NCT06300502
Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)
Enrolling by Invitation
PHASE1Ages 18–85InterventionalMassachusetts General HospitalInvestigator-initiated
~15 participants
Updated 2026-04-22 on ClinicalTrials.gov
What's tested:Kybella
At a glance
Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Measured over 3 months after treatment
Conditions
Where it's being run
1 sites across 1 statesMassachusetts1
Study leadership
- Richard R Anderson, MD · PRINCIPAL_INVESTIGATOR · Massachusetts General Hospital
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
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. 3. Participant is seeking treatment for cNF. 4. Participant has ≥ 6 paired cNF that are visible and measure 2 mm or more in size. The target treatment area must be amenable to both deoxycholate injections and surveillance with digital and 3D photography. Preferred locations are trunk (back or chest), arms and legs. 5. Participant is able and willing to comply with all visit, treatment and evaluation schedules and requirements. 6. Participant is able to understand and provide written informed consent. 7. Participant has no known allergy to deoxycholate. 8. Participant has no concurrent injury or wound in the target area.
What this trial measures
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]3 months after treatment
Treatment will be considered tolerable if \<40% of participants treated have a \>grade 2 CTCAE v5 adverse event (AE).