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

NCT ID: NCT06120036

Last Updated: 2025-02-21

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-12-06
• Study Completion Date: 2025-01-09

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: OTHER
• Blinding Strategy: NONE

Study Groups

Kybella Injection

Group Type: ACTIVE_COMPARATOR

Kybella

Intervention Type: DRUG

Injection into the cutaneous Neurofibromas lesion.

Asclera Injection

Group Type: ACTIVE_COMPARATOR

Asclera

Intervention Type: DRUG

Injection into the cutaneous Neurofibromas lesion.

Interventions

Kybella

Injection into the cutaneous Neurofibromas lesion.

Intervention Type: DRUG

Asclera

Injection into the cutaneous Neurofibromas lesion.

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Johns Hopkins University

OTHER

Sponsor Role: collaborator

Massachusetts General Hospital

OTHER

Sponsor Role: lead

Responsible Party

Richard Rox Anderson, MD

Director, Wellman Center for Photomedicine

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Richard R Anderson, MD

Role: PRINCIPAL_INVESTIGATOR

Massachusetts General Hospital

Locations

Wellman Center for Photomedicine

Boston, Massachusetts, United States

Countries

United States

Other Identifiers

2022P001946

Identifier Type: -

Identifier Source: org_study_id