Trial Outcomes & Findings for A Study of Lymphoseek® as a Lymphoid Tissue Targeting Agent in Pediatric Patients With Melanoma, Rhabdomyosarcoma, or Other Solid Tumors Who Are Undergoing Lymph Node Mapping (NCT NCT02509598)

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

24 participants

Primary outcome timeframe

1 Day

Results posted on

2024-06-28

Participant Flow

Participant milestones

Participant milestones
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Overall Study STARTED	24
Overall Study COMPLETED	23
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Overall Study Withdrawal by Subject	1

Baseline Characteristics

A Study of Lymphoseek® as a Lymphoid Tissue Targeting Agent in Pediatric Patients With Melanoma, Rhabdomyosarcoma, or Other Solid Tumors Who Are Undergoing Lymph Node Mapping

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=23 Participants 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Age, Continuous	11.6 years STANDARD_DEVIATION 4.9 • n=5 Participants
Sex: Female, Male Female	15 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	21 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Tumor type Melanoma	5 Participants n=5 Participants
Tumor type Rhabdomyosarcoma	4 Participants n=5 Participants
Tumor type Other solid tumors	14 Participants n=5 Participants

PRIMARY outcome

Timeframe: 1 Day

Outcome measures

Outcome measures
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=23 Participants 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Average Number of Lymph Nodes Identified Intraoperatively Per Subject	3.30 nodes per subject Interval 1.95 to 4.65

PRIMARY outcome

Timeframe: 1 Day

The proportion of subjects with Lymphoseek-identified lymph nodes

Outcome measures

Outcome measures
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=23 Participants 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Subject Localization Rates	22 Participants

SECONDARY outcome

Timeframe: 1 Day

Outcome measures

Outcome measures
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=23 Participants 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Proportion of Subjects Who Underwent Preoperative SPECT or SPECT/CT	21 Participants

SECONDARY outcome

Timeframe: 1 day

Population: Number of subjects who underwent preoperative SPECT/CT

Outcome measures

Outcome measures
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=21 Participants 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Proportion of Subjects With a Lymph Node Identified Preoperatively Using SPECT or SPECT/CT	20 Participants

SECONDARY outcome

Timeframe: 1 Day

Outcome measures

Outcome measures
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=21 Participants 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Average Number of Lymph Nodes Identified Preoperatively Using SPECT or SPECT/CT Per Subject	2.75 nodes per subject Interval 1.81 to 3.68

SECONDARY outcome

Timeframe: 1 Day

Average difference in number of LNs identified preoperatively with SPECT/CR and number of LNs identified intraoperatively per subject

Outcome measures

Outcome measures
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=21 Participants 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Agreement of the Number of Nodes Identified by Preoperative SPECT or SPECT/CT to Intraoperative Localization	-0.86 nodes per subject Interval -2.6 to 0.89

SECONDARY outcome

Timeframe: 1 Day

Population: Number of subjects with pathology-positive nodes

The proportion of patients with pathology-positive lymph nodes who had at least one pathology-positive lymph node that was identified by Lymphoseek and had no other pathology-positive lymph nodes (i.e., identified by any other method)

Outcome measures

Outcome measures
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=2 Participants 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Upstaging	2 Participants

SECONDARY outcome

Timeframe: 1 Day

Population: Subjects with pathology-positive nodes

The number of pathology-positive lymph nodes that were missed intraoperatively by mapping agent divided by the total number of pathology-positive lymph nodes for that mapping agent. Population is all subjects with pathology-positive nodes. False negative rate represents the failure rate of the mapping agent to identify sentinel lymph nodes.

Outcome measures

Outcome measures
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=4 pathology-positive nodes 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Nodal False Negative Rate for Nodes Identified	0 proportion Interval 0.0 to 0.6

SECONDARY outcome

Timeframe: 1 Day

Population: Subjects with pathology-positive nodes

The number of pathology-positive lymph nodes that were identified intraoperatively by mapping agent divided by the total number of pathology-positive lymph nodes for that mapping agent. Population is all subjects with pathology-positive nodes. Sensitivity rate represents the success rate of the mapping agent to identify sentinel lymph nodes.

