OsteosarcomaPhase 1CytaluxNCT06235125
Safety and Feasibility of Intraoperative Visualization With Cytalux in Children
A Phase 1 study testing Cytalux in people with Osteosarcoma and Pulmonary Metastasis, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial summary
Pediatric subjects aged 6-17 with biopsy confirmed cancer and imaging findings suspicious for pulmonary metastatic disease scheduled to undergo pulmonary metastasectomy via and open or minimally invasive approach. Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Osteosarcoma, Pulmonary Metastasis, Fluorescence
- What is being tested?
- Cytalux
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Apr 2024to 31 Aug 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients 6-17 years of age at the time of study enrollment
- Willingness of research participant or legal guardian/representative to give written informed consent
- Willingness of patients (subjects) age 12-17 to provide written adolescent assent
- Patient weight greater than or equal to 20 kg
- Histologically confirmed diagnosis of osteosarcoma, synovial sarcoma, hepatoblastoma, rhabdomyosarcoma, Ewing sarcoma, Wilms tumor or other non-rhabdomyosarcoma soft tissue sarcoma
- Imaging findings highly suspicious for pulmonary metastatic disease based on CT, PET-CT or other imaging and warranting pulmonary surgery based on the judgment of the treating team. At least one nodule ≥4mm measured by preoperative imaging.
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- Female (assigned female at birth) participant is not pregnant and agrees to an acceptable form of contraception from the time of consent through 30 days after study intervention. Confirmed abstinence is an acceptable form of contraception.
- Female (assigned female at birth) participant must agree to not donate ova from time of consent until 30 days after study intervention
- Male (assigned male at birth) participant must agree to not donate sperm from time of consent until 30 days after study intervention.
Who cannot join
- Any medical condition that in the opinion of the investigators could potentially jeopardize the safety of the subject
- History of anaphylactic reactions to products containing indocyanine green for near infrared imaging. Subjects with a medical history of 'idiopathic anaphylaxis' will also be excluded.
- History of allergy to any of the components of CYTALUX™ (PAFOLACIANINE) INJECTION
- Presence of any psychological, familial, sociological condition or geographical challenges potentially hampering compliance with the study protocol or follow-up schedule
- Impaired renal function defined as eGFR< 50 mL/min/1.73m2
- Impaired liver function defined as values > 3x the upper limit of normal (ULN) for alanine aminotransferase (ALT) or aspartate aminotransferase (AST), alkaline phosphatase (ALP), or >2x ULN for total bilirubin except in subjects with Gilbert's syndrome.
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- Patient unable or unwilling to discontinue folate, folic acid, or folate-containing supplements 48 hours before study drug administration
- History of drug-related serious adverse event with prior Cytalux administration will be an exclusion for re-enrollment for contralateral surgery (see section 5.7).
- Participants will be excluded if their 12th or 18th birthday would occur during study participation
- Male sex at birth and commitment to acceptable form of contraception from time of consent through 30 days after study intervention with confirmed abstinence as an acceptable form of contraception as an inclusion criterion.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611
Timothy Lautz, MD, 312-227-4210, tlautz@luriechildrens.org
Seth D Goldstein, MD, 312-227-4210, sdgoldstein@luriechildens.org
About this study
Official title: A Pilot Study of Near-Infrared Imaging Using the Novel Imaging Agent Cytalux for Adolescent Patients With Metastatic Osteosarcoma Undergoing Pulmonary Metastasectomy
Pediatric subjects aged 6-17 with biopsy confirmed cancer and imaging findings suspicious for pulmonary metastatic disease scheduled to undergo pulmonary metastasectomy via and open or minimally invasive approach.
Read the detailed description
Pulmonary metastasectomy, or surgery to remove cancer which has spread to the lungs, plays a key role in the treatment of children with metastatic solid tumors. Fluorescence-guided surgery (FGS) has been shown to be a promising technique to highlight cancer cells and enable real-time surgical guidance (Stummer 2006, Hernot 2019, Goldstein 2021). Current techniques for fluorescence-guided surgery rely on indocyanine green (ICG), a non-specific fluorescent molecular agent which tends to accumulate in cancer cells because of increased angiogenesis and decreased lymphatic clearance. Molecular agents targeted to tumor-specific receptors offer the hope of increased sensitivity and specificity for detecting even very small metastatic nodules, and thus enhancing surgical clearance of disease.
