Trial Outcomes & Findings for Intra-Arterial Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma and Choroid Plexus Carcinoma Prior to Second-Look Surgery (NCT NCT04994977)

Results Overview

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

1 participants

Primary outcome timeframe

about 6 months since the start of therapy

Results posted on

2026-06-08

Participant Flow

Participant milestones

Participant milestones
Measure	Intra-arterial Chemotherapy 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. Melphalan: Given at 0.5 mg/ml. Carboplatin: Given at 5 mg/ml. Topotecan: Given at 0.2 mg/ml.
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Intra-arterial Chemotherapy n=1 Participants 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. Melphalan: Given at 0.5 mg/ml. Carboplatin: Given at 5 mg/ml. Topotecan: Given at 0.2 mg/ml.
Age, Categorical <=18 years	1 Participants n=9 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=9 Participants
Age, Categorical >=65 years	0 Participants n=9 Participants
Sex: Female, Male Female	0 Participants n=9 Participants
Sex: Female, Male Male	1 Participants n=9 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=9 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	0 Participants n=9 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=9 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=9 Participants
Race (NIH/OMB) Asian	0 Participants n=9 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=9 Participants
Race (NIH/OMB) Black or African American	0 Participants n=9 Participants
Race (NIH/OMB) White	1 Participants n=9 Participants
Race (NIH/OMB) More than one race	0 Participants n=9 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=9 Participants

PRIMARY outcome

Timeframe: about 6 months since the start of therapy

Outcome measures

Outcome measures
Measure	Intra-arterial Chemotherapy n=1 Participants 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. Melphalan: Given at 0.5 mg/ml. Carboplatin: Given at 5 mg/ml. Topotecan: Given at 0.2 mg/ml.
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	0 Count of SAEs

SECONDARY outcome

Timeframe: On Day 1 of the trial for each subject

Outcome measures

Outcome measures
Measure	Intra-arterial Chemotherapy n=1 Participants 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. Melphalan: Given at 0.5 mg/ml. Carboplatin: Given at 5 mg/ml. Topotecan: Given at 0.2 mg/ml.
The Number of Successful Angiography Procedures, Determined by Examination of Vasculature and Assessment of Catheter Placement	1 Count of procedure

SECONDARY outcome

Timeframe: Between 4-6 weeks after intra-arterial chemotherapy

Outcome measures

Outcome measures
Measure	Intra-arterial Chemotherapy n=1 Participants 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. Melphalan: Given at 0.5 mg/ml. Carboplatin: Given at 5 mg/ml. Topotecan: Given at 0.2 mg/ml.
The Number of Patients With a Tumor Volume Reduction Response, Determined by MRI Assessments	1 Participants

SECONDARY outcome

Timeframe: Between 4-6 weeks after intra-arterial chemotherapy

Outcome measures

Outcome measures
Measure	Intra-arterial Chemotherapy n=1 Participants 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. Melphalan: Given at 0.5 mg/ml. Carboplatin: Given at 5 mg/ml. Topotecan: Given at 0.2 mg/ml.
The Number of Patients With a Tumor Volume Reduction Response, Determined by MRI Assessments	1 Participants

SECONDARY outcome

Timeframe: Between 4-6 weeks after intra-arterial chemotherapy

Outcome measures

Outcome measures
Measure	Intra-arterial Chemotherapy n=1 Participants 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. Melphalan: Given at 0.5 mg/ml. Carboplatin: Given at 5 mg/ml. Topotecan: Given at 0.2 mg/ml.
The Number of Patients With a Tumor Vascularity Reduction Response, Determined by MRI Assessments	1 Participants

SECONDARY outcome

Timeframe: Between 4-6 weeks after intra-arterial chemotherapy

Outcome measures

Outcome measures
Measure	Intra-arterial Chemotherapy n=1 Participants 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. Melphalan: Given at 0.5 mg/ml. Carboplatin: Given at 5 mg/ml. Topotecan: Given at 0.2 mg/ml.
The Number of Patients With a Tumor Vascularity Reduction Response, Determined by MRI Assessments	1 Participants

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	Intra-arterial Chemotherapy n=1 Participants 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. Melphalan: Given at 0.5 mg/ml. Carboplatin: Given at 5 mg/ml. Topotecan: Given at 0.2 mg/ml.
The Success of Second Look Surgery Determined by Measuring the Extent of Tumor Resection	1 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Intra-arterial Chemotherapy n=1 Participants 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. Melphalan: Given at 0.5 mg/ml. Carboplatin: Given at 5 mg/ml. Topotecan: Given at 0.2 mg/ml.
The Success of Second Look Surgery Determined by Amount of Blood Loss	1 Participants

SECONDARY outcome

Timeframe: Around 7 weeks after intra-arterial chemotherapy, during second look surgery

Outcome measures

Outcome measures
Measure	Intra-arterial Chemotherapy n=1 Participants 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. Melphalan: Given at 0.5 mg/ml. Carboplatin: Given at 5 mg/ml. Topotecan: Given at 0.2 mg/ml.
The Success of Second Look Surgery Determined by Percent of Blood Loss	5.5 percent blood loss

OTHER_PRE_SPECIFIED outcome

Timeframe: Around 7 weeks after intra-arterial chemotherapy

Determined by comparison of pathology reports and surgical outcomes

Outcome measures

Outcome data not reported

Adverse Events

Intra-arterial Chemotherapy

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Mark Souweidane

Weill Cornell Medicine

Phone: (212) 746-7232

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place