Apply to Trial

Compensation: Varies

Number of Visits: 1

Duration: Day 0 to 1

20 Participants Needed

Pafolacianine for Identifying Lesions in Childhood Cancer

Overseen ByGina Lewis

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Mayo Clinic

Must not be taking: Folic acid

Disqualifiers: Renal failure, Liver failure, Pregnancy, others

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

Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval

Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Will I have to stop taking my current medications?

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

How is the drug Pafolacianine unique in identifying lesions in childhood cancer?

Pafolacianine is unique because it is designed to help identify cancerous lesions during surgery by making them glow, which can improve the precision of surgical removal. This approach is different from traditional treatments that focus on shrinking or eliminating tumors through chemotherapy or radiation.¹ ² ³ ⁴ ⁵

What is the purpose of this trial?

This phase III trial studies how well pafolacianine works for identifying cancerous lesions in children and adolescent patients with primary solid tumors or solid tumors that have spread from where they first started (primary site) to other places in the body (metastatic). Pafolacianine is a fluorescent imaging agent that targets folate receptors which are overexpressed in many cancers and is used with near infrared (NIR) imaging during surgery to identify tumor cells. NIR uses a special camera that uses wavelengths in the infrared range to visualize and locate the tumor cells that are lit up by the pafolacianine. Giving pafolacianine for NIR imaging may work better than other imaging agents in identifying cancerous lesions in pediatric patients with solid tumors.

Research Team

Stephanie F. Polites, MD, MPH

Principal Investigator

Mayo Clinic

Eligibility Criteria

This trial is for children and adolescents with primary or metastatic solid tumors. Participants must have a type of cancer that could potentially overexpress folate receptors, making them suitable for the imaging agent being tested.

Inclusion Criteria

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

I am between 6 months and 17 years old.

I am suspected to have or diagnosed with a solid tumor and surgery is planned.

Exclusion Criteria

My kidneys are not working properly.

I am being evaluated for liver failure or am on the liver transplant list.

I cannot or will not use birth control.

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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive pafolacianine IV and undergo NIR fluorescent imaging during standard of care surgery

Day 0 to 1

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of adverse events

Up to 30 days after surgery

Treatment Details

Interventions

Pafolacianine

Trial Overview The study tests pafolacianine, an imaging agent used during surgery to light up cancer cells in kids. It's paired with near-infrared (NIR) imaging to help surgeons see and remove tumors more effectively than with standard methods.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Diagnostic (pafolacianine + NIR)Experimental Treatment4 Interventions

Patients receive pafolacianine IV over 60 to 90 minutes and undergo NIR fluorescent imaging during standard of care (SOC) surgery on study. Patients also undergo collection of tissue during SOC surgery on study.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

Findings from Research

Cure rates for children with cancer have plateaued around 70%, highlighting the need for new drug approaches, especially for hard-to-treat cancers like stage IV neuroblastoma and relapsed leukemia.

Novel agents, including cell cycle inhibitors like clofarabine and nelarabine, as well as targeted therapies such as tyrosine kinase inhibitors and monoclonal antibodies, show promise in improving treatment outcomes for high-risk pediatric cancers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Educational paper. The development of new therapies for pediatric oncology.Horton, TM., Berg, SL.[2021]

The Patient-derived xenograft (PDX) for Childhood Cancer Therapeutics (PCAT) portal provides a comprehensive resource for studying pediatric cancer, featuring 324 PDX models along with clinical and genomic data to aid in the development of new treatments.

PCAT includes curated preclinical testing results for 68 models and 79 therapeutic agents, allowing researchers to identify effective therapies and compare patient tumors with PDX models, enhancing the understanding of pediatric cancer biology.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

PCAT: an integrated portal for genomic and preclinical testing data of pediatric cancer patient-derived xenograft models.Yang, J., Li, Q., Noureen, N., et al.[2021]