Atypical Choroid Plexus PapillomaPhase 1NCT04994977

Intra-Arterial Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma and Choroid Plexus Carcinoma Prior to Second-Look Surgery

A Phase 1 study testing Melphalan, Carboplatin and Topotecan in people with Atypical Choroid Plexus Papilloma and Choroid Plexus Carcinoma, run by Weill Medical College of Cornell University at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

This study will test the safety and efficacy of intra-arterial chemotherapy in subjects with newly diagnosed, residual, or recurrent atypical choroid plexus papilloma and choroid plexus carcinoma prior to a second surgery. Read more about this study

Who is it for?: Ages 0 to 100 years
Conditions the study is for: Atypical Choroid Plexus Papilloma, Choroid Plexus Carcinoma
What is being tested?: Melphalan, Carboplatin and Topotecan
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 1 participants
Where: 1 siteUnited States
Study dates: 4 May 2023to 1 Dec 2025
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 0 to 100 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Subjects with a histologically confirmed diagnosis of ACPP or CPC that is newly diagnosed, residual or recurrent.
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.
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:
Absolute neutrophil count ≥ 1,000/μL
Platelets ≥ 100,000/μL (transfusion independent, defined as not receiving platelet transfusions within a 7-day period prior to enrollment)
Hemoglobin ≥ 8 g/dL (may receive PRBC transfusions)

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Total bilirubin < 1.5 times upper limit of normal for age
AST (SGOT)/ALT(SGPT) < 2.5 X institutional upper limit of normal for age
Creatinine clearance or radioisotope GFR ≥ 70 ml/min/1.73m2 or a serum creatinine WNL for age as determined using the Schwartz formula.36
Sodium, Potassium, Calcium and Magnesium < 1.5x institutional ULN
Albumin ≥ 3 g/dL
Subjects who are receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to enrollment.
Subjects with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment.
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
If subject has received any previous treatment, all treatment related toxicities should have recovered to < grade 2
Subject or parent must sign a written informed consent document according to institutional guidelines.

Who cannot join

Females who are pregnant or lactating.
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.
Subjects who are receiving any other anticancer or investigational agents.
Subjects with uncontrolled seizures.
Subjects receiving enzyme inducing anticonvulsants.
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.

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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.
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.
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.

Trial locations and contacts

1 site in 1 country.

United States1 site

Weill Cornell Medicine
New York, New York, 10025

Other trials in United States

About this study

Official title: Intra-Arterial (IA) Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma (ACPP) and Choroid Plexus Carcinoma (CPC) Prior to Second-Look Surgery

It is believed that intra-arterial chemotherapy will be safe and feasible for this population and will result in decreased tumor size, which may further improve the goals of a second-look surgery.

Read the detailed description

The intent of this study is to determine if intra-arterial chemotherapy is a safe and effective option for subjects with atypical choroid plexus papilloma and choroid plexus carcinoma prior to receiving second-look surgery. Angiography occurs when a catheter is inserted through the subjects vasculature to the major vessels that supply cerebral circulation. Subjects on this trial will undergo a a cerebral angiogram to determine the ideal arteries to use for drug infusion. Once identified, chemotherapy will be delivered through the catheter directly to the site of tumor.

Conditions: Atypical Choroid Plexus Papilloma, Choroid Plexus Carcinoma
Being tested: Melphalan, Carboplatin, Topotecan
Medical subject headings: Choroid Plexus Carcinoma

What participants receive

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

MelphalanDrug
Given at 0.5 mg/ml.
CarboplatinDrug
Given at 5 mg/ml.
TopotecanDrug
Given at 0.2 mg/ml.

Study groups in NCT04994977
Study group	Role	What they receive
Intra-arterial Chemotherapy	Experimental	Subjects are pre-treated with heparin, and then given single doses of Melphalan, Carboplatin, and Topotecan consecutively via intra-arterial infusion. Multiple arteries may be used, with the total dose of the drugs remaining the same. Receives: Drug: Melphalan; Drug: Carboplatin; Drug: Topotecan

What is being measured

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

Safety of Intra-arterial Chemotherapy in Subjects With ACPP and CPC, Measured by the Number of Serious Adverse Events That Are Reported as at Least Possible Related to the Intervention That Occur in Subjects on the Trial
Measured: about 6 months since the start of therapy

Secondary outcomes (6)

The Number of Successful Angiography Procedures, Determined by Examination of Vasculature and Assessment of Catheter Placement
Measured: On Day 1 of the trial for each subject
The Number of Patients With a Tumor Volume Reduction Response, Determined by MRI Assessments
Measured: Between 4-6 weeks after intra-arterial chemotherapy
The Number of Patients With a Tumor Vascularity Reduction Response, Determined by MRI Assessments
Measured: Between 4-6 weeks after intra-arterial chemotherapy
The Success of Second Look Surgery Determined by Measuring the Extent of Tumor Resection
The number of participants with successful second look surgery defined by gross total resection of residual tumor
Measured: Around 7 weeks after intra-arterial chemotherapy, during second look surgery
The Success of Second Look Surgery Determined by Amount of Blood Loss
The number of participants with successful second look surgery determined by amount of blood loss. If the amount of blood loss is insignificant i.e. the surgery does not necessitate blood transfusion, then the surgery is deemed successful.
Measured: Around 7 weeks after intra-arterial chemotherapy, during second look surgery
The Success of Second Look Surgery Determined by Percent of Blood Loss
Measured: Around 7 weeks after intra-arterial chemotherapy, during second look surgery

