Relapsed Solid TumorPhase 1/2NCT03690869

REGN2810 in Pediatric Patients With Relapsed, Refractory Solid, or Central Nervous System (CNS) Tumors and Safety and Efficacy of REGN2810 in Combination With Radiotherapy in Pediatric Patients With Newly Diagnosed or Recurrent Glioma

A Phase 1/2 study testing cemiplimab (monotherapy), cemiplimab (maintenance) and Conventional or hypofractionated in people with Relapsed Solid Tumor and Refractory Solid Tumor, run by Regeneron Pharmaceuticals at 20 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Phase 1: To confirm the safety and anticipated recommended phase 2 dose (RP2D) of REGN2810 (cemiplimab) for children with recurrent or refractory solid or Central Nervous System (CNS) tumors To characterize the pharmacokinetics (PK) of REGN2810 given in children with recurrent or refractory solid or CNS tumors Phase 2… Read more about this study

Who is it for?: Ages Up to 25 years
Conditions the study is for: Relapsed Solid Tumor, Refractory Solid Tumor, Relapsed Central Nervous System Tumor
What is being tested?: cemiplimab (monotherapy), cemiplimab (maintenance) and Conventional or hypofractionated
Type of treatment: Injection or infusion, Surgery or procedure
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 57 participants
Where: 20 sitesUnited States
Study dates: 24 Sep 2018to 10 May 2023
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 Up to 25 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Age 0 to <18 years of age (Phase 1)
Age ≥3 and ≤25 years of age (Efficacy Phase)
Karnofsky performance status ≥50 (patients >16 years) or Lansky performance status ≥50 (patients ≤ 16 years)
Life expectancy >8 weeks
Adequate Bone Marrow Function
Adequate Renal Function
Adequate Liver Function
Adequate Neurologic Function

Who cannot join

Patients with bulky metastatic disease of the CNS causing Uncal herniation or symptomatic midline shift, significant, symptomatic mass effect, or uncontrolled neurological symptoms such as seizures or altered mental status
Patients with metastatic spine disease and gliomatosis as documented by diffuse involvement of >2 lobes
Patients who are receiving any other investigational anticancer agent(s)
Patients on greater than dexamethasone 0.1 mg/kg/day (maximum 4 mg/day) or equivalent dose in alternate corticosteroid, or actively undergoing corticosteroid dose escalation in the last 7 days
Patients with a history of allogeneic stem cell transplant
Prior treatment with an agent that blocks the PD-1/PD-L1/PD-L2 pathway
Note: Other protocol-defined Inclusion/Exclusion criteria apply

Trial locations and contacts

20 sites in 1 country.

City, postcode or hospital

20 sites in 1 country

United States20 sites

C. S. Mott/University of Michigan
Ann Arbor, Michigan, 48109
Johns Hopkins - Pediatric Oncology
Baltimore, Maryland, 21287
Massachusetts General Hospital
Boston, Massachusetts, 02114
Dana Farber Cancer Institute/ Boston Children's Hospital
Boston, Massachusetts, 02215
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Florida- Neurosurgery
Gainesville, Florida, 32610
Texas Children's Cancer & Hematology Centers Baylor College of Medicine
Houston, Texas, 77030
Children's Hospital Los Angeles (CHLA)
Los Angeles, California, 90054
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404
University of Minnesota / Masonic Cancer Center
Minneapolis, Minnesota, 55455
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Oregon Health & Science University (OHSU) - Doernbecher Children's Hospital
Portland, Oregon, 97239
University of Utah
Salt Lake City, Utah, 84113
Rady Children's Hospital
San Diego, California, 92123
UCSF Benioff Children's Hospital
San Francisco, California, 94158
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University School of Medicine
St Louis, Missouri, 63110
Children's National Health System (Children's National Medical Center)
Washington D.C., District of Columbia, 20010

Other trials in United States

About this study

Official title: A Safety and Pharmacokinetic Study of Single Agent REGN2810 in Pediatric Patients With Relapsed or Refractory Solid or Central Nervous System (CNS) Tumors and a Safety and Efficacy Trial of REGN2810 in Combination With Radiotherapy in Pediatric Patients With Newly Diagnosed Diffuse Intrinsic Pontine Glioma, Newly Diagnosed High-Grade Glioma, or Recurrent High-Grade Glioma

Phase 1:

