NeurofibromatosesPhase 1NCT01682811
Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)
A Phase 1 study testing Part 1 Levulan injection, Part 1 Levulan surface application and Part 1 Levulan surface application twice in people with Neurofibromatoses, run by Harry T Whelan, MD at 1 site in United States.
Trial summary
GENERAL OBJECTIVE The general objective is to assess the safety and efficacy of photodynamic therapy (PDT) in the treatment of neurofibromatosis 1 (NF1) tumors in the skin. SPECIFIC OBJECTIVE This is a light dose escalation pilot study to determine the safety and efficacy of PDT using 5-aminolevulinic acid (ALA) and… Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Neurofibromatoses
- What is being tested?
- Part 1 Levulan injection, Part 1 Levulan surface application and Part 1 Levulan surface application twice
- 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
- 12 Mar 2012to 7 Jul 2016
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 90 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects with NF1 will be selected for photodynamic therapy on the following criteria.
- Age: 18 years or older.
- NF1 will be diagnosed by American Academy of Neurology guidelines.
- Location of tumor: cutaneous, trunk or limbs only.
- Tumor type: superficial dermal neurofibromas, less than or equal to 4 mm deep.
- Growth confirmation: direct measurement for the dermal neurofibromas, ruler and photo-volumetric method.
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- Informed consent of subject.
- Absence of any other malignancy.
- Only failures to meet criteria 1-6 due to the primary disease will be disqualifying
Who cannot join
- Subjects will be excluded from participation in the study on the basis of the following:
- Life expectancy less than 1 year.
- Pregnancy.
- Inability to consent.
- Cutaneous photosensitivity to the wavelengths used to activate PDT.
- A diagnosis of porphyria.
Show all 9Show fewer
- 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.
United States1 site
The Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
About this study
Official title: Photodynamic Therapy for Benign Dermal Neurofibromas Using Levulan Kerastick For Topical Solution, Plus Illumination With Red Light
GENERAL OBJECTIVE The general objective is to assess the safety and efficacy of photodynamic therapy (PDT) in the treatment of neurofibromatosis 1 (NF1) tumors in the skin.
SPECIFIC OBJECTIVE This is a light dose escalation pilot study to determine the safety and efficacy of PDT using 5-aminolevulinic acid (ALA) and 633 nm light in the treatment of benign dermal neurofibromas.
Specifically, the primary goal of the current study is to determine the maximum tolerable light doses that can be administered to subjects undergoing topical photoillumination photodynamic therapy with standard application of Levulan Kerastick (ALA) for Topical Solution.
Read the detailed description
STUDY DESIGN This protocol is a Phase I light dose escalation pilot study to determine the safety and, secondarily, the efficacy of PDT using Levulan and 633 nm light in the treatment of benign dermal neurofibromas. This protocol represents the first two parts of a planned three part study including both pediatric and adult subjects. Part 1 will consist of studying the penetration and uptake of the PS in neurofibromas that are scheduled for excision. These tumors will be excised for therapeutic reasons unrelated to this study, and so this study will place no further burden on the subject other than a 3-24 hr incubation of the Levulan on the tumor prior to excision. The primary hypothesis to be tested is whether Levulan will accumulate, and be converted to PpIX, by the tumor tissue more than by the surrounding normal tissue. Secondary hypotheses are that tumors incubated with Levulan will show greater fluorescence than untreated tumors and tumors incubated with vehicle only (placebo application).
As the Institutional Review Boards involved generally desire pilot data on adult populations first, we will with then proceed with the adult clinical trial portion of this protocol as part 2. Part 2 will use the optimum incubation time, if one has been identified in part 1, and add a dose escalation study of the amount of red light used to activate the Levulan. Part 3, with pediatric subjects, will commence at a future date, pending review of the initial adult study results.
