instances, the only other allowed form of treatment is surgical.

Who cannot join

The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study IP.
The subject has current, recent (within five half lives of the experimental drug or if half life not known, within the past 6 months prior to the Screening Visit), or planned (while enrolled in this study) participation in an experimental drug study (excluding Study 301).
The subject is a WOCBP who is unwilling or unable to comply with pregnancy prevention measures.
The subject is pregnant or breastfeeding.

Trial locations and contacts

37 sites in 9 countries.

Country

City, postcode or hospital

37 sites in 9 countries

United States17 sites

Pellepharm Investigative Site
Ann Arbor, Michigan, 48109
PellePharm Investigative Site
Chicago, Illinois, 60637
Cleveland Clinic
Cleveland, Ohio, 44195
Duke University Medical Center
Durham, North Carolina, 27710
PellePharm Investigative Site
Fremont, California, 94538
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
MD Anderson Cancer Center
Houston, Texas, 77030
Laser & Skin Surgery Center of Indiana
Indianapolis, Indiana, 46260
Pellepharm Investigative Site
Miami, Florida, 33125
University of Minnesota
Minneapolis, Minnesota, 55455
University of Utah, Midvalley Dermatology
Murray, Utah, 84107
Yale University
New Haven, Connecticut, 06519
Columbia University Irving Medical Center
New York, New York, 10032
PellePharm Investigative Site
Newport Beach, California, 92660
PellePharm Investigative Site
Ormond Beach, Florida, 32174
Oregon Health and Science University
Portland, Oregon, 97239
Saint Louis University
St Louis, Missouri, 63104

Other trials in United States

Spain4 sites

PellePharm Investigative Site
Barcelona, 08036
PellePharm Investigative Site
Madrid, 28023
PellePharm Investigative Site
Madrid, 28034
PellePharm Investigative Site
Seville, 41009

Other trials in Spain

United Kingdom4 sites

PellePharm Investigative Site
Glasgow
PellePharm Investigative Site
London
PellePharm Investigative Site
Manchester
PellePharm Investigative Site
Oxford

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France3 sites

PellePharm Investigative Site
Lille
PellePharm Investigative Site
Nantes, 44093
Hopital Saint Louis
Paris, 75010

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Germany3 sites

PellePharm Investigative Site
Berlin, 10117
PellePharm Investigative Site
Munich, 80337
PellePharm Investigative Site
Münster, 48149

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Italy3 sites

AOU Luigi Vanvitelli
Naples, 80131
PellePharm Investigative Site
Rozzano, 20089
PellePharm Investigative Site
Vicenza, 36100

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Belgium1 site

PellePhram Investigative Site
Leuven, 3000

Other trials in Belgium

Denmark1 site

PellePharm Investigative Site
Copenhagen, DK-2400

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Netherlands1 site

PellePharm Investigative Site
Maastricht, 6229 HX

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About this study

Official title: A Phase 3, Multicenter, Open-Label Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)

This is a multicenter, open label extension study evaluating the safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult subjects with Gorlin syndrome.

Conditions: Basal Cell Nevus Syndrome
Being tested: Patidegib Topical Gel, 2%
Keywords: Gorlin syndrome, Patidegib, Basal cell nevus syndrome, Nevoid basal cell carcinoma syndrome, Basal cell carcinoma, Hedgehog, Surgically eligible basal cell carcinomas, BCC, BCNS
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.

Patidegib Topical Gel, 2%Drug
Patidegib Topical Gel, 2%

Study groups in NCT04308395
Study group	Role	What they receive
Patidegib Topical Gel, 2%	Experimental	Patidegib Topical Gel, 2% Receives: Drug: Patidegib Topical Gel, 2%

What is being measured

The primary outcome is the main question the study is designed to answer.

