Drug Trials Snapshot: LUMISIGHT
HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.
LIMITATIONS OF THIS SNAPSHOT:
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your healthcare provider about the benefits and risks of a drug.
Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the LUMISIGHT Prescribing Information for all the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).
Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).
LUMISIGHT (pegulicianine)
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Lumicell, Inc
Original Approval date: April 17, 2024
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
LUMISIGHT is a drug used for fluorescence imaging in adults with breast cancer to help detect cancerous tissue within the resection cavity following removal of the primary specimen during lumpectomy surgery.
How is this drug used?
LUMISIGHT is an injection given by a health care provider in the vein (intravenous) two to six hours before a surgeon images the resection cavity during lumpectomy surgery. If positive LUMISIGHT signal is seen, the surgeon may remove additional tissue.
Who participated in the clinical trials?
Evidence of effectiveness was primarily provided by a clinical trial (CL0007, NCT03686215) that enrolled 406 patients with confirmed invasive breast cancer, ductal carcinoma in situ (DCIS), or both. Patients who received chemotherapy or radiation therapy before surgery were not enrolled. This trial was conducted at 14 sites within the United States.
The safety of LUMISIGHT was evaluated in 726 patients who received a single dose of 1 mg/kg of LUMISIGHT. Among these 726 patients, 703 (97%) had breast cancer and 23 (3%) had other types of cancer. The number of patients representing efficacy findings differs from the number of patients representing safety findings due to different pools of study participants analyzed for efficacy and safety.
How were the trials designed?
CL0007 was a randomized, multicenter, intra-patient controlled clinical trial in patients with confirmed invasive breast cancer, DCIS, or both who had not yet received chemotherapy or radiation therapy. All patients received a dose of LUMISIGHT before proceeding to lumpectomy surgery. Patients randomized to the LUMISIGHT imaging group had fluorescence imaging of the lumpectomy cavity following standard of care (SOC) surgery and removal of additional tissue if positive signal was seen. The trial measured the proportion of these patients who had residual breast cancer detected and removed following SOC lumpectomy surgery and the sensitivity and specificity of LUMISIGHT for residual breast cancer.
How were the trials designed?
The efficacy and safety of LUMISIGHT for the intraoperative detection of cancerous tissue within the resection cavity following removal of the primary specimen during lumpectomy surgery in patients with breast cancer were evaluated in a randomized, multicenter, intra-patient controlled clinical trial. A total of 406 adult patients with confirmed invasive breast cancer, DCIS, or both received 1 mg/kg LUMISIGHT by intravenous injection two to six hours prior to imaging with the Lumicell Direct Visualization System (DVS) intraoperative imaging device. Among them, 357 patients underwent LUMISIGHT-guided imaging after completion of the SOC lumpectomy procedure. Patients who received neoadjuvant chemotherapy or radiotherapy prior to surgery were excluded from study. After the SOC lumpectomy was completed, the lumpectomy cavity was divided into six regions based on anatomic orientation and each region was imaged with the Lumicell DVS. When positive fluorescence signal was detected, the tissue was resected with a cavity shave procedure and the region was re-imaged. A maximum of two LUMISIGHT-guided shaves were obtained from a single region. Histopathology analysis of excised tissue served as the reference standard. The reference standard for cavity regions that did not have a LUMISIGHT-guided cavity shave or tissue from a second surgery was histopathological status of the corresponding outermost surface of the lumpectomy specimen or SOC cavity shave. Positive margins were defined as tumor on ink for invasive cancers (with or without DCIS) or within 2 mm of the linked margin for DCIS alone.
The mean age of patients was 62 years (range: 36 to 83 years). Distribution by race was 82% White, 7% Black or African American, 6% Asian, and 5% other. Distribution by ethnicity was 3% Hispanic or Latino, 94% not Hispanic or Latino, and 3% unknown or unreported. In the study group, 70% of patients had invasive ductal carcinoma (with or without DCIS), 20% had DCIS only, 10% had invasive lobular carcinoma (with or without DCIS), and <1% had both invasive ductal and invasive lobular carcinoma.
The study assessed the proportion of patients receiving LUMISIGHT who had residual breast cancer detected and removed using the Lumicell DVS after completion of SOC lumpectomy and the image-level performance of LUMISIGHT for detection of residual breast cancer in the lumpectomy cavity after completion of SOC lumpectomy.
DEMOGRAPHICS SNAPSHOT
Figure 1 summarizes how many male and female patients were enrolled in the clinical trial used to evaluate the efficacy of LUMISIGHT.
Figure 1. Baseline Demographics by Sex, mITT Population
Source: Adapted from FDA Review
Abbreviations: mITT, modified intent-to-treat
Figure 2 summarizes how many patients by race were enrolled in the clinical trial used to evaluate the efficacy of LUMISIGHT.
