Cutaneous Neurofibroma, Neurofibromatosis 1
Conditions
Keywords
NFX-179
Brief summary
This study will enroll and treat subjects with cutaneous neurofibromas with NFX-179, a topical study drug. Eligible subjects will receive treatment for 28 days and be observed by a study doctor for approximately 56 days. Subjects will be randomly assigned to 1 of 4 treatment groups. 3 of the treatment groups will receive a specific dose NFX-179, and 1 group will receive placebo. The subject, study doctor, and NFlection Therapeutics will not know what treatment group each subject is assigned. Study participation requires at least 7 clinic visits, blood, urine, and tissue collection, images of the treated cutaneous neurofibromas, electrocardiograms, and information regarding the subject's medical and disease history.
Interventions
DRUGNFX-179 Gel
gel for topical administration
DRUGVehicle Gel
vehicle gel for topical administration
Sponsors
NFlection Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Subject is at least 18 years of age 2. Subject must provide written informed consent prior to any study procedures 3. Subject must have a clinical diagnosis of NF1 4. Subject has 6 Study cNF Tumors (5 Target cNF Tumors \[1 on the face; 4 on the anterior trunk or upper extremities\] that will be treated with the assigned study medication;1 Untreated cNF Tumor on the anterior trunk or upper extremities) that each meet the following criteria: * Has, in the investigator's opinion, a clinically typical appearance * Is dome shaped * Is not pedunculated * Is a discrete tumor * Is not irritated * Is not in an area subject to repeated trauma (e.g., area that is shaved, on the beltline, under a bra strap, etc.) * Does not have an active cutaneous infection * Has a diameter that is ≥5mm and ≤10mm * Has a height of ≥2mm * Is, when centered in the center of the provided template, the only cNF tumor visible * Is not within 5mm of the orbital rim. 5. Subject is willing to have the 5 Target cNF Tumors and the 1 Untreated cNF Tumor excised at the end of the treatment period 6. Subject is willing to have hair in the area surrounding the Target cNF Tumors shaved, if necessary, to obtain photographs 7. Subject is willing to minimize exposure of each Target cNF to natural and artificial ultraviolet radiation 8. Subject is willing to forego treatment of the Target cNF Tumors, except protocol specified therapy, during the study 9. Female subjects who are women of childbearing potential must have a negative urine pregnancy test result and be willing to use a protocol approved, contraceptive method for the duration of the study 10. Subject is willing and able to follow all study instructions and to attend all study visits.
Exclusion criteria
1. Subject has applied any of the following topical products in the previous 30 days on or in proximity to any Study cNF Tumor that, in the investigator's opinion, impairs evaluation of any the tumor or which exposes the subject to an unacceptable risk by study participation: * Corticosteroids * Retinoids (e.g., tazarotene, tretinoin, adapalene) * \> 5% of an alpha-hydroxy acid (e.g., glycolic acid, lactic acid) * Fluorouracil * Imiquimod 2. Any Study cNF Tumor has ever been treated with an MEK inhibitor or a BRAF inhibitor 3. The subject has used any of the following systemic medications in the noted time period: * Retinoids (e.g., etretinate, isotretinoin) within the previous 90 days * MEK inhibitors within the previous 180 days * BRAF inhibitors within the previous 180 days 4. Subject has a history of hypersensitivity to any of the ingredients in the study medications 5. Subject has any known intercurrent illness or physical condition that would, in the investigator's opinion, impair evaluation of a Target cNF Tumor or which exposes the subject to an unacceptable risk by study participation 6. Subject has, in the investigator's opinion, clinically relevant history of liver disease, including viral hepatitis, current alcohol abuse, or cirrhosis 7. Subject has a history of metastatic disease, or active cancer (excluding nonmelanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of the breast, or Stage 0 chronic lymphocytic lymphoma) within the previous 5 years 8. Subject has any condition (e.g., other skin conditions or diseases, metabolic dysfunction, physical examination findings, clinical laboratory findings) or situation (e.g., vacation, scheduled surgery) that would, in the investigator's opinion, impair evaluation of a Target cNF Tumor or which exposes the subject to an unacceptable risk by study participation 9. Subject has participated in an investigational drug trial in which administration of an investigational study medication occurred within the previous 30 days
