Primary Extra-cranial Solid TumorPhase 1NCT04928677
A Study of Codrituzumab in Children and Young Adults With Solid Tumors and Have Not Responded to Treatment or Have Come Back After Treatment
A Phase 1 study testing Codrituzumab in people with Primary Extra-cranial Solid Tumor and Recurrent or Refractory Glypican 3 (GPC3), run by Memorial Sloan Kettering Cancer Center at 5 sites in United States.
Trial summary
The purpose of this study to find out whether codrituzumab is a safe treatment that causes few or mild side effects in children and young adults who have solid tumors that express the protein GPC3. The researchers also want to study the way codrituzumab is absorbed, distributed, and cleared from the body. Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Primary Extra-cranial Solid Tumor, Recurrent or Refractory Glypican 3 (GPC3)
- What is being tested?
- Codrituzumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 5 sitesUnited States
- Study dates
- 9 Jun 2021to Jun 2027
- 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 1 year to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Consent/Assent: All patients and/or their parents or legally authorized representatives must sign written informed consent; assent, when appropriate, will be obtained according to institutional guidelines.
- Age: Patients must be aged 12 months up to but not including 22 years at the time of study registration. A patient who is 21 at the time of enrollment but turns 22 thereafter will still be considered eligible for the purposes of this study.
- Diagnosis:
- Patients must have a diagnosis of a primary extra-cranial solid tumor that is recurrent or refractory to standard therapy.
- For the purposes of this study, the following definitions will be used:
- Refractory is defined as any tumor which progresses despite maximal standard therapies
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- Recurrent (relapsed) is defined as a completion of planned therapy after which point the tumor recurs within 5 years of treatment. Additionally, any tumor which recurs twice is considered relapsed.
- Tumor GPC3 Expression: Patients must have demonstrated positive GPC3 expression via immunohistochemistry (IHC) on any prior tumor sample. Confirmation of GPC3 expression may include a diagnostic or relapsed sample, at a primary or metastatic site. There is no limit to how long ago this sample was collected. This GPC3 expression via IHC will be centrally confirmed by Ventana as in prior studies of codrituzumab and described in the treatment. Patients may choose to enroll on the prescreening portion, which allows for assessment of GPC3 expression only, prior to enrollment on the full clinical trial.
- Tumor GPC3 Expression: Patients must have demonstrated a minimum of 1+ GPC3 expression via immunohistochemistry (IHC) on any prior tumor sample. Confirmation of GPC3 expression may include a diagnostic or relapsed sample, at a primary or metastatic site. There is no limit to how long ago this sample was collected. This GPC3 expression via IHC will be centrally confirmed by Ventana as in prior studies of codrituzumab and described in the treatment plan section. Patients may choose to enroll on the prescreening portion, which allows for assessment of GPC3 expression only, prior to enrollment on the full clinical trial.
- Disease Status: Patients must have measurable disease based on RECIST 1.1.
- Performance Level: Patients must have Karnofsky Performance Score (for patients > 16 years of age) or Lansky Performance Score (for patients ≤ 16 years of age) ≥ 50% assessed within 2 weeks of study enrollment.
- Neurological Deficits: Patients with neurologic deficits must have been stable and off of steroids for a minimum of 1 week prior to study entry; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
- Pregnancy/Contraception: Patients must not be pregnant or breast-feeding; females, excluding pre-menstrual, must have a negative serum or urine pregnancy test within 7 days prior to enrollment; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method, which includes abstinence, for 90 days after the last dose of study drug.
- Prior Therapy: Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study; recovery is defined as all AEs, attributable to prior therapy, having improved to grade 2 or better or as outlined below. For agents that have known adverse events occurring beyond 28 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair.
- Systemic Anticancer Therapy (e.g. Chemotherapy):
- Not Myelosuppressive: > 7 days must have elapsed from their last dose of anticancer therapy not known to be myelosuppressive (e.g. not associated with reduced platelet or ANC counts).
- Myelosuppressive: > 14 days must have elapsed from their last dose of known myelosuppressive anticancer therapy.
- Antibodies: > 21 days must have elapsed from the infusion of last anticancer antibody.
- Cellular Therapies: > 42 days must have elapsed from the completion of any type of cellular therapy, including modified T cells, NK cells, dendritic cells, etc.
- Radiation: Patients who have had radiation must have had their last fraction of:
- Local irradiation to the primary tumors or other limited sites (cumulative dose < 40Gy) > 14 days prior to registration.
- Local irradiation to the primary tumors or other sites (cumulative dose ≥ 40Gy), therapeutic 131I-MIBG or other radiopharmaceutical, and other substantial bone marrow radiation, > 42 days prior to registration.
- Craniospinal irradiation, radiation to > 50% of pelvis, or total body irradiation > 120 days prior to registration.
- Stem Cell Infusions: With or without TBI
- 84 days must have elapsed from an allogeneic bone marrow or stem cell transplant, or any stem cell infusion including DLI or boost infusion and patients must also not have any evidence of acute or chronic GvHD.
