Neurofibromatosis 1Phase 1NCT06120036
Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas
A Phase 1 study testing Kybella and Asclera in people with Neurofibromatosis 1, run by Massachusetts General Hospital at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Neurofibromatosis 1
- What is being tested?
- Kybella and Asclera
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Dec 2022to 9 Jan 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- Adult males and females ≥18 years of age
- Have 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
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- 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
- Patients must be seeking treatment for cNF
- 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.
- cNF must be located on the trunk, arms or legs of the patient
- Able and willing to comply with all visit, treatment and evaluation schedules and requirements
- Able to understand and provide written informed consent
Who cannot join
- Patients who are undergoing other treatment modalities or investigational agents for their cNF lesions
- Individuals who cannot give informed consent or adhere to study schedule
- Actively tanning during the course of the study
- 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;
- Known allergy to injectable anesthetics, polidocanol or deoxycholic acid
- Those with acute thromboembolic diseases
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- Those with bleeding abnormalities or those who are currently being treated with antiplatelet or anticoagulant therapy
- Those with dysphagia
- Women who are pregnant
- 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.
United States1 site
Wellman Center for Photomedicine
Boston, Massachusetts, 02114
About this study
Official title: Dosing and Tolerability of Deoxycholic Acid Versus Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas
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
- Being tested
- Kybella, Asclera
- Medical subject headings
- Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KybellaDrug
Injection into the cutaneous Neurofibromas lesion.
AscleraDrug
Injection into the cutaneous Neurofibromas lesion.
| Study group | Role | What they receive |
|---|---|---|
| Kybella Injection | Active comparator | Not described Receives: Drug: Kybella |
| Asclera Injection | Active comparator | Not described Receives: Drug: Asclera |
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]
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.
Measured: 3 months after treatment
Secondary outcomes (3)
Treatment specific patient reported outcomes (PRO)
NRS11, modality specific satisfaction assessment
Measured: 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
Clinician reported outcomes (ClinRO)
Clinician assessment of cNF via questionnaire. Physician rates degree of change of treated and control cNFs on a scale from -3 (no change) to 3 (very large improvement).
Measured: Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment
Modified SkinDex for cNF
Health-related quality of life measure. Asks how much participants have been bothered by cNFs over the past week from 0 (never bothered) to 5 (always bothered).
Measured: Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2023
- Study start6 Dec 2022
- Primary completion9 Jan 2025
- Study completion9 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis 1Studies 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
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
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
- NCT06120036
- Sponsor's protocol ID
- 2022P001946
- Status last verified
- Feb 2025
- First submitted
- 1 Nov 2023
- Last updated
- 21 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dosing and Tolerability of Deoxycholic Acid Versus Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas
- ClinicalTrials.gov ID
- NCT06120036
- Acronym / sponsor ID
- 2022P001946
- Conditions
- Neurofibromatosis 1
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- Johns Hopkins University
- Study start
- 6 Dec 2022 (actual)
- Primary completion
- 9 Jan 2025 (actual)
- Study completion
- 9 Jan 2025 (actual)
- First posted
- 7 Nov 2023 (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.
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.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Kybella Injection | Active comparator | Drug: Kybella |
| Asclera Injection | Active comparator | Drug: Asclera |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Adult males and females ≥18 years of age
- Have a diagnosis of NF1 based on germline genetic testing or by meeting ≥ 2 the following criteria:
- Patients must be seeking treatment for cNF
- 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.
- cNF must be located on the trunk, arms or legs of the patient
Key exclusion criteria (10 listed)
- Patients who are undergoing other treatment modalities or investigational agents for their cNF lesions
- Individuals who cannot give informed consent or adhere to study schedule
- Actively tanning during the course of the study
- 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;
- Known allergy to injectable anesthetics, polidocanol or deoxycholic acid
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 |
|---|---|---|
| KybellaInjection into the cutaneous Neurofibromas lesion. | Drug | Kybella Injection |
| AscleraInjection into the cutaneous Neurofibromas lesion. | Drug | Asclera Injection |
Primary outcome
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: 3 months after treatment
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 secondary outcomes
Treatment specific patient reported outcomes (PRO)
Time frame: 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
Clinician reported outcomes (ClinRO)
Time frame: Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment
Modified SkinDex for cNF
Time frame: Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2025.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
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 and Asclera in people with 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 14 inclusion and 10 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: finished, as of the sponsor's last update on 21 Feb 2025. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 21 Feb 2025.