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NFX-179 Topical Gel Treatment in 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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04435665
Recruitment Status : Recruiting
First Posted : June 17, 2020
Last Update Posted : October 9, 2020
Sponsor:
Information provided by (Responsible Party):
NFlection Therapeutics, Inc.

Brief Summary:
This study will enroll and treat subjects with cutaneous neurofibromas with NFX-179, a topical study drug. Eligible subjects will receive treatment for 28 days and be observed by a study doctor for approximately 56 days. Subjects will be randomly assigned to 1 of 4 treatment groups. 3 of the treatment groups will receive a specific dose NFX-179, and 1 group will receive placebo. The subject, study doctor, and NFlection Therapeutics will not know what treatment group each subject is assigned. Study participation requires at least 7 clinic visits, blood, urine, and tissue collection, images of the treated cutaneous neurofibromas, electrocardiograms, and information regarding the subject's medical and disease history.

Condition or disease Intervention/treatment Phase
Neurofibromatosis 1 Cutaneous Neurofibroma Drug: NFX-179 Gel Drug: Vehicle Gel Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 48 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 2a Study to Determine Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Activity of NFX-179 Gel in Subjects With Cutaneous Neurofibromas
Actual Study Start Date : August 21, 2020
Estimated Primary Completion Date : July 2021
Estimated Study Completion Date : July 2021


Arm Intervention/treatment
Experimental: NFX-179 Gel Low
NFX-179 Gel for topical administration, once daily for 28 days
Drug: NFX-179 Gel
gel for topical administration

Experimental: NFX-179 Gel Mid
NFX-179 Gel for topical administration, once daily for 28 days
Drug: NFX-179 Gel
gel for topical administration

Experimental: NFX-179 Gel High
NFX-179 Gel for topical administration, once daily for 28 days
Drug: NFX-179 Gel
gel for topical administration

Placebo Comparator: Vehicle Arm
Vehicle Gel, for topical administration, once daily for 28 days
Drug: Vehicle Gel
vehicle gel for topical administration




Primary Outcome Measures :
  1. 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

  2. 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).

  3. Assessment of adverse events/serious adverse events [ Time Frame: Baseline through Week 8 ]
    Assessment of adverse events (AEs)/serious adverse events (SAEs).


Secondary Outcome Measures :
  1. 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, ultrasound measurements and digital images. All modes of tumor volume measurement are reported in cubic millimeters.

  2. 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.

  3. Physician Tumor Assessment of the average overall severity of cNF tumor [ 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.

  4. Subject Self-Assessment of the average overall severity of each Target cNF tumor [ 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.



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.


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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 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.
  5. Subject is willing to have the 5 Target cNF Tumors and the 1 Untreated cNF Tumor excised at the end of the treatment period
  6. Subject is willing to have hair in the area surrounding the Target cNF Tumors shaved, if necessary, to obtain photographs
  7. Subject is willing to minimize exposure of each Target cNF to natural and artificial ultraviolet radiation
  8. Subject is willing to forego treatment of the Target cNF Tumors, except protocol specified therapy, during the study
  9. 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
  10. Subject is willing and able to follow all study instructions and to attend all study visits.

Exclusion Criteria:

  1. 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
  2. Any Study cNF Tumor has ever been treated with an MEK inhibitor or a BRAF inhibitor
  3. 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
  4. Subject has a history of hypersensitivity to any of the ingredients in the study medications
  5. 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
  6. Subject has, in the investigator's opinion, clinically relevant history of liver disease, including viral hepatitis, current alcohol abuse, or cirrhosis
  7. 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
  8. 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
  9. 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): NCT04435665


Contacts
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Contact: Christopher O'Mara, MS 7078532199 comara@nflectionrx.com

Locations
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United States, California
Center for Dermatology Clinical Research, Inc. Recruiting
Fremont, California, United States, 94538
Contact: Mele Tupou    510-797-0140 ext 4    melet@ctr4derm.com   
Contact: Natalia Cobian    510-797-0140 ext 8    nataliac@ctr4derm.com   
Principal Investigator: Sunil Dhawan, MD         
Sub-Investigator: Namrata Patyal, MD         
United States, Minnesota
Minnesota Clinical Study Center Recruiting
New Brighton, Minnesota, United States, 55112
Contact: Allison Horita    763-571-4200    ahorita@associatedskincare.com   
Contact: Buffi Elliott, LPN    (763) 571-4200    belliott@associatedskincare.com   
Principal Investigator: Steven Kempers, MD         
Sub-Investigator: Jane Lindholm, MD         
United States, New York
Skin Search of Rochester, Inc. Recruiting
Rochester, New York, United States, 14623
Contact: Denise Forster    585-697-1818    dforster@dermrochester.com   
Contact: Angela Malizia    (585) 697-1818    amalizia@dermrochester.com   
Principal Investigator: John Tu, MD         
Sub-Investigator: Lesley Loss, MD         
United States, Texas
DermResearch Recruiting
Austin, Texas, United States, 78758
Contact: Karina Garcia    512-349-9889    karina@driresearchsite.com   
Contact: Kelsey Peterson    (512) 349-9889    kelsey@driresearchsite.com   
Principal Investigator: Janet DuBois, MD         
Sub-Investigator: David Bushore, MD         
Sub-Investigator: Michael Edmond, MD         
University of Texas Southwestern Medical Center Recruiting
Dallas, Texas, United States, 75390
Contact: Seli Gyau    214-645-8968    Seli.Gyau@utsouthwestern.edu   
Principal Investigator: Lu Le, MD, PhD         
Sponsors and Collaborators
NFlection Therapeutics, Inc.
Investigators
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Study Director: Guy Webster, MD, PhD NFlection Therapeutics
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Responsible Party: NFlection Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04435665    
Other Study ID Numbers: NFX-179-NF1-201
First Posted: June 17, 2020    Key Record Dates
Last Update Posted: October 9, 2020
Last Verified: October 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by NFlection Therapeutics, Inc.:
NFX-179
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