Posterior Fossa Ependymoma (PFEPN)Phase 1NCT02774421
Pilot Study of the Effect of Trastuzumab and GM-CSF on Children With Recurrent Ependymoma
A Phase 1 study testing Trastuzumab after SubQ GM-CSF and Trastuzumab in combination with SubQ GM-CSF in people with Posterior Fossa Ependymoma (PFEPN), run by University of Colorado, Denver at 1 site in United States.
Trial summary
This study plans to detect the presence of trastuzumab by mass spectroscopy in relapsed posterior fossa ependymoma (PFEPN) tumor specimen pre-treated with a single dose of intrathecal (IT) trastuzumab, as well as to evaluate toxicity of intrathecal trastuzumab in combination with subcutaneous (subQ)… Read more about this study
- Who is it for?
- Ages 12 months to 21 years
- Conditions the study is for
- Posterior Fossa Ependymoma (PFEPN)
- What is being tested?
- Trastuzumab after SubQ GM-CSF and Trastuzumab in combination with SubQ GM-CSF
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Jun 2017to 31 Mar 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 12 months to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age ≥ 12 months and < 21 years at the time of study enrollment
- Patients must be diagnosed with relapse of previously histologically confirmed PFEPN
- Magnetic resonance (MR) imaging of the brain (performed within 14 days of enrollment) must demonstrate no evidence of diffuse leptomeningeal spread beyond the primary relapse site in posterior fossa and no obstruction of cerebrospinal fluid flow (CSF).
- MR imaging of the total spine (performed within 14 days of enrollment) demonstrates no evidence of spinal metastatic disease.
- Patients must have clinical indication for standard of care surgical resection of relapsed PFEPN tumor for enrollment in Stratum 1
- Patients must meet one of the following performance scores:
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- Eastern Cooperative Oncology Group (ECOG) performance status scores of 0, 1, or 2;
- Karnofsky score of ≥ 50 for patients > 16 years of age; or
- Lansky score of ≥ 50 for patients ≤ 16 years of age.
- Informed Consent: All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.
- Organ Function Requirements:
- Adequate Renal Function defined as:
- 1) Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥70ml/min/1.73 m2 or 2) A serum creatinine based on age/gender as follows: Maximum Serum Creatinine (mg/dL) Age: Male Female
- 1 month to < 6 months 0.4 0.4 6 months to < 1 year 0.5 0.5
- 1 to < 2 years 0.6 0.6 2 to < 6 years 0.8 0.8 6 to < 10 years 1 1 10 to < 13 years 1.2 1.2 13 to < 16 years 1.5 1.4
- ≥ 16 years 1.7 1.4
- Adequate Liver Function defined as:
- Total bilirubin ≤1.5 x upper limit of normal (ULN) for age, and
- Serum glutamic oxaloacetic transaminase (SGOT) (AST) or serum glutamate pyruvate transaminase (SGPT) (ALT) < 3 x upper limit of normal (ULN) for age
- Adequate Bone Marrow Function defined as:
- Peripheral absolute neutrophil count (ANC) ≥1,000/µL
- Platelet count ≥100,000/µL (transfusion independent)
- Adequate Cardiac Function defined as:
- Shortening fraction > 28% by echocardiogram or
- Ejection fraction > 50% by echocardiogram or radionuclide study
Who cannot join
- Patients with a diagnosis of:
- spinal cord ependymoma,
- myxopapillary ependymoma,
- subependymoma,
- ependymoblastoma,
- supratentorial ependymoma, or
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- mixed glioma are NOT eligible for either Stratum.
- Patients with evidence of nodular metastatic spinal disease by MRI are NOT eligible for Stratum 1 but may be eligible for STRATUM 2 if CSF flow is not obstructed based on appropriate imaging studies, and the patient is deemed safe for lumbar puncture by the medical team.
- Patients with clinical contraindications against lumbar puncture are NOT eligible for either Stratum.
- Prior Therapy:
- Radiation therapy: At least 28 days must have elapsed (at time of starting protocol therapy) since completion of focal radiation therapy for current recurrence for Stratum 2; Patients who have already undergone radiation therapy for current recurrence are NOT eligible for Stratum 1.
- Myelosuppressive chemotherapy: At least 21 days must have elapsed after the last dose of myelosuppressive chemotherapy; Patients who have been treated with chemotherapy at time of recurrence are NOT eligible for either Stratum.
- Monoclonal antibodies: At least three (3) half-lives of the antibody must have elapsed since the last dose of a monoclonal antibody (see Appendix I for list of half-lives)
- Surgical resection: Must have fully healed from any surgical procedure to be safe for lumbar puncture according to treating neurosurgeon and at least 14 days should have elapsed since the surgical procedure.
- Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are NOT eligible for either Stratum.
- Concomitant Medications
- Corticosteroids: Patients receiving systemic corticosteroids are NOT eligible for either Stratum.
- Investigational Drugs: Patients who are currently receiving another investigational drug are NOT eligible for either Stratum.
- Anti-cancer Agents: Patients who are currently receiving other anti-cancer agents are NOT eligible for either Stratum.
- Pregnancy, Breast-Feeding, and Contraception
- Pregnant or breast-feeding women are NOT eligible. Pregnancy tests must be obtained in females who are post-menarchal.
- Women of childbearing potential and male participants with partners of childbearing potential must agree to:
- use a "highly effective," non-hormonal form of contraception (including abstinence), or
- two "effective" forms of non-hormonal contraception by the patient and/or partner, and
- Contraception must continue for the duration of study treatment and for at least seven (7) months after the last dose of study treatment.
- Patients who have an uncontrolled serious infection are NOT eligible for either Stratum.
- Patients who have previously received solid organ transplantation are NOT eligible for either Stratum.
- Patients who have a history of:
- significant cardiac disease,
- cardiac disease risk factors, or
- uncontrolled arrhythmias are NOT eligible for either Stratum.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital Colorado
Aurora, Colorado, 80045
About this study
This study plans to detect the presence of trastuzumab by mass spectroscopy in relapsed posterior fossa ependymoma (PFEPN) tumor specimen pre-treated with a single dose of intrathecal (IT) trastuzumab, as well as to evaluate toxicity of intrathecal trastuzumab in combination with subcutaneous (subQ) Granulocyte-macrophage colony-stimulating factor (GM-CSF) in children with relapsed PFEPN
- Conditions
- Posterior Fossa Ependymoma (PFEPN)
- Being tested
- Trastuzumab after SubQ GM-CSF, Trastuzumab in combination with SubQ GM-CSF
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Trastuzumab after SubQ GM-CSFDrug
Administration of trastuzumab following subcutaneous granulocyte-macrophage colony-stimulating factor (GM-CSF). Trastuzumab is a monoclonal antibody targeting HER2-positive cancers, commonly used in breast and gastric cancer treatment.
Trastuzumab in combination with SubQ GM-CSFDrug
Combine trastuzumab, a monoclonal antibody targeting HER2-positive cancers, with subcutaneous granulocyte-macrophage colony-stimulating factor (GM-CSF). GM-CSF functions as an immune system modulator, promoting the activation and proliferation of dendritic cells, macrophages, and granulocytes.
| Study group | Role | What they receive |
|---|---|---|
| IT trastuzumab after subQ GM-CSF | Experimental | Patients with localized recurrent PFEPN will be treated with IT trastuzumab following 5-day course of subQ GM-CSF. Surgical resection should be planned (based on institutional surgical scheduling standards) for ideally 2-7 days after IT trastuzumab dosage. Receives: Drug: Trastuzumab after SubQ GM-CSF |
| IT trastuzumab in combination with subQ GM-CSF | Experimental | Patients with localized recurrent PFEPN will be treated with IT trastuzumab in combination with GM-CSF to establish a maximum tolerated dosage. GM-CSF will be administered at 250 mcg/m2/dose subQ daily for three (3) days prior to the IT trastuzumab dose. Receives: Drug: Trastuzumab in combination with SubQ GM-CSF |
What is being measured
The primary outcome is the main question the study is designed to answer.
Stratum 1: Detection of trastuzumab in tumor following IT administration
The presence of trastuzumab will be measured as a binary (yes/no) outcome.
Measured: 2 weeks
Stratum 2: Determine the Maximum Tolerated Dose (MTD) for IT trastuzumab in combination with fixed doses of subQ GM-CSF
The estimated MTD would be the highest dose at which 0/3 or 1/6 subjects experience a Dose Limiting Toxicity (DLT).
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2016 (estimated)
- Study start12 Jun 2017
- Primary completion27 Feb 2025
- Study completion31 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Posterior Fossa Ependymoma (PFEPN)Studies of the same condition, open and under way.
- More trials run by University of Colorado, DenverThe 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Colorado, Denver
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Hospital Colorado
- Responsible party
- Sponsor
- Investigators
- Margaret Macy, MD, Principal investigator, University of Colorado, Denver
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02774421
- Sponsor's protocol ID
- 14-1076.cc
- Status last verified
- Aug 2026
- First submitted
- 6 May 2016
- Last updated
- 31 Aug 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 31 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of the Effect of Trastuzumab and GM-CSF on Children With Recurrent Ependymoma
- ClinicalTrials.gov ID
- NCT02774421
- Acronym / sponsor ID
- 14-1076.cc
- Conditions
- Posterior Fossa Ependymoma (PFEPN)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University of Colorado, DenverUniversity, hospital or other
- Collaborators
- Children's Hospital Colorado
- Study start
- 12 Jun 2017 (actual)
- Primary completion
- 27 Feb 2025 (actual)
- Study completion
- 31 Mar 2026 (actual)
- First posted
- 17 May 2016 (estimated)
- 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.