To confirm the safety and anticipated recommended phase 2 dose (RP2D) of REGN2810 (cemiplimab) for children with recurrent or refractory solid or Central Nervous System (CNS) tumors
To characterize the pharmacokinetics (PK) of REGN2810 given in children with recurrent or refractory solid or CNS tumors

Phase 2 (Efficacy Phase):

To confirm the safety and anticipated RP2D of REGN2810 to be given concomitantly with conventionally fractionated or hypofractionated radiation among patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG)
To confirm the safety and anticipated RP2D of REGN2810 given concomitantly with conventionally fractionated or hypofractionated radiation among patients with newly diagnosed high-grade glioma (HGG)
To confirm the safety and anticipated RP2D of REGN2810 given concomitantly with re-irradiation in patients with recurrent HGG
To assess PK of REGN2810 in pediatric patients with newly diagnosed DIPG, newly diagnosed HGG, or recurrent HGG when given in combination with radiation
To assess anti-tumor activity of REGN2810 in combination with radiation in improving overall survival at 12 months (OS12) among patients with newly diagnosed DIPG
To assess anti-tumor activity of REGN2810 in combination with radiation in improving progression-free survival at 12 months (PFS12) among patients with newly diagnosed HGG
To assess anti-tumor activity of REGN2810 in combination with radiation in improving overall survival at OS12 among patients with recurrent HGG

Conditions: Relapsed Solid Tumor, Refractory Solid Tumor, Relapsed Central Nervous System Tumor, Refractory Central Nervous System Tumor, Diffuse Intrinsic Pontine Glioma, High Grade Glioma
Being tested: cemiplimab (monotherapy), cemiplimab (maintenance), Conventional or hypofractionated, Re-irradiation
Keywords: Newly Diagnosed, Recurrent, Refractory
Medical subject headings: Central Nervous System Neoplasms, Diffuse Intrinsic Pontine Glioma, Glioma, Recurrence

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

cemiplimab (monotherapy)Drug
Also called REGN2810, Libtayo
To be administered intravenously as monotherapy in Phase 1
cemiplimab (maintenance)Drug
Also called REGN2810, Libtayo
To be administered intravenously in combination with radiation and then used as maintenance therapy
Conventional or hypofractionatedRadiation
Combined with cemiplimab IV administration
Re-irradiationRadiation
Combined with cemiplimab IV administration

Study groups in NCT03690869
Study group	Role	What they receive
Phase 1	Experimental	Patients in both the Solid Tumor Cohort and the CNS Cohort will receive cemiplimab monotherapy. Each Cohort will have 2 subgroups by age (0 to <12 years, 12 to <18 years). Receives: Drug: cemiplimab (monotherapy)
Efficacy with Newly Diagnosed DIPG	Experimental	≥ 3 to < 12 years cohort and 12 to ≤ 25 years cohort with combination of cemiplimab and radiation therapy Receives: Drug: cemiplimab (maintenance); Radiation: Conventional or hypofractionated
Efficacy with Newly Diagnosed HGG	Experimental	≥ 3 to < 12 years cohort and 12 to ≤ 25 years cohort with combination of cemiplimab and radiation therapy Receives: Drug: cemiplimab (maintenance); Radiation: Conventional or hypofractionated
Efficacy with Recurrent HGG	Experimental	≥ 3 to < 12 years cohort and 12 to ≤ 25 years cohort with combination of cemiplimab and radiation therapy Receives: Drug: cemiplimab (maintenance); Radiation: Re-irradiation

