AzTrials
AzTrials

Ependymoma, Recurrent ChildhoodEarly Phase 1NCT04408092

Study of the Effect of GM-CSF on Macrophages in Ependymoma

A Early Phase 1 study testing Granulocyte Macrophage Colony Stimulation Factor in people with Ependymoma, Recurrent Childhood and Ependymoma, run by University of Colorado, Denver at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This study plans to learn more about the use of Granulocyte Macrophage Colony Stimulation Factor (GM-CSF) on ependymoma tumors. The use of GM-CSF is a potential way of increasing the infiltration of immune cells and this study is looking at whether or not this will improve the outcome of patients with an ependymoma Read more about this study

Who is it for?
Ages 12 months to 21 years
Conditions the study is for
Ependymoma, Recurrent Childhood, Ependymoma
What is being tested?
Granulocyte Macrophage Colony Stimulation Factor
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
2 sitesUnited States
Study dates
25 Jun 2013to 21 Jul 2023
Phase
Early 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 one of the following:
  • Newly diagnosed with posterior fossa ependymoma with a subtotal resection at initial surgery. These patients will be eligible for stratum 1.
  • Be in first relapse of their posterior fossa ependymoma. These patients will be eligible for stratum 2.
  • Histologically confirmed diagnosis of intracranial ependymoma .
  • Pre or post-operative MR imaging of the brain demonstrates no evidence of non-contiguous spread beyond the primary site
Show all 27Show fewer
  • Pre or post-operative MR imaging of the spine demonstrates no evidence of non-contiguous spread beyond the primary site
  • Pre-operative CSF cytology obtained from the lumbar CSF space demonstrated no evidence of non-contiguous spread beyond the primary site.
  • The requirement for lumbar CSF examination may be waived if deemed to be medically contraindicated.
  • Patients must meet one of the following performance scores.
  • 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
  • Organ Function Requirements:
  • Adequate renal function defined as:
  • Creatinine clearance or radioisotope GFR ³ 70ml/min/1.73 m2 or
  • A serum creatinine based on age/gender as follows:
  • Age Maximum Serum Creatinine (mg/dL) 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 The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR (Schwartz et al. J. Peds, 106:522, 1985) utilizing child length and stature data published by the CDC.
  • Adequate liver function defined as:
  • Total bilirubin =< 1.5 x upper limit of normal (ULN) for age, and
  • SGOT (AST) or SGPT (ALT) < 3 x upper limit of normal (ULN) for age.
  • Patients with Gilbert syndrome or hemolytic anemia are eligible if total bilirubin is < 3 x upper limit of normal (ULN) for age.
  • Adequate Bone Marrow Function defined as:
  • Peripheral absolute neutrophil count (ANC) >= 1,000/uL
  • Platelet count >= 100,000/uL (transfusion independent).

Who cannot join

  • Patients with a diagnosis of spinal cord ependymoma, myxopapillary ependymoma, subependymoma, ependymoblastoma, supratentorial ependymoma, or mixed glioma are NOT eligible.
  • Patients with evidence of metastatic disease by MRI or CSF cytology are NOT eligible

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • Children's Hospital Colorado

    Denver, Colorado, 80045

  • Arnold Palmer Hospital for Children

    Orlando, Florida, 32806

Other trials in United States

About this study

Official title: Pilot Study of the Effect of GM-CSF on Macrophages in Incompletely Resected or Recurrent Ependymoma

This study plans to learn more about the use of Granulocyte Macrophage Colony Stimulation Factor (GM-CSF) on ependymoma tumors. The use of GM-CSF is a potential way of increasing the infiltration of immune cells and this study is looking at whether or not this will improve the outcome of patients with an ependymoma

Read the detailed description

To study whether Granulocyte Macrophage Colony Stimulation Factor (GM-CSF) increases macrophage infiltration in children with ependymoma (EPN) who are to have planned surgery as a standard procedure for incomplete resection or recurrent tumor. To correlate the extent of macrophage infiltration with other immune markers of the tumor at subsequent surgery with outcome. This is intended as a pilot protocol with the potential to be incorporated in the next COG (Children's Oncology Group) national ependymoma studies.

