- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04435665
NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
A Randomized, Double-Blind, Vehicle-Controlled, Parallel Group Phase 2a Study to Determine Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Activity of NFX-179 Gel in Subjects With Cutaneous Neurofibromas
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
-
California
-
Fremont, California, United States, 94538
- Center for Dermatology Clinical Research, Inc.
-
-
Minnesota
-
New Brighton, Minnesota, United States, 55112
- Minnesota Clinical Study Center
-
-
New York
-
Rochester, New York, United States, 14623
- Skin Search of Rochester, Inc.
-
-
Texas
-
Austin, Texas, United States, 78758
- DermResearch
-
Dallas, Texas, United States, 75390
- University of Texas Southwestern Medical Center
-
-
Virginia
-
Lynchburg, Virginia, United States, 24501
- The Education & Research Foundation, Inc.
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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 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.
- Subject is willing to have the 5 Target cNF Tumors and the 1 Untreated cNF Tumor excised at the end of the treatment period
- Subject is willing to have hair in the area surrounding the Target cNF Tumors shaved, if necessary, to obtain photographs
- Subject is willing to minimize exposure of each Target cNF to natural and artificial ultraviolet radiation
- Subject is willing to forego treatment of the Target cNF Tumors, 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 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
- Any Study cNF Tumor has ever been treated with an MEK inhibitor or a BRAF inhibitor
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
- 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 Tumor 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 Tumor 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: NFX-179 Gel Low
NFX-179 Gel for topical administration, once daily for 28 days
|
gel for topical administration
|
Experimental: NFX-179 Gel Mid
NFX-179 Gel for topical administration, once daily for 28 days
|
gel for topical administration
|
Experimental: NFX-179 Gel High
NFX-179 Gel for topical administration, once daily for 28 days
|
gel for topical administration
|
Placebo Comparator: Vehicle Arm
Vehicle Gel, for topical administration, once daily for 28 days
|
vehicle gel for topical administration
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
Time Frame: 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
|
Baseline through Week 4
|
Safety and Tolerability of NFX-179 Gel Measured by Local Tolerability Assessment
Time Frame: 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).
|
Baseline through Week 4
|
Assessment of Adverse Events
Time Frame: Baseline through Week 8
|
Assessment of adverse events (AEs)
|
Baseline through Week 8
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percent Change in cNF Tumor Volume (Cubic Millimeters)
Time Frame: 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.
|
Baseline through Week 4
|
Systemic Exposure of NFX-179 Gel Measured by Plasma Concentration
Time Frame: 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.
|
Baseline through Week 4
|
Change in Physician Assessment of Tumor Severity Score
Time Frame: 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.
|
Baseline through Week 4
|
Change in Subject Self-Assessment of Tumor Severity Score
Time Frame: 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.
|
Baseline through Week 4
|
Collaborators and Investigators
Sponsor
Investigators
- Study Director: Guy Webster, MD, PhD, NFlection Therapeutics
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Nervous System Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Genetic Diseases, Inborn
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Neoplasms, Nerve Tissue
- Peripheral Nervous System Diseases
- Nervous System Neoplasms
- Heredodegenerative Disorders, Nervous System
- Neoplastic Syndromes, Hereditary
- Nerve Sheath Neoplasms
- Neurocutaneous Syndromes
- Peripheral Nervous System Neoplasms
- Neurofibromatoses
- Neurofibromatosis 1
- Neurofibroma
Other Study ID Numbers
- NFX-179-NF1-201
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Neurofibromatosis 1
-
University of UtahUniversity of British Columbia; Children's Hospital Medical Center, Cincinnati and other collaboratorsTerminatedNeurofibromatosis Type 1 (NF1)United States, Canada
-
University of Alabama at BirminghamCompletedNeurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PNUnited States
-
Novartis PharmaceuticalsTerminatedPlexiform Neurofibroma Associated With Neurofibromatosis Type 1Israel
-
SpringWorks Therapeutics, Inc.Active, not recruitingPlexiform Neurofibroma | Neurofibromatosis Type 1 (NF1)United States
-
AstraZenecaCompletedHealthy Participants | Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs)United States
-
Johns Hopkins UniversityNeurofibromatosis Therapeutic Acceleration ProgramRecruitingNeurofibromatosis 1 | Neurofibromatosis Type 1 | Cutaneous Neurofibroma | Neurofibromatosis (Nonmalignant)United States
-
SpringWorks Therapeutics, Inc.AvailableNeurofibromatosis Type 1-Associated Plexiform Neurofibromas | Histiocytic Neoplasm | Other MAP-K Pathway Driven Diseases
-
National Cancer Institute (NCI)CompletedPlexiform Neurofibroma | Neurofibromatosis Type IUnited States
-
Ann & Robert H Lurie Children's Hospital of ChicagoNational Institute of Neurological Disorders and Stroke (NINDS); Children's...RecruitingNeurofibromatosis 1 | Neurofibromatosis Type 1 | NF1United States
-
Guy's and St Thomas' NHS Foundation TrustRecruitingNeurofibromatosis Type 1United Kingdom
Clinical Trials on NFX-179 Gel
-
NFlection Therapeutics, Inc.CompletedNeurofibromatosis 1 | Cutaneous NeurofibromaUnited States
-
Albert ChiouNFlection Therapeutics, Inc.Not yet recruitingEpidermal Nevi | Nevus SebaceusUnited States
-
Wyeth is now a wholly owned subsidiary of PfizerWithdrawnAdvanced Malignant Solid Tumors
-
OnKure, Inc.University of Colorado, DenverCompleted
-
OnKure, Inc.Active, not recruitingMelanoma | NRAS Gene Mutation | RAS MutationUnited States
-
Brii Biosciences LimitedVBI Vaccines Inc.; Vir Biotechnology, Inc.CompletedHepatitis B, ChronicAustralia, China, Korea, Republic of, New Zealand, Singapore, Thailand
-
Laboratorios Sophia S.A de C.V.Completed
-
Glyciome, LLCBrigham and Women's Hospital; University of Puerto RicoCompletedSensory Perceptual Characteristics | User Acceptability of Gel Delivery SystemUnited States, Puerto Rico
-
Starpharma Pty LtdCompletedBacterial VaginosisUnited States
-
Novan, Inc.CompletedAcne VulgarisDominican Republic, Honduras, Panama