Trial · NCT02762084
Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients
NCT02762084, Phase 2, Completed, PellePharm, Inc.; results first posted 2019-07-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients. Trials, record NCT02762084. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02762084
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients
- NCT number
- NCT02762084
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 17 participants, actual
- Lead sponsor
- PellePharm, Inc.
- Conditions
- Basal Cell Nevus Syndrome
- Interventions
- Patidegib, Vehicle gel
- First posted
- 2016-05-04
- Last updated
- 2020-07-23
- Other registries' IDs
- eudract:2015-004274-15
- Results first posted
- 2019-07-15
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Clinical Efficacy: Percent Change in Tumor Size of Treatment-targeted Surgically Eligible Basal Cell Carcinomas (SEBs) From Baseline | Baseline, Week 26 | SEBs were defined as clinically diagnosed basal cell carcinoma (BCC) 5 millimeters (mm) or greater in diameter on the face, excluding the nose and periorbital skin, and 9 mm or greater at sites other than the face. The percent change in greatest diameters of treatment-targeted surgically eligible basal cell carcinomas (SEBs) from Baseline to Week 26 was calculated as follows: (sum \[Baseline\] - sum \[Week 26\] / sum \[Baseline\] \* 100), where sum = the greatest diameters of Baseline treatment-targeted SEBs and positive numbers represent decrease in tumor size and negative numbers to represent increase in tumor size. Missing values were imputed using Last-Observation Carried Forward (LOCF). |
| Primary | Molecular Efficacy: Percent Change in the Hedgehog (HH) Signaling Pathway Target Gene Glioma-associated Oncogene Homolog 1 (GLI1) Messenger Ribonucleic Acid (mRNA) Levels From Baseline | Baseline, Week 6 | SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin, and 9-mm or greater at sites other than the face. A single baseline SEB designated as a treatment targeted tumor at Baseline was biopsied first at Baseline and again following 6 weeks of treatment. This was used to assess percent change in GLI1 mRNA levels as follows: (Baseline - Week 6) / Baseline \* 100, where positive numbers to represent decrease in GL1 mRNA level and negative numbers to represent increase in GL1 mRNA level. Any missing values were not imputed; all available data is summarized. |
| Primary | Safety and Tolerability Assessment of Treatment With Patidegib Gel: Number of Participants With a Treatment-emergent Adverse Event Causally Related to Study Drug | Baseline through Week 26 | All serious adverse events (SAEs) and all other non-serious adverse events (AEs) regardless of causality are located in the Reported AE Module. AEs considered as related where categorized by the Investigator as either definitely related, probably related, or possibly related. Treatment-emergent AEs are those with an onset after use of study drug. |
| Primary | Safety and Tolerability Assessment of Treatment With Patidegib Gel: Number of Participants With Treatment-emergent Administrative Site Skin Condition AEs Causally Related to Study Drug | Baseline through Week 26 | All SAEs and all other non-serious AEs, regardless of causality, are located in the Reported AE Module. The number of participants reporting administrative-site, skin condition treatment-emergent AEs considered related to study drug by the Investigator are presented below. Treatment-emergent AEs are those with an onset after use of study drug. |
| Secondary | The Number of Participants Reporting New SEBs on the Face From Baseline for the Combined Patidegib Treatment Groups | Baseline, Week 26 | Facial SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin. A new facial SEB was defined as an SEB first noted on the face after Week 2 that developed at a site where there was no visible BCC of any size at Baseline or Week 2. New facial SEBs were investigated for participants on vehicle gel versus participants on patidegib 2% and 4% gel. Missing values were imputed using LOCF. |
| Secondary | The Mean Number of New SEBs on the Face for the Combined Patidegib Treatment Groups | Baseline, Week 26 | Facial SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin. A new facial SEB was defined as an SEB first noted on the face after Week 2 that developed at a site where there was no visible BCC of any size at Baseline or Week 2. New facial SEBs were investigated for participants on vehicle gel versus participants on patidegib 2% and 4% gel. Missing values were imputed using LOCF. The mean number of new SEBs (number per participant) are presented. No measure of dispersion/precision was calculated. |
| Secondary | The Mean Number of New SEBs on the Face for the Combined Patidegib Treatment Groups by Tumor Population | Baseline, Week 26 | Facial SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin. A new facial SEB was defined as an SEB first noted on the face after Week 2 that developed at a site where there was no visible BCC of any size at Baseline or Week 2. New facial SEBs were investigated for participants on vehicle gel versus participants on patidegib 2% and 4% gel. Missing values were imputed using LOCF. The mean number of new SEBs (number per participant) are presented. No measure of dispersion/precision was calculated. |