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Treatment-emergent Adverse Events (TEAEs)
TEAEs are AEs that developed or worsened during the on-treatment period and any treatment-related AEs that occur during the post-treatment period but prior to initiation of other anticancer therapy. Number of TEAEs reported.
Measured: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
Number of Severe (National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE] Grade 3/4/5) TEAEs
NCI CTCAE version 4.0 was utilized for AE grading of severity: Grade 1 (Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated); Grade 2 (Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental ADL); Grade 3 (Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL); Grade 4 (Life-threatening consequences; urgent intervention indicated); Grade 5 (Death related to AE). Number of NCI grade 3/4/5 Treatment-Emergent Adverse Events (AEs) reported
Measured: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
Number of Treatment-emergent Sponsor Identified Immune-related Adverse Events (irAEs)
Number of sponsor-identified irAEs (all grades) reported.
Measured: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
Number of Severe (NCI CTCAE Grade 3/4/5) Treatment-emergent Sponsor Identified irAEs
Number of severe treatment-emergent sponsor-identified irAEs reported.
Measured: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
Number of Treatment-emergent AEs of Special Interest (AESI)
AEs of special interest (AESI) are AEs required to be monitored, documented, and managed in a pre-specified manner as described in the protocol. Number of treatment-emergent AESI reported.
Measured: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
Number of NCI Grade 3/4/5 Treatment-emergent AESI
Number of NCI grade 3/4/5 treatment-emergent AESI reported
Measured: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
Number of Participants With Any TEAE Resulting in Death
Number of participants with any TEAE resulting in death reported
Measured: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
Number of Participants With at Least One Lab Abnormality (NCI-CTCAE All Grades) in Hematology, Electrolytes, Liver, Chemistry
Number of participants with new or worsened laboratory abnormalities (NCI-CTCAE All Grades) reported in Hematology, Electrolytes, Liver, Chemistry; Grade 1 (Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated); Grade 2 (Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental ADL); Grade 3 (Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL); Grade 4 (Life-threatening consequences; urgent intervention indicated); Grade 5 (Death related to AE).
Measured: Up to 36 months
Number of Participants Who Developed Dose Limiting Toxicities (DLTs) (Phase 1)
Number of participants who developed dose limiting toxicities (DLTs) in phase 1 reported
Measured: Baseline to 28 days
Number of Participants Who Developed DLTs (Efficacy Phase)
Measured: Up to 4 weeks post radiation therapy
Elimination Half-life (t1/2) of Functional Cemiplimab (REGN2810) in Serum
Measured: Up to 24 months
Trough Concentration (Ctrough) of Functional Cemiplimab (REGN2810) in Serum
Ctrough (trough concentration) of functional cemiplimab in serum reported.
Measured: Up to Week 16
Peak Concentration (Cmax) of Functional Cemiplimab (REGN2810) in Serum
Cmax (peak concentration) of functional cemiplimab in serum reported.
Measured: Up to Week 16
Area Under the Concentration-time Curve (AUC) of Functional Cemiplimab (REGN2810) in Serum
Measured: Up to 24 months
Percentage of Progression-free Survival (PFS) at 12 Months for Participants With Newly Diagnosed HGG (ndHGG)
PFS was defined as the time from randomization to the date of the first documented tumor progression, as determined per Response Assessment in Neuro-Oncology (RANO)/Immunotherapy Response Assessment in Neuro-Oncology (iRANO) criteria, or death due to any cause.
Measured: At 12 months
Percentage of Overall Survival (OS) at 12 Months for Participants With ndDIPG and Recurrent HGG (rHGG)
OS was defined as the time from randomization to the date of death due to any cause. A participant who had not died was censored at the last date that participant was documented to be alive. 95% CI is based on Kaplan-Meier method.
Measured: Up to 12 months

Secondary outcomes (2)

Objective Response Rate (ORR) for Participants Who Have a Confirmed Complete Response (CR) or Partial Response (PR)
ORR was defined as the percentage of participants who have a confirmed complete response (CR) or partial response (PR), as determined per standard criteria between the date of first study treatment and the date of the first objectively documented progression or the date of receiving another anti-cancer systemic therapy, whichever came first. Clopper-Person exact confidence interval
Measured: Approximately 24 months
Number of Participants With Anti-REGN2810 Antibodies (ADA)
ADA status classified as: Positive; Pre-existing (baseline [BL] sample positive \& all post BL ADA titers reported as < 9-fold BL titer value); Negative (all samples negative); ADA positive: Treatment-boosted (positive result at BL with ≥1 post BL titer result ≥9-fold BL titer value); Treatment-emergent (TE) (negative result or missing result at BL with ≥1 positive post BL result); TE: Persistent (positive result detected in ≥2 consecutive post BL samples separated by ≥ a 12/16-week post BL period with no ADA-negative results in-between, regardless of any missing samples; Indeterminate (positive result in last collection, regardless of any missing samples); Transient (not persistent or indeterminate, regardless of any missing samples)
Measured: 1st follow-up visit, approximately 25 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

24 Sep 201810 May 2023

First posted1 Oct 2018
Study start24 Sep 2018
Primary completion10 May 2023
Study completion10 May 2023

