EpendymomaPhase 1NCT07061626
Determine Maximum Tolerated Dose, Safety, and Tolerability of Rhenium (186Re) in Pediatric Recurrent, Refractory or Progressive Ependymoma and High-Grade Glioma
A Phase 1 study testing Rhenium-186 Nanoliposome in people with Ependymoma and High Grade Gliomas, run by Cerenome at 1 site in United States.
Trial summary
Pediatric patients 6-21 years of age with supratentorial recurrent, refractory, or progressive pediatric ependymoma and high-grade glioma (HGG) will be included in this study of treatment with Rhenium-186 Nanoliposome (186RNL). Read more about this study
- Who is it for?
- Ages 6 to 21 years
- Conditions the study is for
- Ependymoma, High Grade Gliomas
- What is being tested?
- Rhenium-186 Nanoliposome
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2026to Mar 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 6 years to 21 years* of age.
- Lesion number and size:
- Phase 1a/b only: A single lesion (less than or equal to) ≤3.5 cm (longest axis) and volume of (less than or equal to) ≤22.4 mL as the largest tumor (subsequent to individual Cohort lesion size requirements).
- Phase 2a only: A single lesion or any number of multiple lesions separated by (less than or equal to) ≤3 cm; each lesion (less than or equal to) ≤3.5 cm (longest axis) and volume of (less than or equal to) ≤22.4 mL as the largest tumor.
- Diagnosis:
- Documented recurrent, refractory, or progressive ependymoma or HGG not eligible for resection or no longer receiving standard of care.
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- Phase 2a only: May include patients with recurrent, refractory, or progressive ependymoma or HGG where SOC surgery could be safely delayed four (4) weeks post-infusate.
- Documented histologically confirmed high-grade glioma [following 2021 WHO CNS5 glioma nomenclature, e.g., Anaplastic astrocytoma, Anaplastic pleomorphic xanthoastrocytoma (PXA), Anaplastic ganglioglioma, Anaplastic oligodendroglioma, Glioblastoma, Diffuse midline glioma, H3K27M mutant].
- Karnofsky Performance Status ≥ 60. For subjects <16 years of age, Lansky score ≥ 60.
- Acceptable liver function:
- Bilirubin ≤ 1.5 times the upper limit of normal
- AST (SGOT) and ALT (SGPT) ≤ 3.0 times the upper limit of normal (ULN)
- 6) Acceptable renal function:
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- Serum creatinine ≤1.5xULN
- 7. Acceptable hematologic status (without hematologic support):
- ANC ≥1000 cells/uL
- Platelet count ≥100,000/uL
- Hemoglobin ≥9.0 g/dL
- 8. All subjects of childbearing potential must have a negative serum pregnancy test, and subjects must agree to use effective means of contraception (for example, surgical sterilization or the use of barrier contraception with either a condom or diaphragm in conjunction with spermicidal gel or an IUD) with their partner from entry into the study through 6 months after the last dose.
- 9. Life expectancy of at least 2 months.
- Will consider treatment of subjects up to 25 years of age on a per-patient basis if no other co-morbidities are present that require subspecialty consultation outside of neurosurgical and oncologic care.
Who cannot join
- Spinal disease.
- Infratentorial location of tumor.
- Involvement of the leptomeninges.
- Serious intercurrent illness, as determined by the treating physician, which would compromise either patient safety or study outcomes such as:
- Hypertension (two or more blood pressure readings performed at screening of systolic blood pressure (SBP) or diastolic blood pressure (DBP) above 95th percentile for age) despite optimal treatment.
- Active medically significant infection unresponsive to antibiotics (e.g., non-healing wound, ulcer), uncontrolled systemic infection, or bone fracture.
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- Clinically significant cardiac arrhythmias.
- Untreated hypothyroidism.
- Congestive heart failure.
- Myocarditis.
- Inherited bleeding diathesis or coagulopathy with the risk of bleeding.
- Known active malignancy other than ependymoma or high-grade glioma.
- Any of the following prior anticancer therapy:
- Prior treatment with Bevacizumab or other VEGF agents within 12 months prior to study registration.
- Non-standard radiation therapy such as brachytherapy, systemic radioisotope therapy, or intra-operative radiotherapy (IORT) to the target site at any time prior to study registration.
- Standard radiation therapy within 12 weeks prior to study registration.
- Any systemic therapy within 28 days or 2 half-lives, whichever is longer, prior to study registration (this may include investigational agents, small-molecule kinase inhibitors, non-cytotoxic hormonal therapy, biologic agents, metronomic/protracted low-dose chemotherapy, etc.).
- Nitrosoureas or mitomycin C within 42 days prior to study registration.