Many of the tumors which metastasize to the lung are known to express the folate receptor and may therefore benefit from utilizing the tumor imaging agent CYTALUX (pafolacianine). CYTALUX (Pafolacianine) as a diagnostic tool during cancer resection has already been investigated in adults (approved NDA 214907), and no age-specific differences are expected in the pediatric population. Findings from the ELUCIDATE trial in adults demonstrated that intraoperative molecular imaging with CYTALUX (pafolacianine) improves surgical outcomes by identifying occult tumors and close surgical margins and it therefore gained FDA approval for adults with cancer in the lung (both primary and metastatic) as an adjunct with near infrared (NIR) imaging for detection of disease during surgery. 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 is a pilot study in pediatric subjects aged 6-17 with biopsy confirmed cancer and imaging findings suspicious for pulmonary metastatic disease scheduled to undergo pulmonary metastasectomy via an open or minimally invasive approach.
In the proposed study, subjects and their parents or guardians will give informed consent prior to commencement of any study procedures. Subjects will be dosed with 0.025 mg/kg CYTALUX (pafolacianine) injection intravenously from 4 hours to up to 24 hours prior to surgery. Subjects will be considered evaluable if they are exposed to study drug and/or NIR fluorescent light imaging. During surgery, all subjects will first undergo evaluation by normal surgical techniques (white light, palpation, and/or other localization techniques) and all suspicious nodules and lesions identified under standard surgical approach will be recorded as such. Following standard surgical assessment, subjects will undergo assessment with NIR fluorescent light imaging prior to and after resection.
- Conditions
- Osteosarcoma, Pulmonary Metastasis, Fluorescence, Metastatic Sarcoma, Pediatrics
- Being tested
- Cytalux
- Medical subject headings
- Osteosarcoma, Sarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CytaluxDrug
Also called Pafolacianine injection, OTL38 injection
Folate analog ligand conjugated with an indole cyanine-like green dye as a solution in vials containing 3 mL at 2 mg/mL
| Study group | Role | What they receive |
|---|---|---|
| Cytalux with Near Infrared Imaging | Experimental | All participants will receive Cytalux and undergo near infrared imaging. Receives: Drug: Cytalux |
What is being measured
The primary outcome is the main question the study is designed to answer.
Sensitivity of Near-infrared (NIR) imaging after pafolacianine injection for metastatic nodule identification
In children and adolescents with histologically-confirmed cancer undergoing pulmonary surgery for suspected metastases, each excised pulmonary lesion will be classified by the surgeon as positive or negative on NIR imaging after administration CYTALUX™ (PAFOLACIANINE) and then subjected to routine histologic examination. Sensitivity will be assessed as the fraction of histologically-confirmed metastatic nodules which demonstrated positive NIR fluorescence.
Measured: 1 day
False-Positive Rate of Near-infrared (NIR) imaging after pafolacianine injection for metastatic nodule identification
In children and adolescents with histologically-confirmed cancer undergoing pulmonary surgery for suspected metastases, each excised pulmonary lesion will be classified by the surgeon as positive or negative on NIR imaging after administration CYTALUX™ (PAFOLACIANINE) and then subjected to routine histologic examination. The false positive rate will be assessed as the fraction of NIR-positive nodules that have no viable malignancy on histologic examination.
