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

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ClinicalTrials.gov Identifier: NCT05005845
Recruitment Status : Recruiting
First Posted : August 16, 2021
Last Update Posted : June 7, 2022
Sponsor:
Information provided by (Responsible Party):
NFlection Therapeutics, Inc.

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 168 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
Estimated Primary Completion Date : January 2023
Estimated Study Completion Date : February 2023


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 :
  1. 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.

  2. 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 :
  1. 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.

  2. 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.

  3. 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.

  4. 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:
  1. 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

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

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
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Sponsors and Collaborators
NFlection Therapeutics, Inc.
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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: June 7, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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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