NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)

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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

, , 

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

, , 


Verification Date

November 2021