Timeline

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

4 May 20231 Dec 2025

First posted6 Aug 2021
Study start4 May 2023
Primary completion23 Jul 2024
Study completion1 Dec 2025

Why it stopped: Study terminated due to low accrual

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Atypical Choroid Plexus PapillomaStudies of the same condition, open and under way.
More trials run by Weill Medical College of Cornell UniversityThe 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: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 1 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04994977
Sponsor's protocol ID: 20-08022610
Status last verified: May 2026
First submitted: 17 Jul 2021
Last updated: 8 Jun 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Study at a glance

From the registry record, last updated by the sponsor on 8 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Intra-Arterial (IA) Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma (ACPP) and Choroid Plexus Carcinoma (CPC) Prior to Second-Look Surgery
ClinicalTrials.gov ID: NCT04994977
Acronym / sponsor ID: 20-08022610
Conditions: Atypical Choroid Plexus Papilloma, Choroid Plexus Carcinoma
Phase: Phase 1
Study type: Interventional
Recruitment status: TerminatedReason stopped: Study terminated due to low accrual
Enrolment: 1 participants (actual)
Lead sponsor: Weill Medical College of Cornell UniversityUniversity, hospital or other
Study start: 4 May 2023 (actual)
Primary completion: 23 Jul 2024 (actual)
Study completion: 1 Dec 2025 (actual)
First posted: 6 Aug 2021 (actual)
Results: Posted 8 Jun 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

It is believed that intra-arterial chemotherapy will be safe and feasible for this population and will result in decreased tumor size, which may further improve the goals of a second-look surgery.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Intra-arterial Chemotherapy	Experimental	Drug: Melphalan, Drug: Carboplatin, Drug: TopotecanSubjects are pre-treated with heparin, and then given single doses of Melphalan, Carboplatin, and Topotecan consecutively via intra-arterial infusion. Multiple arteries may be used, with the total dose of the drugs remaining the same.

Who may qualify

Ages: 0 to 100 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (20 listed)

Subjects with a histologically confirmed diagnosis of ACPP or CPC that is newly diagnosed, residual or recurrent.
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.
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:
Subjects who are receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to enrollment.
Subjects with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment.

Key exclusion criteria (9 listed)

Females who are pregnant or lactating.
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.
Subjects who are receiving any other anticancer or investigational agents.
Subjects with uncontrolled seizures.
Subjects receiving enzyme inducing anticonvulsants.

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
MelphalanGiven at 0.5 mg/ml.	Drug	Intra-arterial Chemotherapy
CarboplatinGiven at 5 mg/ml.	Drug	Intra-arterial Chemotherapy
TopotecanGiven at 0.2 mg/ml.	Drug	Intra-arterial Chemotherapy

Primary outcome

Safety of Intra-arterial Chemotherapy in Subjects With ACPP and CPC, Measured by the Number of Serious Adverse Events That Are Reported as at Least Possible Related to the Intervention That Occur in Subjects on the Trial
Time frame: about 6 months since the start of therapy

6 secondary outcomes

The Number of Successful Angiography Procedures, Determined by Examination of Vasculature and Assessment of Catheter Placement
Time frame: On Day 1 of the trial for each subject
The Number of Patients With a Tumor Volume Reduction Response, Determined by MRI Assessments
Time frame: Between 4-6 weeks after intra-arterial chemotherapy
The Number of Patients With a Tumor Vascularity Reduction Response, Determined by MRI Assessments
Time frame: Between 4-6 weeks after intra-arterial chemotherapy
The Success of Second Look Surgery Determined by Measuring the Extent of Tumor Resection
Time frame: Around 7 weeks after intra-arterial chemotherapy, during second look surgery
The Success of Second Look Surgery Determined by Amount of Blood Loss
Time frame: Around 7 weeks after intra-arterial chemotherapy, during second look surgery
The Success of Second Look Surgery Determined by Percent of Blood Loss
Time frame: Around 7 weeks after intra-arterial chemotherapy, during second look surgery

Results and safety

Results posted to the registry on 8 Jun 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Intra-arterial Chemotherapy	1	1

Primary outcome: Safety of Intra-arterial Chemotherapy in Subjects With ACPP and CPC, Measured by the Number of Serious Adverse Events That Are Reported as at Least Possible Related to the Intervention That Occur in Subjects on the Trial

Time frame: about 6 months since the start of therapy. Number. Unit: Count of SAEs

Measure	Intra-arterial Chemotherapy n = 1
Value	0

8 secondary outcomes are reported in the full results.

Adverse events

Time frame: ~ 6 months. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Intra-arterial Chemotherapy	0 of 1 (0%)	0 of 1 (0%)	0 of 1

Limitations and caveats (from the sponsor): Early termination due to low enrollment

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

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.

Mark Souweidane, M.D. principal investigator

Weill Medical College of Cornell University

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 20 inclusion and 9 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: stopped early, as of the sponsor's last update on 8 Jun 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?

Yes. Results are posted in the registry (first posted 8 Jun 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Safety of Intra-arterial Chemotherapy in Subjects With ACPP and CPC, Measured by the Number of Serious Adverse Events That Are Reported as at Least Possible Related to the Intervention That Occur in Subjects on the Trial (time frame: about 6 months since the start of therapy). 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 NCT04994977.

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 8 Jun 2026.