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Phospho-erk (p-ERK) Levels of Target cNF Tumors in NFX-179 Gel Group and Vehicle Gel Group After 28 Days of Once-daily (QD) Application | Baseline through Week 4 | Pharmacodynamic activity (biochemical and physiologic effects of drugs) of NFX-179 Gel as defined by suppression of phospho-ERK (p-ERK) levels in Target cNF Tumors in each NFX-179 Gel group compared with the Vehicle Gel group after 28 days of once-daily (QD) application will be measured at Week 4 |
| Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | Baseline through Week 4 | Safety and tolerability will be measured via a local tolerability assessment. The investigator will assess erythema, edema, scabbing/crusting, vesiculation, and erosion. The subject will assess stinging, burning, and pruritus. All assessments are performed using a 4-point scale (0 none, 1 mild, 2 moderate, 3 severe). |
| Assessment of Adverse Events | Baseline through Week 8 | Assessment of adverse events (AEs) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in cNF Tumor Volume (Cubic Millimeters) | Baseline through Week 4 | Percent change in cNF tumor volume after 28 days of QD applications of NFX-179 gel based on tumor volume derived from ruler measurements. |
| Change in Subject Self-Assessment of Tumor Severity Score | Baseline through Week 4 | The Subject Self-Assessment is the subject's assessment of the average overall severity of each Target cNF at a particular time point and is not a comparison with any other time point. The Subject Self-Assessment is a 5-point measuring tumor severity (0 clear/none, 1 almost clear, 2 mild, 3 moderate, 4 severe). The assessment is performed at the Baseline visit and week 4 visit. |
| Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Baseline through Week 4 | Systemic exposure of NFX-179 will be measured during the 28 days of QD applications at select investigative sites. Pharmacokinetic samples will be drawn at the baseline visit and week 4 visit. 5 time point samples will be collected at the week 4 visit. |
| Change in Physician Assessment of Tumor Severity Score | Baseline through Week 4 | Effect of treatment with The Physician Tumor Assessment is the investigator's assessment of the average overall severity of each Target cNF tumor at a particular time point. The Physician Tumor Assessment is a 5-point measuring tumor severity (0 clear/none, 1 almost clear, 2 mild, 3 moderate, 4 severe). The assessment is performed at the Baseline visit and week 4 visit. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| NFX-179 Gel Low (0.05%) NFX-179 Gel for topical administration, once daily for 28 days
NFX-179 Gel 0.05%
NFX-179 Gel: gel for topical administration | 12 |
| NFX-179 Gel Mid (0.15%) NFX-179 Gel for topical administration, once daily for 28 days
NFX-179 Gel 0.15%
NFX-179 Gel: gel for topical administration | 11 |
| NFX-179 Gel High (0.50%) NFX-179 Gel for topical administration, once daily for 28 days
NFX-179 Gel 0.50%
NFX-179 Gel: gel for topical administration | 12 |
| Vehicle Arm (Placebo) Vehicle Gel, for topical administration, once daily for 28 days
Vehicle Gel placebo
Vehicle Gel: vehicle gel for topical administration | 13 |
| Total | 48 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| Overall Study | Lost to Follow-up | 0 | 0 | 0 | 1 |
Baseline characteristics
| Characteristic | NFX-179 Gel Low (0.05%) | NFX-179 Gel Mid (0.15%) | NFX-179 Gel High (0.50%) | Vehicle Arm (Placebo) | Total |
|---|---|---|---|---|---|
| Age, Continuous | 51.1 years | 49.5 years | 43.5 years | 45.2 years | 47.8 years |