Measured: 1 day
Secondary outcomes (2)
Detection of occult metastatic lesion(s) with Near-infrared (NIR) imaging after pafolacianine injection
The proportion of subjects with one or more NIR-positive lesion(s), not detected under normal light and/or palpation, and subsequently determined on histologic evaluation to contain viable metastatic tumor
Measured: 1 day
Number of patients with treatment-emergent adverse events (TEAEs)
Adverse events (AEs) will be coded using MedDRA. An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. A TEAE is defined as an AE observed after administration of the study intervention and up to the follow-up period. The number of TEAEs will be collected to asses the safety and tolerability of single intravenous infusions/injection of CYTALUX™ (PAFOLACIANINE) INJECTION in children and adolescents
Measured: 30 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2024
- Study start8 Apr 2024
- Primary completion31 Aug 2026 (estimated)
- Study completion31 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for OsteosarcomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Collaborators
- On Target Laboratories, LLC
- Responsible party
- Principal investigator, Timothy Lautz, Ann & Robert H Lurie Children's Hospital of Chicago
- Investigators
- Timothy Lautz, MD, Principal investigator, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06235125
- Sponsor's protocol ID
- 2023-6263
- Acronym
- Cytalux
- Status last verified
- Jan 2026
- First submitted
- 6 Jan 2024
- Last updated
- 5 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Sarkaria IS, Martin LW, Rice DC, Blackmon SH, Slade HB, Singhal S; ELUCIDATE Study Group. Pafolacianine for intraoperative molecular imaging of cancer in the lung: The ELUCIDATE trial. J Thorac Cardiovasc Surg. 2023 Dec;166(6):e468-e478. doi: 10.1016/j.jtcvs.2023.02.025. Epub 2023 Mar 3.
PubMed 37019717
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Near-Infrared Imaging Using the Novel Imaging Agent Cytalux for Adolescent Patients With Metastatic Osteosarcoma Undergoing Pulmonary Metastasectomy
- ClinicalTrials.gov ID
- NCT06235125
- Acronym / sponsor ID
- Cytalux / 2023-6263
- Conditions
- Osteosarcoma, Pulmonary Metastasis, Fluorescence, Metastatic Sarcoma, Pediatrics
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Collaborators
- On Target Laboratories, LLC
- Study start
- 8 Apr 2024 (actual)
- Primary completion
- 31 Aug 2026 (estimated)
- Study completion
- 31 Aug 2026 (estimated)
- First posted
- 31 Jan 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Pediatric subjects aged 6-17 with biopsy confirmed cancer and imaging findings suspicious for pulmonary metastatic disease scheduled to undergo pulmonary metastasectomy via and open or minimally invasive approach.
Detailed description
Pulmonary metastasectomy, or surgery to remove cancer which has spread to the lungs, plays a key role in the treatment of children with metastatic solid tumors. Fluorescence-guided surgery (FGS) has been shown to be a promising technique to highlight cancer cells and enable real-time surgical guidance (Stummer 2006, Hernot 2019, Goldstein 2021). Current techniques for fluorescence-guided surgery rely on indocyanine green (ICG), a non-specific fluorescent molecular agent which tends to accumulate in cancer cells because of increased angiogenesis and decreased lymphatic clearance. Molecular agents targeted to tumor-specific receptors offer the hope of increased sensitivity and specificity for detecting even very small metastatic nodules, and thus enhancing surgical clearance of disease.
Many of the tumors which metastasize to the lung are known to express the folate receptor and may therefore benefit from utilizing the tumor imaging agent CYTALUX (pafolacianine). CYTALUX (Pafolacianine) as a diagnostic tool during cancer resection has already been investigated in adults (approved NDA 214907), and no age-specific differences are expected in the pediatric population. Findings from the ELUCIDATE trial in adults demonstrated that intraoperative molecular imaging with CYTALUX (pafolacianine) improves surgical outcomes by identifying occult tumors and close surgical margins and it therefore gained FDA approval for adults with cancer in the lung (both primary and metastatic) as an adjunct with near infrared (NIR) imaging for detection of disease during surgery. 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 is a pilot study in pediatric subjects aged 6-17 with biopsy confirmed cancer and imaging findings suspicious for pulmonary metastatic disease scheduled to undergo pulmonary metastasectomy via an open or minimally invasive approach.