- Psychiatric illness/social situations that would limit compliance with the study requirements.
- A tumor located within 1.0cm of a ventricle AND it is determined by the surgeon, PI, and Sponsor to be a risk for drug extravasation to the subarachnoid space if given catheter placement and drug administration.
- A tumor within 1.5cm of critical structures, including the optic chiasm, optic nerves, or brainstem.
- Evidence of acute intracranial or intratumoral hemorrhage either by magnetic resonance imaging (MRI) or computerized tomography (CT) scan (subjects with resolving hemorrhage changes, punctate hemorrhage, or hemosiderin are eligible).
- Treatment with antiepileptic medications must have a two-week history of a stable dose of antiepileptic without seizures prior to study registration.
- Patients with corticosteroid requirements to control cerebral edema must be maintained at a stable or decreasing dose for a minimum of two weeks without progression of clinical symptoms prior to study registration.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Ashley Plant, 1-312-277-4090, aplant@luriechildrens.org
Monica Newmark, MNewmark@luriechildrens.org
About this study
Official title: A Two-Part, Phase 1/2a Trial to Determine the Maximum Tolerated Dose, Safety, and Tolerability of Rhenium (186Re) Obisbemeda (Rhenium-186 NanoLiposome, 186RNL) Delivered Via Convection Enhanced Delivery (CED) in Supratentorial Recurrent, Refractory, or Progressive Pediatric Ependymoma and High-Grade Glioma (HGG)
Pediatric patients 6-21 years of age with supratentorial recurrent, refractory, or progressive pediatric ependymoma and high-grade glioma (HGG) will be included in this study of treatment with Rhenium-186 Nanoliposome (186RNL). Phase 1 of the study will look to determine the maximum tolerated dose (MTD) of 186RNL in this patient population. Phase 2 of the study will use the recommended dose determined in Phase 1 to continue to look at overall response rate and progression-free survival following 186RNL treatment.
- Conditions
- Ependymoma, High Grade Gliomas
- Being tested
- Rhenium-186 Nanoliposome
- Medical subject headings
- Ependymoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rhenium-186 NanoliposomeDrug
Rhenium (186Re) Obisbemeda (Rhenium-186 NanoLiposome, 186RNL), BMEDA-chelated-186rhenium encapsulated within liposomes, allows the 186Re to be directly delivered to the site of the tumor through CED and maintain localization at the site of infusion.
| Study group | Role | What they receive |
|---|---|---|
| Phase 1a - Cohort A | Experimental | Up to six subjects will be enrolled in Cohort A. Tumor size will be limited to a maximum diameter of (less than or equal to) ≤2 cm and a maximum volume of (less than or equal to) ≤4.2 mL. Up to two CED catheters will be utilized in Cohort A at the discretion and consensus of the PI, neurosurgeon, and/or study team. The concentration of 186RNL infusate in Cohort A is 0.5 mCi/mL, with a corresponding estimated absorbed dose of ~87.5Gy. Receives: Drug: Rhenium-186 Nanoliposome |
| Phase 1a - Cohort B | Experimental | Up to six subjects will be enrolled in Cohort B. Tumor size will be limited to a maximum diameter of 3.5 cm in the longest axis and a maximum volume of 22.4 mL. Up to 5 total catheters may be used for Cohort B at the discretion and consensus of the PI, neurosurgeon, and/or study team to ensure total tumor volume coverage. The concentration of 186RNL infusate in Cohort B is 1.0 mCi/mL with a corresponding estimated absorbed dose of ~176Gy (~2x Cohort A). Receives: Drug: Rhenium-186 Nanoliposome |
| Phase 1b | Experimental | Phase 1b continued dosing escalation will be defined following review of the safety data from Phase 1a. Receives: Drug: Rhenium-186 Nanoliposome |
| Phase 2 | Experimental | The Phase 2 expansion study will utilize the maximum tolerable dose (MFD) determined from the Phase 1 dose escalation study. If no maximal tolerable dose is found, tumors may be treated up to an estimated total absorbed dose of 176 Gy. Varying concentrations of 186RNL infusate (0.5-2.5 mCi/mL) may be used to achieve this maximum tolerable dose while maintaining total tumor volume coverage. Phase 2a will enroll an additional 12 patients with a diagnosis of recurrent, refractory, or progressive ependymoma and an additional 20 patients with a diagnosis of recurrent, refractory, or progressive HGG. Receives: Drug: Rhenium-186 Nanoliposome |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (MTD)
Determine the maximum tolerated dose (MTD) of 186RNL administered by convection-enhanced delivery (CED) in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma or HGG.
Measured: 28 days
Overall Response Rate (ORR) by RANO in Ependymoma
Determine the best overall response rate (ORR) by Radiographic Assessment in Neuro-Oncology (RANO) criteria following 186RNL administration in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma.