| Race/Ethnicity, Customized Asian | 0 Participants | 3 Participants | 0 Participants | 1 Participants | 4 Participants |
| Race/Ethnicity, Customized Black/African American | 1 Participants | 1 Participants | 2 Participants | 1 Participants | 5 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 2 Participants | 0 Participants | 1 Participants | 0 Participants | 3 Participants |
| Race/Ethnicity, Customized Non-white | 1 Participants | 4 Participants | 2 Participants | 2 Participants | 9 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 9 Participants | 7 Participants | 9 Participants | 11 Participants | 36 Participants |
| Race/Ethnicity, Customized Not reported | 1 Participants | 4 Participants | 2 Participants | 2 Participants | 9 Participants |
| Race/Ethnicity, Customized White | 11 Participants | 7 Participants | 10 Participants | 11 Participants | 39 Participants |
| Sex: Female, Male Female | 9 Participants | 8 Participants | 8 Participants | 7 Participants | 32 Participants |
| Sex: Female, Male Male | 3 Participants | 3 Participants | 4 Participants | 6 Participants | 16 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 13 | 0 / 12 | 0 / 11 | 0 / 12 |
| other Total, other adverse events | 2 / 13 | 2 / 12 | 2 / 11 | 4 / 12 |
| serious Total, serious adverse events | 0 / 13 | 0 / 12 | 0 / 11 | 0 / 12 |
Outcome results
Primary
Assessment of Adverse Events
Assessment of adverse events (AEs)
Time frame: Baseline through Week 8
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Vehicle Arm (Placebo) | Assessment of Adverse Events | Number of subjects withdrawn due to AEs | 0 Subjects |
| Vehicle Arm (Placebo) | Assessment of Adverse Events | Subjects with severe treatment-emergent AEs | 0 Subjects |
| Vehicle Arm (Placebo) | Assessment of Adverse Events | Subjects with moderate treatment-emergent AEs | 1 Subjects |
| Vehicle Arm (Placebo) | Assessment of Adverse Events | Number of subjects with treatment-emergent AEs | 2 Subjects |
| Vehicle Arm (Placebo) | Assessment of Adverse Events | Number of subects with related treatment-emergent AEs | 0 Subjects |
| Vehicle Arm (Placebo) | Assessment of Adverse Events | Subjects with mild treatment-emergent AEs | 1 Subjects |
| NFX-179 Gel Low (0.05%) | Assessment of Adverse Events | Subjects with moderate treatment-emergent AEs | 2 Subjects |
| NFX-179 Gel Low (0.05%) | Assessment of Adverse Events | Subjects with mild treatment-emergent AEs | 0 Subjects |
| NFX-179 Gel Low (0.05%) | Assessment of Adverse Events | Number of subjects with treatment-emergent AEs | 2 Subjects |
| NFX-179 Gel Low (0.05%) | Assessment of Adverse Events | Number of subjects withdrawn due to AEs | 0 Subjects |
| NFX-179 Gel Low (0.05%) | Assessment of Adverse Events | Subjects with severe treatment-emergent AEs | 0 Subjects |
| NFX-179 Gel Low (0.05%) | Assessment of Adverse Events | Number of subects with related treatment-emergent AEs | 0 Subjects |
| NFX-179 Gel Mid (0.15%) | Assessment of Adverse Events | Subjects with mild treatment-emergent AEs | 0 Subjects |
| NFX-179 Gel Mid (0.15%) | Assessment of Adverse Events | Subjects with moderate treatment-emergent AEs | 1 Subjects |
| NFX-179 Gel Mid (0.15%) | Assessment of Adverse Events | Number of subjects withdrawn due to AEs | 0 Subjects |
| NFX-179 Gel Mid (0.15%) | Assessment of Adverse Events | Number of subects with related treatment-emergent AEs | 0 Subjects |
| NFX-179 Gel Mid (0.15%) | Assessment of Adverse Events | Subjects with severe treatment-emergent AEs | 0 Subjects |
| NFX-179 Gel Mid (0.15%) | Assessment of Adverse Events | Number of subjects with treatment-emergent AEs | 1 Subjects |
| NFX-179 Gel High (0.50%) | Assessment of Adverse Events | Subjects with severe treatment-emergent AEs | 0 Subjects |
| NFX-179 Gel High (0.50%) | Assessment of Adverse Events | Subjects with mild treatment-emergent AEs | 3 Subjects |
| NFX-179 Gel High (0.50%) | Assessment of Adverse Events | Subjects with moderate treatment-emergent AEs | 0 Subjects |
| NFX-179 Gel High (0.50%) | Assessment of Adverse Events | Number of subjects with treatment-emergent AEs | 3 Subjects |
