The Role of 5-Aminolevulinic Acid Fluorescence-Guided Surgery in Head and Neck Cancers: a Pilot Trial

NCT05101798 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: STUDY-20-00713
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

Neoplasm; Skull Base Neoplasm

Keywords

head and neck tumor; skull base tumor; neoplasm

Outcome Measures

Primary Outcomes (1)

• 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.

  Through completion of surgery, up to 24 hours

Study Arms (1)

5-aminolevulinic acid hydrochloride (Gleolan®)

EXPERIMENTAL

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

Interventions

5-Aminolevulinic acid Hydrochloride DRUG

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®

5-aminolevulinic acid hydrochloride (Gleolan®)

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects included in this trial must have had documentation of a new or recurrent head and neck or skull base tumor for which surgical resection is indicated and has been planned. These patients will include those with newly diagnosed or recurrent malignancies.
• Age 18-80
• Karnofsky score \>60%
• Subjects must have normal organ and marrow function as defined below:
• leukocytes ≥ 3,000/mcL
• absolute neutrophil count ≥ 1,500/mcL
• platelets ≥ 100,000/mcl
• total bilirubin within normal institutional limits
• AST(SGOT)/ALT(SPGT) ≤ 2.5 X institutional upper limit of normal
• creatinine within normal institutional limits OR Creatinine clearance \>60 mL/min/1.73m2 for patients with creatinine levels above institutional normal as defined per institution.

You may not qualify if:

• Ability to understand and the willingness to sign a written informed consent.
• all upper aerodigestive head and neck cancers
• Patients with non-resectable tumors or not deemed surgical candidates
• History of allergic reactions attributed to compounds of similar chemical/biologic composition to ALA.
• Personal or family history of porphyria
• Uncontrolled concurrent illness including but not limited to: ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness//social situations that would limit compliance with study requirements
• Women who are pregnant or become pregnant will be excluded from the trial as it is unknown if ALA is teratogenic or has abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with 5-ALA breastfeeding should be discontinued if the mother is treated with 5-ALA.
• Prior history of GI perforation, diverticulitis, and or/peptic ulcer disease

Sponsors & Collaborators

• Icahn School of Medicine at Mount Sinai: lead
• NX Development Corp: collaborator

Study Sites (3)

Mount Sinai Union Square

New York, New York, 10003, United States

Location

New York Eye and Ear Infirmary of Mount Sinai

New York, New York, 10003, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

MeSH Terms

Conditions

Neoplasms; Skull Base Neoplasms; Head and Neck Neoplasms

Interventions

Aminolevulinic Acid

Condition Hierarchy (Ancestors)

Skull Neoplasms; Bone Neoplasms; Neoplasms by Site; Bone Diseases; Musculoskeletal Diseases

Intervention Hierarchy (Ancestors)

Levulinic Acids; Keto Acids; Carboxylic Acids; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Limitations and Caveats

The renewal arm of the study (2022-2025) depended on materials from NX Development Corporation (Aladuck endoscope and Gleolan (5-ALA) drug). Thus, when NX Development Corporation decided to close the study, the researchers were unable to continue study activities.

Results Point of Contact

• Title: Orli Weiss
• Organization: Icahn School of Medicine at Mount Sinai

orli.weiss@icahn.ismms.edu

9086700847

Study Officials

• Alfred-Marc Iloreta, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: October 28, 2021
• First Posted: November 1, 2021
• Study Start: September 14, 2021
• Primary Completion: December 25, 2022
• Study Completion: December 25, 2022
• Last Updated: July 28, 2025
• Results First Posted: July 28, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)