Why it stopped: Sponsor Decision

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Relapsed Solid TumorStudies of the same condition, open and under way.
More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
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 1/2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 57 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03690869
Sponsor's protocol ID: R2810-ONC-1690
Other IDs: PNOC 013 (CC#160825)
Status last verified: Apr 2025
First submitted: 17 Sep 2018
Last updated: 8 Apr 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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PEEL-224, Vincristine and Temozolomide in Pediatric Solid Tumors
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All Relapsed Solid Tumor trials

Study at a glance

From the registry record, last updated by the sponsor on 8 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Safety and Pharmacokinetic Study of Single Agent REGN2810 in Pediatric Patients With Relapsed or Refractory Solid or Central Nervous System (CNS) Tumors and a Safety and Efficacy Trial of REGN2810 in Combination With Radiotherapy in Pediatric Patients With Newly Diagnosed Diffuse Intrinsic Pontine Glioma, Newly Diagnosed High-Grade Glioma, or Recurrent High-Grade Glioma
ClinicalTrials.gov ID: NCT03690869
Acronym / sponsor ID: R2810-ONC-1690
Conditions: Relapsed Solid Tumor, Refractory Solid Tumor, Relapsed Central Nervous System Tumor, Refractory Central Nervous System Tumor, Diffuse Intrinsic Pontine Glioma, High Grade Glioma
Keywords: Newly Diagnosed, Recurrent, Refractory
Phase: Phase 1/2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Sponsor Decision
Enrolment: 57 participants (actual)
Lead sponsor: Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
Collaborators: Pacific Pediatric Neuro-Oncology Consortium
Study start: 24 Sep 2018 (actual)
Primary completion: 10 May 2023 (actual)
Study completion: 10 May 2023 (actual)
First posted: 1 Oct 2018 (actual)
Results: Posted 9 Oct 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Phase 1:

To confirm the safety and anticipated recommended phase 2 dose (RP2D) of REGN2810 (cemiplimab) for children with recurrent or refractory solid or Central Nervous System (CNS) tumors
To characterize the pharmacokinetics (PK) of REGN2810 given in children with recurrent or refractory solid or CNS tumors

Phase 2 (Efficacy Phase):

To confirm the safety and anticipated RP2D of REGN2810 to be given concomitantly with conventionally fractionated or hypofractionated radiation among patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG)
To confirm the safety and anticipated RP2D of REGN2810 given concomitantly with conventionally fractionated or hypofractionated radiation among patients with newly diagnosed high-grade glioma (HGG)
To confirm the safety and anticipated RP2D of REGN2810 given concomitantly with re-irradiation in patients with recurrent HGG
To assess PK of REGN2810 in pediatric patients with newly diagnosed DIPG, newly diagnosed HGG, or recurrent HGG when given in combination with radiation
To assess anti-tumor activity of REGN2810 in combination with radiation in improving overall survival at 12 months (OS12) among patients with newly diagnosed DIPG
To assess anti-tumor activity of REGN2810 in combination with radiation in improving progression-free survival at 12 months (PFS12) among patients with newly diagnosed HGG
To assess anti-tumor activity of REGN2810 in combination with radiation in improving overall survival at OS12 among patients with recurrent HGG

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 4

Arm	Type	Receives
Phase 1	Experimental	Drug: cemiplimab (monotherapy)Patients in both the Solid Tumor Cohort and the CNS Cohort will receive cemiplimab monotherapy. Each Cohort will have 2 subgroups by age (0 to <12 years, 12 to <18 years).
Efficacy with Newly Diagnosed DIPG	Experimental	Drug: cemiplimab (maintenance), Radiation: Conventional or hypofractionated≥ 3 to < 12 years cohort and 12 to ≤ 25 years cohort with combination of cemiplimab and radiation therapy
Efficacy with Newly Diagnosed HGG	Experimental	Drug: cemiplimab (maintenance), Radiation: Conventional or hypofractionated≥ 3 to < 12 years cohort and 12 to ≤ 25 years cohort with combination of cemiplimab and radiation therapy
Efficacy with Recurrent HGG	Experimental	Drug: cemiplimab (maintenance), Radiation: Re-irradiation≥ 3 to < 12 years cohort and 12 to ≤ 25 years cohort with combination of cemiplimab and radiation therapy

Who may qualify

Ages: Up to 25 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (8 listed)

Age 0 to <18 years of age (Phase 1)
Age ≥3 and ≤25 years of age (Efficacy Phase)
Karnofsky performance status ≥50 (patients >16 years) or Lansky performance status ≥50 (patients ≤ 16 years)
Life expectancy >8 weeks
Adequate Bone Marrow Function