Measured: 90 days
Progression-Free Suvival at 12 months (PFS12) in HGG
Determine progression-free survival at 12 months (PFS12) in subjects with supratentorial recurrent, refractory, or progressive pediatric HGG.
Measured: 12 months
Secondary outcomes (9)
Safety of 186RNL Dose
Assess the safety of single dose 186RNL administered by CED in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma or HGG, as determined by the most recent version of the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE).
Measured: 28 days
Dose Distribution of 186RNL
Assess the dose distribution of 186RNL administered by CED in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma or HGG.
Measured: 8 days
Neuropsychologic Outcome
Assess the neuropsychologic outcomes of subjects exposed to 186RNL at 1 year in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma or HGG using the following assessments. Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V; ages 6-16:11) OR Wechsler Adult Intelligence Scale, Fourth Edition (WAIS-5; age >17): Vocabulary, Block Design, Symbol Search, Coding, Digit Span
These provide an IQ score on the standard scale with a higher score indicating a better outcome
Measured: 1 year
Progression-Free Survival at 24 Months (PFS24) in Ependymoma
Determine the progression-free survival at 24 months (PFS24) in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma.
Measured: 24 months
Overall Response Rate (ORR) by RANO in HGG
Determine the best overall response rate (ORR) by Radiographic Assessment in Neuro-Oncology (RANO) criteria following 186RNL administration in subjects with supratentorial recurrent, refractory, or progressive pediatric HGG.
Measured: 90 days
Overall Survival at 24 Months (OS24)
Determine overall survival at 24 months (OS24) in participants with supratentorial recurrent, refractory, or progressive pediatric ependymoma and in participants with supratentorial recurrent, refractory, or progressive pediatric HGG.
Measured: 24 months
Neuropsychologic Outcome
Assess the neuropsychologic outcomes of subjects exposed to 186RNL at 1 year in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma or HGG using the California Verbal Learning Test, Children's Version (CVLT-C; ages 6-16:11) OR California Verbal Learning Test, Third Edition (CVLT-3; age >17)
Each sub-test is scored out of 9 with a higher score indicating a better outcome.
Measured: 12 months
Neuropsychologic Outcome
Assess the neuropsychologic outcomes of subjects exposed to 186RNL at 1 year in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma or HGG using the Wide Range Assessment of Memory \& Learning, Third Edition (WRAML3; all ages): Story Memory, Design Learning.
Within each sub category a higher score indicates a better outcome
Measured: 12 months
Neuropsychologic Outcome
Assess the neuropsychologic outcomes of subjects exposed to 186RNL at 1 year in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma or HGG using the Beery-Buktenica Developmental Test of Visual-Motor Integration (Beery VMI; all ages).
Within each sub category a higher score indicates a better outcome
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2025
- Study start1 Aug 2026
- Primary completionMar 2028 (estimated)
- Study completionMar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for EpendymomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 56 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cerenome
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ann & Robert H Lurie Children's Hospital of Chicago
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07061626
- Sponsor's protocol ID
- CA-2024-PBC-001
- Other IDs
- HT9425-24-1-1035
- Status last verified
- Sep 2026
- First submitted
- 3 May 2025
- Last updated
- 11 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Two-Part, Phase 1/2a Trial to Determine the Maximum Tolerated Dose, Safety, and Tolerability of Rhenium (186Re) Obisbemeda (Rhenium-186 NanoLiposome, 186RNL) Delivered Via Convection Enhanced Delivery (CED) in Supratentorial Recurrent, Refractory, or Progressive Pediatric Ependymoma and High-Grade Glioma (HGG)
- ClinicalTrials.gov ID
- NCT07061626
- Acronym / sponsor ID
- CA-2024-PBC-001
- Conditions
- Ependymoma, High Grade Gliomas
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 56 participants (estimated)
- Lead sponsor
- CerenomeIndustry (pharma, biotech, devices)
- Collaborators
- Ann & Robert H Lurie Children's Hospital of Chicago
- Study start
- 1 Aug 2026 (actual)
- Primary completion
- Mar 2028 (estimated)
- Study completion
- Mar 2028 (estimated)
- First posted