This study plans to detect the presence of trastuzumab by mass spectroscopy in relapsed posterior fossa ependymoma (PFEPN) tumor specimen pre-treated with a single dose of intrathecal (IT) trastuzumab, as well as to evaluate toxicity of intrathecal trastuzumab in combination with subcutaneous (subQ) Granulocyte-macrophage colony-stimulating factor (GM-CSF) in children with relapsed PFEPN
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| IT trastuzumab after subQ GM-CSF | Experimental | Drug: Trastuzumab after SubQ GM-CSFPatients with localized recurrent PFEPN will be treated with IT trastuzumab following 5-day course of subQ GM-CSF. Surgical resection should be planned (based on institutional surgical scheduling standards) for ideally 2-7 days after IT trastuzumab dosage. |
| IT trastuzumab in combination with subQ GM-CSF | Experimental | Drug: Trastuzumab in combination with SubQ GM-CSFPatients with localized recurrent PFEPN will be treated with IT trastuzumab in combination with GM-CSF to establish a maximum tolerated dosage. GM-CSF will be administered at 250 mcg/m2/dose subQ daily for three (3) days prior to the IT trastuzumab dose. |
Who may qualify
- Ages
- 12 months to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (25 listed)
- Age ≥ 12 months and < 21 years at the time of study enrollment
- Patients must be diagnosed with relapse of previously histologically confirmed PFEPN
- Magnetic resonance (MR) imaging of the brain (performed within 14 days of enrollment) must demonstrate no evidence of diffuse leptomeningeal spread beyond the primary relapse site in posterior fossa and no obstruction of cerebrospinal fluid flow (CSF).
- MR imaging of the total spine (performed within 14 days of enrollment) demonstrates no evidence of spinal metastatic disease.
- Patients must have clinical indication for standard of care surgical resection of relapsed PFEPN tumor for enrollment in Stratum 1
Key exclusion criteria (31 listed)
- Patients with a diagnosis of:
- Patients with evidence of nodular metastatic spinal disease by MRI are NOT eligible for Stratum 1 but may be eligible for STRATUM 2 if CSF flow is not obstructed based on appropriate imaging studies, and the patient is deemed safe for lumbar puncture by the medical team.
- Patients with clinical contraindications against lumbar puncture are NOT eligible for either Stratum.
- Prior Therapy:
- Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are NOT eligible for either Stratum.
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 |
|---|---|---|
| Trastuzumab after SubQ GM-CSFAdministration of trastuzumab following subcutaneous granulocyte-macrophage colony-stimulating factor (GM-CSF). Trastuzumab is a monoclonal antibody targeting HER2-positive cancers, commonly used in breast and gastric cancer treatment. | Drug | IT trastuzumab after subQ GM-CSF |
| Trastuzumab in combination with SubQ GM-CSFCombine trastuzumab, a monoclonal antibody targeting HER2-positive cancers, with subcutaneous granulocyte-macrophage colony-stimulating factor (GM-CSF). GM-CSF functions as an immune system modulator, promoting the activation and proliferation of dendritic cells, macrophages, and granulocytes. | Drug | IT trastuzumab in combination with subQ GM-CSF |
Primary outcome
Stratum 1: Detection of trastuzumab in tumor following IT administration
Time frame: 2 weeks
The presence of trastuzumab will be measured as a binary (yes/no) outcome.
Stratum 2: Determine the Maximum Tolerated Dose (MTD) for IT trastuzumab in combination with fixed doses of subQ GM-CSF
Time frame: 1 month
The estimated MTD would be the highest dose at which 0/3 or 1/6 subjects experience a Dose Limiting Toxicity (DLT).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2026.
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.
Margaret Macy, MD principal investigator
University of Colorado, Denver
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 Trastuzumab after SubQ GM-CSF and Trastuzumab in combination with SubQ GM-CSF in people with Posterior Fossa Ependymoma (PFEPN), run by University of Colorado, Denver 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 25 inclusion and 31 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: finished, as of the sponsor's last update on 31 Aug 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Stratum 1: Detection of trastuzumab in tumor following IT administration (time frame: 2 weeks). The study lists 2 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 31 Aug 2026.