Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas

NCT06120036 | Completed Phase 1 FDA Drug

• 1 other identifier: 2022P001946
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

This study will evaluate the tolerability and effectiveness of two treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: Kybella and Asclera injection. Each patient will have a treatment and a control site.

Conditions

Neurofibromatosis 1

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

  Device based treatment will be considered tolerable if \<40% of participants treated have a \>grade 2 adverse event (AE). A grade 2 AE is defined as an adverse event that requires treatment.

  3 months after treatment

Secondary Outcomes (3)

• Treatment specific patient reported outcomes (PRO)

  Baseline, 1 day after treatment, 1 week after treatment, 1 month after treatment, 2 months after treatment, 3 months after treatment, 6 months after treatment, 12 months after treatment

• Clinician reported outcomes (ClinRO)

  Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment

• Modified SkinDex for cNF

  Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment

Other Outcomes (3)

• Rate of healing

  Baseline, 3 months after treatment, 6 months after treatment, and 12 months after treatment

• cNF appearance

  Baseline, 3 months after treatment, 6 months after treatment, and 12 months after treatment

• Biologic effect

  3 months after treatment

Study Arms (2)

Kybella Injection

ACTIVE COMPARATOR

Drug: Kybella

Asclera Injection

ACTIVE COMPARATOR

Drug: Asclera

Interventions

Kybella DRUG

Injection into the cutaneous Neurofibromas lesion.

Kybella Injection

Asclera DRUG

Injection into the cutaneous Neurofibromas lesion.

Asclera Injection

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult males and females ≥18 years of age
• Have a diagnosis of NF1 based on germline genetic testing or by meeting ≥ 2 the following criteria:
• Family history of NF1
• Six or more light brown ("cafe-au-lait") spots on the skin
• Presence of two or more neurofibromas of any type, or one or more plexiform neurofibromas
• Freckling under the arms or in the groin area
• Two or more pigmented, benign bumps on the eye's iris (Lisch nodules)
• A distinctive bony lesion: dysplasia (abnormal growth) of the sphenoid bone behind the eye, or dysplasia of long bones, often in the lower leg
• Tumor on the optic nerve that may interfere with vision
• Patients must be seeking treatment for cNF
• Patients must have ≥ 6 paired cNF per modality (3 treated and 3 untreated). cNF should be visible and measure between 2-8mm in size. These must be in areas amenable to treatment and surveillance with digital photography.
• cNF must be located on the trunk, arms or legs of the patient
• Able and willing to comply with all visit, treatment and evaluation schedules and requirements
• Able to understand and provide written informed consent

You may not qualify if:

• Patients who are undergoing other treatment modalities or investigational agents for their cNF lesions
• Individuals who cannot give informed consent or adhere to study schedule
• Actively tanning during the course of the study
• Adverse reactions to compounds of any external agent (e.g., gels, lotions or anesthetic creams) required for use in the study, if no alternative to the said agent exists;
• Known allergy to injectable anesthetics, polidocanol or deoxycholic acid
• Those with acute thromboembolic diseases
• Those with bleeding abnormalities or those who are currently being treated with antiplatelet or anticoagulant therapy
• Those with dysphagia
• Women who are pregnant
• Any condition which, in the Investigator's opinion, would make it unsafe (for the participant or study personnel) to treat the participant as part of this research study

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Johns Hopkins University: collaborator

Study Sites (1)

Wellman Center for Photomedicine

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Neurofibromatosis 1

Interventions

Deoxycholic Acid; Polidocanol

Condition Hierarchy (Ancestors)

Neurofibromatoses; 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; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Cholic Acids; Bile Acids and Salts; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Cholanes; Polyethylene Glycols; Ethylene Glycols; Glycols; Alcohols; Organic Chemicals; Polymers; Macromolecular Substances; Biomedical and Dental Materials; Manufactured Materials; Technology, Industry, and Agriculture

Study Officials

• Richard R Anderson, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Wellman Center for Photomedicine

Study Record Dates

• First Submitted: November 1, 2023
• First Posted: November 7, 2023
• Study Start: December 6, 2022
• Primary Completion: January 9, 2025
• Study Completion: January 9, 2025
• Last Updated: February 21, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)