NEUROFIBROMATOSIS 1Phase 2NCT02728388
Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II
A Phase 2 study testing aminolevulinic acid in people with NEUROFIBROMATOSIS 1, run by Donald Basel at 1 site in United States.
Trial summary
The investigators wish to determine the time to disease progression for benign neurofibromas treated with Levulan Kerastick topical photosensitizer and red light photodynamic therapy (PDT) in patients with neurofibromatosis type 1 (NF1). Read more about this study
- Who is it for?
- Ages 14 to 30 years
- Conditions the study is for
- NEUROFIBROMATOSIS 1
- What is being tested?
- aminolevulinic acid
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Sep 2016to 28 Mar 2026
- Phase
- Phase 2
- 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 14 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patient is 14 years or older.
- Diagnosed NF1, determined by American Academy of Neurology Guidelines (see Diagnosis section).
- Tumor Location: cutaneous, trunk, or limbs only.
- Tumor Type: superficial dermal neurofibromas ≤4mm deep.
- Patient has provided written informed consent.
- Patient is willing to and can comply with study follow-up requirements.
- Absence of any other malignancy.
Who cannot join
- Life expectancy less than 3 years.
- Pregnancy.
- Cutaneous photosensitivity to the wavelengths used to active PDT.
- A diagnosis of porphyria.
- Allergy to aminolevulinic acid or any of the topical solution vehicle components.
- Previous chemotherapy within 6 weeks of proposed PDT.
- Other concurrent tumor therapy.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Topical Photodynamic Therapy (PDT) With Levulan® Kerastick® for Benign Dermal Neurofibromas Phase II
The investigators wish to determine the time to disease progression for benign neurofibromas treated with Levulan Kerastick topical photosensitizer and red light photodynamic therapy (PDT) in patients with neurofibromatosis type 1 (NF1).
The investigators also wish to measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates.
Read the detailed description
Neurofibromas contain a large quantity of fibrous matter, and it is not anticipated that significant reductions in tumor size can be achieved in large, long established, tumors. Cutaneous neurofibromas, which do not usually become apparent until puberty, continue to increase in size and number throughout adulthood. The psychosocial burden of these disfiguring tumors is significant, and the targeted age group (14-30) is at a life period associated with an acceleration in tumor growth. It is for these reasons that the investigators hope to affect the growth rate of less established tumors, in order to prevent or lessen this burden as the patient progresses further into adulthood.
Therefore, the investigators wish to determine the time to disease progression (defined as 50% growth in size over baseline) for benign neurofibromas treated with PDT in patients with neurofibromatosis type 1 (NF1) in subjects aged 14-30.
The treatment will consist of choosing several neurofibromas of similar size, and applying a topical drug called Levulan, or just the topical application alone (a placebo). Within 24 hours of drug treatment, the neurofibromas will be illuminated with red light (both Levulan and placebo). The Levulan is what is known as a photosensitizer, and will be activated by the red light to potentially kill some of the tumor cells. Approximately every 6 months after, for three years, the tumors will be measured by digital photography and ultrasound to see if they are growing more slowly than the ones with the placebo application alone.
- Conditions
- NEUROFIBROMATOSIS 1
- Being tested
- aminolevulinic acid
- Keywords
- NF1, Neurofibroma, Neurofibromatosis Type 1
- Medical subject headings
- Neurofibromatosis 1, Neurofibroma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
aminolevulinic acidDrug
Also called Photodynamic therapy
Drug: Levulan Kerastick
Drug: Topical placebo
Device: Omnilux Revive
Procedure: Photodynamic therapy
Other: Tumor growth rate measurements
| Study group | Role | What they receive |
|---|---|---|
| PDT Treatment | Experimental | Each subject will have either placebo or Levulan Kerastick topical application applied to matched sets of neurofibromas. Each subject will have both sets, in order to serve as his/her own control subject.
16 to 24 hours post study drug treatment, both sets of neurofibromas, Levulan and placebo treated, will be irradiated with red light (630 nm) from an Omnilux Revive light device at 100 mW/cm2 for 1000 seconds (16.7 minutes). Receives: Drug: aminolevulinic acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to disease progression
The time it takes for 50% growth in tumor size over baseline measurements.
