Cytalux for Lung Metastasis in Children

(Cytalux Trial)

Overseen By Timothy Lautz, MD

Age: < 18

Sex: Any

Trial Phase: Phase 1

Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago

Must not be taking: Folate supplements

Disqualifiers: Anaphylaxis, Allergy, Renal impairment, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness of Cytalux for children with cancer that has spread to the lungs. It focuses on using a special imaging technique to improve the visibility and removal of lung tumors during surgery. Children aged 6 to 17 with certain cancers, such as osteosarcoma or Wilms tumor, and who have lung nodules that may require surgery, might be suitable candidates. Participants will receive Cytalux and undergo near-infrared imaging to help surgeons identify cancerous tissue. As a Phase 1 trial, the research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial requires participants to stop taking folate, folic acid, or folate-containing supplements 48 hours before the study drug is given. Other medications are not specifically mentioned, so it's best to discuss with the study team.

Is there any evidence suggesting that Cytalux is likely to be safe for children?

Research has shown that Cytalux is already approved for use in adults with lung and ovarian cancers, indicating some level of safety for humans in these cases. However, its safety and effectiveness for children remain unknown.

In adults, Cytalux is usually well-tolerated, but researchers are still gathering safety information for children. This trial is in the early stages and primarily focuses on assessing the treatment's safety for kids. Researchers will closely monitor for any side effects or problems.

Prospective participants should discuss any concerns with the medical team. They can provide more details on what to expect and how they will ensure participant safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Cytalux is unique because it uses near-infrared imaging to help surgeons see lung metastases more clearly during surgery. Most treatments for lung metastasis, like chemotherapy or radiation, focus on killing cancer cells but don't assist directly in surgery. Cytalux, however, acts as a fluorescent dye that makes cancerous tissues glow when exposed to a special light, allowing surgeons to precisely target and remove tumors. Researchers are excited because this precision could lead to more successful surgeries with fewer cancer cells left behind, potentially improving outcomes for children with lung metastases.

What evidence suggests that Cytalux might be an effective treatment for lung metastasis in children?

Research has shown that Cytalux, also known as pafolacianine, can make lung tumors more visible during surgery. This treatment attaches to a specific part of cancer cells called the folate receptor. In previous studies, Cytalux successfully highlighted lung tumors, aiding surgeons in locating and removing them. Although this trial is still investigating its safety and effectiveness in children, early results from adults are promising. This suggests the treatment might also help identify lung tumors in children. ^{1 6 7 8 9}

Who Is on the Research Team?

Timothy Lautz, MD

Principal Investigator

Ann & Robert H Lurie Children's Hospital of Chicago

Are You a Good Fit for This Trial?

This trial is for children aged 6-17 with certain types of cancer like osteosarcoma, who are scheduled for lung surgery to remove suspected metastases. They must weigh at least 20 kg and agree to use contraception or practice abstinence if applicable. Children under 12 need guardian consent, while those 12-17 must also give assent.

Inclusion Criteria

Willingness of research participant or legal guardian/representative to give written informed consent

Female participants must agree to not donate ova from consent until 30 days after study intervention

I am between 6 and 17 years old.

Exclusion Criteria

I am allergic to ingredients in CYTALUX™.

My liver tests are significantly higher than normal, but I might have Gilbert's syndrome.

Presence of any psychological, familial, sociological condition, or geographical challenges potentially hampering compliance with the study protocol or follow-up schedule

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-Surgery Preparation

Subjects will be dosed with 0.025 mg/kg CYTALUX (pafolacianine) injection intravenously from 4 hours to up to 24 hours prior to surgery.

1 day

Surgery and Imaging

Subjects undergo pulmonary metastasectomy with near-infrared imaging using CYTALUX to identify and resect metastatic nodules.

1 day

Follow-up

Participants are monitored for treatment-emergent adverse events (TEAEs) and safety of the intervention.

30 days

What Are the Treatments Tested in This Trial?

Interventions

• Cytalux

Trial Overview

The trial is testing the safety and feasibility of using Cytalux during surgery in pediatric patients. Cytalux helps surgeons see cancerous cells better to potentially improve the removal process during pulmonary metastasectomy.

How Is the Trial Designed?

Group I: Cytalux with Near Infrared Imaging Experimental Treatment 1 Intervention

All participants will receive Cytalux and undergo near infrared imaging.

Cytalux is already approved in United States for the following indications:

Approved in United States as Cytalux for:

• Ovarian cancer
• Lung cancer

Who Is Running the Clinical Trial?

Ann & Robert H Lurie Children's Hospital of Chicago

Lead Sponsor

Trials

275

Recruited

5,182,000+

On Target Laboratories, LLC

Industry Sponsor

Trials

8

Recruited

470+

Published Research Related to This Trial

In a study of 30 patients with brain metastases from non-small cell lung cancer (NSCLC), combination chemotherapy with cisplatin, ifosfamide, and irinotecan resulted in a 50% response rate in brain lesions, indicating significant efficacy.

The median survival time for these patients was 382 days, with a 1-year survival rate of 56.1%, suggesting that this chemotherapy regimen may improve outcomes for patients with brain metastases from NSCLC.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Combination chemotherapy of cisplatin, ifosfamide, and irinotecan with rhG-CSF support in patients with brain metastases from non-small cell lung cancer. Fujita, A., Fukuoka, S., Takabatake, H., et al.[2018]

A patient with brain metastases from small cell lung cancer initially responded well to cranial irradiation, indicating that this treatment can be effective for such cases.