Figure 2. Baseline Demographics by Race , mITT Population
Source: Adapted from FDA Review
Abbreviations: mITT, modified intent-to-treat
Figure 3 summarizes how many patients by age were enrolled in the clinical trial used to evaluate the efficacy of LUMISIGHT.
Figure 3. Baseline Demographics by Age, mITT Population
Source: Adapted from FDA Review
Abbreviations: mITT, modified intent-to-treat
Figure 4 summarizes how many patients by ethnicity were enrolled in the clinical trial used to evaluate the efficacy of LUMISIGHT.
Figure 4. Baseline Demographics by Ethnicity (Efficacy Population)
Source: Adapted from FDA Review
Abbreviations: mITT, modified intent-to-treat
Who participated in the trials?
Table 1. Baseline Demographics of CL0007
| Demographic | Enrolled Population N=406 |
mITT Population N=357 |
Control Population N=35 |
| Age, years | |||
| Mean (SD) | 62.3 (9.7) | 62.4 (9.6) | 61.6 (9.9) |
| Median (min, max) | 64 (36, 83) | 64 (36, 83) | 62 (37, 82) |
| Age group, years, n (%) | |||
| <65 | 211 (52%) | 184 (52%) | 19 (54%) |
| ≥65 | 195 (48%) | 173 (48%) | 16 (46%) |
| ≥75 | 35 (9%) | 30 (8%) | 2 (6%) |
| Race, n (%) | |||
| American Indian or Alaska Native | 1 (<1%) | 0 | 1 (3%) |
| Asian | 22 (5%) | 21 (6%) | 1 (3%) |
| Black or African American | 26 (6%) | 22 (6%) | 4 (11%) |
| Native Hawaiian or Pacific Islander | 1 (<1%) | 1 (<1%) | 0 |
| White | 337 (83%) | 297 (83%) | 27 (77%) |
| Other | 4 (1%) | 4 (1%) | 0 |
| Unknown or not reported | 15 (4%) | 12 (3%) | 2 (6%) |
| Ethnicity, n (%) | |||
| Hispanic or Latino | 12 (3%) | 11 (3%) | 1 (3%) |
| Non-Hispanic or Latino | 383 (94%) | 336 (94%) | 34 (97%) |
| Unknown or not reported | 11 (3%) | 10 (3%) | 0 |
Source: Adapted from FDA Review
Abbreviations: mITT, modified intent-to-treat; SD, standard deviatio
What are the benefits of this drug?
In CL0007, 7.6% (27 out of 357) of patients with LUMISIGHT imaging had cancer found in additional tissue removed due to positive LUMISIGHT signal after completion of SOC lumpectomy surgery. Among the images considered positive for cancer by the reference standard, 49.1% had positive LUMISIGHT signal, and among the images considered negative for cancer by the reference standard, 86.5% had negative LUMISIGHT signal.
What are the benefits of this drug (results of trials used to assess efficacy)?
CL0007 assessed the proportion of patients receiving LUMISIGHT who had residual breast cancer detected and removed using the Lumicell DVS after completion of SOC lumpectomy. There were 166 of 357 (46%) patients who had at least one LUMISIGHT-guided shave. A total of 27 of 357 patients had residual breast cancer confirmed by histopathology in at least one LUMISIGHT-guided shave (7.6%; 95% CI: 5.0%, 10.8%).
Table 2 shows sensitivity and specificity of LUMISIGHT for residual breast cancer after completion of SOC lumpectomy calculated from the 2,346 evaluable images in the study. Regions of the lumpectomy cavity from which LUMISIGHT-guided shaves were taken contributed more than one image to the analysis.
Table 2. Image-Level Performance of LUMISIGHT for Detection of Residual Breast Cancer in the Lumpectomy Cavity After Completion of Standard of Care Lumpectomy
| Parameter | Reference Standard Positive | Reference Standard Negative | Total | |
| LUMISIGHT imaging result1 Positive | 34 | 337 | 371 | |
| LUMISIGHT imaging result1 Negative | 35 | 1940 | 1975 | |
| Total | 69 | 2277 | 2346 | |
| Diagnostic performance2 % (95% CI) | Sensitivity 49.1 (36.4, 61.9) | Specificity 86.5 (84.5, 88.3) | NA | |
Source: LUMISIGHT Prescribing Information
1 Regions of the lumpectomy cavity from which LUMISIGHT-guided shaves were taken contributed more than one image.
2 Sensitivity and specificity were calculated using a generalized estimating equation method to account for within-patient correlation.
Abbreviations: CI, confidence interval; NA, not applicable
A total of 155 of 357 (43%) patients had at least one false positive image, and 28 of 357 (8%) patients had at least one false negative image. Among the 166 patients with at least one LUMISIGHT-guided shave, the mean total LUMISIGHT-guided shave volume was 22 cm3 ±20 cm3, accounting for 20% ±15% of the total volume of resection.
