A Phase 1 interventional study of WSD0628 in Glioblastoma, sponsored by Mayo Clinic. Recruiting at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-04-21.
Sponsored by Mayo Clinic · Phase 1, Interventional, and Treatment
The purpose of this study is to test WSD0628 in combination with radiation therapy for recurrent brain tumors.
High grade gliomas are the most common primary brain tumor in adults. Despite aggressive treatment including surgery, chemotherapy, and radiation, these tumors have a dismal prognosis. Following a radiation therapy, almost 80% of them recur locally. The focus of this project is the development of a radiation sensitizer (a small molecule ATM inhibitor, WSD0628) with the goal to enhance the efficacy of radiation therapy. The first step will be to establish a pre-clinical PK→PD→efficacy model to describe WSD0628 plasma and tumor concentrations associated with robust ATM inhibition and radiosensitizing effects. This model will be instrumental in interpreting the pharmacokinetic (PK) data and dosage selection in the proposed first-in-human, Phase 1, open-label, multicenter, single-arm, dose-escalation, and dose-expansion study in approximately 42 adult patients with recurrent high-grade glioma. The aims of the study are to assess the safety, tolerability, PKs and preliminary anti-tumor activity of WSD0628 in combination with radiation therapy. The dose-escalation portion of the study (Part A) will enroll approximately 24 patients and is comprised of Bayesian Optimal Interval (BOIN) design with target toxicity rate of 22%-33%. Once the recommended Phase 2 dose (RP2D) is established, Part B of the study will commence in which an additional 12 patients will be enrolled and treated at the RP2D for further evaluation of safety and efficacy (standard expansion cohort), and an additional 6 patients will have a tissue evaluation of tumor penetrance after a one-time dose of study drug prior to radiosurgery and surgical resection (Phase 0, tumor penetrance cohort). Tumor response will be assessed, using brain magnetic resonance imaging (MRI) with assessment based on the Response Assessment in Neuro-Oncology (RANO) criteria, and safety will include analysis of adverse events (AEs) and laboratory data. Additionally, PK, pharmacodynamic (PD), overall survival, progression-free survival, overall response rate, and patient-reported outcomes will be evaluated. The maximum duration of Part A will be 32 months and Part B,12 months. Funding Source - FDA OOPD
1,920 studies on the registry are indexed under Glioblastoma; 450 are open to participants now.
This study's planned enrollment of 94 is above the median of 36 across 1,618 interventional studies indexed under Glioblastoma.
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Of its 445 completed or terminated interventional studies of FDA-regulated products, 313 (70%) have results posted.
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Histological confirmation of one of the following:
The following laboratory values obtained ≤15 days prior to registration:
Calculated creatinine clearance ≥45 ml/min using the Cockcroft-Gault formula below:
Exclusion Criteria:
Any of the following because this study involves an investigational agent, the genotoxic, mutagenic, and teratogenic effects of which on the developing fetus and newborn are unknown:
Uncontrolled intercurrent illness including, but not limited to:
Any of the following cardiac criteria:
Any of the following medications:
Any of the following prior therapies:
WSD0628 treatment should be started the day before radiation therapy starts (Day 1). Radiation therapy is given for 10 consecutive business days (Day 2-15, not including weekends and holidays), and WSD0628 will only be given on those 10 consecutive business days ≥30 minutes but ≤2 hours before radiation.
Drug: WSD0628
WSD0628 treatment should be started the day before radiation therapy starts (Day 1). Radiation therapy is given for 10 consecutive business days (Day 2-15, not including weekends and holidays), and WSD0628 will only be given on those 10 consecutive business days ≥30 minutes but ≤2 hours before radiation. The Group B (Dose Expansion) portion of the study will be opened after the Group A (Dose Escalation) is complete.
Drug: WSD0628
One treatment of WSD0628 will be given prior to radiation and surgical resection will be performed on the same day. The two doses given will be determined by Group A and Group B. Patients will be randomized in a 1:1 fashion. * Dose level 1: minimally radiosensitizing concentration of WSD0628 will be achieved. * Dose level 2: selected based on a prediction that a maximally radiosensitizing concentration of WSD0628 will be achieved. This portion of the study will open after Group A (Dose Escalation) is complete. This portion of the study will open to patients with recurrent high-grade glioma to further evaluate the efficacy, safety, tolerability, pharmacokinetics and biological activity of WSD0628 when combined with radiation therapy in specific patient subgroups
Drug: WSD0628
A non-toxic compound and inhibits the DNA damage response associated with radiation therapy. • WSD-0628 radio sensitizes Glioblastoma cells.
Determine the maximum tolerated dose of WSD0628 in combination with radiation therapy for patients with recurrent high-grade glioma.
Use Bayesian Optimal Interval (BOIN) design to inform dose escalation and de-escalation decisions and to ultimately determine the maximum tolerated dose level (MTD) of WSD0628 in patient population.
Time frame: 4 weeks after last day of RT (up to 60 days)
Determine the recommended phase 2 dose of WSD0628 in combination with radiation therapy for patients with recurrent high-grade glioma.
Evaluate biologic activity measures using rank-based desirability scores (RDS) generated for each of the dose levels to identify which of these scores best when looking jointly at toxicity and biologic activity. The rank-based desirability scores (RDS) for this BOIN12 design will be used to help identify the best dose
Time frame: 4 weeks after last day of RT (up to 60 days)
Measure the incidence of acute adverse effects related to WSD0628 delivered concurrently with radiation
All AEs must be documented in the subject's medical record
Time frame: 4 weeks after last day of RT (up to 60 days)
Assess anti-tumor activity of WSD0628 delivered concurrently with radiation, including intracranial overall response rate (ORR)
Overall response rate defined as the proportion of patients who achieve a partial response (PR) or a complete response (CR) divided by the total number of patients who received therapy. Exact binomial 95% confidence intervals for the overall response rate will be calculated.
Time frame: 4 weeks after last day of RT (up to 60 days)
Assess anti-tumor activity of WSD0628 delivered concurrently with radiation, including progression-free survival (PFS)
The primary measure of response will be by serial measures of the product of the two largest cross-sectional diameters (bidirectional product) using the RANO and iRANO criteria.
Time frame: Beginning of study therapy until the first occurrence of progression or death
Assess anti-tumor activity of WSD0628 delivered concurrently with radiation, including volumetric change in tumor size
measured by MRI/CT scan evaluations
Time frame: Beginning of study therapy until the first occurrence of progression or death
Plan to share: No
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