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

Learn more about:
Related Clinical Trial
NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) Mechanism of Action of Transcranial Direct Current Stimulation in Neurofibromatosis Type 1 Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1 Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1 Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial Pilot Randomized Control Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1 Open Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1 Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial Intelligence A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN) Innovation in the Treatment of Persistent Pain in Adults With NF1: Implementation of the iCanCope Mobile Application- Clinical Trial Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1 Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1 A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) Analysis of Data Collected From Individuals Administered Neurobehavioral Assessments Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia Comparison of Gastrointestinal Motility in Healthy Children and Children With Constipation AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas Medical Treatment of “High-Risk” Neurofibromas Fludeoxyglucose F 18 Positron Emission Tomography and Magnetic Resonance Perfusion Imaging in Patients With Neurofibromatosis 1 and Plexiform Neurofibroma Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1 Use of Topical Liquid Diclofenac Following Laser Microporation of Cutaneous Neurofibromas in Patients With NF1 Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (PNs) Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas Treatment of NF1-related Plexiform Neurofibroma With Trametinib Subtle Myocardial Deformation Abnormalities in Asymptomatic Nf-1 Patients R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas Use of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1 AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the Spine MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform Neurofibromas Neurofibromatosis Type 1 Brain Tumor Genetic Risk Acceptance and Commitment Therapy for Adolescents and Young Adults With Neurofibromatosis and Chronic Pain Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas Adaptation and Quality of Life Among Adults With Neurofibromatosis Type I Reliability of Functional Outcome Measures in Neurofibromatosis 1 Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1 (NF1) on Clinical Treatment Trials Targeting the Mechanisms Underlying Cutaneous Neurofibroma Formation in NF1: A Clinical Translational Approach. Sorafenib to Treat Children and Young Adults With Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST) Quality of Friendships in Children With Neurofibromatosis Study of Disease Severity in Adults With Neurofibromatosis Type 1 (NF1) MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas Interventions for Reading Disabilities in NF1 Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1 Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic Pain Neurobiology and Treatment of Reading Disability in NF-1 From Molecules to Cognition: Inhibitory Mechanisms in ASD and NF1 Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1) Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1 Analysis of Plasma for Diagnosis and Follow-up of Neurofibromatosis Type 1 Study About Annoucement of the Diagnosis of Neurofibromatosis 1 in de Novo Forms MicroRNAs in Patients With Neurofibromatosis Type 1 Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas Stem Cells in NF1 Patients With Tumors of the Central Nervous System Function of the Pigment Epithelium in Patients With Type 1 Neurofibromatosis NF1-Attention: Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1) Internet Support Group for Parents of a Child With Neurofibromatosis Type 1 Reading Disability in Children With NF1 Multi-center Project: Spinal Abnormalities in Neurofibromatosis Type1 (NF1) Patients Functional Imaging and Reading Deficit in Children With NF1 Effects of Physical Training on Bone and Muscle Quality, Muscle Strength, and Motor Coordination in Children With NF1 Effect of Lamotrigine on Cognition in NF1 A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1 Neurofibromatosis Type 1 (NF1) and Tibial Dysplasia Non-invasive Stimulation in Neurofibromatosis Type 1 Modifying Genes in Neurofibromatosis 1 Natural History and Biology of Skin Neurofibromas in Neurofibromatosis Type 1 Vision, Attention and Reading in Neurofibromatosis Type 1 (NF1) Children Neuropsychological Impairment and Quality of Life in Neurofibromatosis Type 1 Spinal Abnormalities in Neurofibromatosis Type 1 (NF1) Neurofibromatosis Type 1 Patient Registry Frameshift Peptides of Children With NF1 Prevalence of Constitutional Mismatch-repair Deficiency Among Suspected Neurofibromatosis Type 1/Legius Syndrome Children Without a Malignancy and Without a NF1 or SPRED1 Mutation How Neurofibromatosis Type 1 (NF1) Affects Schoolwork and Self-Esteem

Brief Title

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

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

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.
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

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

Secondary Outcome

 Percent change in cNF tumor volume (cubic millimeters)

Condition

Neurofibromatosis 1

Intervention

NFX-179 Gel

Study Arms / Comparison Groups

 NFX-179 Gel Low
Description:  NFX-179 Gel for topical administration, once daily for 28 days

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

48

Start Date

August 21, 2020

Completion Date

April 14, 2021

Primary Completion Date

April 8, 2021

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 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
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Guy Webster, MD, PhD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04435665

Organization ID

NFX-179-NF1-201


Responsible Party

Sponsor

Study Sponsor

NFlection Therapeutics, Inc.


Study Sponsor

Guy Webster, MD, PhD, Study Director, NFlection Therapeutics


Verification Date

January 2021