Subsequent recurrences were successfully managed with high-dose intravenous etoposide and teniposide, suggesting that epipodophyllotoxins may be a promising treatment option for CNS metastases of small cell lung cancer.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Podophyllotoxins for brain metastases of small cell lung cancer. Haaxma-Reiche, H., Berendsen, HH., Postmus, PE.[2019]

Topotecan showed some efficacy in treating brain metastases from small cell lung cancer (SCLC), with objective responses in 2 out of 3 patients, but had limited effectiveness in other types of brain metastases, leading to early termination of the study with 19 patients.

The treatment was associated with significant hematological toxicity, including severe neutropenia (37%), thrombocytopenia (21%), and anemia (16%), which raises concerns about its safety, particularly in elderly patients or those with poor health status.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Topotecan in the treatment of brain metastases. A phase II study of GOIM (Gruppo Oncologico dell'Italia Meridionale). Lorusso, V., Galetta, D., Giotta, F., et al.[2013]

In a trial involving 43 chemonaive non-small-cell lung cancer patients with brain metastases, the combination of pemetrexed and cisplatin showed a significant objective response rate of 41.9% for brain metastases, indicating its efficacy as a first-line treatment.

The treatment was generally well-tolerated, with manageable side effects, including some grade 3-4 hematological toxicities, and resulted in a median survival time of 7.4 months, which is promising for a population with typically poor prognosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pemetrexed and cisplatin as first-line chemotherapy for advanced non-small-cell lung cancer (NSCLC) with asymptomatic inoperable brain metastases: a multicenter phase II trial (GFPC 07-01). Barlesi, F., Gervais, R., Lena, H., et al.[2022]

In a study of 159 patients with advanced non-small cell lung cancer, those treated with bevacizumab plus chemotherapy (BV+CT) had a significantly lower incidence of brain metastases (14%) compared to those receiving chemotherapy alone (31%).

Patients receiving BV+CT had better survival rates (33.89%) compared to those on chemotherapy alone (18.64%), particularly benefiting VEGF-positive patients, while high expression of carbonic anhydrase-9 (CA9) was associated with reduced effectiveness of BV treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Bevacizumab plus chemotherapy versus chemotherapy alone for preventing brain metastasis derived from advanced lung cancer. Fu, Y., Hu, J., Du, N., et al.[2022]

Citations

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clinicaltrials.gov/study/NCT06235125?term=PAFOLACIANINE&viewType=Table&rank=1

Study Details | NCT06235125 | Safety and Feasibility of ...

We anticipate that this agent will have similar safety and efficacy in children and to similarly demonstrate applicable for all types of metastatic tumors. This ...

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ontargetlabs.com/wp-content/uploads/2024/11/Let-it-glow-Intraoperative-visualization-of-pulmonary-metastases-using-pafolacianine-a-next-generation-fluorescent-agent-for-young-adults-undergoing-pulmonary-met-002.pdf

Intraoperative visualization of pulmonary metastases using ...

Among these emerging agents, pafolacianine (CYTALUX) has gar- nered attention for its ability to target the folate receptor, which is frequently ...

3.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12507096/

Pafolacianine for intraoperative molecular imaging of cancer ...

In the FS, 25 participants (22.3%) had a history of 31 cancerous lung tumors. Sites of origin of pulmonary metastases included colon and rectum, ovary, pancreas ...

4.

clinicaltrials.gov

clinicaltrials.gov/study/NCT02602119

Intraoperative Imaging of Pulmonary Nodules by OTL38

The age groups are: Child (birth-17); Adult (18-64); Older Adult (65+) ... A measure of all deaths, due to any cause, that occur during a clinical study. ... A ...

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cytaluxhcp.com/wp-content/uploads/2025/08/cytalux-uspi-july-2025.pdf

HIGHLIGHTS OF PRESCRIBING INFORMATION These ...

Safety and effectiveness of CYTALUX in pediatric patients have not been established. ... The depth of primary lung lesions detected by CYTALUX ranged from 0 to 38 ...

6.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/label/2022/214907s002lbl.pdf

Cytalux - accessdata.fda.gov

Safety and effectiveness of CYTALUX in pediatric patients have not been established. ... cancer in 65%, metastasis to the lung in 21%, benign lung lesions ...

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connect.careboxhealth.com

connect.careboxhealth.com/en-US/trial/listing/455635

Safety and Feasibility of Intraoperative Visualization With ...

Pediatric subjects aged 6-17 with biopsy confirmed cancer and imaging findings suspicious for pulmonary metastatic disease scheduled to undergo pulmonary ...

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Safety and Feasibility of Intraoperative Visualization With ...

Pediatric subjects aged 6-17 with biopsy confirmed cancer and imaging findings suspicious for pulmonary metastatic disease scheduled to ...

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ontargetlabs.com

ontargetlabs.com/new-case-report-demonstrates-the-promise-of-cytalux-in-detection-and-resection-of-metastatic-osteosarcoma-lesions/

New Case Report Demonstrates the Promise of CYTALUX ...

“While CYTALUX has already been approved for adult lung and ovarian cancers, this report demonstrates a proof of concept for pediatric surgeons ...