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

Neurofibromatosis 1 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06120036
• Other study ID #: 2021P001946
• Status: Recruiting
• Phase: Phase 1
• Start date: December 6, 2022
• Est. completion date: September 30, 2024

Study information

• Verified date: November 2023
• Source: Massachusetts General Hospital
• Contact: Devon Gunter
• Phone: 617-724-2168
• Email: wellmancrc[@]partners.org
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: September 30, 2024
• Est. primary completion date: April 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 85 Years

Eligibility

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

Related Conditions & MeSH terms

• Neurofibroma
• Neurofibromatoses
• Neurofibromatosis 1

Intervention

Drug:
• Kybella
Injection into the cutaneous Neurofibromas lesion.
• Asclera
Injection into the cutaneous Neurofibromas lesion.

Locations

Country	Name	City	State
United States	Wellman Center for Photomedicine	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Massachusetts General Hospital	Johns Hopkins University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Rate of healing	Measured clinically via photography completed by a member of the study team at baseline, 3-month, 6-month, and 12-month post-treatment	Baseline, 3-months after treatment, 6-month after treatment, and 12-months after treatment
Other	cNF appearance	Clinically completed 2D and 3D photography Cherry Imaging.	Baseline, 3 months after treatment, 6 months after treatment, and 12 months after treatment
Other	Biologic effect	Degree of tissue necrosis on skin lesion biopsy at 3 months	3 months after treatment
Primary	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	Treatment specific patient reported outcomes (PRO)	NRS11, modality specific satisfaction assessment	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
Secondary	Clinician reported outcomes (ClinRO)	Clinician assessment of cNF via questionnaire	Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment
Secondary	Modified SkinDex for cNF	Health-related quality of life measure	Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment