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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04924075




Registration number
NCT04924075
Ethics application status
Date submitted
8/06/2021
Date registered
11/06/2021

Titles & IDs
Public title
Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2a Related Genetic Alterations (MK-6482-015)
Scientific title
A Phase 2 Study to Evaluate the Efficacy and Safety of Belzutifan (MK-6482, Formerly PT2977) Monotherapy in Participants With Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Advanced Solid Tumors With HIF-2a Related Genetic Alterations
Secondary ID [1] 0 0
MK-6482-015
Secondary ID [2] 0 0
6482-015
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pheochromocytoma/Paraganglioma 0 0
Pancreatic Neuroendocrine Tumor 0 0
Von Hippel-Lindau Disease 0 0
Advanced Gastrointestinal Stromal Tumor 0 0
HIF-2a Mutated Cancers 0 0
Condition category
Condition code
Cancer 0 0 0 0
Neuroendocrine tumour (NET)
Cancer 0 0 0 0
Stomach
Cancer 0 0 0 0
Bowel - Small bowel (duodenum and ileum)
Cancer 0 0 0 0
Bowel - Back passage (rectum) or large bowel (colon)
Cancer 0 0 0 0
Pancreatic
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Belzutifan

Experimental: Belzutifan - Belzutifan, 120 mg, oral, once daily (QD) until progressive disease or discontinuation.


Treatment: Drugs: Belzutifan
Belzutifan, 120 mg, oral, once daily (QD) until progressive disease or discontinuation.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Objective Response Rate (ORR) as Assessed by Blinded Independent Central Review (BICR)
Timepoint [1] 0 0
Up to approximately 5.5 years
Secondary outcome [1] 0 0
Duration of Response (DOR) as Assessed by BICR
Timepoint [1] 0 0
Up to approximately 5.5 years
Secondary outcome [2] 0 0
Time to Response (TTR) as Assessed by BICR
Timepoint [2] 0 0
Up to approximately 5.5 years
Secondary outcome [3] 0 0
Disease Control Rate (DCR) as Assessed by BICR
Timepoint [3] 0 0
Up to approximately 5.5 years
Secondary outcome [4] 0 0
Progressive Free Survival (PFS) as Assessed by BICR
Timepoint [4] 0 0
Up to approximately 5.5 years
Secondary outcome [5] 0 0
Overall Survival (OS)
Timepoint [5] 0 0
Up to approximately 5.5 years
Secondary outcome [6] 0 0
Number of Participants Experiencing Adverse Events (AEs)
Timepoint [6] 0 0
Up to approximately 5.5 years
Secondary outcome [7] 0 0
Number of Participants Discontinuing Study Drug due to an AE
Timepoint [7] 0 0
Up to approximately 5.5 years
Secondary outcome [8] 0 0
Time to Surgery (TTS)
Timepoint [8] 0 0
Up to approximately 5.5 years

Eligibility
Key inclusion criteria
The main inclusion criteria include but are not limited to the following:

* Male and female participants at least 12 years of age (at least 18 years of age for Cohort B1)
* Diagnosis of one of the following: Advanced/metastatic pheochromocytoma/paraganglioma (PPGL), pancreatic neuroendocrine tumors (pNET), von Hippel-Lindau (VHL) disease associated localized tumors, or advanced wild-type gastrointestinal stromal tumor (wt GIST) or advanced solid tumors with Hypoxia Inducible Factor- 2 alpha subunit (HIF-2a) related genetic alterations
* Cohort BI: VHL Disease-associated tumors:

* Have a diagnosis of VHL disease as determined by a germline test locally and/or clinical diagnosis
* Must be =18 years of age
* Has a life expectancy of at least 3 months

The main exclusion criteria include but are not limited to the following:

