Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II
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|ClinicalTrials.gov Identifier: NCT02728388|
Recruitment Status : Recruiting
First Posted : April 5, 2016
Last Update Posted : January 4, 2023
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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.
|Condition or disease||Intervention/treatment||Phase|
|NEUROFIBROMATOSIS 1||Drug: aminolevulinic acid||Phase 2|
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 Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Topical Photodynamic Therapy (PDT) With Levulan® Kerastick® for Benign Dermal Neurofibromas Phase II|
|Study Start Date :||August 2016|
|Estimated Primary Completion Date :||December 2025|
|Estimated Study Completion Date :||December 2025|
Experimental: PDT Treatment
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).
Drug: aminolevulinic acid
Drug: Levulan Kerastick
Drug: Topical placebo
Device: Omnilux Revive
Procedure: Photodynamic therapy
Other: Tumor growth rate measurements
Other Name: Photodynamic therapy
- Time to disease progression [ Time Frame: 3 years ]The time it takes for 50% growth in tumor size over baseline measurements.
- Tumor growth rate [ Time Frame: 3 years ]Measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates.
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|Ages Eligible for Study:||14 Years to 30 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- Life expectancy less than 3 years.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02728388
|Contact: Harry T Whelan, MDfirstname.lastname@example.org|
|Contact: Marsha Malloy, RN, BSN, MBAemail@example.com|
|United States, Wisconsin|
|The Medical College of Wisconsin||Recruiting|
|Milwaukee, Wisconsin, United States, 53226|
|Contact: Harry T Whelan, MD 414-266-7540 firstname.lastname@example.org|
|Principal Investigator:||Harry T Whelan, MD||Medical College of Wisconsin|
|Responsible Party:||Harry T Whelan, MD, Bleser Professor of Neurology, Medical College of Wisconsin|
|Other Study ID Numbers:||
|First Posted:||April 5, 2016 Key Record Dates|
|Last Update Posted:||January 4, 2023|
|Last Verified:||January 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Neurofibromatosis Type 1
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplastic Syndromes, Hereditary
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Peripheral Nervous System Neoplasms
Nervous System Neoplasms