Key exclusion criteria (7 listed)

Patients with bulky metastatic disease of the CNS causing Uncal herniation or symptomatic midline shift, significant, symptomatic mass effect, or uncontrolled neurological symptoms such as seizures or altered mental status
Patients with metastatic spine disease and gliomatosis as documented by diffuse involvement of >2 lobes
Patients who are receiving any other investigational anticancer agent(s)
Patients on greater than dexamethasone 0.1 mg/kg/day (maximum 4 mg/day) or equivalent dose in alternate corticosteroid, or actively undergoing corticosteroid dose escalation in the last 7 days
Patients with a history of allogeneic stem cell transplant

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
cemiplimab (monotherapy)Also: REGN2810, LibtayoTo be administered intravenously as monotherapy in Phase 1	Drug	Phase 1
cemiplimab (maintenance)Also: REGN2810, LibtayoTo be administered intravenously in combination with radiation and then used as maintenance therapy	Drug	Efficacy with Newly Diagnosed DIPG, Efficacy with Newly Diagnosed HGG, Efficacy with Recurrent HGG
Conventional or hypofractionatedCombined with cemiplimab IV administration	Radiation	Efficacy with Newly Diagnosed DIPG, Efficacy with Newly Diagnosed HGG
Re-irradiationCombined with cemiplimab IV administration	Radiation	Efficacy with Recurrent HGG

Primary outcome

Number of Treatment-emergent Adverse Events (TEAEs)
Time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
TEAEs are AEs that developed or worsened during the on-treatment period and any treatment-related AEs that occur during the post-treatment period but prior to initiation of other anticancer therapy. Number of TEAEs reported.
Number of Severe (National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE] Grade 3/4/5) TEAEs
Time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
NCI CTCAE version 4.0 was utilized for AE grading of severity: Grade 1 (Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated); Grade 2 (Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental ADL); Grade 3 (Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL); Grade 4 (Life-threatening consequences; urgent intervention indicated); Grade 5 (Death related to AE). Number of NCI grade 3/4/5 Treatment-Emergent Adverse Events (AEs) reported
Number of Treatment-emergent Sponsor Identified Immune-related Adverse Events (irAEs)
Time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
Number of sponsor-identified irAEs (all grades) reported.
Number of Severe (NCI CTCAE Grade 3/4/5) Treatment-emergent Sponsor Identified irAEs
Time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
Number of severe treatment-emergent sponsor-identified irAEs reported.
Number of Treatment-emergent AEs of Special Interest (AESI)
Time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
AEs of special interest (AESI) are AEs required to be monitored, documented, and managed in a pre-specified manner as described in the protocol. Number of treatment-emergent AESI reported.
Number of NCI Grade 3/4/5 Treatment-emergent AESI
Time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
Number of NCI grade 3/4/5 treatment-emergent AESI reported
Number of Participants With Any TEAE Resulting in Death
Time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)
Number of participants with any TEAE resulting in death reported
Number of Participants With at Least One Lab Abnormality (NCI-CTCAE All Grades) in Hematology, Electrolytes, Liver, Chemistry
Time frame: Up to 36 months
Number of participants with new or worsened laboratory abnormalities (NCI-CTCAE All Grades) reported in Hematology, Electrolytes, Liver, Chemistry; Grade 1 (Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated); Grade 2 (Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental ADL); Grade 3 (Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL); Grade 4 (Life-threatening consequences; urgent intervention indicated); Grade 5 (Death related to AE).
Number of Participants Who Developed Dose Limiting Toxicities (DLTs) (Phase 1)
Time frame: Baseline to 28 days
Number of participants who developed dose limiting toxicities (DLTs) in phase 1 reported
Number of Participants Who Developed DLTs (Efficacy Phase)
Time frame: Up to 4 weeks post radiation therapy
Elimination Half-life (t1/2) of Functional Cemiplimab (REGN2810) in Serum
Time frame: Up to 24 months
Trough Concentration (Ctrough) of Functional Cemiplimab (REGN2810) in Serum
Time frame: Up to Week 16
Ctrough (trough concentration) of functional cemiplimab in serum reported.
Peak Concentration (Cmax) of Functional Cemiplimab (REGN2810) in Serum
Time frame: Up to Week 16
Cmax (peak concentration) of functional cemiplimab in serum reported.
Area Under the Concentration-time Curve (AUC) of Functional Cemiplimab (REGN2810) in Serum
Time frame: Up to 24 months
Percentage of Progression-free Survival (PFS) at 12 Months for Participants With Newly Diagnosed HGG (ndHGG)
Time frame: At 12 months
PFS was defined as the time from randomization to the date of the first documented tumor progression, as determined per Response Assessment in Neuro-Oncology (RANO)/Immunotherapy Response Assessment in Neuro-Oncology (iRANO) criteria, or death due to any cause.
Percentage of Overall Survival (OS) at 12 Months for Participants With ndDIPG and Recurrent HGG (rHGG)
Time frame: Up to 12 months
OS was defined as the time from randomization to the date of death due to any cause. A participant who had not died was censored at the last date that participant was documented to be alive. 95% CI is based on Kaplan-Meier method.

