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

NCT04994977 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: 20-08022610
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Atypical Choroid Plexus Papilloma; Choroid Plexus Carcinoma

Outcome Measures

Primary Outcomes (1)

• 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

  Through study completion, a little over a year for each subject

Secondary Outcomes (8)

• The number of successful angiography procedures, determined by examination of vasculature and assessment of catheter placement

  On Day 1 of the trial for each subject

• The number of patients with a tumor volume reduction response, determined by MRI assessments

  Between 4-6 weeks after intra-arterial chemotherapy

• The proportion of patients with a tumor volume reduction response, determined by MRI assessments

  Between 4-6 weeks after intra-arterial chemotherapy

• The number of patients with a tumor vascularity reduction response, determined by MRI assessments

  Between 4-6 weeks after intra-arterial chemotherapy

• The proportion of patients with a tumor vascularity reduction response, determined by MRI assessments

  Between 4-6 weeks after intra-arterial chemotherapy

Other Outcomes (1)

• Extent of pathology correlation to tumor vascularity and tumor viability

  Around 7 weeks after intra-arterial chemotherapy

Study Arms (1)

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.

Drug: Melphalan; Drug: Carboplatin; Drug: Topotecan

Interventions

Melphalan DRUG

Given at 0.5 mg/ml.

Intra-arterial Chemotherapy

Carboplatin DRUG

Given at 5 mg/ml.

Intra-arterial Chemotherapy

Topotecan DRUG

Given at 0.2 mg/ml.

Intra-arterial Chemotherapy

Eligibility Criteria

• Age: 0 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• 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:
• weeks post-focal RT (radiation therapy), 3 months post-CSI (craniospinal irradiation)

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Weill Medical College of Cornell University: lead

Study Sites (1)

Weill Cornell Medicine

New York, New York, 10025, United States

Location

MeSH Terms

Conditions

Choroid Plexus Carcinoma

Interventions

Melphalan; Carboplatin; Topotecan

Intervention Hierarchy (Ancestors)

Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Coordination Complexes; Camptothecin; Alkaloids; Heterocyclic Compounds

Study Officials

• Mark Souweidane, M.D.

  Weill Medical College of Cornell University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 17, 2021
• First Posted: August 6, 2021
• Study Start: May 4, 2023
• Primary Completion: July 23, 2024
• Study Completion: December 1, 2025
• Last Updated: March 25, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)