Experimental Artery-Directed chemo for rare brain tumors: tiny trial ends early
NCT ID NCT04994977
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tested giving chemotherapy drugs directly into the brain's arteries to treat two rare types of choroid plexus tumors. The goal was to shrink the tumor before a second surgery. Only 1 person was enrolled, and the study was terminated, so we have very little information about safety or effectiveness.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Melphalan, carboplatin, and topotecan (chemotherapy drugs given directly into an artery)
- What this could lead to
- If it works, this approach could shrink these rare brain tumors before surgery, making the operation safer and more effective.
- What could go wrong
- This was a very early Phase 1 trial with only 1 participant, so we don't know if it's safe or effective. The study was terminated, meaning results are limited.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
-
1 person
The number who actually took part.
- Started
-
May 2023
- Finished
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Dec 2025
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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0 to 100 years
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Subjects with a histologically confirmed diagnosis of ACPP or CPC that is newly diagnosed, residual or recurrent. 2. Subjects must have a Karnofsky or Lansky Performance Score ≥ 60 % assessed within two weeks prior to enrollment. Karnofsky is used for patients ≥ 16 years and Lansky for those \< 16. 3. Subjects must have normal organ and marrow function documented within 14 days of enrollment and within 7 days of the start of treatment as noted below: 1. Absolute neutrophil count ≥ 1,000/μL 2. Platelets ≥ 100,000/μL (transfusion independent, defined as not receiving platelet transfusions within a 7-day period prior to enrollment) 3. Hemoglobin ≥ 8 g/dL (may receive PRBC transfusions) 4. Total bilirubin \< 1.5 times upper limit of normal for age 5. AST (SGOT)/ALT(SGPT) \< 2.5 X institutional upper limit of normal for age 6. Creatinine clearance or radioisotope GFR ≥ 70 ml/min/1.73m2 or a serum creatinine WNL for age as determined using the Schwartz formula.36 7. Sodium, Potassium, Calcium and Magnesium \< 1.5x institutional ULN 8. Albumin ≥ 3 g/dL 4. Subjects who are receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to enrollment. 5. Subjects with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment. 6. If the subject has any of the following therapies, must be at least: * 4 weeks post-focal RT (radiation therapy), 3 months post-CSI (craniospinal irradiation) * 4 weeks post-myelosuppressive chemotherapy (if post-nitrosoureas, must have 6 weeks therapy) * 4 weeks post-monoclonal antibodies * 1 week post-targeted therapy 7. If subject has received any previous treatment, all treatment related toxicities should have recovered to \< grade 2 8. Subject or parent must sign a written informed consent document according to institutional guidelines. Exclusion Criteria: 1. Females who are pregnant or lactating. 2. Subjects with any clinically significant unrelated systemic illness (serious infections or significant cardiac, pulmonary, hepatic or other organ dysfunction) likely to interfere with the study procedures or results. 3. Subjects who are receiving any other anticancer or investigational agents. 4. Subjects with uncontrolled seizures. 5. Subjects receiving enzyme inducing anticonvulsants. 6. Subjects with other factors that increase the risk of QT prolongation or arrhythmic events (e.g., heart failure, hypokalemia, family history of long QT interval syndrome) including heart failure that meets New York Heart Association (NYHA) class II or above. 7. Subjects who have had an allogenic bone marrow transplant \< 6 months prior to enrollment or an autologous bone marrow/stem cell transplant \< 3 months prior to enrollment. 8. Subjects with multifocal disease or disease that has been disseminated will not be eligible for this study. They will undergo systemic chemotherapy and their disease will be further evaluated prior to be eligible for 2nd look surgery. 9. This study will only enroll subjects with ACPP or CPC and will not enroll subjects with choroid plexus papilloma (CPP). ACPP or CPC subjects with symptomatic hydrocephalus will not be eligible for this study. These subjects will have to be treated for their hydrocephalus and be re-evaluated according to our eligibility criteria in order to be enrolled.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Weill Cornell Medicine
New York, New York, 10025, United States
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