Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ACT001 · 1 trial · 1 indication
A DLT is defined as any of the following * Haematological toxicity * Grade 4 neutropenia * Grade ≥ 3 febrile neutropenia or neutropenic infection * Grade 4 thrombocytopenia * Grade 3 thrombocytopenia with clinically significant bleeding * Non-haematological toxicity * Grade ≥ 3 nausea, vomiting or diarrhoea despite optimal supportive therapy * Grade ≥ 3 hypertension despite appropriate intervention * Other grade ≥ 3 non-haematological toxicity, except grade 3 fatigue or transient events (such as allergic reactions or electrolyte disturbances) that are readily controlled with medical therapy and/or are of no clinical concern
Mean change from baseline in vital sign measurements reported in beats/min (inclusive) at each study timepoint.
Mean change from baseline in vital sign measurements reported in mmHg at each study timepoint
Mean change from baseline in vital sign measurements reported in degrees Celsius (0C) at each study timepoint.
Mean change from baseline in vital sign measurements reported in bpm at each study timepoint
Mean change from baseline in electrocardiogram (ECG) parameters of heart rate, ventricular rate, PR interval, QRS duration, and QTcF.
Mean change from baseline in Karnofsky Performance Scale score
| Arm | Type | Description |
|---|---|---|
| 1b dose exploration | EXPERIMENTAL | 1b dose exploration for 18 patients - The starting dose of ACT001 will be administered in combination with a single intravenous (IV) infusion of Pembrolizumab. After recovery from surgical resection, dosing will resume on a 3 weekly cycle and will consist of Pembrolizumab (standard dosing) and daily ACT001. Evaluation of a dose level of at least three (3) patients after completing one cycle of treatment post-surgery is required prior to commencing the next dose level. |
| 2a- Randomized/Two-treatment Arm | EXPERIMENTAL | 30 Patients will be randomized to Arm A or Arm B at a ratio of 1 (Arm A) : 2 (Arm B). 10 patients will be randomized to the Pembrolizumab only arm (Arm A) and 20 patients will be randomized to the ACT001 plus Pembrolizumab arm (Arm B). |
| Name | Type | Description |
|---|---|---|
| ACT001 | DRUG | Phase1b - Starting approximately 2 weeks prior to the scheduled surgery, patient will receive an assigned dose of ACT001 by mouth in combination with a single dose of 200 mg pembrolizumab via an intravenous (IV-through a tube in vain) infusion in the clinic. Then patient will self administer ACT001 capsules twice daily by mouth, at the dose to which patient is assigned, until the evening prior to scheduled surgery. Patient will then undergo surgery to remove all or part of tumor. This a standard 3+3 dose escalation. |
| ACT001 + Pembrolizumab | DRUG | Phase 2a - Starting approximately 2 weeks prior to the scheduled surgery, patient will receive a single dose of 200 mg pembrolizumab via an intravenous (IV) infusion in the clinic (an IV infusion means the drug will be delivered through a tube in your vein). Patient will then self-administer ACT001 capsules twice daily by mouth, at the dose to which patient is assigned, until the evening prior to scheduled surgery. Patient then will undergo surgery to remove all or part of tumor. |
Inclusion Criteria: 1. Patient has provided written informed consent. 2. ≥ 18 years old at time of screening visit. 3. Histologically confirmed GBM at the time of diagnosis. 4. First or second relapse by the time of consenting. 5. Tumor progression (magnetic resonance imaging \[MRI\], defined by RA...
ACT001 is an investigational small molecule being studied for the treatment of recurrent glioblastoma multiforme (GBM), a type of brain cancer. It is currently in Phase 1 clinical development, meaning it has not yet been approved by regulatory authorities and is still being evaluated for safety and efficacy in patients.
ACT001 is a small molecule being studied in combination with an anti-PD-1 agent for recurrent glioblastoma multiforme. The specific molecular target of ACT001 is not disclosed in the available information, but the ongoing trial evaluates its safety and efficacy when used alongside an immune checkpoint inhibitor.
ACT001 is being developed by BrainsWay Ltd., a company publicly traded under the ticker symbol BWAY. The company is conducting a Phase 1 clinical trial of ACT001 in the United States for recurrent glioblastoma multiforme.
ACT001 is in Phase 1 clinical development. It is an investigational drug, not yet approved by the FDA or other regulatory bodies. The ongoing Phase 1 trial is active but not recruiting participants, and it is designed to evaluate the safety and efficacy of ACT001 in combination with an anti-PD-1 agent.
ACT001 is being studied in a Phase 1 clinical trial with the identifier NCT05053880. This trial, titled "A Study to Evaluate Safety and Efficacy of ACT001 and Anti-PD-1 in Patients With Surgically Accessible Recurrent Glioblastoma Multiforme," is active but not recruiting. It enrolls 48 participants aged 18 years and older in the United States.
ACT001 is not known to have alternative names in the available information. It is being studied as a single agent in combination with an anti-PD-1 therapy for recurrent glioblastoma multiforme, and no other names for this drug have been disclosed.