| NFX-179 Gel High (0.50%) | Assessment of Adverse Events | Number of subects with related treatment-emergent AEs | 1 Subjects |
| NFX-179 Gel High (0.50%) | Assessment of Adverse Events | Number of subjects withdrawn due to AEs | 0 Subjects |
Primary
Phospho-erk (p-ERK) Levels of Target cNF Tumors in NFX-179 Gel Group and Vehicle Gel Group After 28 Days of Once-daily (QD) Application
Pharmacodynamic activity (biochemical and physiologic effects of drugs) of NFX-179 Gel as defined by suppression of phospho-ERK (p-ERK) levels in Target cNF Tumors in each NFX-179 Gel group compared with the Vehicle Gel group after 28 days of once-daily (QD) application will be measured at Week 4
Time frame: Baseline through Week 4
Population: The p-ERK analysis included all available p-ERK data from tumors determined by histology to be cNF tumors. Missing data were due to missing or non-analyzable tumor samples or tumor samples shown to come from non-cNF tumors upon histologic review. Therefore the number of participants is not the same as those in the participant flow.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Vehicle Arm (Placebo) | Phospho-erk (p-ERK) Levels of Target cNF Tumors in NFX-179 Gel Group and Vehicle Gel Group After 28 Days of Once-daily (QD) Application | 0.3054 % reduction in pERK to total ERK | Standard Deviation 0.1369 |
| NFX-179 Gel Low (0.05%) | Phospho-erk (p-ERK) Levels of Target cNF Tumors in NFX-179 Gel Group and Vehicle Gel Group After 28 Days of Once-daily (QD) Application | 0.2734 % reduction in pERK to total ERK | Standard Deviation 0.1257 |
| NFX-179 Gel Mid (0.15%) | Phospho-erk (p-ERK) Levels of Target cNF Tumors in NFX-179 Gel Group and Vehicle Gel Group After 28 Days of Once-daily (QD) Application | 0.2267 % reduction in pERK to total ERK | Standard Deviation 0.1272 |
| NFX-179 Gel High (0.50%) | Phospho-erk (p-ERK) Levels of Target cNF Tumors in NFX-179 Gel Group and Vehicle Gel Group After 28 Days of Once-daily (QD) Application | 0.1627 % reduction in pERK to total ERK | Standard Deviation 0.0686 |
Primary
Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment
Safety and tolerability will be measured via a local tolerability assessment. The investigator will assess erythema, edema, scabbing/crusting, vesiculation, and erosion. The subject will assess stinging, burning, and pruritus. All assessments are performed using a 4-point scale (0 none, 1 mild, 2 moderate, 3 severe).
Time frame: Baseline through Week 4
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Vehicle Arm (Placebo) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of moderate erythema | 0 events |
| Vehicle Arm (Placebo) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of mild edema | 0 events |
| Vehicle Arm (Placebo) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of mild burning | 6 events |
| Vehicle Arm (Placebo) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of scabbing/crusting | 0 events |
| Vehicle Arm (Placebo) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of mild pruritus | 2 events |
| Vehicle Arm (Placebo) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of moderate pruritus | 0 events |
| Vehicle Arm (Placebo) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of mild erythema | 0 events |
| Vehicle Arm (Placebo) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of mild stinging | 0 events |
| NFX-179 Gel Low (0.05%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of mild edema | 0 events |
| NFX-179 Gel Low (0.05%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of moderate erythema | 0 events |
| NFX-179 Gel Low (0.05%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of mild erythema | 2 events |
| NFX-179 Gel Low (0.05%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of mild pruritus | 5 events |
| NFX-179 Gel Low (0.05%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of mild stinging | 0 events |
| NFX-179 Gel Low (0.05%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of scabbing/crusting | 0 events |
| NFX-179 Gel Low (0.05%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of mild burning | 0 events |
| NFX-179 Gel Low (0.05%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of moderate pruritus | 1 events |
