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

Phase 1

Completed

• Conditions: Neurofibromatosis 1
• Interventions: Drug: Kybella; Drug: Asclera

• Registration Number: NCT06120036
• Lead Sponsor: Massachusetts General Hospital

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2022-12-06
• Study First Submitted: 2023-11-01
• Study First Submitted QC: 2023-11-01
• Study First Posted: 2023-11-07
• Primary Completion: 2025-01-09
• Study Completion: 2025-01-09
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-19
• Last Update Posted: 2025-02-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1. Adult males and females ≥18 years of age

2. Have a diagnosis of NF1 based on germline genetic testing or by meeting ≥ 2 the following criteria:

   1. Family history of NF1
   2. Six or more light brown ("cafe-au-lait") spots on the skin
   3. Presence of two or more neurofibromas of any type, or one or more plexiform neurofibromas
   4. Freckling under the arms or in the groin area
   5. Two or more pigmented, benign bumps on the eye's iris (Lisch nodules)
   6. A distinctive bony lesion: dysplasia (abnormal growth) of the sphenoid bone behind the eye, or dysplasia of long bones, often in the lower leg
   7. Tumor on the optic nerve that may interfere with vision

3. Patients must be seeking treatment for cNF

4. 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.

5. cNF must be located on the trunk, arms or legs of the patient

6. Able and willing to comply with all visit, treatment and evaluation schedules and requirements

7. Able to understand and provide written informed consent

Exclusion Criteria

1. Patients who are undergoing other treatment modalities or investigational agents for their cNF lesions
2. Individuals who cannot give informed consent or adhere to study schedule
3. Actively tanning during the course of the study
4. 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;
5. Known allergy to injectable anesthetics, polidocanol or deoxycholic acid
6. Those with acute thromboembolic diseases
7. Those with bleeding abnormalities or those who are currently being treated with antiplatelet or anticoagulant therapy
8. Those with dysphagia
9. Women who are pregnant
10. 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Kybella Injection	Kybella	-
Asclera Injection	Asclera	-

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]	3 months after treatment	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.

Secondary Outcome Measures

Name	Time	Method
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	NRS11, modality specific satisfaction assessment
Clinician reported outcomes (ClinRO)	Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment	Clinician assessment of cNF via questionnaire. Physician rates degree of change of treated and control cNFs on a scale from -3 (no change) to 3 (very large improvement).
Modified SkinDex for cNF	Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment	Health-related quality of life measure. Asks how much participants have been bothered by cNFs over the past week from 0 (never bothered) to 5 (always bothered).

Trial Locations

Locations (1)

Wellman Center for Photomedicine; 🇺🇸 Boston, Massachusetts, United States