In the proposed study, subjects and their parents or guardians will give informed consent prior to commencement of any study procedures. Subjects will be dosed with 0.025 mg/kg CYTALUX (pafolacianine) injection intravenously from 4 hours to up to 24 hours prior to surgery. Subjects will be considered evaluable if they are exposed to study drug and/or NIR fluorescent light imaging. During surgery, all subjects will first undergo evaluation by normal surgical techniques (white light, palpation, and/or other localization techniques) and all suspicious nodules and lesions identified under standard surgical approach will be recorded as such. Following standard surgical assessment, subjects will undergo assessment with NIR fluorescent light imaging prior to and after resection.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cytalux with Near Infrared Imaging | Experimental | Drug: CytaluxAll participants will receive Cytalux and undergo near infrared imaging. |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Patients 6-17 years of age at the time of study enrollment
- Willingness of research participant or legal guardian/representative to give written informed consent
- Willingness of patients (subjects) age 12-17 to provide written adolescent assent
- Patient weight greater than or equal to 20 kg
- Histologically confirmed diagnosis of osteosarcoma, synovial sarcoma, hepatoblastoma, rhabdomyosarcoma, Ewing sarcoma, Wilms tumor or other non-rhabdomyosarcoma soft tissue sarcoma
Key exclusion criteria (10 listed)
- Any medical condition that in the opinion of the investigators could potentially jeopardize the safety of the subject
- History of anaphylactic reactions to products containing indocyanine green for near infrared imaging. Subjects with a medical history of 'idiopathic anaphylaxis' will also be excluded.
- History of allergy to any of the components of CYTALUX™ (PAFOLACIANINE) INJECTION
- Presence of any psychological, familial, sociological condition or geographical challenges potentially hampering compliance with the study protocol or follow-up schedule
- Impaired renal function defined as eGFR< 50 mL/min/1.73m2
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CytaluxAlso: Pafolacianine injection, OTL38 injectionFolate analog ligand conjugated with an indole cyanine-like green dye as a solution in vials containing 3 mL at 2 mg/mL | Drug | Cytalux with Near Infrared Imaging |
Primary outcome
Sensitivity of Near-infrared (NIR) imaging after pafolacianine injection for metastatic nodule identification
Time frame: 1 day
In children and adolescents with histologically-confirmed cancer undergoing pulmonary surgery for suspected metastases, each excised pulmonary lesion will be classified by the surgeon as positive or negative on NIR imaging after administration CYTALUX™ (PAFOLACIANINE) and then subjected to routine histologic examination. Sensitivity will be assessed as the fraction of histologically-confirmed metastatic nodules which demonstrated positive NIR fluorescence.
False-Positive Rate of Near-infrared (NIR) imaging after pafolacianine injection for metastatic nodule identification
Time frame: 1 day
In children and adolescents with histologically-confirmed cancer undergoing pulmonary surgery for suspected metastases, each excised pulmonary lesion will be classified by the surgeon as positive or negative on NIR imaging after administration CYTALUX™ (PAFOLACIANINE) and then subjected to routine histologic examination. The false positive rate will be assessed as the fraction of NIR-positive nodules that have no viable malignancy on histologic examination.
2 secondary outcomes
Detection of occult metastatic lesion(s) with Near-infrared (NIR) imaging after pafolacianine injection
Time frame: 1 day
Number of patients with treatment-emergent adverse events (TEAEs)
Time frame: 30 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2026.
- Background: PubMed 37019717Sarkaria IS, Martin LW, Rice DC, Blackmon SH, Slade HB, Singhal S; ELUCIDATE Study Group. Pafolacianine for intraoperative molecular imaging of cancer in the lung: The ELUCIDATE trial. J Thorac Cardiovasc Surg. 2023 Dec;166(6):e468-e478. doi: 10.1016/j.jtcvs.2023.02.025. Epub 2023 Mar 3.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Timothy Lautz, MD principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cytalux in people with Osteosarcoma and Pulmonary Metastasis, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Timothy Lautz, MD (312-227-4210, tlautz@luriechildrens.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Sensitivity of Near-infrared (NIR) imaging after pafolacianine injection for metastatic nodule identification (time frame: 1 day). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06235125, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jun 2026.