Recombinant Granulocyte Macrophage Colony Stimulation Factor (rGM-CSF) is a hematopoietic growth factor which supports survival, clonal expansion, and differentiation of hematopoietic progenitor cells. rGM-CSF induces partially committed progenitor cells to divide and differentiate in the granulocyte-macrophage pathways. rGM-CSF stimulates the production of monocytes, granulocytes, erythrocytes, and sometimes, megakaryocytes in the bone marrow. It also induces mature macrophages to increase phagocytosis, superoxide generation, Antibody Dependent Cell-mediated Cytotoxicity (ADCC), tumoricidal killing and cytokine production (IL-1 and tumor necrosis factor).

Registration of all participants must occur before any study-related procedures. Staff will be available to register participants Monday thru Friday, from 8:00 AM to 5:00 PM Mountain Standard Time.

Being tested
Granulocyte Macrophage Colony Stimulation Factor
Keywords
granulocyte macrophage colony stimulation factor, Incomplete resection
Medical subject headings
Ependymoma

What participants receive

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

  • Granulocyte Macrophage Colony Stimulation FactorDrug

    Also called GM-CSF

    Recombinant Granulocyte Macrophage Colony Stimulation Factor (rGM-CSF) is a hematopoietic growth factor which supports survival, clonal expansion, and differentiation of hematopoietic progenitor cells. rGM-CSF induces partially committed progenitor cells to divide and differentiate in the granulocyte-macrophage pathways. rGM-CSF stimulates the production of monocytes, granulocytes, erythrocytes, and sometimes, megakaryocytes in the bone marrow. It also induces mature macrophages to increase phagocytosis, superoxide generation, Antibody Dependent Cell-mediated Cytotoxicity (ADCC), tumoricidal killing and cytokine production (IL-1 and tumor necrosis factor). GM-CSF was used in the first successful phase III trial of immunotherapy in the pediatric COG protocol, ANBL0931, a national study in high risk neuroblastoma.

Study groups in NCT04408092
Study groupRoleWhat they receive
GM-CSF treatment at second-look surgery armExperimentalNewly diagnosed patients with EPN who have a subtotal resection at initial presentation and are without evidence of metastatic tumor will be enrolled in this stratum. Total patient population in this stratum will be 10 patients. It should be noted that prior experience suggests that about 1/3 of newly presenting patients still have residual tumor after the initial surgery
Receives: Drug: Granulocyte Macrophage Colony Stimulation Factor
GM-CSF treatment at recurrence arm.ExperimentalEPN patients with a first regional relapse and without evidence of metastatic tumor will be enrolled in this stratum. Total patient population will be 10 patients. Patients with a first recurrence will have the recurrence confirmed by the local institutional neuro-radiologists. They will have the entire neuro-axis scanned and a spinal tap performed (where safe) to exclude metastatic tumor. They will then receive 5 days of GM-CSF and then proceed to surgery if deemed clinically indicated by the treating physician
Receives: Drug: Granulocyte Macrophage Colony Stimulation Factor

What is being measured

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

  1. Number of participants with increased M/M infiltration compared to institutional and ACNS0121 controls

    Outcome measure is met if cell count is >50 AIF1+ microglia per high power field (40x)

    Measured: 8 years

Timeline

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

  1. First posted29 May 2020
  2. Study start25 Jun 2013
  3. Primary completion21 Sep 2021
  4. Study completion21 Jul 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Model description
standard treatment of EPN includes repeat surgeries at (1) second-look surgery to remove residual tumor after first-look surgery at initial diagnosis, and (2) optimal debulking of recurrent tumor, providing a rare opportunity to measure the effect of experimental therapy directly in children's brain tumors. Ten patients each will be entered into the second-look and recurrence strata of this study.
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04408092
Sponsor's protocol ID
13-0133.cc
Other IDs
P30CA046934
Status last verified
Mar 2024
First submitted
10 Dec 2019
Last updated
15 Mar 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing Granulocyte Macrophage Colony Stimulation Factor in people with Ependymoma, Recurrent Childhood and Ependymoma, run by University of Colorado, Denver at 2 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 27 inclusion and 2 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 15 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 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?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

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?

Number of participants with increased M/M infiltration compared to institutional and ACNS0121 controls (time frame: 8 years). 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 NCT04408092.

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 15 Mar 2024.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in