Neurofibromas, CutaneousPhase 1NCT06300502
Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)
A Phase 1 study testing Kybella in people with Neurofibromas, Cutaneous and Neurofibromatosis 1, run by Massachusetts General Hospital at 1 site in United States.
Trial summary
The goal of this clinical trial is to evaluate the tolerability and effectiveness of multiple treatments of an FDA-approved drug in those with Neurofibromatosis Type 1 (NF1) Cutaneous Neurofibromas (cNFs). Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Neurofibromas, Cutaneous, Neurofibromatosis 1
- What is being tested?
- Kybella
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Apr 2025to Feb 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant is an adult ≥18 years of age.
- Participant has a diagnosis of NF1 based on germline genetic testing or by meeting ≥ 2 the following criteria:
- 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,
Show all 15Show fewer
- 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.
- Participant is seeking treatment for cNF.
- 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.
- Participant is able and willing to comply with all visit, treatment and evaluation schedules and requirements.
- Participant is able to understand and provide written informed consent.
- Participant has no known allergy to deoxycholate.
- Participant has no concurrent injury or wound in the target area.
Who cannot join
- Participant cannot give informed consent or adhere to study schedule.
- Participant is actively tanning during the course of the study.
- Participant has a known allergy to deoxycholic acid.
- For female participants: those who are pregnant.
- Participant has 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Wellman Center for Photomedicine
Boston, Massachusetts, 02114
About this study
Official title: Assessing the Efficacy of Repeat, Monthly Treatments of Cutaneous Neurofibromas (cNFs)
The goal of this clinical trial is to evaluate the tolerability and effectiveness of multiple treatments of an FDA-approved drug in those with Neurofibromatosis Type 1 (NF1) Cutaneous Neurofibromas (cNFs). The main questions it aims to answer are:
Will performing:
Read more
- Up to 6 months treatment sessions
- A minimum of 30 days apart
- With up to 50 injections of deoxycholate into a maximum of 50 cNFs in a single region of the body (for a maximum total dose of 10 mL per monthly treatment session) result in tolerable local skin reactions and reduction in both individual cNF size by >50% as well as improved cNF appearance in the treated field?
Researchers will compare treated tumors and control tumors to see if the treatment is effective.
Participants will:
- Receive up to 6 monthly treatments with Kybella (deoxycholate). Treatment for a given tumor will be stopped when the tumor is assessed as clear clinically.
- Complete surveys asking about pain during and after treatments.
- Complete surveys asking about satisfaction with the treatments.
- Undergo 2D photography and 3D imaging of treatment areas.
- Optionally, receive biopsies of up to 6 treated lesions to investigate characteristics of tumors that respond well to treatment as well as non-respondent tumors.
- Conditions
- Neurofibromas, Cutaneous, Neurofibromatosis 1
- Being tested
- Kybella
- Medical subject headings
- Neurofibroma, Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KybellaDrug
Also called Deoxycholate, Deoxycholic acid
Kybella is sterile 1% deoxycholic acid provided in a 2 mL single-use vial.
| Study group | Role | What they receive |
|---|---|---|
| Treated cutaneous neurofibromas (cNFs) | Active comparator | Injection into up to 50 cutaneous Neurofibromas (for at total maximum of 10 mL injected) with Kybella (1% deoxycholic acid). Receives: Drug: Kybella |
| Control cutaneous neurofibromas (cNFs) | No intervention | A complementary region of cNFs of similar characteristics to the treatment area in the same body region will be selected to serve as an untreated control group. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Treatment will be considered tolerable if <40% of participants treated have a >grade 2 CTCAE v5 adverse event (AE).
Measured: 3 months after treatment
Secondary outcomes (7)
Patient Reported Outcomes
Using questionnaires we will determine the patients' reported outcomes. Patient Global Assessment: Patient rates degree of change of treated and control cNFs on a scale from -3 (no change) to 3 (very large improvement).
Measured: Baseline, after treatment session #4, and at 3 months after final treatment.
Clinician Reported Outcomes
Using questionnaires we will determine the clinicians' reported outcomes. Physician Global Assessment: Clinician rates degree of change of treated and control cNFs on a scale from -3 (no change) to 3 (very large improvement).
Measured: Baseline, after treatment session #4, and at 3 months after final treatment.
VAS Pain Scale
Participants will be asked to rate their pain using a visual analog scale (0 to 10) at each treatment visit and 1 week post-treatment.
Measured: Baseline, after each treatment session, 1 week after each treatment session, and 3 months after final treatment.
Patient Satisfaction
Satisfaction with each treatment modality is rated from 1 (very unsatisfied) to 5 (very satisfied).
Measured: Baseline, after treatment session #4, and at 3 months after final treatment.
Rate of Healing
Measured clinically via photography completed by a member of the study team at baseline, and all in-person visits.
Measured: Baseline, 1 month, 2 months, 3 months, 4 months, 5 months, 6 months after baseline; 3 months after final treatment.
cNF Appearance (Height)
Clinically completed 2D photography and 3D Cherry Imaging. Change from baseline in height of cNFs.
Measured: Baseline, after treatment session #4, and at 3 months after final treatment.
cNF Appearance (Volume)
Clinically completed 2D photography and 3D Cherry Imaging. Change from baseline in volume of cNFs.
Measured: Baseline, after treatment session #4, and at 3 months after final treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2024
- Study start8 Apr 2025
- Primary completionFeb 2027 (estimated)
- Study completionFeb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromas, CutaneousStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Johns Hopkins University
- Responsible party
- Principal investigator, Richard Rox Anderson, MD, Massachusetts General Hospital
- Investigators
- Richard R Anderson, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06300502
- Sponsor's protocol ID
- 2023P003620
- Status last verified
- Apr 2026
- First submitted
- 13 Feb 2024
- Last updated
- 22 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessing the Efficacy of Repeat, Monthly Treatments of Cutaneous Neurofibromas (cNFs)
- ClinicalTrials.gov ID
- NCT06300502
- Acronym / sponsor ID
- 2023P003620
- Conditions
- Neurofibromas, Cutaneous, Neurofibromatosis 1
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- Johns Hopkins University
- Study start
- 8 Apr 2025 (actual)
- Primary completion
- Feb 2027 (estimated)
- Study completion
- Feb 2027 (estimated)
- First posted
- 8 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to evaluate the tolerability and effectiveness of multiple treatments of an FDA-approved drug in those with Neurofibromatosis Type 1 (NF1) Cutaneous Neurofibromas (cNFs). The main questions it aims to answer are:
Will performing:
- Up to 6 months treatment sessions
- A minimum of 30 days apart
- With up to 50 injections of deoxycholate into a maximum of 50 cNFs in a single region of the body (for a maximum total dose of 10 mL per monthly treatment session) result in tolerable local skin reactions and reduction in both individual cNF size by >50% as well as improved cNF appearance in the treated field?
Researchers will compare treated tumors and control tumors to see if the treatment is effective.
Participants will:
- Receive up to 6 monthly treatments with Kybella (deoxycholate). Treatment for a given tumor will be stopped when the tumor is assessed as clear clinically.
- Complete surveys asking about pain during and after treatments.
- Complete surveys asking about satisfaction with the treatments.
- Undergo 2D photography and 3D imaging of treatment areas.
- Optionally, receive biopsies of up to 6 treated lesions to investigate characteristics of tumors that respond well to treatment as well as non-respondent tumors.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treated cutaneous neurofibromas (cNFs) | Active comparator | Drug: KybellaInjection into up to 50 cutaneous Neurofibromas (for at total maximum of 10 mL injected) with Kybella (1% deoxycholic acid). |
| Control cutaneous neurofibromas (cNFs) | No intervention | –A complementary region of cNFs of similar characteristics to the treatment area in the same body region will be selected to serve as an untreated control group. |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Participant is an adult ≥18 years of age.
- Participant has a diagnosis of NF1 based on germline genetic testing or by meeting ≥ 2 the following criteria:
- Participant is seeking treatment for cNF.
- 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.
- Participant is able and willing to comply with all visit, treatment and evaluation schedules and requirements.
Key exclusion criteria (5 listed)
- Participant cannot give informed consent or adhere to study schedule.
- Participant is actively tanning during the course of the study.
- Participant has a known allergy to deoxycholic acid.
- For female participants: those who are pregnant.
- Participant has 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KybellaAlso: Deoxycholate, Deoxycholic acidKybella is sterile 1% deoxycholic acid provided in a 2 mL single-use vial. | Drug | Treated cutaneous neurofibromas (cNFs) |
Primary outcome
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: 3 months after treatment
Treatment will be considered tolerable if <40% of participants treated have a >grade 2 CTCAE v5 adverse event (AE).
7 secondary outcomes
Patient Reported Outcomes
Time frame: Baseline, after treatment session #4, and at 3 months after final treatment.
Clinician Reported Outcomes
Time frame: Baseline, after treatment session #4, and at 3 months after final treatment.
VAS Pain Scale
Time frame: Baseline, after each treatment session, 1 week after each treatment session, and 3 months after final treatment.
Patient Satisfaction
Time frame: Baseline, after treatment session #4, and at 3 months after final treatment.
Rate of Healing
Time frame: Baseline, 1 month, 2 months, 3 months, 4 months, 5 months, 6 months after baseline; 3 months after final treatment.
cNF Appearance (Height)
Time frame: Baseline, after treatment session #4, and at 3 months after final treatment.
cNF Appearance (Volume)
Time frame: Baseline, after treatment session #4, and at 3 months after final treatment.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Richard R Anderson, MD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Kybella in people with Neurofibromas, Cutaneous and Neurofibromatosis 1, run by Massachusetts General Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 22 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: 3 months after treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Apr 2026.