5-Aminolevulinic Acid (5-ALA) to Enhance Visualization of Malignant Tumor

In support of the US marketing application for 5-ALA, this single arm trial is being
conducted to establish the efficacy and safety of Gliolan (5-ALA) in patients with newly
diagnosed or recurrent malignant gliomas. The hypothesis of the study is Gliolan (5-ALA), as
an adjunct to tumor resection, is safe and that real-time tissue fluorescence correlates with
malignant histopathology. The primary objective in this single arm study is to define the
positive predictive value (PPV) of Gliolan-induced PPIX fluorescence for malignant tumor at
the time of initial resection and first use of FGS by taking a biopsy of tissue presenting
with red fluorescence when observed during the course of resection of new or recurrent
malignant gliomas. The functionality and performance reliability of the blue light excitation
microscope platforms will be assessed.

Eligibility Criteria

Inclusion Criteria:

- Subjects included must have an MRI documenting a primary brain tumor for which
resection is indicated and has been planned. These patients will include those with
newly diagnosed or recurrent malignant gliomas. Standard criteria for diagnosis will
include a distinct ring-like pattern of contrast enhancement with thick irregular
walls on MRI for patients with a presumed newly diagnosed malignant glioma.

- Age 18-80.

- Karnofsky>60%.

- Subjects must have normal organ and marrow function as defined below:

Leukocytes >3,000/mL Platelets >100,000/mL Total bilirubin below upper limit of normal AST
(SGOT)/ALT (SGPT) <2.5 X institutional upper limit of normal Creatinine below upper limit
of normal OR Creatinine clearance >60 mL/min/1.73 m2 for patients with creatinine levels
above institutional normal.

- The effects of 5-aminolevulinic Acid (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 document.
Translation will be provided as appropriate by institution.

- Inclusion of Women and Minorities: Both men and women and members of all ethnic groups
are eligible for this trial.

Exclusion Criteria:

- Patients with radiographic tumors of, or involving, nonresectable midline, the basal
ganglia, or brain stem as assessed by MRI.

- History of allergic reactions attributed to compounds of similar chemical or biologic
composition to aminolevulinic acid (ALA). Patients should refrain from use of other
potential phototoxic substances (e.g. tetracyclines, sulfonamides, fluoroquinolones,
hypericin extracts) for 72 h.

- Personal or family history of porphyria.

- Uncontrolled intercurrent 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. . Because there is an unknown but potential risk for adverse
events in nursing infants secondary to treatment of the mother with 5-aminolevulinic
acid (5-ALA), breastfeeding should be discontinued if the mother is treated with
5-aminolevulinic acid (5-ALA).

- Women who are pregnant will be excluded from the trial as aminolevulinic acid (ALA) is
unknown to be teratogenic or have abortifacient effects Prior history of GI
perforation, diverticulitis, and/or peptic ulcer disease.

Principal Investigator

Roukoz B Chamoun, MD

Study Contact

Constantinos G Hadjipanayis, MD, PhD, constantinos.hadjipanayis@mountsinai.org, 212-844-6922

Estimated Completion Date

Friday, December 1, 2017

ClinicalTrials.gov #

NCT02632370
08/07/2017