A Study of Belzutifan in Adolescent Participants With Solid Tumors (MK-9999-01E/LIGHTBEAM-U01)
8 septembre 2026 mis à jour par: Merck Sharp & Dohme LLC
LIGHTBEAM-U01 Substudy 01E: A Phase 2 Substudy to Evaluate the Safety and Efficacy of Belzutifan in Participants With Solid Tumors
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.
Aperçu de l'étude
Statut
Pas encore de recrutement
Les conditions
Intervention / Traitement
Type d'étude
Interventionnel
Inscription (Estimé)
15
Phase
- Phase 2
Contacts et emplacements
Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.
Coordonnées de l'étude
- Nom: Toll Free Number
- Numéro de téléphone: 1-888-577-8839
- E-mail: Trialsites@msd.com
Critères de participation
Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.
Critère d'éligibilité
Âges éligibles pour étudier
- Enfant
Accepte les volontaires sains
Non
La description
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 exclusion criteria include but are not limited to the following:
- 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
- Has had an allogenic tissue/solid organ transplant
- Has a history of autologous stem cell transplant within 6 months of start of study intervention
- Has known additional malignancy that is progressing or has required active treatment within the past 2 years
- Has known active central nervous system metastases and/or carcinomatous meningitis
- Has an active infection requiring systemic therapy
Plan d'étude
Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: Belzutifan
Participants will receive belzutifan 80 mg (body weight <40 kg) or 120 mg (body weight ≥40 kg) orally once daily for approximately 2 years.
|
Administered once daily via oral tablet
Autres noms:
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Number of Participants Who Experience One or More Adverse Events (AEs)
Délai: 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.
|
Up to approximately 5 years
|
|
Number of Participants Who Discontinue Study Intervention Due to an AE
Délai: 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.
|
Up to approximately 5 years
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Area Under the Concentration-Time Curve From Time 0 to 24 hours of Belzutifan
Délai: 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.
|
At designated timepoints (up to 5 weeks)
|
|
Minimum Plasma Concentration (Cmin) of Belzutifan
Délai: At designated timepoints (up to 5 weeks)
|
Blood samples will be collected at specified intervals to determine the Cmin of belzutifan.
|
At designated timepoints (up to 5 weeks)
|
|
Maximum Plasma Concentration (Cmax) of Belzutifan
Délai: At designated timepoints (up to 5 weeks)
|
Blood samples will be collected at specified intervals to determine the Cmax of belzutifan.
|
At designated timepoints (up to 5 weeks)
|
|
Objective Response Rate (ORR)
Délai: 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.
|
Up to approximately 5 years
|
|
Duration of Response (DOR)
Délai: 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.
|
Up to approximately 5 years
|
Collaborateurs et enquêteurs
C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.
Parrainer
Les enquêteurs
- Directeur d'études: Medical Director, Merck Sharp & Dohme LLC
Publications et liens utiles
La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.
Liens utiles
Dates d'enregistrement des études
Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.
Dates principales de l'étude
Début de l'étude (Estimé)
23 novembre 2026
Achèvement primaire (Estimé)
2 janvier 2034
Achèvement de l'étude (Estimé)
2 janvier 2034
Dates d'inscription aux études
Première soumission
8 juillet 2026
Première soumission répondant aux critères de contrôle qualité
8 juillet 2026
Première publication (Réel)
14 juillet 2026
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
11 septembre 2026
Dernière mise à jour soumise répondant aux critères de contrôle qualité
8 septembre 2026
Dernière vérification
1 septembre 2026
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- 9999-01E
- 2025 (Subvention/contrat des NIH des États-Unis: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
- LIGHTBEAM-U01 (Autre identifiant: MSD)
- U1111-1330-9370 (Identificateur de registre: UTN)
- 2025-524515-37-00 (Identificateur de registre: EU CT)
- MK-9999-01E (Autre identifiant: MSD)
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
OUI
Description du régime IPD
https://trialstransparency.msdclinicaltrials.com/pdf/ProcedureAccessClinicalTrialData.pdf
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
Oui
Étudie un produit d'appareil réglementé par la FDA américaine
Non
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