Measured: 3 years
Secondary outcomes (1)
Tumor growth rate
Measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2016 (estimated)
- Study start16 Sep 2016
- Primary completion31 Dec 2025
- Study completion28 Mar 2026
Why it stopped: data analysis unequivocal - requires study methodology re-evaluation
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.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Donald Basel
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Donald Basel, Medical College of Wisconsin
- Investigators
- Donald G Basel, MD, Study director, Medical College of Wisconsin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02728388
- Sponsor's protocol ID
- PRO00026795
- Status last verified
- Jul 2026
- First submitted
- 25 Mar 2016
- Last updated
- 10 Jul 2026
- 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 10 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Topical Photodynamic Therapy (PDT) With Levulan® Kerastick® for Benign Dermal Neurofibromas Phase II
- ClinicalTrials.gov ID
- NCT02728388
- Acronym / sponsor ID
- PRO00026795
- Conditions
- NEUROFIBROMATOSIS 1
- Keywords
- NF1, Neurofibroma, Neurofibromatosis Type 1
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: data analysis unequivocal - requires study methodology re-evaluation
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Donald BaselUniversity, hospital or other
- Study start
- 16 Sep 2016 (actual)
- Primary completion
- 31 Dec 2025 (actual)
- Study completion
- 28 Mar 2026 (actual)
- First posted
- 5 Apr 2016 (estimated)
- 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 investigators wish to determine the time to disease progression for benign neurofibromas treated with Levulan Kerastick topical photosensitizer and red light photodynamic therapy (PDT) in patients with neurofibromatosis type 1 (NF1).
The investigators also wish to measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates.
Detailed description
Neurofibromas contain a large quantity of fibrous matter, and it is not anticipated that significant reductions in tumor size can be achieved in large, long established, tumors. Cutaneous neurofibromas, which do not usually become apparent until puberty, continue to increase in size and number throughout adulthood. The psychosocial burden of these disfiguring tumors is significant, and the targeted age group (14-30) is at a life period associated with an acceleration in tumor growth. It is for these reasons that the investigators hope to affect the growth rate of less established tumors, in order to prevent or lessen this burden as the patient progresses further into adulthood.
Therefore, the investigators wish to determine the time to disease progression (defined as 50% growth in size over baseline) for benign neurofibromas treated with PDT in patients with neurofibromatosis type 1 (NF1) in subjects aged 14-30.
The treatment will consist of choosing several neurofibromas of similar size, and applying a topical drug called Levulan, or just the topical application alone (a placebo). Within 24 hours of drug treatment, the neurofibromas will be illuminated with red light (both Levulan and placebo). The Levulan is what is known as a photosensitizer, and will be activated by the red light to potentially kill some of the tumor cells. Approximately every 6 months after, for three years, the tumors will be measured by digital photography and ultrasound to see if they are growing more slowly than the ones with the placebo application alone.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PDT Treatment | Experimental | Drug: aminolevulinic acidEach subject will have either placebo or Levulan Kerastick topical application applied to matched sets of neurofibromas. Each subject will have both sets, in order to serve as his/her own control subject. 16 to 24 hours post study drug treatment, both sets of neurofibromas, Levulan and placebo treated, will be irradiated with red light (630 nm) from an Omnilux Revive light device at 100 mW/cm2 for 1000 seconds (16.7 minutes). |
Who may qualify
- Ages
- 14 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Patient is 14 years or older.
- Diagnosed NF1, determined by American Academy of Neurology Guidelines (see Diagnosis section).
- Tumor Location: cutaneous, trunk, or limbs only.
- Tumor Type: superficial dermal neurofibromas ≤4mm deep.
- Patient has provided written informed consent.
Key exclusion criteria (7 listed)
- Life expectancy less than 3 years.
- Pregnancy.
- Cutaneous photosensitivity to the wavelengths used to active PDT.
- A diagnosis of porphyria.
- Allergy to aminolevulinic acid or any of the topical solution vehicle components.
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 |
|---|---|---|
| aminolevulinic acidAlso: Photodynamic therapyDrug: Levulan Kerastick Drug: Topical placebo Device: Omnilux Revive Procedure: Photodynamic therapy Other: Tumor growth rate measurements | Drug | PDT Treatment |
Primary outcome
Time to disease progression
Time frame: 3 years
The time it takes for 50% growth in tumor size over baseline measurements.
1 secondary outcome
Tumor growth rate
Time frame: 3 years
Results and safety
The study was stopped early (reason given: data analysis unequivocal - requires study methodology re-evaluation). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2026.
- 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.
Donald G Basel, MD study director
Medical College of Wisconsin
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing aminolevulinic acid in people with NEUROFIBROMATOSIS 1, run by Donald Basel 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 7 inclusion and 7 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: stopped early, as of the sponsor's last update on 10 Jul 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was stopped early (reason given: data analysis unequivocal - requires study methodology re-evaluation). Results are not available in the source record.
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?
Time to disease progression (time frame: 3 years). 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 10 Jul 2026.