- Conditions
- Neurofibromatoses
- Being tested
- Part 1 Levulan injection, Part 1 Levulan surface application, Part 1 Levulan surface application twice, Part 1 Levulan surface application twice with microneedling, Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 1
- Medical subject headings
- Neurofibromatoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Part 1 Levulan injectionDrug
Control or treatment lesions will be injected with Levulan vehicle only or active drug and incubated under occlusion for 3 or 24 hrs. A minimum of three lesions per group on the same subject will be treated. Lesions will then be excised in the normal manner, sectioned vertically, and checked for Protoporphyrin IX using fluorescence microscopy.
Part 1 Levulan surface applicationDrug
Control or treatment lesions will be painted with Levulan vehicle only or active drug, allowed to dry, and incubated under occlusion for 3 or 24 hrs. A minimum of three lesions per group on the same subject will be treated. Lesions will then be excised in the normal manner, sectioned vertically, and checked for Protoporphyrin IX using fluorescence microscopy.
Part 1 Levulan surface application twiceDrug
Control or treatment lesions will be painted twice with Levulan vehicle only or active drug, allowed to dry between and after applications, and incubated under occlusion for 3 hrs. A minimum of three tumors per group on the same subject will be treated. Lesions will then be excised in the normal manner, sectioned vertically, and checked for Protoporphyrin IX using fluorescence microscopy.
Part 1 Levulan surface application twice with microneedlingDrug
Control or treatment lesions will be prepared with microneedling, painted twice with Levulan vehicle only or active drug, allowed to dry between and after applications, and incubated under occlusion for 24 hrs. A minimum of three tumors per group on the same subject will be treated. Lesions will then be excised in the normal manner, sectioned vertically, and checked for Protoporphyrin IX using fluorescence microscopy.
Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 1Drug
2-6 adult subjects with 3-8 lesions per (Levulan or control) group per subject. Controls will consist of lesions treated with vehicle only and light illumination, and will be paired with treatment lesions by the study doctor. Control lesions will be treated on the same subject as study lesions. Levulan will be incubated for 3-24 hours under occlusion, then gently rinsed with water and patted dry. Photoactivation of lesions treated with Levulan is then accomplished with 633 nm red light illumination. 633 nm light will be applied for 8 minutes to achieve a dose of 50 J/cm^2. There will be one treatment session per subject. Treatments will include a minimum of three test lesions and an additional three control lesions.
Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 2Drug
2-6 adult subjects with 3-8 lesions per (Levulan or control) group per subject. Controls will consist of lesions treated with vehicle only and light illumination, and will be paired with treatment lesions by the study doctor. Control lesions will be treated on the same subject as study lesions. Levulan will be incubated for 3-24 hours under occlusion, then gently rinsed with water and patted dry. Photoactivation of lesions treated with Levulan is then accomplished with 633 nm red light illumination. 633 nm light will be applied for 16 minutes to achieve a dose of 100 J/cm^2. There will be one treatment session per subject. Treatments will include a minimum of three test lesions and an additional three control lesions.
Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 3Drug
2-6 adult subjects with 3-8 lesions per (Levulan or control) group per subject. Controls will consist of lesions treated with vehicle only and light illumination, and will be paired with treatment lesions by the study doctor. Control lesions will be treated on the same subject as study lesions. Levulan will be incubated for 3-24 hours under occlusion, then gently rinsed with water and patted dry. Photoactivation of lesions treated with Levulan is then accomplished with 633 nm red light illumination. 633 nm light will be applied for 32 minutes to achieve a dose of 200 J/cm^2. There will be one treatment session per subject. Treatments will include a minimum of three test lesions and an additional three control lesions.