| Secondary | Percent Change in Baseline Treatment-targeted SEBs Tumor Size From Baseline | Baseline and Weeks 6, 10, 14, 18, and 22 | SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin, and 9 mm or greater at sites other than the face. The percent change in greatest diameters of Baseline treatment-targeted SEBs from Baseline to Week x (Week 6, 10, 14, 18, or 22) was calculated as follows: (sum \[Baseline\] - sum \[Week x\] / sum \[Baseline\] \* 100), where sum = the greatest diameters of Baseline treatment-targeted SEBs, and positive numbers to represent decrease in tumor size and negative numbers to represent increase in tumor size. Missing values were imputed using LOCF. |
| Secondary | Percent Change in Central Facial SEBs From Baseline | Baseline and Weeks 6, 10, 14, 18, 22, and 26 | Central facial SEBs were defined as those located on the nose or periorbital area (eyelids) which were 3 mm or greater at Baseline. The percent change from Baseline to Week x (Week x = Weeks 6, 10, 14, 18, 22, or 26) in central facial SEBs was calculated as follows: \[sum (Baseline) - sum (Week x)\] / \[sum (Baseline)\] \* 100 where sum = the greatest diameters of Baseline treatment-targeted SEBs where positive numbers to represent decrease in tumor size and negative numbers to represent increase in tumor size. Missing values were imputed using LOCF. |
| Secondary | Proportion of Non-central Facial BCCs Increasing to ≥ 5 mm From Baseline | Baseline and Weeks 6, 10, 14, 18, 22, and 26 | The proportion of non-central facial BCCs that at Baseline measured a greatest diameter of \< 5 mm and increased to a diameter of ≥ 5 mm by Week x (Week x = Weeks 6, 10, 14, 18, 22, or 26) were calculated for each participant as follows: (Number of non-central facial BCCs with greatest diameter ≥ 5 mm at Week x) / (Number of non-central facial BCCs with greatest diameter \< 5 mm at Baseline). Missing values were imputed using LOCF. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Clinical Efficacy: Percent Change in Tumor Size of Treatment-targeted Surgically Eligible Basal Cell Carcinomas (SEBs) From Baseline | Baseline, Week 26 | By tumor per protocol population includes all treatment-targeted tumors except tumors that were biopsied and excludes tumors that did not meet protocol criteria. | percentage change | MEAN |
| PRIMARY | Molecular Efficacy: Percent Change in the Hedgehog (HH) Signaling Pathway Target Gene Glioma-associated Oncogene Homolog 1 (GLI1) Messenger Ribonucleic Acid (mRNA) Levels From Baseline | Baseline, Week 6 | Participants who received at least 1 dose of study drug who had evaluable GLI1 mRNA data at Baseline and Week 6. | percentage change | MEAN |
| PRIMARY | Safety and Tolerability Assessment of Treatment With Patidegib Gel: Number of Participants With a Treatment-emergent Adverse Event Causally Related to Study Drug | Baseline through Week 26 | All enrolled participants who were randomized, received at least 1 confirmed dose of study drug, and had at least 1 post-baseline safety assessment. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Safety and Tolerability Assessment of Treatment With Patidegib Gel: Number of Participants With Treatment-emergent Administrative Site Skin Condition AEs Causally Related to Study Drug | Baseline through Week 26 | All enrolled participants who were randomized, received at least 1 confirmed dose of study drug, and had at least 1 post-baseline safety assessment. | participants | NUMBER |
| SECONDARY | The Number of Participants Reporting New SEBs on the Face From Baseline for the Combined Patidegib Treatment Groups | Baseline, Week 26 | Participants who received at least 1 dose of study drug. | participants | NUMBER |
| SECONDARY | The Mean Number of New SEBs on the Face for the Combined Patidegib Treatment Groups | Baseline, Week 26 | Participants who received at least 1 dose of study drug. | new SEBs | NUMBER |
| SECONDARY | The Mean Number of New SEBs on the Face for the Combined Patidegib Treatment Groups by Tumor Population | Baseline, Week 26 | By tumor per protocol population includes all treatment-targeted tumors except tumors that were biopsied and excludes tumors that did not meet protocol criteria. | new SEBs | NUMBER |
| SECONDARY | Percent Change in Baseline Treatment-targeted SEBs Tumor Size From Baseline | Baseline and Weeks 6, 10, 14, 18, and 22 | Participants who received at least 1 dose of study drug. | percentage change | MEAN |
| SECONDARY | Percent Change in Central Facial SEBs From Baseline | Baseline and Weeks 6, 10, 14, 18, 22, and 26 | Participants who received at least 1 dose of study drug and who had a central facial SEB at Baseline. | percent change | MEAN |
