Malignant Neoplasm
Conditions
Brief summary
Researchers are looking for new ways to treat adolescents with locally advanced, unresectable, or metastatic solid tumors. Participants were enrolled into pheochromocytoma/paraganglioma (PPGL), wild type gastrointestinal stromal tumor (wtGIST), and Von Hippel-Lindau (VHL) disease-associated localized tumors cohorts: * PPGL are rare cancers that start in cells that make hormones in the adrenal glands * wtGIST is a less common type of cancer that starts in the digestive tract. Wild type means it does not have certain gene mutations (changes) * VHL disease-associated localized tumors are rare tumors caused by a certain gene mutation that may be passed down from parents to children * Locally advanced means the cancer has spread into nearby tissue * Unresectable means the cancer cannot be removed by surgery * Metastatic means the cancer has spread to other parts of the body The goal of the study is to learn about the safety of belzutifan and if people tolerate it.
Interventions
DRUGBelzutifan
Administered once daily via oral tablet
Sponsors
Merck Sharp & Dohme LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
The main inclusion criteria include but are not limited to the following: * Has a diagnosis of one of the following: locally advanced, unresectable, or metastatic pheochromocytoma/paraganglioma or wild-type gastrointestinal stromal tumors, or localized tumors associated with von Hippel-Lindau disease * Has measurable disease per RECIST 1.1
Exclusion criteria
The main
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Who Experience One or More Adverse Events (AEs) | Up to approximately 5 years | An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The number of participants that experience AEs will be reported. |
| Number of Participants Who Discontinue Study Intervention Due to an AE | Up to approximately 5 years | An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The number of participants that discontinue study intervention due to an AE will be reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Area Under the Concentration-Time Curve From Time 0 to 24 hours of Belzutifan | At designated timepoints (up to 5 weeks) | Blood samples will be collected at specified intervals to determine the area under the concentration-time curve from time 0 to 24 hours of belzutifan. |
| Minimum Plasma Concentration (Cmin) of Belzutifan | At designated timepoints (up to 5 weeks) | Blood samples will be collected at specified intervals to determine the Cmin of belzutifan. |
| Maximum Plasma Concentration (Cmax) of Belzutifan | At designated timepoints (up to 5 weeks) | Blood samples will be collected at specified intervals to determine the Cmax of belzutifan. |
| Objective Response Rate (ORR) | Up to approximately 5 years | ORR is defined as the percentage of participants with Complete Response (CR: disappearance of all target lesions) or Partial Response (PR: at least a 30% decrease in the sum of diameters of target lesions) per Response Evaluation Criteria In Solid Tumors Version 1.1 (RECIST 1.1). The percentage of participants who experience CR or PR as assessed by investigator will be reported. |
| Duration of Response (DOR) | Up to approximately 5 years | For participants who demonstrate a confirmed Complete Response (CR: disappearance of all target lesions) or Partial Response (PR: at least a 30% decrease in the sum of diameters of target lesions) per Response Evaluation Criteria In Solid Tumors Version 1.1 (RECIST 1.1), DOR is defined as the time from first documented evidence of CR or PR until progressive disease (PD) or death. Per RECIST 1.1, PD is defined as at least a 20% increase in the sum of diameters of target lesions. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered PD. DOR as assessed by investigator will be reported. |
Contacts
CONTACTToll Free Number
STUDY_DIRECTORMedical Director
Merck Sharp & Dohme LLC
Outcome results
None listed