| NFX-179 Gel Mid (0.15%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of moderate pruritus | 2 events |
| NFX-179 Gel Mid (0.15%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of mild edema | 1 events |
| NFX-179 Gel Mid (0.15%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of mild stinging | 0 events |
| NFX-179 Gel Mid (0.15%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of mild pruritus | 7 events |
| NFX-179 Gel Mid (0.15%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of mild burning | 0 events |
| NFX-179 Gel Mid (0.15%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of moderate erythema | 0 events |
| NFX-179 Gel Mid (0.15%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of mild erythema | 1 events |
| NFX-179 Gel Mid (0.15%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of scabbing/crusting | 0 events |
| NFX-179 Gel High (0.50%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of scabbing/crusting | 1 events |
| NFX-179 Gel High (0.50%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of moderate pruritus | 0 events |
| NFX-179 Gel High (0.50%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of mild erythema | 1 events |
| NFX-179 Gel High (0.50%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of mild edema | 0 events |
| NFX-179 Gel High (0.50%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of mild pruritus | 4 events |
| NFX-179 Gel High (0.50%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of mild burning | 0 events |
| NFX-179 Gel High (0.50%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | subject reported instances of mild stinging | 4 events |
| NFX-179 Gel High (0.50%) | Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment | investigator reported instances of moderate erythema | 1 events |
Secondary
Change in Physician Assessment of Tumor Severity Score
Effect of treatment with The Physician Tumor Assessment is the investigator's assessment of the average overall severity of each Target cNF tumor at a particular time point. The Physician Tumor Assessment is a 5-point measuring tumor severity (0 clear/none, 1 almost clear, 2 mild, 3 moderate, 4 severe). The assessment is performed at the Baseline visit and week 4 visit.
Time frame: Baseline through Week 4
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Vehicle Arm (Placebo) | Change in Physician Assessment of Tumor Severity Score | Visit 2 | 2.74 Change in Investigator Assessment score | Standard Deviation 0.53 |
| Vehicle Arm (Placebo) | Change in Physician Assessment of Tumor Severity Score | Visit 5 | 2.51 Change in Investigator Assessment score | Standard Deviation 0.58 |
| NFX-179 Gel Low (0.05%) | Change in Physician Assessment of Tumor Severity Score | Visit 5 | 3.58 Change in Investigator Assessment score | Standard Deviation 0.62 |
| NFX-179 Gel Low (0.05%) | Change in Physician Assessment of Tumor Severity Score | Visit 2 | 2.46 Change in Investigator Assessment score | Standard Deviation 0.62 |
| NFX-179 Gel Mid (0.15%) | Change in Physician Assessment of Tumor Severity Score | Visit 2 | 2.60 Change in Investigator Assessment score | Standard Deviation 0.57 |
| NFX-179 Gel Mid (0.15%) | Change in Physician Assessment of Tumor Severity Score | Visit 5 | 2.50 Change in Investigator Assessment score | Standard Deviation 0.59 |
| NFX-179 Gel High (0.50%) | Change in Physician Assessment of Tumor Severity Score | Visit 2 | 2.65 Change in Investigator Assessment score | Standard Deviation 0.7 |
| NFX-179 Gel High (0.50%) | Change in Physician Assessment of Tumor Severity Score | Visit 5 | 2.70 Change in Investigator Assessment score | Standard Deviation 0.67 |
Secondary
Change in Subject Self-Assessment of Tumor Severity Score
The Subject Self-Assessment is the subject's assessment of the average overall severity of each Target cNF at a particular time point and is not a comparison with any other time point. The Subject Self-Assessment is a 5-point measuring tumor severity (0 clear/none, 1 almost clear, 2 mild, 3 moderate, 4 severe). The assessment is performed at the Baseline visit and week 4 visit.