| Study group | Role | What they receive |
|---|---|---|
| Part 1 Levulan injection | Experimental | 5-aminolevulinic acid uptake by control lesions after Levulan vehicle (placebo) injection and 3 hr incubation, and by Levulan treated lesions after 3 or 24 hr incubation. Receives: Drug: Part 1 Levulan injection |
| Part 1 Levulan painting | Experimental | 5-aminolevulinic acid uptake by control lesions after Levulan vehicle (placebo) surface application and 3 hr incubation, and by Levulan treated lesions and 3 or 24 hr incubation. Receives: Drug: Part 1 Levulan surface application |
| Part 1 Levulan painted twice | Experimental | 5-aminolevulinic acid uptake by control lesions after Levulan vehicle (placebo) surface application twice or by Levulan treated lesions twice and 24 hr incubation. Receives: Drug: Part 1 Levulan surface application twice |
| Part 1 Levulan painted twice with microneedling | Experimental | 5-aminolevulinic acid uptake by control lesions after Levulan vehicle (placebo) surface application twice or by Levulan treated lesions twice and 24 hr incubation. All lesions prepared with microneedling. Receives: Drug: Part 1 Levulan surface application twice with microneedling |
| Part 2 Dose level 1 50 J/cm^2 | Experimental | Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 1 - 50 J/cm^2 633 nm red light to Levulan vehicle (placebo) treated control lesions or Levulan treated lesions and 24 hr incubation. Follow-ups on day 2 for punch biopsies, 14-28 days for evaluation of cutaneous adverse events, and every 3 months up to one year for lesion measurements. Receives: Drug: Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 1 |
| Part 2 Dose level 2 100 J/cm^2 | Experimental | Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 2 - 100 J/cm^2 633 nm red light to Levulan vehicle (placebo) treated control lesions or Levulan treated lesions and 24 hr incubation. Follow-ups on day 2 for punch biopsies, 14-28 days for evaluation of cutaneous adverse events, and every 3 months up to one year for lesion measurements. Receives: Drug: Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 2 |
| Part 2 Dose level 3 200 J/cm^2 | Experimental | Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 3 - 200 J/cm^2 633 nm red light to Levulan vehicle (placebo) treated control lesions or Levulan treated lesions and 24 hr incubation. Follow-ups on day 2 for punch biopsies, 14-28 days for evaluation of cutaneous adverse events, and every 3 months up to one year for lesion measurements. Receives: Drug: Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 3 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1: Photosensitizer Uptake and Conversion to Protoporphyrin IX
The average fluorescence value for PpIX positive tumor areas in excised, sectioned tumors, as determined by fluorescence microscopy. PpIX signals were detected with excitation at 405 nm and emission with a 600 nm long pass filter. PpIX positive areas were determined to be those exhibiting fluorescence above background levels.
Measured: 24 hours
Part 2: Maximum Tolerated Dose (MTD) of 633 nm Red Light
MTD was determined by testing increasing doses up to 200 J/cm^2 on dose escalation cohorts 1 to 3 with 3 to 6 participants each. MTD reflects the highest dose of red light that did not cause a Dose-Limiting Toxicity (DLT) in > 33% of participants. DLT was defined as pain during irradiation requiring cessation of the light treatment, or any serious cutaneous adverse events.
Measured: 48 hours
Secondary outcomes (4)
Part 1: Optimal Occlusion Time
An optimal occlusion time may be apparent from the results of the three time points. If no optimal occlusion time is seen, any occlusion time from 3-24 hours may be chosen for part 2. This secondary outcome measure is not critical to continuing the study, but may be useful in guiding treatment protocols
Measured: 24 hours
Part 2: Efficacy - Lesion Area Growth Rate
Average lesion growth rates observed in ALA-treated lesions compared to vehicle-treated lesions within the same subjects.
Measured: 12 weeks
Part 2: Cosmetic Improvement
Potential cosmetic improvement using subject satisfaction scale.
