TerminatedPhase 1/2

Testing the Safety and Tolerability of CX-4945 in Patients With Recurrent Medulloblastoma Who May or May Not Have Surgery

Stopped: The decision to permanently close PBTC-053 was made following communication from the NCI that the PBTC grant will not be extended beyond March 31, 2026.

United States21 participantsStarted 2019-07-25

Plain-language summary

This is a multi center, Phase I, Phase II and surgical study of the CX-4945 drug (silmitasertib sodium) for patients with recurrent SHH (Sonic Hedgehog) medulloblastoma

Who can participate

Age range

3 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Phase I Skeletally-immature:
. Patient must be skeletally-immature at the time of study enrollment, defined as females with a bone age \< 14 years and males with a bone age \< 16 years.
. Patients who participate in the expansion cohort must have bi-dimensionally measurable disease, defined as at least one lesion that can be accurately measured in at least two dimensions (Section 12.2.2). Patients with measurable extraneural disease only are also eligible.
. Patient must be ≥ 3 and ≤ 18 years of age at the time of enrollment.
. Patients enrolled on the Phase I study must have a BSA as noted below:
. Phase II Skeletally-mature:

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Phase I: Maximum tolerated dose of CX-4945

Timeframe: 4 weeks

Phase I: Plasma pharmacokinetics of CX-4945 in skeletally-immature children

Timeframe: 4 weeks

Surgical Study: Intratumoral PK concentrations

Timeframe: Prior to starting CX-4945 (Day -5 or -7), prior to dose on Day -3, prior to dose on Day -1, day of surgery and during surgery at the time of tissue collection

Phase II: Sustained objective response rate (PR-CR) rate in the skeletally mature cohort

Timeframe: Up to 2 years from enrollment

Trial details

NCT IDNCT03904862

SponsorPediatric Brain Tumor Consortium

Sponsor typeNETWORK

Study typeINTERVENTIONAL

Primary completion2025-08-11

View on ClinicalTrials.gov More Medulloblastoma, Childhood trials

Who can participate

Age range

3 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Phase I Skeletally-immature:
. Patient must be skeletally-immature at the time of study enrollment, defined as females with a bone age \< 14 years and males with a bone age \< 16 years.
. Patients who participate in the expansion cohort must have bi-dimensionally measurable disease, defined as at least one lesion that can be accurately measured in at least two dimensions (Section 12.2.2). Patients with measurable extraneural disease only are also eligible.
. Patient must be ≥ 3 and ≤ 18 years of age at the time of enrollment.
. Patients enrolled on the Phase I study must have a BSA as noted below:
. Phase II Skeletally-mature:

What they're measuring

Phase I: Maximum tolerated dose of CX-4945

Timeframe: 4 weeks

Phase I: Plasma pharmacokinetics of CX-4945 in skeletally-immature children

Timeframe: 4 weeks

Surgical Study: Intratumoral PK concentrations

Timeframe: Prior to starting CX-4945 (Day -5 or -7), prior to dose on Day -3, prior to dose on Day -1, day of surgery and during surgery at the time of tissue collection

Phase II: Sustained objective response rate (PR-CR) rate in the skeletally mature cohort

Timeframe: Up to 2 years from enrollment

Testing the Safety and Tolerability of CX-4945 in Patients With Recurrent Medulloblastoma Who May or May Not Have Surgery

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Testing the Safety and Tolerability of CX-4945 in Patients With Recurrent Medulloblastoma Who May or May Not Have Surgery

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Exclusion criteria