Trial Outcomes & Findings for A Phase II Trial of Poly-ICLC for Low-Grade Gliomas (NCT NCT04544007)
NCT ID: NCT04544007
Last Updated: 2025-03-21
Results Overview
Evaluate the efficacy of poly-ICLC in pediatric NF1 patients with progressive low-grade glioma (LGG) as measured by objective tumor response rate (CR+PR), using Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria. Complete Response (CR) is complete disappearance of the target lesion on T2 weighted and T2 weighted fluid attenuated inversion recovery (FLAIR) imaging and contrast imaging using baseline MRI or best recorded response for comparison; Partial Response (PR) is 50% or greater reduction in the target lesion on T2 weighted and T2 weighted-FLAIR imaging using the baseline MRI for comparison. Response will be assessed through the time of the post-12th cycle tumor assessment, or through the last tumor assessment time during this nominal period for patients in follow-up but who have withdrawn from protocol therapy, or through the last available tumor assessment for patients who are lost to follow-up during this time.
TERMINATED
PHASE2
3 participants
First 48 weeks
2025-03-21
Participant Flow
Participant milestones
| Measure |
Administer Poly-ICLC
Enrolled participants will receive poly-ICLC 20 mcg/kg/dose twice weekly IM (using Monday/Thursday or Tuesday/Friday schedule if possible).
Poly ICLC: Enrolled participants will receive poly-ICLC 20 mcg/kg/dose twice weekly IM (using Monday/Thursday or Tuesday/Friday schedule if possible).
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|---|---|
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Overall Study
STARTED
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3
|
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Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Administer Poly-ICLC
Enrolled participants will receive poly-ICLC 20 mcg/kg/dose twice weekly IM (using Monday/Thursday or Tuesday/Friday schedule if possible).
Poly ICLC: Enrolled participants will receive poly-ICLC 20 mcg/kg/dose twice weekly IM (using Monday/Thursday or Tuesday/Friday schedule if possible).
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|---|---|
|
Overall Study
Withdrawal by Subject
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1
|
|
Overall Study
Confirmed Progressive Disease
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1
|
Baseline Characteristics
A Phase II Trial of Poly-ICLC for Low-Grade Gliomas
Baseline characteristics by cohort
| Measure |
Poly-ICLC
n=3 Participants
This is a phase II, prospective, longitudinal, multi-center trial of poly-ICLC (Hiltonol) treatment for progressive low-grade gliomas in pediatric patients with NF1. The primary objective is to evaluate the efficacy of poly-ICLC in pediatric NF1 patients with progressive low-grade glioma (LGG) as measured by objective tumor response rate (CR+PR) within the first 48 weeks (12 cycles) of therapy.
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|---|---|
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Age, Categorical
<=18 years
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3 Participants
n=5 Participants
|
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Age, Categorical
Between 18 and 65 years
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0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
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0 Participants
n=5 Participants
|
|
Age, Continuous
|
11 years
STANDARD_DEVIATION 6.56 • n=5 Participants
|
|
Sex: Female, Male
Female
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2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
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1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
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2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
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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
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1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
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2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
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3 Participants
n=5 Participants
|
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Poly-ICLC
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3 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: First 48 weeksEvaluate the efficacy of poly-ICLC in pediatric NF1 patients with progressive low-grade glioma (LGG) as measured by objective tumor response rate (CR+PR), using Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria. Complete Response (CR) is complete disappearance of the target lesion on T2 weighted and T2 weighted fluid attenuated inversion recovery (FLAIR) imaging and contrast imaging using baseline MRI or best recorded response for comparison; Partial Response (PR) is 50% or greater reduction in the target lesion on T2 weighted and T2 weighted-FLAIR imaging using the baseline MRI for comparison. Response will be assessed through the time of the post-12th cycle tumor assessment, or through the last tumor assessment time during this nominal period for patients in follow-up but who have withdrawn from protocol therapy, or through the last available tumor assessment for patients who are lost to follow-up during this time.
Outcome measures
| Measure |
Poly-ICLC
n=3 Participants
Enrolled participants will receive poly-ICLC 20 mcg/kg/dose twice weekly IM
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|---|---|
|
Evaluate the Efficacy of Poly-ICLC
Achieved Complete or Partial Response
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0 Participants
|
|
Evaluate the Efficacy of Poly-ICLC
Failed to Achieve Complete or Partial Response
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3 Participants
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: 1 patient progressed on day 170. The other 2 patients are censored on day 134 and day 849 respectively.
Determine progression free survival (PFS) in pediatric NF1 patients with progressive LGG treated with poly-ICLC. Progression free survival (PFS), defined as the time from start of treatment to the first occurrence of tumor recurrence, tumor progression, occurrence of a second malignancy, or death from any cause. Patients event-free at last follow-up will be censored in analysis. PFS will be determined at 12, 24 and 60 months.