Time frame: Baseline through Week 4
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Vehicle Arm (Placebo) | Change in Subject Self-Assessment of Tumor Severity Score | Visit 2 | 2.75 change in subject assessment score | Standard Deviation 0.73 |
| Vehicle Arm (Placebo) | Change in Subject Self-Assessment of Tumor Severity Score | Visit 5 | 2.50 change in subject assessment score | Standard Deviation 0.6 |
| NFX-179 Gel Low (0.05%) | Change in Subject Self-Assessment of Tumor Severity Score | Visit 5 | 2.76 change in subject assessment score | Standard Deviation 0.55 |
| NFX-179 Gel Low (0.05%) | Change in Subject Self-Assessment of Tumor Severity Score | Visit 2 | 2.81 change in subject assessment score | Standard Deviation 0.53 |
| NFX-179 Gel Mid (0.15%) | Change in Subject Self-Assessment of Tumor Severity Score | Visit 2 | 2.71 change in subject assessment score | Standard Deviation 0.38 |
| NFX-179 Gel Mid (0.15%) | Change in Subject Self-Assessment of Tumor Severity Score | Visit 5 | 2.50 change in subject assessment score | Standard Deviation 0.56 |
| NFX-179 Gel High (0.50%) | Change in Subject Self-Assessment of Tumor Severity Score | Visit 2 | 2.38 change in subject assessment score | Standard Deviation 0.71 |
| NFX-179 Gel High (0.50%) | Change in Subject Self-Assessment of Tumor Severity Score | Visit 5 | 2.39 change in subject assessment score | Standard Deviation 0.61 |
Secondary
Percent Change in cNF Tumor Volume (Cubic Millimeters)
Percent change in cNF tumor volume after 28 days of QD applications of NFX-179 gel based on tumor volume derived from ruler measurements.
Time frame: Baseline through Week 4
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Vehicle Arm (Placebo) | Percent Change in cNF Tumor Volume (Cubic Millimeters) | -8.0 percentage change | Standard Deviation 18.2 |
| NFX-179 Gel Low (0.05%) | Percent Change in cNF Tumor Volume (Cubic Millimeters) | -1.6 percentage change | Standard Deviation 22.1 |
| NFX-179 Gel Mid (0.15%) | Percent Change in cNF Tumor Volume (Cubic Millimeters) | -11.9 percentage change | Standard Deviation 29.4 |
| NFX-179 Gel High (0.50%) | Percent Change in cNF Tumor Volume (Cubic Millimeters) | -16.7 percentage change | Standard Deviation 30.3 |
Secondary
Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration
Systemic exposure of NFX-179 will be measured during the 28 days of QD applications at select investigative sites. Pharmacokinetic samples will be drawn at the baseline visit and week 4 visit. 5 time point samples will be collected at the week 4 visit.
Time frame: Baseline through Week 4
Population: Pharmacokinetic data from subjects treated with active NFX-179 gel was performed, which does not include samples from subjects treated with Vehicle gel (placebo). As such, no analysis pharmacokinetic analysis was performed in the Vehicle Arm.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| NFX-179 Gel Low (0.05%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, post dose 30 min | NA ng/mL |
| NFX-179 Gel Low (0.05%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Baseline, pre dose (0 hour) | NA ng/mL |
| NFX-179 Gel Low (0.05%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, post dose 1 hour | NA ng/mL |
| NFX-179 Gel Low (0.05%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, post dose 4 hours | NA ng/mL |
| NFX-179 Gel Low (0.05%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, pre dose (0 hour) | NA ng/mL |
| NFX-179 Gel Low (0.05%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, post dose 2 hours | NA ng/mL |
| NFX-179 Gel Mid (0.15%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, pre dose (0 hour) | NA ng/mL |
| NFX-179 Gel Mid (0.15%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Baseline, pre dose (0 hour) | NA ng/mL |
| NFX-179 Gel Mid (0.15%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, post dose 30 min | NA ng/mL |
| NFX-179 Gel Mid (0.15%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, post dose 1 hour | NA ng/mL |
| NFX-179 Gel Mid (0.15%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, post dose 2 hours | NA ng/mL |
| NFX-179 Gel Mid (0.15%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, post dose 4 hours | NA ng/mL |
| NFX-179 Gel High (0.50%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, pre dose (0 hour) | 0.439 ng/mL |
| NFX-179 Gel High (0.50%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, post dose 4 hours | 0.528 ng/mL |
| NFX-179 Gel High (0.50%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, post dose 2 hours | 0.718 ng/mL |
| NFX-179 Gel High (0.50%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, post dose 30 min | 0.887 ng/mL |
| NFX-179 Gel High (0.50%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Baseline, pre dose (0 hour) | NA ng/mL |
| NFX-179 Gel High (0.50%) | Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration | Week 4, post dose 1 hour | 0.573 ng/mL |