| SECONDARY | Proportion of Non-central Facial BCCs Increasing to ≥ 5 mm From Baseline | Baseline and Weeks 6, 10, 14, 18, 22, and 26 | Participants who received at least 1 dose of study drug with non-central facial BCCs \< 5 mm at Baseline. | proportion of BCCs | MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group117
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Clinical Efficacy: Percent Change in Tumor Size of Treatment-targeted Surgically Eligible Basal Cell Carcinomas (SEBs) From Baseline | Patidegib Gel 2% | 51.29 | Standard Deviation 41.780 | percentage change | |
| Clinical Efficacy: Percent Change in Tumor Size of Treatment-targeted Surgically Eligible Basal Cell Carcinomas (SEBs) From Baseline | Patidegib Gel 4% | 26.63 | Standard Deviation 41.270 | percentage change | |
| Clinical Efficacy: Percent Change in Tumor Size of Treatment-targeted Surgically Eligible Basal Cell Carcinomas (SEBs) From Baseline | Vehicle Gel | 21.82 | Standard Deviation 25.213 | percentage change | |
| Molecular Efficacy: Percent Change in the Hedgehog (HH) Signaling Pathway Target Gene Glioma-associated Oncogene Homolog 1 (GLI1) Messenger Ribonucleic Acid (mRNA) Levels From Baseline | Patidegib Gel 2% | 53.83 | Standard Deviation 27.197 | percentage change | |
| Molecular Efficacy: Percent Change in the Hedgehog (HH) Signaling Pathway Target Gene Glioma-associated Oncogene Homolog 1 (GLI1) Messenger Ribonucleic Acid (mRNA) Levels From Baseline | Patidegib Gel 4% | 20.69 | Standard Deviation 34.730 | percentage change | |
| Molecular Efficacy: Percent Change in the Hedgehog (HH) Signaling Pathway Target Gene Glioma-associated Oncogene Homolog 1 (GLI1) Messenger Ribonucleic Acid (mRNA) Levels From Baseline | Vehicle Gel | 28.53 | Standard Deviation 43.096 | percentage change | |
| Safety and Tolerability Assessment of Treatment With Patidegib Gel: Number of Participants With a Treatment-emergent Adverse Event Causally Related to Study Drug | Patidegib Gel 2% | 2 | Participants | ||
| Safety and Tolerability Assessment of Treatment With Patidegib Gel: Number of Participants With a Treatment-emergent Adverse Event Causally Related to Study Drug | Patidegib Gel 4% | 5 | Participants | ||
| Safety and Tolerability Assessment of Treatment With Patidegib Gel: Number of Participants With a Treatment-emergent Adverse Event Causally Related to Study Drug | Vehicle Gel | 2 | Participants | ||
| Safety and Tolerability Assessment of Treatment With Patidegib Gel: Number of Participants With Treatment-emergent Administrative Site Skin Condition AEs Causally Related to Study Drug | Patidegib Gel 2% | Application site alopecia | 0 | participants |
1 to 10 of 117Source ClinicalTrials.gov
See all 117 results by groupParticipant flow15
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Patidegib Gel 2% | 6 | |
| Overall Study | STARTED | Patidegib Gel 4% | 6 | |
| Overall Study | STARTED | Vehicle Gel | 5 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Patidegib Gel 2% | 6 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Patidegib Gel 4% | 6 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Vehicle Gel | 5 | |
| Overall Study | By Tumor Per Protocol Population (PP) | Patidegib Gel 2% | 6 | |
| Overall Study | By Tumor Per Protocol Population (PP) | Patidegib Gel 4% | 6 | |
| Overall Study | By Tumor Per Protocol Population (PP) | Vehicle Gel | 4 | |
| Overall Study | COMPLETED | Patidegib Gel 2% | 5 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 participant flowAdverse events111
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Hyperthyroidism | Endocrine disorders | Patidegib Gel 2% | 1 | 6 | |
| other | Hyperthyroidism | Endocrine disorders | Patidegib Gel 4% | 0 | 6 | |
| other | Hyperthyroidism | Endocrine disorders | Vehicle Gel | 0 | 5 | |
| other | Hypothyroidism | Endocrine disorders | Patidegib Gel 2% | 0 | 6 | |
| other | Hypothyroidism | Endocrine disorders | Patidegib Gel 4% | 0 | 6 | |
| other | Hypothyroidism | Endocrine disorders | Vehicle Gel | 1 | 5 | |
| other | Episcleritis | Eye disorders | Patidegib Gel 2% | 0 | 6 | |
| other | Episcleritis | Eye disorders | Patidegib Gel 4% | 1 | 6 | |
| other | Episcleritis | Eye disorders | Vehicle Gel | 0 | 5 | |
| other | Constipation | Gastrointestinal disorders | Patidegib Gel 2% | 0 | 6 |
1 to 10 of 111Source ClinicalTrials.gov
See all 111 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Patidegib Gel 2% | 0 | 6 |
| Total, all-cause mortality | Patidegib Gel 4% | 0 | 6 |
| Total, all-cause mortality | Vehicle Gel | 0 | 5 |
| Total, other adverse events | Patidegib Gel 2% | 4 | 6 |
| Total, other adverse events | Patidegib Gel 4% | 6 | 6 |
| Total, other adverse events | Vehicle Gel | 3 | 5 |
| Total, serious adverse events | Patidegib Gel 2% | 0 | 6 |
| Total, serious adverse events | Patidegib Gel 4% | 0 | 6 |
| Total, serious adverse events | Vehicle Gel | 2 | 5 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Patidegib Gel vs Vehicle Gel: The Number of Participants Reporting New SEBs on the Face From Baseline for the Combined Patidegib Treatment Groups |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02762084
GET https://conductscience.org/api/trials/v1/NCT02762084/results?page=2