Trial · NCT01682811
Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)
NCT01682811, Phase 1, Completed, Harry T Whelan, MD; results first posted 2021-06-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1). Trials, record NCT01682811. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01682811
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)
- NCT number
- NCT01682811
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 20 participants, actual
- Lead sponsor
- Harry T Whelan, MD
- Conditions
- Neurofibromatoses
- Interventions
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, Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 2, Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 3
- First posted
- 2012-09-11
- Last updated
- 2021-06-18
- Results first posted
- 2021-06-18
- Limitations and caveats
No participants in part 2 completed 1 year of follow-up visits, and are reported as lost to follow-up. This necessarily limits all outcome measurements for part 2 other than the maximum tolerable dose measurement.
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part 1: Photosensitizer Uptake and Conversion to Protoporphyrin IX | 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. |
| Primary | Part 2: Maximum Tolerated Dose (MTD) of 633 nm Red Light | 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. |
| Secondary | Part 1: Optimal Occlusion Time | 24 hours | 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 |
| Secondary | Part 2: Efficacy - Lesion Area Growth Rate | 12 weeks | Average lesion growth rates observed in ALA-treated lesions compared to vehicle-treated lesions within the same subjects. |
| Secondary | Part 2: Cosmetic Improvement | 1 year | Potential cosmetic improvement using subject satisfaction scale. |
| Secondary | Part 2: Pain Reduction | 1 year | Potential pain reduction, as measured by standard visual analog 1-10 scale. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part 1: Photosensitizer Uptake and Conversion to Protoporphyrin IX | 24 hours | The first participant contributed three lesions to each of the first three groups. The second participant likewise for the second three groups. The next two participants contributed three lesions each to each of the next two groups. The next two participants contributed likewise for the last two groups. PpIX positive areas were detected only in the last group. | Densitometry units/micrometer squared | MEAN |
| PRIMARY | Part 2: Maximum Tolerated Dose (MTD) of 633 nm Red Light | 48 hours | Of the 14 enrolled in Part 2, 5 did not receive red light treatment per protocol. No subjects were enrolled in cohort 3 (200 J/cm\^2) due to marked pain in cohort 2. In the opinion of the clinical dermatologist, higher dose levels would likely not be tolerable, and it would be unethical to escalate the light dose further. It was decided that the MTD would be set at 100 J/cm\^2, and the highest dose level foregone. | joules/cm squared | NUMBER |
| SECONDARY | Part 1: Optimal Occlusion Time | 24 hours | The first participant contributed three lesions to each of the first three groups. The second participant contributed three lesions to each of the second three groups. Only the six groups in the first two arms are relevant to this outcome measure. | hours | NUMBER |
| SECONDARY | Part 2: Efficacy - Lesion Area Growth Rate | 12 weeks | Only 5 participants from Part 2 dose level 1 of the study had lesion sizes monitored during follow-up visits. | millimeter squared/day post-treatment | MEAN |
| SECONDARY | Part 2: Cosmetic Improvement | 1 year | No cosmetic improvement data was collected. | ||
| SECONDARY | Part 2: Pain Reduction | 1 year | No pain reduction data was collected. | ||
