About this trial
This study will gather data from new and existing patients with patient medical records, and patient/family/caregiver reported information to establish a clear natural history of disease suitable to serve as an external, contemporary or historical control arm for future therapeutic development programs of drugs, devices, or biologic interventions in DMG or DIPG.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Diagnosis of diffuse midline glioma according to the WHO 2021 Classification of Tumors of the Central Nervous System diagnostic criteria including diffuse intrinsic pontine glioma (DIPG). In the absence of a pathologically confirmed diagnosis, a grade IV glioma involving the thalamus, hypothalamus, brainstem, cerebellum, midbrain, or spinal cord, or with a pontine epicenter and diffuse involvement of the pons.
Patients with any performance status, comorbidity or disease severity are eligible
Patients or their legally-authorized representative must be willing and able to provide electronic, informed consent (and assent, if applicable)
Informed consent obtained for the XCELSIOR longitudinal outcomes registry (NCT03793088).
Disqualifiers
Patient or legally-authorized representative is unable to provide informed consent.
Patient or caregiver is unable to complete the PRO and ClinRO by an electronic platform.
Trial population
Up to 400 patients of any age who have been diagnosed with diffuse midline glioma (DMG) or diffuse intrinsic pontine glioma (DIPG).
Trial design
Cohort
Other
Treatments tested in this trial
Not listed
Trial groups
Trial outcomes
Primary outcomes
Overall Survival (OS)
OS is defined as time from first dose of treatment to death due to any cause.
Secondary outcomes
Objective Response Rate (ORR)
ORR is defined as the proportion of patients with a documented complete response (CR) or partial response (PR) as assessed by RANO or RAPNO.
Clinical Benefit Rate (CBR)
CBR defined as the proportion of patients with a best overall response of complete response (CR), partial response (PR), or stable disease (SD) as assessed by RANO or RAPNO