- 42 days must have elapsed from an autologous stem cell infusion including boost infusion.
- Supportive Therapies: Patients must be off all growth factor(s) that support platelet, red blood cell, or white blood cell count, number or function for at least 7 days prior to registration (e.g. filgrastim, sargramostim, erythropoietin, romiplostim, eltrombopag, etc.) as well as off Pegylated G-CSF for at least 14 days prior to registration.
- Organ Function: Patients must have documented within 14 days of registration and within 7 days of starting treatment the following:
- Hgb > 8 gm/dL (may be transfusion-supported)
- Platelet count > 50,000/mm^3 (transfusion independent)
- Absolute neutrophil count (ANC) > 1000/mm^3
- INR ≤ 2.5
- Total Bilirubin (sum of conjugated + unconjugated) ≤ 3 times institutional upper limit of normal (ULN) for age
- Aspartate aminotransferase (AST) ≤ 5 times institutional ULN for age
- Alanine Aminotransferase (ALT) ≤ 5 times institutional ULN for age
- Serum albumin ≥ 2 g/dL
- GFR ≥ 50 mL/min/1.73 m^2 as measured using urine creatinine clearance, serum cystatin c, radioisotope GFR, or serum creatinine as measured by the Schwartz equation (Refer to Schwartz, et al. J Am Soc Neph, 2009)
Who cannot join
- Patients receiving current anti-cancer therapy or investigational agents are not eligible for study entry.
- Patients who do not have tumor tissue available for GPC3 testing are not eligible for study entry.
- Patients who have received any prior GPC3-directed immunotherapy are not eligible for study entry.
- Patients with uncontrolled seizures are not eligible for study entry.
- Subjects with a condition requiring systemic treatment with either corticosteroids (>0.15 mg/kg daily prednisone equivalents) or other immunosuppressive medications, if used to modify immune adverse events related to prior therapy, > 14 days must have elapsed since last dose of corticosteroid or immunosuppressive agent. Inhaled or topical steroids, and adrenal replacement doses ≤ 0.15 mg/kg daily prednisone equivalents are permitted in the absence of active autoimmune disease.
- Patients with documented CNS tumor, CNS metastasis, CNS ischemia and/or infarction, whether symptomatic or discovered incidentally without clinical symptoms, will be excluded from study participation.
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- Patients with a baseline QTc > 480. (as measured using Bazett formula; Refer to Bazett, Heart, 1920).
- Patients with an inability to return for follow-up visits, obtain follow-up studies required to assess toxicity to therapy, or comply with the safety monitoring requirements.
- Patients who have an uncontrolled infection are not eligible.
- Patients who have received a prior solid organ transplantation are not eligible.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Julia Glade Bender, MD contact
5 sites in 1 country
United States5 sites, 5 recruiting
- Recruiting
Children's Healthcare of Atlanta (Data Collection Only)
Atlanta, Georgia, 30322
Thomas Cash, MD, 404-785-0910, thomas.cash@emory.edu
- Recruiting
Dana Farber Cancer Institute (Data Collection Only)
Boston, Massachusetts, 02115
Allison O'Neill, MD, 617-632-4202
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Brian Turpin, Do, (513) 517-2234
- Recruiting
Children's Hospital of Los Angeles (Data Collection Only)
Los Angeles, California, 90027
Rachana Shah, MD, (323) 361-4624
- Recruiting
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Michael Ortiz, MD, 1-833-675-5437
About this study
Official title: A Multi-Center Phase I Study of Codrituzumab in Pediatric Patients With Relapsed or Refractory Glypican 3 (GPC3) Expressing Extra-cranial Solid Tumors
The purpose of this study to find out whether codrituzumab is a safe treatment that causes few or mild side effects in children and young adults who have solid tumors that express the protein GPC3. The researchers also want to study the way codrituzumab is absorbed, distributed, and cleared from the body.
- Being tested
- Codrituzumab
- Keywords
- Codrituzumab, Hepatoblastoma, 20-489
- Medical subject headings
- Recurrence, Simpson-Golabi-Behmel syndrome, Hepatoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CodrituzumabDrug
For Phase A (Dose Escalation), the starting dose (dose level 1) will be 10 mg/kg with 1 planned dose escalation to 20 mg/kg (dose level 2) if dose level 1 is determined to be safe and tolerable. Once a RP2D/MTD is identified in Phase A, then Phase B (hepatoblastoma expansion cohort) will open and allow for enrollment of up to 10 additional patients.For both phases, patients will receive codrituzumab IV once per week for a period of 21 days per cycle. Patients will be eligible for up to 25 cycles.
| Study group | Role | What they receive |
|---|---|---|
| Codrituzumab | Experimental | For Phase A of the study, we will use the 3+3 study design, with 2 planned dose levels, starting at 50% of the adult RP2D to confirm the pediatric RP2D/MTD. 6-9,43 Phase B will include an expansion cohort for patients with hepatoblastoma. Receives: Drug: Codrituzumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
estimate the MTD
The Phase A dose escalation scheme will follow a 3+3 design.