Incidence of Treatment-emergent Adverse Events (TEAEs)
IMP=Investigational medicinal product; Unique adverse event = adverse event of a certain preferred term, counted only once within each subject.
Related adverse event = adverse event with relationship as Definitely, Probably or Possibly.
Measured: 12 months

Secondary outcomes (5)

Change in Number of Facial BCCs Removed by Surgery
Measured: 12 months
Change in Number of New Lesions Suspicious for BCC
Measured: 12 months
Change in Advanced Basal Cell Carcinoma Index (aBCCdex) Lesion Score
Measured: 12 months
Change in Dermatology Life Quality Index (DLQI)
Measured: 12 months
Change in EuroQol Group 5-level EQ 5D (EQ 5D 5L)
Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

3 Jun 202014 Jul 2021

First posted16 Mar 2020
Study start3 Jun 2020
Primary completion14 Jul 2021
Study completion14 Jul 2021

Why it stopped: Study was terminated early due to low blinded event rate and this study termination is not related to safety of the drug Patidegib Topical Gel, 2%.

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 Basal Cell Nevus SyndromeStudies 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 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: All patients will receive Patidegib Topical Gel, 2%,
Masking: None (open label)
Enrolment: 108 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04308395
Sponsor's protocol ID: Pelle-926-301E
Status last verified: Jul 2024
First submitted: 11 Mar 2020
Last updated: 24 Sep 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

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

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All Basal Cell Nevus Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 24 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 3, Multicenter, Open-Label Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)
ClinicalTrials.gov ID: NCT04308395
Acronym / sponsor ID: Pelle-926-301E
Conditions: Basal Cell Nevus Syndrome
Keywords: Gorlin syndrome, Patidegib, Basal cell nevus syndrome, Nevoid basal cell carcinoma syndrome, Basal cell carcinoma, Hedgehog, Surgically eligible basal cell carcinomas, BCC
Phase: Phase 3
Study type: Interventional
Recruitment status: TerminatedReason stopped: Study was terminated early due to low blinded event rate and this study termination is not related to safety of the drug Patidegib Topical Gel, 2%.
Enrolment: 108 participants (actual)
Lead sponsor: Sol-Gel Technologies, Ltd.Industry (pharma, biotech, devices)
Study start: 3 Jun 2020 (actual)
Primary completion: 14 Jul 2021 (actual)
Study completion: 14 Jul 2021 (actual)
First posted: 16 Mar 2020 (actual)
Results: Posted 24 Sep 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This is a multicenter, open label extension study evaluating the safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult subjects with Gorlin syndrome.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: All patients will receive Patidegib Topical Gel, 2%,

Arm	Type	Receives
Patidegib Topical Gel, 2%	Experimental	Drug: Patidegib Topical Gel, 2%Patidegib Topical Gel, 2%

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

The subject must have completed PellePharm Study Pelle-926-201 or Pelle-926-301.
Study Pelle-926-301 subjects must have completed the End of Treatment Visit in Study 301, prior to the Screening Visit in this study. They must also complete all Study 301 related procedures prior to the Baseline Visit of this study.
The subject must be willing to abstain from application of a non-study topical medication (prescription or over the counter) to facial skin for the duration of the trial except as prescribed by the Investigator. Moisturizers and emollients are allowed. Subjects will be encouraged to use their preferred sunscreen with a sunscreen protection factor (SPF) of at least 30 daily on all exposed skin sites.
Female subjects must have a negative pregnancy test. For Study 301 subjects a negative serum pregnancy test result from Study 301 is acceptable if the test was done within 7 days of the Screening Visit of this study.
If the subject is a woman of child bearing potential (WOCBP), she must be willing to use birth control methods which may be considered highly effective. Hormonal contraception must be supplemented with a barrier method (preferably condom). Birth control must start prior to Baseline, continue through the duration of the study, and for 30 days after last application of investigational product (IP).

