Kybella vs Asclera for Neurofibroma

Phase 1

Recruiting

Led By Richard R Anderson, MD

Research Sponsored by Massachusetts General Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Seeking treatment for cNF

Diagnosis of NF1 based on germline genetic testing or meeting specific clinical criteria

Must not have

Unable to give informed consent or adhere to study schedule

Dysphagia

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment

Awards & highlights

No Placebo-Only Group

Summary

This trial will look at if Kybella & Asclera injections are effective & safe for treating Neurofibromatosis Type 1 Cutaneous Neurofibromas. Patients will have one site treated & one as a control.

Who is the study for?

Adults over 18 with Neurofibromatosis Type 1 and at least six paired cutaneous neurofibromas on their trunk, arms, or legs can join this trial. Participants must not be pregnant, have a history of severe allergies to the study substances, bleeding disorders, or be undergoing other treatments for their skin lesions.

What is being tested?

The trial is testing Kybella and Asclera injections on different sites of the body to see which one is better tolerated and more effective in treating Neurofibromatosis Type 1 related skin tumors.

What are the potential side effects?

Possible side effects include pain at the injection site, swelling, bruising, numbness, redness and hardness in the treatment area. There may also be risks of nerve injury leading to facial muscle weakness or an uneven smile.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am seeking treatment for cutaneous neurofibromatosis.

Select...

I have been diagnosed with NF1 through genetic testing or clinical evaluation.

Select...

I have at least 6 noticeable skin growths on my trunk, arms, or legs.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am unable to understand or follow the study's requirements.

Select...

I have difficulty swallowing.

Select...

I have a recent blood clot.

Select...

I have bleeding issues or am on blood thinning medication.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment for reporting.