Measured: 1 year
Secondary outcomes (1)
response rate
partial + complete response according to RECIST v1.1)
Measured: at 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jun 2021
- Study start9 Jun 2021
- Primary completionJun 2027 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Extra-cranial Solid TumorStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- This is a phase I, open-label, dose-escalation, multi-center study in pediatric patients 12 months through 21 years of age with GPC3 expressing extra-cranial solid tumors.
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Chugai Pharma USA
- Responsible party
- Sponsor
- Investigators
- Michael Ortiz, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04928677
- Sponsor's protocol ID
- 20-489
- Status last verified
- Oct 2026
- First submitted
- 10 Jun 2021
- Last updated
- 26 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center Phase I Study of Codrituzumab in Pediatric Patients With Relapsed or Refractory Glypican 3 (GPC3) Expressing Extra-cranial Solid Tumors
- ClinicalTrials.gov ID
- NCT04928677
- Acronym / sponsor ID
- 20-489
- Keywords
- Codrituzumab, Hepatoblastoma, 20-489
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Collaborators
- Chugai Pharma USA
- Study start
- 9 Jun 2021 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 16 Jun 2021 (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.
The purpose of this study to find out whether codrituzumab is a safe treatment that causes few or mild side effects in children and young adults who have solid tumors that express the protein GPC3. The researchers also want to study the way codrituzumab is absorbed, distributed, and cleared from the body.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a phase I, open-label, dose-escalation, multi-center study in pediatric patients 12 months through 21 years of age with GPC3 expressing extra-cranial solid tumors.
| Arm | Type | Receives |
|---|---|---|
| Codrituzumab | Experimental | Drug: CodrituzumabFor Phase A of the study, we will use the 3+3 study design, with 2 planned dose levels, starting at 50% of the adult RP2D to confirm the pediatric RP2D/MTD. 6-9,43 Phase B will include an expansion cohort for patients with hepatoblastoma. |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (37 listed)
- Consent/Assent: All patients and/or their parents or legally authorized representatives must sign written informed consent; assent, when appropriate, will be obtained according to institutional guidelines.
- Age: Patients must be aged 12 months up to but not including 22 years at the time of study registration. A patient who is 21 at the time of enrollment but turns 22 thereafter will still be considered eligible for the purposes of this study.
- Patients must have a diagnosis of a primary extra-cranial solid tumor that is recurrent or refractory to standard therapy.
- For the purposes of this study, the following definitions will be used:
- Tumor GPC3 Expression: Patients must have demonstrated positive GPC3 expression via immunohistochemistry (IHC) on any prior tumor sample. Confirmation of GPC3 expression may include a diagnostic or relapsed sample, at a primary or metastatic site. There is no limit to how long ago this sample was collected. This GPC3 expression via IHC will be centrally confirmed by Ventana as in prior studies of codrituzumab and described in the treatment. Patients may choose to enroll on the prescreening portion, which allows for assessment of GPC3 expression only, prior to enrollment on the full clinical trial.
Key exclusion criteria (10 listed)
- Patients receiving current anti-cancer therapy or investigational agents are not eligible for study entry.
- Patients who do not have tumor tissue available for GPC3 testing are not eligible for study entry.
- Patients who have received any prior GPC3-directed immunotherapy are not eligible for study entry.
- Patients with uncontrolled seizures are not eligible for study entry.
- Subjects with a condition requiring systemic treatment with either corticosteroids (>0.15 mg/kg daily prednisone equivalents) or other immunosuppressive medications, if used to modify immune adverse events related to prior therapy, > 14 days must have elapsed since last dose of corticosteroid or immunosuppressive agent. Inhaled or topical steroids, and adrenal replacement doses ≤ 0.15 mg/kg daily prednisone equivalents are permitted in the absence of active autoimmune disease.
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 |
|---|---|---|
| CodrituzumabFor Phase A (Dose Escalation), the starting dose (dose level 1) will be 10 mg/kg with 1 planned dose escalation to 20 mg/kg (dose level 2) if dose level 1 is determined to be safe and tolerable. Once a RP2D/MTD is identified in Phase A, then Phase B (hepatoblastoma expansion cohort) will open and allow for enrollment of up to 10 additional patients.For both phases, patients will receive codrituzumab IV once per week for a period of 21 days per cycle. Patients will be eligible for up to 25 cycles. | Drug | Codrituzumab |
Primary outcome
estimate the MTD
Time frame: 1 year
The Phase A dose escalation scheme will follow a 3+3 design.
1 secondary outcome
response rate
Time frame: at 6 weeks
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 26 Oct 2026.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
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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.
Julia Glade Bender, MD contact
Michael Ortiz, MD principal investigator
Memorial Sloan Kettering Cancer Center
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Codrituzumab in people with Primary Extra-cranial Solid Tumor and Recurrent or Refractory Glypican 3 (GPC3), run by Memorial Sloan Kettering Cancer Center at 5 sites 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 37 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 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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 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 Michael Ortiz, MD (1-833-675-5437, ortizm2@mskcc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
estimate the MTD (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 26 Oct 2026.