A Phase 2 interventional study of 5-Aminolevulinic acid Hydrochloride in Neoplasm and Skull Base Neoplasm, sponsored by Icahn School of Medicine at Mount Sinai. Terminated at 3 sites in United States. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2025-07-28.
Sponsored by Icahn School of Medicine at Mount Sinai · Phase 2, Interventional, and Diagnostic
This study allows head and neck cancer surgeons to specifically visualize cancerous cells apart from normal healthy tissue. 5-aminolevulinic acid (5-ALA) is a safe and effective FDA-approved agent successfully used by neurosurgeons for FGS of different brain tumors is given to the patients preoperatively. Using specific wavelengths of light as well as specialized magnified lenses the surgeons use this technique to assist in tumor resection.
Treatment for many head and neck cancer involves surgical resection, or removal, of the tumor. A potential outcome of surgery is subtotal resection, in which some cancerous material fails to be removed resulting in tumor recurrence and lower overall survival. Achieving total resection without interfering with normal tissue is a challenge due the critical anatomical structures of the head and neck. Fluorescent-guided surgery (FGS) is a new method of intraoperative imaging that can be used to clearly visualize cancerous tissue aprt from normal tissue during surgery in real-time. Before surgery, an agent that preferentially enters tumor cells is given to the patient. Tumor cells then glow brightly when exposed to specific wavelengths of light during surgery, allowing the surgeon to easily distinguish between healthy and cancerous tissue. 5-aminolevulinic acid (5-ALA) is a safe and effective FDA approved agent successfully used by neurosurgeons for FGS of different brain tumors which has not been assessed for use in head and neck cancers. The aim of the study is to determine if 5-ALA can be effectively used in FGS for different types of head and neck cancers, and if it will improve survival, tumor regrowth, and other outcomes for patients. 23 patients will be enrolled from the Mount Sinai Hospital, New York Eye and Ear Infirmary and Mount Sinai Union Square Faculty Practices over a 1 year period and will be completed by December 2022.
9,365 studies on the registry are indexed under Neoplasms; 2,489 are open to participants now.
This study's enrollment of 7 is below the median of 50 across 7,253 interventional studies indexed under Neoplasms.
Browse Neoplasms studies →Icahn School of Medicine at Mount Sinai is the lead sponsor of 764 studies on the registry; 181 are open to participants now.
Of its 121 completed or terminated interventional studies of FDA-regulated products, 82 (68%) have results posted.
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Subjects must have normal organ and marrow function as defined below:
Exclusion Criteria:
Gleolan® is available in colorless glass vials containing 1.5 g 5-aminolevulinic acid hydrochloride (Gleolan®) Gleolan® is administered orally to patients prior to tumor removal by surgery (20 mg/kg BW).
Drug: 5-Aminolevulinic acid Hydrochloride
The closure consists of colorless glass vial (Type II, Ph Eur) and bromobutyl-rubber stopper ø 20 mm (Type I, Ph Eur) and flip cap. The powder for oral solution is intended for single (partial) use. To prepare the ready-to-use solution the content of one vial is dissolved in 50 ml of water or apple juice. The concentration of the reconstituted solution is 3%. Gleolan® is administered orally to patients prior to tumor removal by surgery (20 mg/kg BW). The reconstituted solution may be prepared up to 24 hours prior to use if protected from light. It should be given 3 hours (range 3-5 hours) prior to planned induction of anesthesia for surgery, although its effect is present for up to 8-12 hours. This step is critical for an optimal visualization of tumor tissue due to pharmacokinetic properties of Gleolan®.
Also known as: Gleolan®
Number of Participants With Positive Test Result of PPIX Tissue Fluorescence
The feasibility of using oral Gleolan® as an adjunct diagnostic imaging tool for malignant tumor tissue fluorescence will be primarily assessed by computing sensitivity of intraoperative Gleolan® induced PPIX tissue fluorescence. PPIX tissue fluorescence will be defined categorically as "no" (score 0), "low" (score 1), "medium" (score 2), and "high" (score 3) by operative surgeon, and images will be recorded. For purposes of computing measures of diagnostic performance a score of 0 will be considered a negative test result and a score of 1, 2 or 3 will be considered a positive test result.
Time frame: Through completion of surgery, up to 24 hours
Enrollment was opened on 9/14/2021 and study was actively recruiting through Jan 14, 2025. The date of the last study visit was Dec 25, 2022. On Jan 14, 2025, the sponsor contacted the study team regarding closing the study. The study was officially closed on Feb 25, 2025.
| Milestone | 5-aminolevulinic Acid Hydrochloride (Gleolan®) |
|---|---|
| Started | 7 |
| Completed | 7 |
| Not completed | 0 |
The feasibility of using oral Gleolan® as an adjunct diagnostic imaging tool for malignant tumor tissue fluorescence will be primarily assessed by computing sensitivity of intraoperative Gleolan® induced PPIX tissue fluorescence. PPIX tissue fluorescence will be defined categorically as "no" (score 0), "low" (score 1), "medium" (score 2), and "high" (score 3) by operative surgeon, and images will be recorded. For purposes of computing measures of diagnostic performance a score of 0 will be considered a negative test result and a score of 1, 2 or 3 will be considered a positive test result.
| Participants | 5-aminolevulinic Acid Hydrochloride (Gleolan®) |
|---|---|
| 0 | 1 |
| 1 | 0 |
| 2 | 0 |
| 3 | 6 |
Collected over 24 hours. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| 5-aminolevulinic Acid Hydrochloride (Gleolan®) | 0/7 (0%) | 0/7 (0%) | 2/7 (28.6%) |
| Event | 5-aminolevulinic Acid Hydrochloride (Gleolan®) |
|---|---|
| Mild photosensitivity reaction (CTCAE grade I)Skin and subcutaneous tissue disorders | 2/7 |
| NauseaGastrointestinal disorders | 1/7 |
| Age, Continuous(years) | 5-aminolevulinic Acid Hydrochloride (Gleolan®) |
|---|---|
| Median | 60 (33 to 82) |
| Sex: Female, Male(Participants) | 5-aminolevulinic Acid Hydrochloride (Gleolan®) |
|---|---|
| Female | 1 |
| Male | 6 |
| Ethnicity (NIH/OMB)(Participants) | 5-aminolevulinic Acid Hydrochloride (Gleolan®) |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 4 |
| Unknown or Not Reported | 2 |
| Race (NIH/OMB)(Participants) | 5-aminolevulinic Acid Hydrochloride (Gleolan®) |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 5 |
| More than one race | 1 |
| Unknown or Not Reported | 1 |
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Plan to share: No — Data will be de-identified and aggregated in all reporting
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Icahn School of Medicine at Mount Sinai