2 secondary outcomes

Objective Response Rate (ORR) for Participants Who Have a Confirmed Complete Response (CR) or Partial Response (PR)
Time frame: Approximately 24 months
Number of Participants With Anti-REGN2810 Antibodies (ADA)
Time frame: 1st follow-up visit, approximately 25 months

Results and safety

Results posted to the registry on 9 Oct 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
SOLID TUMOR < 12 yr REGN2810 3mg/kg	3	0
SOLID TUMOR 12 to <18 yr REGN2810 3mg/kg	5	0
CNS TUMOR <12 yr REGN2810 3mg/kg	3	0
CNS TUMOR <12 yr REGN2810 4.5mg/kg	9	0
CNS TUMOR 12 to <18 yr REGN2810 3mg/kg	5	0
ndDIPG <12 yr REGN2810 4.5mg/kg + Radiotherapy	6	0
ndDIPG >=12 yr REGN2810 3mg/kg + Radiotherapy	5	0
ndHGG >=12 yr REGN2810 3mg/kg + Radiotherapy	12	1
rHGG <12 yr REGN2810 4.5mg/kg + Re-Irradiation	1	0
rHGG >= 12 yr REGN2810 3mg/kg + Re-Irradiation	8	0

Primary outcome: Number of Treatment-emergent Adverse Events (TEAEs)

Time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months). Number. Unit: Events

Measure	SOLID TUMOR < 12 yr REGN2810 3mg/kg n = 3	SOLID TUMOR 12 to <18 yr REGN2810 3mg/kg n = 5	CNS TUMOR <12 yr REGN2810 3mg/kg n = 3	CNS TUMOR <12 yr REGN2810 4.5mg/kg n = 9	CNS TUMOR 12 to <18 yr REGN2810 3mg/kg n = 5	ndDIPG <12 yr REGN2810 4.5mg/kg + Radiotherapy n = 6	ndDIPG >=12 yr REGN2810 3mg/kg + Radiotherapy n = 5	ndHGG >=12 yr REGN2810 3mg/kg + Radiotherapy n = 12	rHGG <12 yr REGN2810 4.5mg/kg + Re-Irradiation n = 1	rHGG >= 12 yr REGN2810 3mg/kg + Re-Irradiation n = 8
Value	19	42	24	56	49	108	102	222	28	175

Population analysed: Safety analysis set (SAF): All enrolled participants who have received any study treatment (at least one dose of any component of study treatment in a combination therapy) in each study phase. Participants analyzed according to the study treatment received (as treated).

Primary outcome: Number of Severe (National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE] Grade 3/4/5) TEAEs

Time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months). Number. Unit: Events

Measure	SOLID TUMOR < 12 yr REGN2810 3mg/kg n = 3	SOLID TUMOR 12 to <18 yr REGN2810 3mg/kg n = 5	CNS TUMOR <12 yr REGN2810 3mg/kg n = 3	CNS TUMOR <12 yr REGN2810 4.5mg/kg n = 9	CNS TUMOR 12 to <18 yr REGN2810 3mg/kg n = 5	ndDIPG <12 yr REGN2810 4.5mg/kg + Radiotherapy n = 6	ndDIPG >=12 yr REGN2810 3mg/kg + Radiotherapy n = 5	ndHGG >=12 yr REGN2810 3mg/kg + Radiotherapy n = 12	rHGG <12 yr REGN2810 4.5mg/kg + Re-Irradiation n = 1	rHGG >= 12 yr REGN2810 3mg/kg + Re-Irradiation n = 8
Value	3	5	0	9	4	25	8	19	4	16

Primary outcome: Number of Treatment-emergent Sponsor Identified Immune-related Adverse Events (irAEs)

