A Phase 2 Study to Test Effects of Using DAY101 in Children with Brain Cancer
- Conditions
- BRAF-Altered, Recurrent or Progressive Low-Grade Glioma in pediatric patientsTherapeutic area: Diseases [C] - Cancer [C04]
- Registration Number
- EUCTR2020-003657-30-GB
- Lead Sponsor
- DOT Therapeutics-1 Inc. (Day One)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 60
- Age 6 months to 25 years with a relapsed or progressive LGG with a documented known activating BRAF alteration.
- Confirmation of histopathologic diagnosis of LGG from either original diagnosis or relapse
- Must have received at least 1 line of systemic therapy prior and have documented evidence of radiographic progression
- Must have at least 1 measurable lesion
- Karnofsky (those 16 years and older) or Lansky (those younger than 16 years) performance score of at least 50.
For the complete list of the inclusion criteria, please refer to the protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range: 60
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Patient has symptoms of clinical progression without radiographically recurrent or radiographically progressive disease.
- History of any major disease, other than the diagnosis of LGG, that might interfere with safe protocol participation
- Major surgery within 14 days (2 weeks) prior to C1D1
- Clinically significant active cardiovascular disease, or history of myocardial infarction, or deep vein thrombosis/pulmonary embolism within 6 months prior to C1D1
- Current enrollment in any other investigational treatment study. Participation in a concurrent observational or bio-sampling study is allowed.
For the complete list of the exclusion criteria, please refer to the protocol.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the efficacy of DAY101 in pediatric patients aged 6 months to 25 years of age with a relapsed or progressive low-grade glioma (LGG) harboring a known activating BRAF alteration.;Secondary Objective: - To assess safety and tolerability of DAY101<br> - To determine the relationship between pharmacokinetics (PK) and drug effects, including efficacy and safety<br> <br>For the complete list of secondary objectives please refer to the protocol Section 2 Study Objectives.;Primary end point(s): Overall Response Rate;Timepoint(s) of evaluation of this end point: End of Cycle 3, end of Cycle 6, and ever 3 cycles thereafter. Please see Appendix A of the protocol.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Type, frequency, and severity of AEs and laboratory abnormalities<br> - Pharmacokinetic profile of DAY101 (e.g., area under the concentration-time curve [AUC], Cmin, etc.)<br> - time following initiation of DAY101 to progression or death in patients treated with DAY101<br> - length of response in patients with best overall confirmed response of CR or PR <br> - time to first response following initiation of DAY101 in patients with best overall confirmed response of CR or PR <br><br> Please see Table 6 in protocol for further information.;Timepoint(s) of evaluation of this end point: AEs: From the start of treatment with DAY101 until 30 days after the last dose. <br>PK profile (blood sampling): C1D1, C1D15, C2D1, C41, Every 3rd cycle, At the time of toxicity, and/or at time of surgery, if clinically indicated. <br>Change from QT interval, baseline PR interval, baseline QRS interval, baseline heart rate, <br><br>Please see Appendix A of the protocol for further details.