Outcome measures
| Measure |
Poly-ICLC
n=3 Participants
Enrolled participants will receive poly-ICLC 20 mcg/kg/dose twice weekly IM
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|---|---|
|
Determine Progression Free Survival (PFS)
Progressed by last visit
|
1 Participants
|
|
Determine Progression Free Survival (PFS)
Censored at the last visit
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2 Participants
|
SECONDARY outcome
Timeframe: 24 MonthsPopulation: 1 patient progressed on day 170. The other 2 patients are censored on day 134 and day 849 respectively.
Determine progression free survival (PFS) in pediatric NF1 patients with progressive LGG treated with poly-ICLC. Progression free survival (PFS), defined as the time from start of treatment to the first occurrence of tumor recurrence, tumor progression, occurrence of a second malignancy, or death from any cause. Patients event-free at last follow-up will be censored in analysis. PFS will be determined at 12, 24 and 60 months.
Outcome measures
| Measure |
Poly-ICLC
n=3 Participants
Enrolled participants will receive poly-ICLC 20 mcg/kg/dose twice weekly IM
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|---|---|
|
Determine Progression Free Survival (PFS)
Progressed by last visit
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1 Participants
|
|
Determine Progression Free Survival (PFS)
Censored at the last visit
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2 Participants
|
SECONDARY outcome
Timeframe: 24 MonthsEvaluate the efficacy of poly-ICLC in pediatric NF1 patients with progressive LGG as measured by best objective tumor response rate (CR+PR) within 24 cycles of therapy. Objective response rate (defined as CR or PR) after 24 cycles of treatment. Response will be assessed through the time of the post-24th cycle tumor assessment, or through the last tumor assessment time during this nominal period for patients in follow-up, but who have withdrawn from protocol therapy, or through the last available tumor assessment for patients who are lost to follow-up during this time.
Outcome measures
| Measure |
Poly-ICLC
n=3 Participants
Enrolled participants will receive poly-ICLC 20 mcg/kg/dose twice weekly IM
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|---|---|
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Number of Participants Who Achieved Best Objective Tumor Response Rate (CR+PR)
Achieved Complete or Partial Response
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0 Participants
|
|
Number of Participants Who Achieved Best Objective Tumor Response Rate (CR+PR)
Failed to Achieve Complete or Partial Response
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3 Participants
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SECONDARY outcome
Timeframe: 12 MonthsEvaluate the efficacy of poly-ICLC in pediatric NF1 patients with progressive LGG as measured by clinical benefit response rate (CR+PR+MR+SD). Clinical benefit defined as (CR+PR+MR+SD). Clinical benefit will be assessed through the time of the post-12 and 24th cycle tumor assessment, or through the last tumor assessment time during this nominal period for patients in follow-up but who have withdrawn from protocol therapy, or through the last available tumor assessment for patients who are lost to follow-up during this time.
Outcome measures
| Measure |
Poly-ICLC
n=3 Participants
Enrolled participants will receive poly-ICLC 20 mcg/kg/dose twice weekly IM
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|---|---|
|
Evaluate Efficacy by Clinical Benefit Response Rate (CR+PR+MR+SD)
Complete Response at 12 Months
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0 Participants
|
|
Evaluate Efficacy by Clinical Benefit Response Rate (CR+PR+MR+SD)
Partial Response at 12 Months
|
0 Participants
|
|
Evaluate Efficacy by Clinical Benefit Response Rate (CR+PR+MR+SD)
Minor Response at 12 Months
|
0 Participants
|
|
Evaluate Efficacy by Clinical Benefit Response Rate (CR+PR+MR+SD)
Stable Disease at 12 Months
|
1 Participants
|
|
Evaluate Efficacy by Clinical Benefit Response Rate (CR+PR+MR+SD)
Progressive Disease at 12 Months
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1 Participants
|
|
Evaluate Efficacy by Clinical Benefit Response Rate (CR+PR+MR+SD)
Failed to Remain on Treatment for 12 Months
|
1 Participants
|
SECONDARY outcome
Timeframe: 24 MonthsEvaluate the efficacy of poly-ICLC in pediatric NF1 patients with progressive LGG as measured by clinical benefit response rate (CR+PR+MR+SD). Clinical benefit defined as (CR+PR+MR+SD). Clinical benefit will be assessed through the time of the post-12 and 24th cycle tumor assessment, or through the last tumor assessment time during this nominal period for patients in follow-up but who have withdrawn from protocol therapy, or through the last available tumor assessment for patients who are lost to follow-up during this time.
