Testing the Safety and Tolerability of CX-4945 in Patients With Recurrent Medulloblastoma Who May… (NCT03904862) | Clinical Trial Compass
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 range3 Years
SexALL
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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:
✓. Surgical Study:
✓. Surgical resection must be clinically indicated.
Exclusion criteria
✕. Nursing mothers due to an unknown but potential risk for adverse events in nursing infants.
✕. Patients with a history of any other malignancy with the exception of patients with a secondary brain tumor if the patient's prior malignancy has been in remission for at least 5 years from the end of treatment.
✕. Patients with any of the following gastrointestinal disorders - difficulty swallowing or active malabsorption, uncontrolled diarrhea, gastritis, ulcerative colitis, Crohn's disease or hemorrhagic coloproctitis, history of gastric or small bowel surgery involving any extent of gastric or small bowel resection.
What they're measuring
1
Phase I: Maximum tolerated dose of CX-4945
Timeframe: 4 weeks
2
Phase I: Plasma pharmacokinetics of CX-4945 in skeletally-immature children
Timeframe: 4 weeks
3
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
4
Phase II: Sustained objective response rate (PR-CR) rate in the skeletally mature cohort
✕. Patients with any clinically significant unrelated systemic illness that would compromise the patient's ability to tolerate therapy, put them at additional risk for toxicity or interfere with the study procedures or results.
✕. Corrected QT (QTc) interval is \>480ms
✕. Patients who are receiving other anti-cancer or investigational drug therapy