After completion of SOC lumpectomy, prior to LUMISIGHT-guided tissue removal, 62 of 357 patients (17%) had at least one cancer-positive margin. After LUMISIGHT-guided surgery, 9 of these 62 (15%) patients changed to having all cancer-negative margins, and 2 of the remaining 295 (1%) patients changed from having all cancer-negative margins to having at least one cancer-positive margin.
Were there any differences in how well the drug worked in clinical trials among sex, race, and age?
- Sex: LUMISIGHT was not evaluated in male patients.
- Race: Because almost all participants were White, differences between races in how LUMISIGHT worked could not be determined.
- Age: The observed effect of LUMISIGHT was similar in patients younger and older than 65 years of age.
Were there any differences in how well the drug worked in clinical trials among sex, race, and age groups?
Table 3. Efficacy Results by Age and Race
| Parameter | Patient-Level Removal of Residual Cancer % (95% CI) |
Image-Level Sensitivity % (95% CI) |
Image-Level Specificity % (95% CI) |
| Age, years | |||
| <65 | 8.7 (5.1, 13.7) | 52.4 (36.4, 68) | 83 (80.7, 85.1) |
| ≥65 | 6.4 (3.2, 11.1) | 44.4 (25.5, 64.7) | 87.6 (85.5, 89.5) |
| Race | |||
| White | 7.1 (4.4, 10.6) | 49.1 (35.1, 63.2) | 86.4 (84.8, 87.9) |
| Other1 | 10 (3.8, 20.5) | 50 (24.7, 75.3) | 79.6 (75.3, 83.4) |
Source: Adapted from FDA Review
1 Other includes American Indian or Alaska Native, Asian, Black or African America, Native Hawaiian or Pacific Islander, other, and unknown or not reported
Note: Tissue-level sensitivity and specificity were derived using exact confidence interval estimates.
Abbreviations: CI, confidence interva
What are the possible side effects?
LUMISIGHT may cause serious and potentially deadly immune reactions. The most common side effect, seen in 85% of patients, was chromaturia (change in color of urine).
What are the possible side effects (results of trials used to assess safety)?
The safety of LUMISIGHT was evaluated in 726 patients who received a single dose of 1 mg/kg of LUMISIGHT. Among these 726 patients, 703 (97%) had breast cancer and 23 (3%) had other types of cancer. The mean age of the patients was 62 years (range: 36 years to 95 years), and 98% of them were female. Distribution by race was 82% White, 7% Black or African American, 6% Asian, and 5% other or unreported. Distribution by ethnicity was 3% Hispanic or Latino, 93% not Hispanic or Latino, and 4% unknown or unreported.
Adverse reactions occurring in ≥1% of patients receiving LUMISIGHT were hypersensitivity (1.4%, including anaphylaxis [4 out of 726]) and chromaturia (85%). Chromaturia resolved within 48 hours after administration in 93% of patients, with the longest time to resolution of 15 days.
Adverse reactions occurring in <1% of patients were skin discoloration after extravasation, nausea, dyspnea, pyrexia, and vomiting.
Were there any differences in side effects among sex, race, and age?
- Sex: Because almost all participants in the safety analysis were female, differences between sexes in the occurrence of side effects could not be determined.
- Race: Because almost all participants in the safety analysis were White, differences between races in the occurrence of side effects could not be determined.
- Age: The observed occurrence of side effects was lower in patients 65 years of age or older than patients younger than 65 years of age. Because of limited data, this difference may be due to chance.
Were there any differences in side effects of the clinical trials among sex, race, and age groups?
Table 4. Side Effects by Age, Safety Population
| Parameter | <65 Years n/Ns (%) |
≥65 Years n/Ns (%) |
| Patients with any non-chromaturia AE | 105/405 (26) | 71/321 (22) |
| Patients with any related non-chromaturia AE | 21/405 (5) | 8/321 (2) |
Source: Adapted from FDA Review
1 Other includes American Indian or Alaska Native, Asian, Black or African America, Native Hawaiian or Pacific Islander, other, and unknown or not reported
Abbreviations: AE, adverse event; n, number of patients with adverse event; Ns, total number of patients for each specific subgroup and were assigned to that specific ar
Table 5. Side Effects by Race, Safety Population
| Parameter | White n/Ns (%) |
Other1 n/Ns (%) |
| Patients with any non-chromaturia AE | 147/597 (25) | 29/129 (22) |
| Patients with any related non-chromaturia AE | 25/597 (4) | 4/129 (3) |
Source: Adapted from FDA Review
1 Other includes American Indian or Alaska Native, Asian, Black or African America, Native Hawaiian or Pacific Islander, other, and unknown or not reported
Abbreviations: AE, adverse event; n, number of patients with adverse event; Ns, total number of patients for each specific subgroup and were assigned to that specific arm
GLOSSARY
CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.