* Unable to swallow orally administered medication or has a disorder that might affect the absorption of belzutifan
* History of a second malignancy, unless potentially curative treatment has been completed with no evidence of malignancy for 2 years
* Any of the following: A pulse oximeter reading <92% at rest, or requires intermittent supplemental oxygen, or requires chronic supplemental oxygen
* Clinically significant cardiac disease, including unstable angina, acute myocardial infarction, or arterial bypass (CABG) or Percutaneous transluminal coronary angioplasty (PTCA) =6 months from study entry, or New York Heart Association Class III or IV congestive heart failure
* Received prior treatment (except somatostatin analogs) with chemotherapy, targeted therapy, biologics, or other investigational therapy within the past 4 weeks of first dose of study intervention
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/08/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
4/06/2029
Actual
Sample size
Target
322
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Prince of Wales Hospital-Medical Oncology ( Site 1601) - Randwick
Recruitment hospital [2] 0 0
The Royal Melbourne Hospital ( Site 1602) - Parkville
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Iowa
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania
Country [9] 0 0
United States of America
State/province [9] 0 0
Tennessee
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
Canada
State/province [11] 0 0
Alberta
Country [12] 0 0
Canada
State/province [12] 0 0
Ontario
Country [13] 0 0
Chile
State/province [13] 0 0
Region M. De Santiago
Country [14] 0 0
China
State/province [14] 0 0
Beijing
Country [15] 0 0
China
State/province [15] 0 0
Guangdong
Country [16] 0 0
China
State/province [16] 0 0
Shanghai
Country [17] 0 0
China
State/province [17] 0 0
Sichuan
Country [18] 0 0
Denmark
State/province [18] 0 0
Hovedstaden
Country [19] 0 0
Denmark
State/province [19] 0 0
Syddanmark
Country [20] 0 0
France
State/province [20] 0 0
Alsace
Country [21] 0 0
France
State/province [21] 0 0
Bouches-du-Rhone
Country [22] 0 0
France
State/province [22] 0 0
Ile-de-France
Country [23] 0 0
France
State/province [23] 0 0
Paris
Country [24] 0 0
France
State/province [24] 0 0
Rhone-Alpes
Country [25] 0 0
Germany
State/province [25] 0 0
Bayern
Country [26] 0 0
Germany
State/province [26] 0 0
Brandenburg
Country [27] 0 0
Germany
State/province [27] 0 0
Nordrhein-Westfalen
Country [28] 0 0
Germany
State/province [28] 0 0
Berlin
Country [29] 0 0
Hungary
State/province [29] 0 0
Budapest
Country [30] 0 0
Israel
State/province [30] 0 0
Ramat Gan
Country [31] 0 0
Israel
State/province [31] 0 0
Tel Aviv
Country [32] 0 0
Italy
State/province [32] 0 0
Campania
Country [33] 0 0
Italy
State/province [33] 0 0
Bologna
Country [34] 0 0
Italy
State/province [34] 0 0
Brescia
Country [35] 0 0
Italy
State/province [35] 0 0
Milano
Country [36] 0 0
Italy
State/province [36] 0 0
Verona
Country [37] 0 0
Japan
State/province [37] 0 0
Hokkaido
Country [38] 0 0
Japan
State/province [38] 0 0
Kanagawa
Country [39] 0 0
Japan
State/province [39] 0 0
Kochi
Country [40] 0 0
Japan
State/province [40] 0 0
Tokyo
Country [41] 0 0
Japan
State/province [41] 0 0
Kyoto
Country [42] 0 0
Korea, Republic of
State/province [42] 0 0
Seoul
Country [43] 0 0
Netherlands
State/province [43] 0 0
Utrecht
Country [44] 0 0
Norway
State/province [44] 0 0
Oslo
Country [45] 0 0
Russian Federation
State/province [45] 0 0
Baskortostan, Respublika
Country [46] 0 0
Russian Federation
State/province [46] 0 0
Leningradskaya Oblast
Country [47] 0 0
Russian Federation
State/province [47] 0 0
Moskva
Country [48] 0 0
Singapore
State/province [48] 0 0
Central Singapore
Country [49] 0 0
Spain
State/province [49] 0 0
Asturias
Country [50] 0 0
Spain
State/province [50] 0 0
Madrid, Comunidad De
Country [51] 0 0
Spain
State/province [51] 0 0
Barcelona
Country [52] 0 0
Sweden
State/province [52] 0 0
Skane Lan
Country [53] 0 0
Sweden
State/province [53] 0 0
Stockholms Lan
Country [54] 0 0
Sweden
State/province [54] 0 0
Uppsala Lan
Country [55] 0 0
Sweden
State/province [55] 0 0
Vastra Gotalands Lan
Country [56] 0 0
Turkey
State/province [56] 0 0
Adana
Country [57] 0 0
Turkey
State/province [57] 0 0
Izmir
Country [58] 0 0
Turkey
State/province [58] 0 0
Ankara
Country [59] 0 0
Turkey
State/province [59] 0 0
Istanbul
Country [60] 0 0
United Kingdom
State/province [60] 0 0
Cambridgeshire
Country [61] 0 0
United Kingdom
State/province [61] 0 0
England
Country [62] 0 0
United Kingdom
State/province [62] 0 0
Glasgow City
Country [63] 0 0
United Kingdom
State/province [63] 0 0
London, City Of

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Merck Sharp & Dohme LLC
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Merck Sharp & Dohme LLC
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Toll Free Number
Address 0 0
Country 0 0
Phone 0 0
1-888-577-8839
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04924075