Measured: 1 year
Part 2: Pain Reduction
Potential pain reduction, as measured by standard visual analog 1-10 scale.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2012 (estimated)
- Study start12 Mar 2012
- Primary completion23 Oct 2015
- Study completion7 Jul 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for NeurofibromatosesStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Harry T Whelan, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Harry T Whelan, MD, Medical College of Wisconsin
- Investigators
- Harry T Whelan, MD, Principal investigator, Medical College of Wisconsin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01682811
- Sponsor's protocol ID
- PRO 14555
- Status last verified
- Jun 2021
- First submitted
- 5 Sep 2012
- Last updated
- 18 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Quirk B, Olasz E, Kumar S, Basel D, Whelan H. Photodynamic Therapy for Benign Cutaneous Neurofibromas Using Aminolevulinic Acid Topical Application and 633 nm Red Light Illumination. Photobiomodul Photomed Laser Surg. 2021 Jun;39(6):411-417. doi: 10.1089/photob.2020.4957. Epub 2021 Jan 20.
PubMed 33470897
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Photodynamic Therapy for Benign Dermal Neurofibromas Using Levulan Kerastick For Topical Solution, Plus Illumination With Red Light
- ClinicalTrials.gov ID
- NCT01682811
- Acronym / sponsor ID
- PRO 14555
- Conditions
- Neurofibromatoses
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Harry T Whelan, MDUniversity, hospital or other
- Study start
- 12 Mar 2012 (actual)
- Primary completion
- 23 Oct 2015 (actual)
- Study completion
- 7 Jul 2016 (actual)
- First posted
- 11 Sep 2012 (estimated)
- Results
- Posted 18 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
GENERAL OBJECTIVE The general objective is to assess the safety and efficacy of photodynamic therapy (PDT) in the treatment of neurofibromatosis 1 (NF1) tumors in the skin.
SPECIFIC OBJECTIVE This is a light dose escalation pilot study to determine the safety and efficacy of PDT using 5-aminolevulinic acid (ALA) and 633 nm light in the treatment of benign dermal neurofibromas.
Specifically, the primary goal of the current study is to determine the maximum tolerable light doses that can be administered to subjects undergoing topical photoillumination photodynamic therapy with standard application of Levulan Kerastick (ALA) for Topical Solution.
Detailed description
STUDY DESIGN This protocol is a Phase I light dose escalation pilot study to determine the safety and, secondarily, the efficacy of PDT using Levulan and 633 nm light in the treatment of benign dermal neurofibromas. This protocol represents the first two parts of a planned three part study including both pediatric and adult subjects. Part 1 will consist of studying the penetration and uptake of the PS in neurofibromas that are scheduled for excision. These tumors will be excised for therapeutic reasons unrelated to this study, and so this study will place no further burden on the subject other than a 3-24 hr incubation of the Levulan on the tumor prior to excision. The primary hypothesis to be tested is whether Levulan will accumulate, and be converted to PpIX, by the tumor tissue more than by the surrounding normal tissue. Secondary hypotheses are that tumors incubated with Levulan will show greater fluorescence than untreated tumors and tumors incubated with vehicle only (placebo application).
As the Institutional Review Boards involved generally desire pilot data on adult populations first, we will with then proceed with the adult clinical trial portion of this protocol as part 2. Part 2 will use the optimum incubation time, if one has been identified in part 1, and add a dose escalation study of the amount of red light used to activate the Levulan. Part 3, with pediatric subjects, will commence at a future date, pending review of the initial adult study results.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 7
| Arm | Type | Receives |
|---|---|---|
| Part 1 Levulan injection | Experimental | Drug: Part 1 Levulan injection5-aminolevulinic acid uptake by control lesions after Levulan vehicle (placebo) injection and 3 hr incubation, and by Levulan treated lesions after 3 or 24 hr incubation. |
| Part 1 Levulan painting | Experimental | Drug: Part 1 Levulan surface application5-aminolevulinic acid uptake by control lesions after Levulan vehicle (placebo) surface application and 3 hr incubation, and by Levulan treated lesions and 3 or 24 hr incubation. |