Outcome measures

Outcome measures
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=4 pathology-positive nodes 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Nodal Sensitivity	1.00 proportion Interval 0.39 to 1.0

SECONDARY outcome

Timeframe: 1 Day

Number of nodes whose local and central pathology results agree divided by the number of nodes with nonmissing local and central pathology results

Outcome measures

Outcome measures
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=58 Nodes with nonmissing pathology 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Nodal Agreement of Central Pathology Assessment With Local Pathology Assessment of the Excised Lymph Node(s) to Confirm the Presence/Absence of Tumor Metastases	57 Nodes with nonmissing pathology

SECONDARY outcome

Timeframe: 1 Day

Change in subject nodal staging was assessed via shift tables to show the number of subjects with each combination of presurgery and postsurgery nodal staging. Presurgery staging was based on standard-of-care clinical assessment. Postsurgery staging was based on the pathology results of the nodes identified.

Outcome measures

Outcome measures
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=23 Participants 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Change in Subject Nodal Staging Before and After Surgery Based Upon Nodes Identified	3 Participants

SECONDARY outcome

Timeframe: 1 Day

Changes to postsurgical treatment plan will be compared to baseline treatment plan.

Outcome measures

Outcome measures
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=23 Participants 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Number of Changes in Postsurgical Treatment Plan in Relation to Nodes Identified by Lymphoseek	2 Participants

Adverse Events

Tc99m Tilmanocept and Vital Blue Dye (Optional)

Serious events: 3 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=23 participants at risk 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Blood and lymphatic system disorders Anaemia	4.3% 1/23 • Number of events 1 • Adverse events beginning on the day of injection through the follow-up assessment (4- to 14-day or 62 ± 7 day, depending on protocol amendment at time of enrollment) were to be reported. Adverse events, blood samples, physical exam, vital signs and ECGs were collected and monitored at protocol-specified times.
Blood and lymphatic system disorders febrile neutropenia	8.7% 2/23 • Number of events 4 • Adverse events beginning on the day of injection through the follow-up assessment (4- to 14-day or 62 ± 7 day, depending on protocol amendment at time of enrollment) were to be reported. Adverse events, blood samples, physical exam, vital signs and ECGs were collected and monitored at protocol-specified times.
Metabolism and nutrition disorders hypokalaemia	4.3% 1/23 • Number of events 1 • Adverse events beginning on the day of injection through the follow-up assessment (4- to 14-day or 62 ± 7 day, depending on protocol amendment at time of enrollment) were to be reported. Adverse events, blood samples, physical exam, vital signs and ECGs were collected and monitored at protocol-specified times.
Renal and urinary disorders acute kidney injury	4.3% 1/23 • Number of events 1 • Adverse events beginning on the day of injection through the follow-up assessment (4- to 14-day or 62 ± 7 day, depending on protocol amendment at time of enrollment) were to be reported. Adverse events, blood samples, physical exam, vital signs and ECGs were collected and monitored at protocol-specified times.
General disorders pyrexia	4.3% 1/23 • Number of events 1 • Adverse events beginning on the day of injection through the follow-up assessment (4- to 14-day or 62 ± 7 day, depending on protocol amendment at time of enrollment) were to be reported. Adverse events, blood samples, physical exam, vital signs and ECGs were collected and monitored at protocol-specified times.

Other adverse events

Other adverse events
Measure	Tc99m Tilmanocept and Vital Blue Dye (Optional) n=23 participants at risk 0.5 mCi, 50 ug of Tc99m tilmanocept single administration. Optionally, 1-3 mL of vital blue dye, single administration (per institution's standard of care). Tc99m tilmanocept: A single dose of 0.5 mCi and 50 ug of Tc99m tilmanocept administered intradermally, peritumorally approximately 15 minutes to 8 hours before surgery Vital Blue Dye (optional): A single dose 1-3 mL of vital blue dye (1%) administered intradermally at the start of or during surgery (optional, per institution's standard of care). Lymph Node Mapping: Intraoperative lymph node mapping will occur 15 minutes to 8 hours after Tc99m tilmanocept injection
Gastrointestinal disorders Nausea	17.4% 4/23 • Adverse events beginning on the day of injection through the follow-up assessment (4- to 14-day or 62 ± 7 day, depending on protocol amendment at time of enrollment) were to be reported. Adverse events, blood samples, physical exam, vital signs and ECGs were collected and monitored at protocol-specified times.
Gastrointestinal disorders Vomiting	13.0% 3/23 • Adverse events beginning on the day of injection through the follow-up assessment (4- to 14-day or 62 ± 7 day, depending on protocol amendment at time of enrollment) were to be reported. Adverse events, blood samples, physical exam, vital signs and ECGs were collected and monitored at protocol-specified times.

Additional Information

Director, Regulatory & Medical Affairs

Cardinal Health 414, LLC

Phone: 6147570226

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place