NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT05005845 |
|
Recruitment Status :
Completed
First Posted : August 16, 2021
Last Update Posted : November 18, 2023
|
Sponsor:
NFlection Therapeutics, Inc.
Information provided by (Responsible Party):
NFlection Therapeutics, Inc.
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Brief Summary:
This is a randomized, double-blind, vehicle-controlled, parallel group dose response study evaluating the safety and effectiveness of 2 concentrations of NFX-179 Gel in subjects with cutaneous neurofibromas. At Visit 1, the investigator will identify 10 Target cNFs that fulfil the enrollment criteria. The Target cNFs must be located on the subject's face, anterior trunk, or upper extremities. Two Target cNFs must be on the face and 8 must be on the anterior trunk or upper extremities. The study medication will be applied topically QD to the Target cNFs for 182days (26 weeks). During the duration of the study subjects will be evaluated for safety and efficacy.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Cutaneous Neurofibroma Neurofibromatosis 1 | Drug: NFX-179 gel Drug: Vehicle gel | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 199 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Randomized, Double-Blind, Vehicle-Controlled, Parallel Group Phase 2 Dose-Response Study to Determine Safety and Effectiveness of Two Concentrations of NFX-179 Gel in Subjects With Cutaneous Neurofibromas |
| Actual Study Start Date : | September 20, 2021 |
| Actual Primary Completion Date : | October 4, 2023 |
| Actual Study Completion Date : | October 4, 2023 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Neurofibromatosis type 1
MedlinePlus related topics:
Neurofibromatosis
| Arm | Intervention/treatment |
|---|---|
|
Active Comparator: 0.5% NFX-179 gel
Topical gel applied once daily to target cNFs
|
Drug: NFX-179 gel
NFX-179 topical gel is the active investigational product being studied
Other Name: NFX-179 topical gel |
|
Active Comparator: 1.5% NFX-179 gel
Topical gel applied once daily to target cNFs
|
Drug: NFX-179 gel
NFX-179 topical gel is the active investigational product being studied
Other Name: NFX-179 topical gel |
|
Placebo Comparator: Vehicle gel
Topical gel applied once daily to target cNFs
|
Drug: Vehicle gel
NFX-179 vehicle gel is the placebo comparator for this study
Other Name: NFX-179 vehicle gel |
Primary Outcome Measures :
- Safety and effectiveness of once daily NFX-179 gel treatment for 182 days measured by local tolerability assessments, safety laboratory tests, and target cNF volume reduction [ Time Frame: 182 days ]Safety will be measured by routine safety laboratory tests (CBC/differential, serum chemistry, urinalysis), and local tolerability assessment where 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). Effectiveness will be measured by the percent of treated subjects with at least a 50% reduction in cNF volume after 6 months of treatment.
- Safety of once daily NFX-179 gel treatment for 182 days by adverse events and serious adverse events assessments [ Time Frame: 182 days ]Safety of NFX-179 gel compared to the vehicle group will be measured by the assessment and occurrence of new and ongoing adverse events (AEs)/serious adverse events (SAEs).
Secondary Outcome Measures :
- Percent of subjects with at least 50% Target cNF volume reduction after 182 days [ Time Frame: 182 days ]Treatment effectiveness will be measured by the percent of treated subjects with at least a 50% reduction in cNF volume after 6 months of treatment. Tumor volume will be measured by ruler measurements and two-dimensional photography. All modes of tumor volume measurement are reported in cubic millimeters.
- Percent change in cNF volume over 182 days [ Time Frame: 182 days ]Percent change of tumor volume will be calculated through ruler measurements and two-dimensional photography throughout the course of treatment. All modes of tumor volume measurement are reported in cubic millimeters.
- Physician's Tumor Assessment of Target cNF severity over 182 days [ Time Frame: 182 days ]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). This assessment is performed at Screening, Baseline/Day 1, Days 58, 183, and 211.
- Subject's Self-Assessment of Target cNF severity over 182 days [ Time Frame: 182 days ]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). This assessment is performed at Screening, Baseline/Day 1, Days 58, 183, and 211.
Other Outcome Measures:
- Patient Reported Outcome Measure to assess Target cNF symptoms over 182 days [ Time Frame: 182 days ]This assessment is performed via the subject's assessment of their experience over the previous week with 9 items for each Target cNF (sensitivity, pain, itch, noticeability, size, appearance, care to avoid irritation, how much the cNF bothers the subject, self-consciousness). Each item is assessed by using a 5-point scale.