| Part 1 Levulan painted twice | Experimental | Drug: Part 1 Levulan surface application twice5-aminolevulinic acid uptake by control lesions after Levulan vehicle (placebo) surface application twice or by Levulan treated lesions twice and 24 hr incubation. |
| Part 1 Levulan painted twice with microneedling | Experimental | Drug: Part 1 Levulan surface application twice with microneedling5-aminolevulinic acid uptake by control lesions after Levulan vehicle (placebo) surface application twice or by Levulan treated lesions twice and 24 hr incubation. All lesions prepared with microneedling. |
| Part 2 Dose level 1 50 J/cm^2 | Experimental | Drug: Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 1Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 1 - 50 J/cm^2 633 nm red light to Levulan vehicle (placebo) treated control lesions or Levulan treated lesions and 24 hr incubation. Follow-ups on day 2 for punch biopsies, 14-28 days for evaluation of cutaneous adverse events, and every 3 months up to one year for lesion measurements. |
| Part 2 Dose level 2 100 J/cm^2 | Experimental | Drug: Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 2Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 2 - 100 J/cm^2 633 nm red light to Levulan vehicle (placebo) treated control lesions or Levulan treated lesions and 24 hr incubation. Follow-ups on day 2 for punch biopsies, 14-28 days for evaluation of cutaneous adverse events, and every 3 months up to one year for lesion measurements. |
| Part 2 Dose level 3 200 J/cm^2 | Experimental | Drug: Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 3Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 3 - 200 J/cm^2 633 nm red light to Levulan vehicle (placebo) treated control lesions or Levulan treated lesions and 24 hr incubation. Follow-ups on day 2 for punch biopsies, 14-28 days for evaluation of cutaneous adverse events, and every 3 months up to one year for lesion measurements. |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Subjects with NF1 will be selected for photodynamic therapy on the following criteria.
- Age: 18 years or older.
- NF1 will be diagnosed by American Academy of Neurology guidelines.
- Location of tumor: cutaneous, trunk or limbs only.
- Tumor type: superficial dermal neurofibromas, less than or equal to 4 mm deep.
Key exclusion criteria (9 listed)
- Life expectancy less than 1 year.
- Pregnancy.
- Inability to consent.
- Cutaneous photosensitivity to the wavelengths used to activate PDT.
- A diagnosis of porphyria.
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 |
|---|---|---|
| Part 1 Levulan injectionControl or treatment lesions will be injected with Levulan vehicle only or active drug and incubated under occlusion for 3 or 24 hrs. A minimum of three lesions per group on the same subject will be treated. Lesions will then be excised in the normal manner, sectioned vertically, and checked for Protoporphyrin IX using fluorescence microscopy. | Drug | Part 1 Levulan injection |
| Part 1 Levulan surface applicationControl or treatment lesions will be painted with Levulan vehicle only or active drug, allowed to dry, and incubated under occlusion for 3 or 24 hrs. A minimum of three lesions per group on the same subject will be treated. Lesions will then be excised in the normal manner, sectioned vertically, and checked for Protoporphyrin IX using fluorescence microscopy. | Drug | Part 1 Levulan painting |
| Part 1 Levulan surface application twiceControl or treatment lesions will be painted twice with Levulan vehicle only or active drug, allowed to dry between and after applications, and incubated under occlusion for 3 hrs. A minimum of three tumors per group on the same subject will be treated. Lesions will then be excised in the normal manner, sectioned vertically, and checked for Protoporphyrin IX using fluorescence microscopy. | Drug | Part 1 Levulan painted twice |
| Part 1 Levulan surface application twice with microneedlingControl or treatment lesions will be prepared with microneedling, painted twice with Levulan vehicle only or active drug, allowed to dry between and after applications, and incubated under occlusion for 24 hrs. A minimum of three tumors per group on the same subject will be treated. Lesions will then be excised in the normal manner, sectioned vertically, and checked for Protoporphyrin IX using fluorescence microscopy. | Drug | Part 1 Levulan painted twice with microneedling |
| Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 12-6 adult subjects with 3-8 lesions per (Levulan or control) group per subject. Controls will consist of lesions treated with vehicle only and light illumination, and will be paired with treatment lesions by the study doctor. Control lesions will be treated on the same subject as study lesions. Levulan will be incubated for 3-24 hours under occlusion, then gently rinsed with water and patted dry. Photoactivation of lesions treated with Levulan is then accomplished with 633 nm red light illumination. 633 nm light will be applied for 8 minutes to achieve a dose of 50 J/cm^2. There will be one treatment session per subject. Treatments will include a minimum of three test lesions and an additional three control lesions. | Drug | Part 2 Dose level 1 50 J/cm^2 |
| Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 22-6 adult subjects with 3-8 lesions per (Levulan or control) group per subject. Controls will consist of lesions treated with vehicle only and light illumination, and will be paired with treatment lesions by the study doctor. Control lesions will be treated on the same subject as study lesions. Levulan will be incubated for 3-24 hours under occlusion, then gently rinsed with water and patted dry. Photoactivation of lesions treated with Levulan is then accomplished with 633 nm red light illumination. 633 nm light will be applied for 16 minutes to achieve a dose of 100 J/cm^2. There will be one treatment session per subject. Treatments will include a minimum of three test lesions and an additional three control lesions. | Drug | Part 2 Dose level 2 100 J/cm^2 |
| Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 32-6 adult subjects with 3-8 lesions per (Levulan or control) group per subject. Controls will consist of lesions treated with vehicle only and light illumination, and will be paired with treatment lesions by the study doctor. Control lesions will be treated on the same subject as study lesions. Levulan will be incubated for 3-24 hours under occlusion, then gently rinsed with water and patted dry. Photoactivation of lesions treated with Levulan is then accomplished with 633 nm red light illumination. 633 nm light will be applied for 32 minutes to achieve a dose of 200 J/cm^2. There will be one treatment session per subject. Treatments will include a minimum of three test lesions and an additional three control lesions. | Drug | Part 2 Dose level 3 200 J/cm^2 |
Primary outcome
Part 1: Photosensitizer Uptake and Conversion to Protoporphyrin IX
Time frame: 24 hours
The average fluorescence value for PpIX positive tumor areas in excised, sectioned tumors, as determined by fluorescence microscopy. PpIX signals were detected with excitation at 405 nm and emission with a 600 nm long pass filter. PpIX positive areas were determined to be those exhibiting fluorescence above background levels.
Part 2: Maximum Tolerated Dose (MTD) of 633 nm Red Light
Time frame: 48 hours
MTD was determined by testing increasing doses up to 200 J/cm^2 on dose escalation cohorts 1 to 3 with 3 to 6 participants each. MTD reflects the highest dose of red light that did not cause a Dose-Limiting Toxicity (DLT) in > 33% of participants. DLT was defined as pain during irradiation requiring cessation of the light treatment, or any serious cutaneous adverse events.
4 secondary outcomes
Part 1: Optimal Occlusion Time
Time frame: 24 hours
Part 2: Efficacy - Lesion Area Growth Rate
Time frame: 12 weeks
Part 2: Cosmetic Improvement
Time frame: 1 year
Part 2: Pain Reduction
Time frame: 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33470897Quirk B, Olasz E, Kumar S, Basel D, Whelan H. Photodynamic Therapy for Benign Cutaneous Neurofibromas Using Aminolevulinic Acid Topical Application and 633 nm Red Light Illumination. Photobiomodul Photomed Laser Surg. 2021 Jun;39(6):411-417. doi: 10.1089/photob.2020.4957. Epub 2021 Jan 20.
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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.
Harry T Whelan, MD principal investigator
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 1 study testing Part 1 Levulan injection, Part 1 Levulan surface application and Part 1 Levulan surface application twice in people with Neurofibromatoses, run by Harry T Whelan, MD 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 9 inclusion and 9 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 18 Jun 2021. 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?
Yes. Results are posted in the registry (first posted 18 Jun 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Part 1: Photosensitizer Uptake and Conversion to Protoporphyrin IX (time frame: 24 hours). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01682811, which also lists 1 linked publication.
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 18 Jun 2021.