- 11 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Pediatric patients 6-21 years of age with supratentorial recurrent, refractory, or progressive pediatric ependymoma and high-grade glioma (HGG) will be included in this study of treatment with Rhenium-186 Nanoliposome (186RNL). Phase 1 of the study will look to determine the maximum tolerated dose (MTD) of 186RNL in this patient population. Phase 2 of the study will use the recommended dose determined in Phase 1 to continue to look at overall response rate and progression-free survival following 186RNL treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Phase 1a - Cohort A | Experimental | Drug: Rhenium-186 NanoliposomeUp to six subjects will be enrolled in Cohort A. Tumor size will be limited to a maximum diameter of (less than or equal to) ≤2 cm and a maximum volume of (less than or equal to) ≤4.2 mL. Up to two CED catheters will be utilized in Cohort A at the discretion and consensus of the PI, neurosurgeon, and/or study team. The concentration of 186RNL infusate in Cohort A is 0.5 mCi/mL, with a corresponding estimated absorbed dose of ~87.5Gy. |
| Phase 1a - Cohort B | Experimental | Drug: Rhenium-186 NanoliposomeUp to six subjects will be enrolled in Cohort B. Tumor size will be limited to a maximum diameter of 3.5 cm in the longest axis and a maximum volume of 22.4 mL. Up to 5 total catheters may be used for Cohort B at the discretion and consensus of the PI, neurosurgeon, and/or study team to ensure total tumor volume coverage. The concentration of 186RNL infusate in Cohort B is 1.0 mCi/mL with a corresponding estimated absorbed dose of ~176Gy (~2x Cohort A). |
| Phase 1b | Experimental | Drug: Rhenium-186 NanoliposomePhase 1b continued dosing escalation will be defined following review of the safety data from Phase 1a. |
| Phase 2 | Experimental | Drug: Rhenium-186 NanoliposomeThe Phase 2 expansion study will utilize the maximum tolerable dose (MFD) determined from the Phase 1 dose escalation study. If no maximal tolerable dose is found, tumors may be treated up to an estimated total absorbed dose of 176 Gy. Varying concentrations of 186RNL infusate (0.5-2.5 mCi/mL) may be used to achieve this maximum tolerable dose while maintaining total tumor volume coverage. Phase 2a will enroll an additional 12 patients with a diagnosis of recurrent, refractory, or progressive ependymoma and an additional 20 patients with a diagnosis of recurrent, refractory, or progressive HGG. |
Who may qualify
- Ages
- 6 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (22 listed)
- 6 years to 21 years* of age.
- Lesion number and size:
- Diagnosis:
- Karnofsky Performance Status ≥ 60. For subjects <16 years of age, Lansky score ≥ 60.
- Acceptable liver function:
Key exclusion criteria (24 listed)
- Spinal disease.
- Infratentorial location of tumor.
- Involvement of the leptomeninges.
- Serious intercurrent illness, as determined by the treating physician, which would compromise either patient safety or study outcomes such as:
- Any of the following prior anticancer therapy:
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 |
|---|---|---|
| Rhenium-186 NanoliposomeRhenium (186Re) Obisbemeda (Rhenium-186 NanoLiposome, 186RNL), BMEDA-chelated-186rhenium encapsulated within liposomes, allows the 186Re to be directly delivered to the site of the tumor through CED and maintain localization at the site of infusion. | Drug | Phase 1a - Cohort A, Phase 1a - Cohort B, Phase 1b, Phase 2 |
Primary outcome
Maximum Tolerated Dose (MTD)
Time frame: 28 days
Determine the maximum tolerated dose (MTD) of 186RNL administered by convection-enhanced delivery (CED) in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma or HGG.
Overall Response Rate (ORR) by RANO in Ependymoma
Time frame: 90 days
Determine the best overall response rate (ORR) by Radiographic Assessment in Neuro-Oncology (RANO) criteria following 186RNL administration in subjects with supratentorial recurrent, refractory, or progressive pediatric ependymoma.
Progression-Free Suvival at 12 months (PFS12) in HGG
Time frame: 12 months
Determine progression-free survival at 12 months (PFS12) in subjects with supratentorial recurrent, refractory, or progressive pediatric HGG.
9 secondary outcomes
Safety of 186RNL Dose
Time frame: 28 days
Dose Distribution of 186RNL
Time frame: 8 days
Neuropsychologic Outcome
Time frame: 1 year
Progression-Free Survival at 24 Months (PFS24) in Ependymoma
Time frame: 24 months
Overall Response Rate (ORR) by RANO in HGG
Time frame: 90 days
Overall Survival at 24 Months (OS24)
Time frame: 24 months
Neuropsychologic Outcome
Time frame: 12 months
Neuropsychologic Outcome
Time frame: 12 months
Neuropsychologic Outcome
Time frame: 12 months
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 11 Sep 2026.
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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.
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Rhenium-186 Nanoliposome in people with Ependymoma and High Grade Gliomas, run by Cerenome at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 22 inclusion and 24 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 11 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Tolerated Dose (MTD) (time frame: 28 days). The study lists 3 primary outcomes in all. 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 11 Sep 2026.