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Subject is at least 18 years of age
- Subject must provide written informed consent prior to any study procedures
- Subject must have a clinical diagnosis of NF1
-
Subject has 10 clinically diagnosed Target cNFs with preferably 2 Target cNFs located on the face and 8 Target cNFs located on the anterior trunk or upper extremities. Alternatively, at least 1 Target cNF is located on the face, in which case 9 Target cNFs must be located on the anterior trunk or upper extremities. Each Target cNF must meet the following criteria:
- Has, in the investigator's opinion, a clinically typical appearance
- Is not within 1 cm of the orbital rim
- Is not covered with hair that might, in the investigator's opinion, interfere with obtaining photographs or impair evaluation of the cNF
- Has a Physician's Tumor Assessment grade ≥2
- Is dome shaped
- Is not pedunculated
- Is a discrete cNF surrounded by sufficient non-affected skin that, in the investigator's opinion:
- The dimensions can be measured
- The perimeter can be outlined in the study photographs
- Is not irritated (e.g., bleeding, inflamed)
- 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
- Target cNFs on the face must have the following tumor dimensions:
- Has a length that is ≥5mm and ≤14mm
- Has a width that is ≥5mm and ≤14mm
- Has a height that is ≥2mm.
- Target cNFs on the anterior trunk or upper extremities must have the following tumor dimensions:
- Has a length that is ≥7mm and ≤14mm
- Has a width that is ≥5mm and ≤14mm
- Has a height that is ≥2mm.
- Subject agrees to avoid exposure of Target cNFs to excessive sunlight and to use her/his routine sunscreen if excessive exposure cannot be avoided
- Subject agrees NOT to use tanning beds
- Subject is willing to forego treatment of each Target cNF, except protocol specified therapy, during the study
- 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
- Subject is willing and able to follow all study instructions and to attend all study visits.
Exclusion Criteria:
-
Subject has used any of the following topical therapies within the specified period prior to Visit 1 on or in proximity to any Target cNF that, in the investigator's opinion, impairs evaluation of any the cNFs or which exposes the subject to an unacceptable risk by study participation:
- Corticosteroids; 30 days
- Prescription retinoids (e.g., tazarotene, tretinoin, adapalene); 30 days
- > 5% of an alpha-hydroxy acid (e.g., glycolic acid, lactic acid); 30 days
- Fluorouracil; 30 days
- Imiquimod; 30 days
- LASER, light (e.g., intense pulsed light [IPL], photo-dynamic therapy [PDT]) or other energy-based therapy; 180 days
- MEK inhibitor or BRAF inhibitor; ever.
-
The subject has used any of the following systemic medications therapies within the specified period prior to Visit 1:
- Retinoids (e.g., etretinate, isotretinoin); 90 days
- MEK inhibitors; 180 days
- BRAF inhibitors; 180 days
- Subject has a history of hypersensitivity to any of the ingredients in the study medications
- Subject has any known intercurrent illness or physical condition that would, in the investigator's opinion, impair evaluation of a Target cNF or which exposes the subject to an unacceptable risk by study participation
- Subject has, in the investigator's opinion, clinically relevant history of liver disease, including viral hepatitis, current alcohol abuse, or cirrhosis
- 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
- 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 or which exposes the subject to an unacceptable risk by study participation
- Subject has participated in an investigational drug trial in which administration of an investigational study medication occurred within the previous 30 days
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05005845
Locations
Show 23 study locations
Sponsors and Collaborators
NFlection Therapeutics, Inc.
| Responsible Party: | NFlection Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT05005845 |
| Other Study ID Numbers: |
NFX-179-NF1-202 |
| First Posted: | August 16, 2021 Key Record Dates |
| Last Update Posted: | November 18, 2023 |
| Last Verified: | November 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Additional relevant MeSH terms:
|
Neurofibromatoses Neurofibromatosis 1 Neurofibroma Nerve Sheath Neoplasms Neoplasms, Nerve Tissue Neoplasms by Histologic Type Neoplasms Neoplastic Syndromes, Hereditary Neurocutaneous Syndromes |
Nervous System Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Peripheral Nervous System Diseases Neuromuscular Diseases Peripheral Nervous System Neoplasms Nervous System Neoplasms |