A Study of Belzutifan in Adolescent Participants With Solid Tumors (MK-9999-01E/LIGHTBEAM-U01)
ConditionMalignant Neoplasm
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12-17
SponsorMerck Sharp & Dohme LLC
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
Has a pulse oximeter reading <92% at rest, requires intermittent supplemental oxygen, or requires chronic supplemental oxygen
Has clinically significant cardiac disease or electrocardiogram indicating uncontrolled cardiac condition or has congenital long QT syndrome
Has a history of human immunodeficiency virus infection
Has received prior treatment with any hypoxia inducible factor-2α inhibitor, including belzutifan
Trial design
Design model
Single group
Treatments tested in this trial
Belzutifan
DrugAdministered once daily via oral tablet
Treatment groups
15 Participants
are divided into 1 treatment groupGroup A: BelzutifanExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Number of Participants Who Experience One or More Adverse Events (AEs)
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.
Time frame
Up to approximately 5 years2
Number of Participants Who Discontinue Study Intervention Due to an AE
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.
Time frame
Up to approximately 5 yearsSecondary outcomes
1
Area Under the Concentration-Time Curve From Time 0 to 24 hours of Belzutifan
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.
Time frame
At designated timepoints (up to 5 weeks)2
Minimum Plasma Concentration (Cmin) of Belzutifan
Blood samples will be collected at specified intervals to determine the Cmin of belzutifan.
Time frame
At designated timepoints (up to 5 weeks)3
Maximum Plasma Concentration (Cmax) of Belzutifan
Blood samples will be collected at specified intervals to determine the Cmax of belzutifan.
Time frame
At designated timepoints (up to 5 weeks)4
Objective Response Rate (ORR)
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.
Time frame
Up to approximately 5 yearsSponsors and contacts
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Source ClinicalTrials.gov