| POST_HOC | TUNEL Assay. | 48 hours | Only 5 participants from Part 2 dose level 1 of the study had biopsies performed and lesions analyzed. | apoptotic cells/visual field | MEAN |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Part 1: Photosensitizer Uptake and Conversion to Protoporphyrin IX | Part 1 Levulan Painted Twice Treated Lesions With Microneedling | 304 | Standard Deviation 94 | Densitometry units/micrometer squared | |
| Part 2: Maximum Tolerated Dose (MTD) of 633 nm Red Light | All Participants | 100 | joules/cm squared | ||
| Part 1: Optimal Occlusion Time | Part 1 Levulan Injection Control Lesions | NA | hours | ||
| Part 1: Optimal Occlusion Time | Part 1 Levulan Injection Treated Lesions 3 Hours | NA | hours | ||
| Part 1: Optimal Occlusion Time | Part 1 Levulan Injection Treated Lesions 24 Hours | NA | hours | ||
| Part 1: Optimal Occlusion Time | Part 1 Levulan Paint Control Lesions | NA | hours | ||
| Part 1: Optimal Occlusion Time | Part 1 Levulan Paint Treated Lesions 3 Hours | NA | hours | ||
| Part 1: Optimal Occlusion Time | Part 1 Levulan Paint Treated Lesions 24 Hours | NA | hours | ||
| Part 2: Efficacy - Lesion Area Growth Rate | Part 2 Dose Level 1 50 J/cm^2 Control | 0.212 | Standard Deviation 0.364 | millimeter squared/day post-treatment | |
| Part 2: Efficacy - Lesion Area Growth Rate | Part 2 Dose Level 1 50 J/cm^2 Treated | 0.081 | Standard Deviation 0.176 | millimeter squared/day post-treatment |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow259
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part 1 Levulan Injection | STARTED | Part 1 Levulan Injection | 1 | 9 |
| Part 1 Levulan Injection | STARTED | Part 1 Levulan Painting | 0 | 0 |
| Part 1 Levulan Injection | STARTED | Experimental: Part 1 Levulan Painted Twice | 0 | 0 |
| Part 1 Levulan Injection | STARTED | Part 1 Levulan Painted Twice With Microneedling | 0 | 0 |
| Part 1 Levulan Injection | STARTED | Part 2 Dose Level 1 50 J/cm^2 | 0 | 0 |
| Part 1 Levulan Injection | STARTED | Part 2 Dose Level 2 100 J/cm^2 | 0 | 0 |
| Part 1 Levulan Injection | STARTED | Part 2 Dose Level 3 200 J/cm^2 | 0 | 0 |
| Part 1 Levulan Injection | Control | Part 1 Levulan Injection | 1 | 3 |
| Part 1 Levulan Injection | Control | Part 1 Levulan Painting | 0 | 0 |
| Part 1 Levulan Injection | Control | Experimental: Part 1 Levulan Painted Twice | 0 | 0 |
1 to 10 of 259Source ClinicalTrials.gov
See all 259 participant flowAdverse events3
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Skin burning with PDT - resolved | Skin and subcutaneous tissue disorders | Part 1 Levulan (5-aminolevulinic Acid) Uptake. | 0 | 6 | 0 |
| other | Skin burning with PDT - resolved | Skin and subcutaneous tissue disorders | Experimental: Part 2 Dose Level 1 50 J/cm^2 | 1 | 11 | 1 |
| other | Skin burning with PDT - resolved | Skin and subcutaneous tissue disorders | Part 2 Dose Level 2 100 J/cm^2 | 0 | 3 | 0 |
Source ClinicalTrials.gov
Adverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part 1 Levulan (5-aminolevulinic Acid) Uptake. | 0 | 6 |
| Total, all-cause mortality | Experimental: Part 2 Dose Level 1 50 J/cm^2 | 0 | 11 |
| Total, all-cause mortality | Part 2 Dose Level 2 100 J/cm^2 | 0 | 3 |
| Total, other adverse events | Part 1 Levulan (5-aminolevulinic Acid) Uptake. | 0 | 6 |
| Total, other adverse events | Experimental: Part 2 Dose Level 1 50 J/cm^2 | 1 | 11 |
| Total, other adverse events | Part 2 Dose Level 2 100 J/cm^2 | 0 | 3 |
| Total, serious adverse events | Part 1 Levulan (5-aminolevulinic Acid) Uptake. | 0 | 6 |
| Total, serious adverse events | Experimental: Part 2 Dose Level 1 50 J/cm^2 | 0 | 11 |
| Total, serious adverse events | Part 2 Dose Level 2 100 J/cm^2 | 0 | 3 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 33470897 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01682811
GET https://conductscience.org/api/trials/v1/NCT01682811/flow?page=2