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CompletedNCT04958486Updated Mar 3, 2025

Combination Intraventricular Chemotherapy Pilot Study: 5-Azacytidine (5-AZA) and Trastuzumab Infusions Into the Fourth Ventricle or Resection Cavity in Children and Adults with Recurrent or Residual Posterior Fossa Ependymoma

An Early Phase 1 interventional study of 5-Azacytidine and trastuzumab infusion in Fossa Ependymoma, sponsored by The University of Texas Health Science Center, Houston. Completed at 1 site in United States. Open to participants aged 1 Year to 80 Years. Per ClinicalTrials.gov, last updated 2025-03-03.

Sponsored by The University of Texas Health Science Center, Houston · Early Phase 1, Interventional, and Treatment

From the registry’s dates

  • Primary completion was Sep 2023, 3 years ago, and no results have been posted to the registry.
Phase
Early Phase 1
Study type
Interventional
Enrollment
4
Allocation
Not applicable
Ages
1 Year to 80 Years
Sex
All
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Study summary

The purpose of this study is to establish the safety and tolerability of simultaneous infusions of 5-Azacytidine and trastuzumab into the fourth ventricle of the brain or resection cavity in patients with recurrent posterior fossa ependymoma and to assess the antitumor activity of simultaneous infusions of 5-Azacytidine and trastuzumab into the fourth ventricle of the brain or resection cavity in patients based upon imaging studies and lumbar cerebrospinal fluid (CSF) cytology.

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Conditions studied

  • Fossa Ependymoma

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Keywords

  • 5-Azacytidine
  • Trastuzumab
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In context

Ependymoma

180 studies on the registry are indexed under Ependymoma; 35 are open to participants now.

This study's enrollment of 4 is below the median of 30 across 149 interventional studies indexed under Ependymoma.

Browse Ependymoma studies →

Lead sponsor

The University of Texas Health Science Center, Houston is the lead sponsor of 880 studies on the registry; 209 are open to participants now.

Of its 170 completed or terminated interventional studies of FDA-regulated products, 140 (82%) have results posted.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
1 Year to 80 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Patients with histologically verified ependymoma, with recurrence or progression anywhere in the brain and/or spine. Patients are also eligible if they have refractory disease, which will be defined as residual tumor which has not been completely cleared despite prior treatments. To be eligible, patients' disease must have originated in the posterior fossa of the brain
  • Patient must have either measurable or evaluable tumor as assessed by MRI of the brain and total spine
  • An implanted catheter in the fourth ventricle or posterior fossa tumor cavity attached to a ventricular access device or agreement to have one placed.
  • A minimum of 4 weeks between any prior radiation treatments or bevacizumab infusions and first infusion of 5-azacytidine or trastuzumab infusions. A minimum of 2 weeks between last dose of any other systemic chemotherapy and first infusion of 5-azacytidine or Trastuzumab into fourth ventricle
  • Life expectancy of at least 12 weeks in the opinion of the principal investigator
  • Lansky score of 50 or greater if ≤16 years of age or Karnofsky score of 50 or greater if > 16 years of age
  • Existing neurological deficits must have been stable for a minimum of 1 week prior to study enrollment
  • Patients must have recovered from the acute toxic effects of all prior anticancer chemotherapy
  • Adequate bone marrow function defined by peripheral absolute neutrophil count (ANC) ≥ 500/µL, platelet count ≥ 50,000/µL (transfusion independent), and hemoglobin ≥ 9.0 gm/dL (may receive red blood cell (RBC) transfusions)
  • Patient or patient's legal representative, parent(s), or guardian able to provide written informed consent.
  • Patient with abnormal cardiac function (Shortening fraction less than 28% on echocardiogram) will need cardiology clearance prior to enrollment
  • Normal renal and liver function on basic metabolic panel. Any patients with abnormal blood urea nitrogen (BUN), creatinine, alanine aminotransferase(AST) or aspartate aminotransferase (ALT) levels will need nephrology and/or gastroenterology clearance prior to enrollment

Exclusion criteria

Exclusion Criteria:

  • Enrolled in another treatment protocol
  • Patients with disease that is completely resectable
  • Has received another investigational or chemotherapy agent within 2 weeks or radiation therapy within 4 weeks prior to 5-azacytidine or trastuzumab infusion into the fourth ventricle
  • Patients with any cardiac issues who are not cleared by cardiology for participation in the study
  • Evidence of untreated infection
  • Pregnant or lactating women
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Study design

Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
4 participants (actual)

Study arms

  • Experimental
    Treatment

    Drug: 5-Azacytidine and trastuzumab infusion

Interventions

  • Drug5-Azacytidine and trastuzumab infusion

    Patients will receive once weekly 10 mg intraventricular 5-Azacytidine infusions for six consecutive weeks followed by observation in the infusion suite for a minimum of 30 minutes and once weekly 21 mg intraventricular trastuzumab infusions for six consecutive weeks followed by observation for a minimum of 2 hour after each infusion for the first 2 infusions. During this monitoring period, temperature, blood pressure, heart rate, and oxygen saturation will be measured. Patients will also have a neurological examination performed to observe for neurological changes. All patients will undergo an MRI of the brain and total spine with and without gadolinium within 7 days after the final 5-AZA and trastuzumab infusion to determine treatment response and to assess for any signal changes in the brain or spine caused by the infusions and they will have a 30 day and a 90 day follow-up assessment by telephone or in person for assessment of outcome measures and safety

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What researchers measure

Primary outcomes

  1. Number of patients with new neurological deficits

    A new neurological deficit will be defined as new cranial neuropathy, change in level of consciousness, motor weakness, gait change or cerebellar finding (ataxia, dysmetria, dysdiadochokinesis) that is attributed by treating physicians to infusions. New neurological deficits graded as Grade 3 or higher after infusions will be defined as serious adverse events (SAE's)

    Time frame: 7 days after treatment

Secondary outcomes

  1. Change in disease progression as assessed by magnetic resonance imaging (MRI)

    Time frame: Baseline(before start of treatment),end of treatment (7 days after treatment)

  2. Change in disease progression as assessed by lumbar CSF cytology

    Time frame: Baseline(before start of treatment),end of treatment (7 days after treatment)

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Study locations

1 site
  • The University of Texas Health Science Center at Houston
    Houston, Texas 77030, United States
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References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Mar 3, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT04958486
Lead sponsor
The University of Texas Health Science Center, Houston
Responsible party
David Ilan Sandberg (Professor, The University of Texas Health Science Center, Houston) — Principal investigator
First posted
Jul 12, 2021
Start date
Jul 8, 2021
Primary completion
Sep 29, 2023
Completion
Sep 29, 2023
Last update
Mar 3, 2025

Study contacts

David I Sandberg, MD
principal investigator · The University of Texas Health Science Center, Houston

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Feb 2025. You cannot join it, but the record below documents what was studied.

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