Atypical Choroid Plexus Papilloma, Choroid Plexus Carcinoma
Conditions
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.
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.
Interventions
DRUGMelphalan
Given at 0.5 mg/ml.
DRUGCarboplatin
Given at 5 mg/ml.
DRUGTopotecan
Given at 0.2 mg/ml.
Sponsors
Weill Medical College of Cornell University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
0 Years to 100 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 | about 6 months since the start of therapy |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 Number of Patients With a Tumor Vascularity Reduction Response, Determined by MRI Assessments | Between 4-6 weeks after intra-arterial chemotherapy | — |
| The Success of Second Look Surgery Determined by Measuring the Extent of Tumor Resection | Around 7 weeks after intra-arterial chemotherapy, during second look surgery | The number of participants with successful second look surgery defined by gross total resection of residual tumor |
| The Success of Second Look Surgery Determined by Amount of Blood Loss | Around 7 weeks after intra-arterial chemotherapy, during second look surgery | 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. |
| The Success of Second Look Surgery Determined by Percent of Blood Loss | Around 7 weeks after intra-arterial chemotherapy, during second look surgery | — |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORMark Souweidane, M.D.
Weill Medical College of Cornell University
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Categorical <=18 years | 1 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 1 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 1 |
| other Total, other adverse events | 0 / 1 |
| serious Total, serious adverse events | 0 / 1 |
Outcome results
None listed