Key exclusion criteria (4 listed)

The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study IP.
The subject has current, recent (within five half lives of the experimental drug or if half life not known, within the past 6 months prior to the Screening Visit), or planned (while enrolled in this study) participation in an experimental drug study (excluding Study 301).
The subject is a WOCBP who is unwilling or unable to comply with pregnancy prevention measures.
The subject is pregnant or breastfeeding.

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
Patidegib Topical Gel, 2%Patidegib Topical Gel, 2%	Drug	Patidegib Topical Gel, 2%

Primary outcome

Incidence of Treatment-emergent Adverse Events (TEAEs)
Time frame: 12 months
IMP=Investigational medicinal product; Unique adverse event = adverse event of a certain preferred term, counted only once within each subject. Related adverse event = adverse event with relationship as Definitely, Probably or Possibly.

5 secondary outcomes

Change in Number of Facial BCCs Removed by Surgery
Time frame: 12 months
Change in Number of New Lesions Suspicious for BCC
Time frame: 12 months
Change in Advanced Basal Cell Carcinoma Index (aBCCdex) Lesion Score
Time frame: 12 months
Change in Dermatology Life Quality Index (DLQI)
Time frame: 12 months
Change in EuroQol Group 5-level EQ 5D (EQ 5D 5L)
Time frame: 12 months

Results and safety

Results posted to the registry on 24 Sep 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Patidegib Topical Gel, 2%	108	3

Primary outcome: Incidence of Treatment-emergent Adverse Events (TEAEs)

Time frame: 12 months. Number. Unit: Number of subjects

Measure	Patidegib Topical Gel, 2% n = 105
Number of subjects with at least one adverse event	46
Number of subjects with at least one adverse event related to IMP	6
Number of subjects with at least leading to treatment discontinuation	0
Number of subjects with at least one AE related to IMP leading to treatment discontinuation	0
Number of subjects with at least one paralesional adverse event	3
Number of subjects with at least one serious adverse event	3
Number of subjects with at least one serious adverse event related to IMP	0
Number of subjects withat least one adverse event leading to death	0

Primary outcome: Incidence of Treatment-emergent Adverse Events (TEAEs)

Time frame: 12 months. Number. Unit: Number of adverse events

Measure	Patidegib Topical Gel, 2% n = 105
Total number of adverse events	82
Number of unique adverse events	72

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: 1 Year. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Patidegib Topical Gel, 2%	3 of 105 (2.9%)	46 of 105 (43.8%)	0 of 105

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Basal Cell Nevus Syndrome

Active, not recruitingPhase 1/2
A Beta-only IL-2 ImmunoTherapY Study
Advanced Solid Tumor, Unresectable Solid Tumor
RecruitingObservational study
Primordial Dwarfism Registry
MOPDII, Meier-Gorlin Syndrome
RecruitingPhase 2
The Cancer Connected Access and Remote Expertise Beyond Walls Program to Provide In-Home Cancer Treatment and Improve Treatment Satisfaction in Cancer Patients Living in the Florida Panhandle and Surrounding Areas
Amyloidosis, Basal Cell Carcinoma
RecruitingObservational study
Familial Investigations of Childhood Cancer Predisposition
Acute Leukemia, Adenomatous Polyposis
RecruitingPhase 1
Blue-Light Photodynamic Therapy and Sonidegib for Multiple Basal Cell Carcinomas
Basal Cell Carcinoma (BCC)

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.

Study Director VP, Clinical Operations study director

PellePharm, Inc.

37 sites in 9 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Patidegib Topical Gel, 2% in people with Basal Cell Nevus Syndrome, run by Sol-Gel Technologies, Ltd. at 37 sites in 9 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 8 inclusion and 4 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: stopped early, as of the sponsor's last update on 24 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 37 sites in United States, Spain, United Kingdom, France, Germany and 4 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 24 Sep 2024). 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?

Incidence of Treatment-emergent Adverse Events (TEAEs) (time frame: 12 months). 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 NCT04308395.

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 24 Sep 2024.