Outcome measures
| Measure |
Poly-ICLC
n=3 Participants
Enrolled participants will receive poly-ICLC 20 mcg/kg/dose twice weekly IM
|
|---|---|
|
Evaluate Efficacy by Clinical Benefit Response Rate (CR+PR+MR+SD)
Minor Response at 24 Months
|
0 Participants
|
|
Evaluate Efficacy by Clinical Benefit Response Rate (CR+PR+MR+SD)
Complete Response at 24 Months
|
0 Participants
|
|
Evaluate Efficacy by Clinical Benefit Response Rate (CR+PR+MR+SD)
Partial Response at 24 Months
|
0 Participants
|
|
Evaluate Efficacy by Clinical Benefit Response Rate (CR+PR+MR+SD)
Stable Disease at 24 Months
|
1 Participants
|
|
Evaluate Efficacy by Clinical Benefit Response Rate (CR+PR+MR+SD)
Progressive Disease at 24 Months
|
0 Participants
|
|
Evaluate Efficacy by Clinical Benefit Response Rate (CR+PR+MR+SD)
Failed to reach at 24 Months
|
2 Participants
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SECONDARY outcome
Timeframe: Up to 24 MonthsAssess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG. The grade and duration of CTCAE toxicities observed during treatment, graded according to the National Cancer Institute Common Terminology Criteria for Adverse Event (CTCAE) version 5.0 for reporting of adverse events.
Outcome measures
| Measure |
Poly-ICLC
n=3 Participants
Enrolled participants will receive poly-ICLC 20 mcg/kg/dose twice weekly IM
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|---|---|
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Assess Toxicity
|
3 Participants
|
Adverse Events
Poly-ICLC
Serious adverse events
| Measure |
Poly-ICLC
n=3 participants at risk
This is a phase II, prospective, longitudinal, multi-center trial of poly-ICLC (Hiltonol) treatment for progressive low-grade gliomas in pediatric patients with NF1. The primary objective is to evaluate the efficay of poly-ICLC in pediatric NF1 patients with progressive low-grade glioma (LGG) as measured by objective tumor response rate (CR+PR) within the first 48 weeks (12 cycles) of therapy.
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|---|---|
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Ear and labyrinth disorders
Middle Ear Inflammation
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33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Musculoskeletal and connective tissue disorders
Back Pain
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33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
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Nervous system disorders
Hydrocephalus
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33.3%
1/3 • Number of events 4 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
Other adverse events
| Measure |
Poly-ICLC
n=3 participants at risk
This is a phase II, prospective, longitudinal, multi-center trial of poly-ICLC (Hiltonol) treatment for progressive low-grade gliomas in pediatric patients with NF1. The primary objective is to evaluate the efficay of poly-ICLC in pediatric NF1 patients with progressive low-grade glioma (LGG) as measured by objective tumor response rate (CR+PR) within the first 48 weeks (12 cycles) of therapy.
|
|---|---|
|
Blood and lymphatic system disorders
White Blood Cell Decreased
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Ear and labyrinth disorders
Middle Ear Inflammation
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Ear and labyrinth disorders
Tinnitus
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Eye disorders
Eye Disorders - Other, Proptosis
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Eye disorders
Periorbital Edema
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Eye disorders
Photophobia
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
General disorders
Fever
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33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
General disorders
Injection Site Reaction
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33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Infections and infestations
Conjunctivitis
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Infections and infestations
Otitis Media
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33.3%
1/3 • Number of events 2 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Infections and infestations
Upper Respiratory Infection
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Injury, poisoning and procedural complications
Fall
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Investigations
Neutrophil Count Decreased
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Investigations
Weight Gain
|
33.3%
1/3 • Number of events 2 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Metabolism and nutrition disorders
Hyperphosphatemia
|
66.7%
2/3 • Number of events 11 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and Connective Tissue Disorder - Other, Specify, Joint Crepitus
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Nervous system disorders
Facial Muscle Weakness
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Nervous system disorders
Headache
|
33.3%
1/3 • Number of events 5 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Nervous system disorders
Hydrocephalus
|
33.3%
1/3 • Number of events 3 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Nervous system disorders
Paresthesia
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Nervous system disorders
Peripheral Sensory Neuropathy
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Psychiatric disorders
Anxiety
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
|
Skin and subcutaneous tissue disorders
Rash Maculo-Papular
|
33.3%
1/3 • Number of events 1 • Up to 24 Months
Assess the toxicity associated with poly-ICLC treatment in pediatric NF1 patients with LGG
|
Additional Information
Coretta Thomas, Scientist & Data Manager for NF Consortium
University of Alabama at Birmingham
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place