NCT02774421

Pilot Study of the Effect of Trastuzumab and GM-CSF on Children With Recurrent Ependymoma

Active, enrolled Phase 1 Last updated 20 April 2026

What this trial tests

Phase 1 trial testing Trastuzumab after SubQ GM-CSF in Posterior Fossa Ependymoma (PFEPN) in 33 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

12 June 2017

Primary endpoint
27 February 2025

1 March 2027

Quick facts

Lead sponsor	University of Colorado, Denver
Phase	Phase 1
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	33
Start date	12 June 2017
Primary completion	27 February 2025
Estimated completion	1 March 2027
Sites	1 location across United States

Drugs / interventions tested

• Trastuzumab after SubQ GM-CSF — full drug profile →
• Trastuzumab in combination with SubQ GM-CSF — full drug profile →

Conditions studied

• Posterior Fossa Ependymoma (PFEPN) — all drugs for Posterior Fossa Ependymoma (PFEPN) →

Sponsor

University of Colorado, Denver

Who can join

Adults 12 Months to 21, any sex, with Posterior Fossa Ependymoma (PFEPN). Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• 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).

Sponsor's own description

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

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. Ependymoma: Evaluation and Management Updates.
   Rudà R, Bruno F, Pellerino A, Soffietti R. · · 2022 · cited 84× · PMID 35384591 · DOI 10.1007/s11912-022-01260-w
2. Trial Watch: Immunostimulation with recombinant cytokines for cancer therapy.
   García-Martínez E, Smith M, Buqué A, Aranda F, et al · · 2018 · cited 38× · PMID 29872569 · DOI 10.1080/2162402x.2018.1433982
3. Reevaluating surgery and re-irradiation for locally recurrent pediatric ependymoma-a multi-institutional study.
   Mak DY, Laperriere N, Ramaswamy V, Bouffet E, et al · · 2021 · cited 7× · PMID 34988448 · DOI 10.1093/noajnl/vdab158
4. Targeted and systemic therapies for recurrent adult ependymomas: real-world outcomes from a single institution and concise literature review.
   Maccari M, Bosio A, Caccese M, Padovan M, et al · · 2026 · PMID 41788986 · DOI 10.3389/fonc.2026.1748353