Time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months). Number. Unit: Events

Measure	SOLID TUMOR < 12 yr REGN2810 3mg/kg n = 3	SOLID TUMOR 12 to <18 yr REGN2810 3mg/kg n = 5	CNS TUMOR <12 yr REGN2810 3mg/kg n = 3	CNS TUMOR <12 yr REGN2810 4.5mg/kg n = 9	CNS TUMOR 12 to <18 yr REGN2810 3mg/kg n = 5	ndDIPG <12 yr REGN2810 4.5mg/kg + Radiotherapy n = 6	ndDIPG >=12 yr REGN2810 3mg/kg + Radiotherapy n = 5	ndHGG >=12 yr REGN2810 3mg/kg + Radiotherapy n = 12	rHGG <12 yr REGN2810 4.5mg/kg + Re-Irradiation n = 1	rHGG >= 12 yr REGN2810 3mg/kg + Re-Irradiation n = 8
Value	0	1	0	0	0	2	5	9	0	9

Primary outcome: Number of Severe (NCI CTCAE Grade 3/4/5) Treatment-emergent Sponsor Identified irAEs

Time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months). Number. Unit: Events

Measure	SOLID TUMOR < 12 yr REGN2810 3mg/kg n = 3	SOLID TUMOR 12 to <18 yr REGN2810 3mg/kg n = 5	CNS TUMOR <12 yr REGN2810 3mg/kg n = 3	CNS TUMOR <12 yr REGN2810 4.5mg/kg n = 9	CNS TUMOR 12 to <18 yr REGN2810 3mg/kg n = 5	ndDIPG <12 yr REGN2810 4.5mg/kg + Radiotherapy n = 6	ndDIPG >=12 yr REGN2810 3mg/kg + Radiotherapy n = 5	ndHGG >=12 yr REGN2810 3mg/kg + Radiotherapy n = 12	rHGG <12 yr REGN2810 4.5mg/kg + Re-Irradiation n = 1	rHGG >= 12 yr REGN2810 3mg/kg + Re-Irradiation n = 8
Value	0	0	0	0	0	0	3	2	0	1

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
SOLID TUMOR < 12 yr REGN2810 3mg/kg	0 of 3 (0%)	3 of 3 (100%)	2 of 3
SOLID TUMOR 12 to <18 yr REGN2810 3mg/kg	0 of 5 (0%)	5 of 5 (100%)	4 of 5
CNS TUMOR <12 yr REGN2810 3mg/kg	0 of 3 (0%)	2 of 3 (66.7%)	2 of 3
CNS TUMOR <12 yr REGN2810 4.5mg/kg	3 of 9 (33.3%)	8 of 9 (88.9%)	8 of 9
CNS TUMOR 12 to <18 yr REGN2810 3mg/kg	0 of 5 (0%)	4 of 5 (80%)	3 of 5
ndDIPG <12 yr REGN2810 4.5mg/kg + Radiotherapy	3 of 6 (50%)	6 of 6 (100%)	6 of 6
ndDIPG >=12 yr REGN2810 3mg/kg + Radiotherapy	3 of 5 (60%)	5 of 5 (100%)	4 of 5
ndHGG >=12 yr REGN2810 3mg/kg + Radiotherapy	7 of 12 (58.3%)	12 of 12 (100%)	7 of 12
rHGG <12 yr REGN2810 4.5mg/kg + Re-Irradiation	0 of 1 (0%)	1 of 1 (100%)	1 of 1
rHGG >= 12 yr REGN2810 3mg/kg + Re-Irradiation	5 of 8 (62.5%)	8 of 8 (100%)	5 of 8

Limitations and caveats (from the sponsor): As all efficacy cohorts for this study have now been closed due to the futility criteria, Regeneron as Sponsor in collaboration with PNOC made a decision to close the study to further enrollment.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
A Plain Language Summary is available on TrialSummaries.comLink provided by the sponsor.

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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. This study is not recruiting at the moment.

Clinical Trial Management study director

Regeneron Pharmaceuticals

20 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 8 inclusion and 7 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: stopped early, as of the sponsor's last update on 8 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 20 sites 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 9 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Treatment-emergent Adverse Events (TEAEs) (time frame: From first dose of study drug up to 90 days after the last dose of study treatment (up to 36 months)). The study lists 16 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 NCT03690869.

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 8 Apr 2025; AzTrials last refreshed its copy on 26 Oct 2026.