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

Primary Purpose

Neoplasm, Skull Base Neoplasm

Status

Recruiting

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

5-Aminolevulinic acid Hydrochloride

About this trial

This is an interventional diagnostic trial for Neoplasm focused on measuring head and neck tumor, skull base tumor, neoplasm

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.
The effects of Gleolan® (5-ALA) on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. A pregnancy test will be performed for all women of childbearing ability prior to surgery (see Exclusion Criteria below). Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
Ability to understand and the willingness to sign a written informed consent.

Exclusion Criteria:

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

Sites / Locations

Mount Sinai Union SquareRecruiting
New York Eye and Ear Infirmary of Mount SinaiRecruiting
Icahn School of Medicine at Mount SinaiRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

5-aminolevulinic acid hydrochloride (Gleolan®)

Arm Description

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).

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Full Information

NCT ID

NCT05101798

First Posted

October 28, 2021

Last Updated

October 12, 2023

Sponsor

Icahn School of Medicine at Mount Sinai

Collaborators

NX Development Corp

1. Study Identification

Unique Protocol Identification Number

NCT05101798

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Recruiting

Study Start Date

September 14, 2021 (Actual)

Primary Completion Date

January 2024 (Anticipated)

Study Completion Date

January 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Icahn School of Medicine at Mount Sinai

Collaborators

NX Development Corp

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

Yes

5. Study Description

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.

Detailed Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Neoplasm, Skull Base Neoplasm

Keywords

head and neck tumor, skull base tumor, neoplasm

7. Study Design

Primary Purpose

Diagnostic

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

26 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

5-aminolevulinic acid hydrochloride (Gleolan®)

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

5-Aminolevulinic acid Hydrochloride

Other Intervention Name(s)

Gleolan®

Intervention Description

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®.

Primary Outcome Measure Information:

Title

PPIX tissue fluorescence

Description

Time Frame

Through completion of surgery, up to 24 hours

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

80 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. The effects of Gleolan® (5-ALA) on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. A pregnancy test will be performed for all women of childbearing ability prior to surgery (see Exclusion Criteria below). Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. Ability to understand and the willingness to sign a written informed consent. all upper aerodigestive head and neck cancers Exclusion Criteria: 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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Chanel Rojas

Phone

(347) 962-6630

Chanel.Rojas@mountsinai.org

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Alfred-Marc Iloreta, MD

Organizational Affiliation

Icahn School of Medicine at Mount Sinai

Official's Role

Principal Investigator

Facility Information:

Facility Name

Mount Sinai Union Square

City

New York

State/Province

New York

ZIP/Postal Code

10003

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Samantha Udondem

Phone

347-978-3973

samantha.udondem@mountsinai.org

Facility Name

New York Eye and Ear Infirmary of Mount Sinai

City

New York

State/Province

New York

ZIP/Postal Code

10003

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Samantha Udondem

Phone

347-978-3973

samantha.udondem@mountsinai.org

Facility Name

Icahn School of Medicine at Mount Sinai

City

New York

State/Province

New York

ZIP/Postal Code

10029

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Alecia Charles

Phone

212-241-7107

Alecia.Charles@mountsinai.org

12. IPD Sharing Statement

Plan to Share IPD

IPD Sharing Plan Description

Data will be de-identified and aggregated in all reporting

Learn more about this trial

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

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