Brief Title
NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
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
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.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
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
Secondary Outcome
Percent of subjects with at least 50% Target cNF volume reduction after 182 days
Condition
Cutaneous Neurofibroma
Intervention
NFX-179 gel
Study Arms / Comparison Groups
0.5% NFX-179 gel
Description: Topical gel applied once daily to target cNFs
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
168
Start Date
September 20, 2021
Completion Date
February 2023
Primary Completion Date
January 2023
Eligibility Criteria
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 10 Target cNFs with 2 cNFs on the face and 8 cNFs on the anterior trunk or
upper extremities, that each 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
- Has a length that is ≥7mm and ≤14mm
- Has a width that is ≥5mm and ≤14mm
- Has a height that is ≥2mm.
5. Subject agrees to avoid exposure of Target cNFs to excessive sunlight and to use
her/his routine sunscreen if excessive exposure cannot be avoided
6. Subject agrees NOT to use tanning beds
7. Subject is willing to forego treatment of each Target cNF, except protocol specified
therapy, during the study
8. 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
9. Subject is willing and able to follow all study instructions and to attend all study
visits.
Exclusion Criteria:
1. 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.
2. 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
3. Subject has a history of hypersensitivity to any of the ingredients in the study
medications
4. 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
5. Subject has, in the investigator's opinion, clinically relevant history of liver
disease, including viral hepatitis, current alcohol abuse, or cirrhosis
6. 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
7. 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
8. Subject has participated in an investigational drug trial in which administration of
an investigational study medication occurred within the previous 30 days
Gender
All
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
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Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT05005845
Organization ID
NFX-179-NF1-202
Responsible Party
Sponsor
Study Sponsor
NFlection Therapeutics, Inc.
Study Sponsor
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Verification Date
November 2021