Belzutifan for von Hippel-Lindau Disease: Pancreatic Lesion Population of the Phase 2 LITESPARK-004 Study.
Tobias Else, Eric Jonasch, Othon Iliopoulos...
https://pubmed.ncbi.nlm.nih.gov/38393723Actively Recruiting
Phase 2
Age: 14Years +
All Genders
ID07167329
The BELIEVE-VHL Trial: Real-World Study on Belzutifan's Effectiveness and Pharmacogenetics in Von Hippel-Lindau Syndrome
Led by José Claudio Casali da Rocha · Updated on 2025-09-11
100
Participants Needed
1
Research Sites
204 weeks
Total Duration
On this page
Sponsors
J
José Claudio Casali da Rocha
Lead Sponsor
A
AC Camargo Cancer Center
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are investigating the use of belzutifan, a HIF2α inhibitor, in people with von Hippel-Lindau (VHL) syndrome, a condition that causes tumors to grow in different parts of the body. This Phase 2 trial aims to evaluate how belzutifan affects tumors, its benefits, possible side effects, timing of treatment response, and disease progression in a real-world setting. The study also explores genetic factors influencing treatment response and conducts a pharmacoeconomic analysis to understand the impact on healthcare costs. Participants with VHL syndrome who have measurable or progressive tumors will receive oral belzutifan. The study monitors hemoglobin and erythropoietin levels during the first six months, assessing the need for erythropoietin supplements in cases of anemia, fatigue, or low oxygen. It also evaluates patient quality of life and perceptions regarding VHL syndrome using questionnaires. The treatment period includes up to 104 weeks of follow-up for tumor response and side effects. Throughout the study, participants will undergo regular assessments including tumor imaging by RECIST 1.1 criteria, blood tests for hemoglobin and erythropoietin, and quality-of-life questionnaires over two years. Researchers will track anemia incidence, need for blood transfusions, and the effects of erythropoietin supplementation on tumor growth. The trial also includes a retrospective and prospective economic evaluation based on healthcare costs before and after starting belzutifan.
CONDITIONS
Brief Title
Real-World Effectiveness and Pharmacogenetics of Belzutifan in VHL Syndrome: The BELIEVE-VHL Trial
Who Can Participate
Age: 14Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Age 14 years or older
- Clinical or genetic confirmation of von Hippel-Lindau (VHL) syndrome
- Measurable or progressive VHL-associated tumors as defined by RECIST 1.1 or specific imaging
- Performance status ECOG 0-2
- Adequate bone marrow, liver, and kidney function
- Ability to swallow oral medication
- Provided written informed consent before enrollment
You will not qualify if you...
- Age under 14 years
- No confirmed diagnosis of von Hippel-Lindau syndrome
- Active malignancy outside VHL tumor types within past 3 years except certain cured skin or cervical cancers
- Allergy or hypersensitivity to belzutifan or its ingredients
- Severe or uncontrolled heart disease including recent heart attack, unstable angina, heart failure, or uncontrolled high blood pressure
- Active infections such as HIV, hepatitis B or C
- Immunosuppressed due to disease or treatment
- Significant bleeding disorders
- Radiotherapy within 4 weeks before enrollment
- Major surgery within 4 weeks before enrollment or need for urgent surgery
- Malabsorption from prior gastrointestinal surgery or active GI disease
- Use of medications that interact significantly with belzutifan
- Expected poor adherence or planned interruption of belzutifan therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - From enrollment and initiation of treatment until the earliest of either documented disease progression or death, with follow-up of up to 104 weeks.
Participants receive oral Belzutifan to treat lesions or neoplasms associated with von Hippel-Lindau syndrome. Treatment effects, benefits, and adverse effects are monitored during this time.
Regular visits during treatment up to 104 weeks
Trial Site Locations
Total: 1 location
1
AC Camargo Cancer Center
São Paulo, São Paulo, Brazil, 01509900
Actively Recruiting
Loading map...
Research Team
J
José Claudio Casali da Rocha, Head of Oncogenetics
J
José Reinaldo De Oliveira Junior
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here
Published Research Related To This Trial
Validity of Antonovsky's sense of coherence scale: a systematic review.
Monica Eriksson, Bengt Lindström
https://pubmed.ncbi.nlm.nih.gov/15911640Electronic Version of the EQ-5D Quality-of-Life Questionnaire: Adaptation to a Brazilian Population Sample.
Ângela Maria Bagattini, Suzi Alves Camey, Sandro René Miguel...
https://pubmed.ncbi.nlm.nih.gov/29754016Belzutifan for Renal Cell Carcinoma in von Hippel-Lindau Disease.
Eric Jonasch, Frede Donskov, Othon Iliopoulos...
https://pubmed.ncbi.nlm.nih.gov/34818478Inhibition of hypoxia-inducible factor-2α in renal cell carcinoma with belzutifan: a phase 1 trial and biomarker analysis.
Toni K Choueiri, Todd M Bauer, Kyriakos P Papadopoulos...
https://pubmed.ncbi.nlm.nih.gov/33888901A Review of Von Hippel-Lindau Syndrome.
Neha Varshney, Amanuel A Kebede, Harry Owusu-Dapaah...
https://pubmed.ncbi.nlm.nih.gov/28785532Benefits of screening in von Hippel-Lindau disease--comparison of morbidity associated with initial tumours in affected parents and children.
M Priesemann, K M Davies, L A Perry...
https://pubmed.ncbi.nlm.nih.gov/16651847Guidelines for surveillance of patients with von Hippel-Lindau disease: Consensus statement of the International VHL Surveillance Guidelines Consortium and VHL Alliance.
Anthony B Daniels, Amit Tirosh, Kristin Huntoon...
https://pubmed.ncbi.nlm.nih.gov/37337409Preventive medicine of von Hippel-Lindau disease-associated pancreatic neuroendocrine tumors.
Tobias Krauss, Alfonso Massimiliano Ferrara, Thera P Links...
https://pubmed.ncbi.nlm.nih.gov/29748190Comprehensive characterization and building of National Registry of von Hippel-Lindau disease in Brazil.
Tabatha Nakakogue Dallagnol, Eduardo Da Cás, Odery Ramos Junior...
https://pubmed.ncbi.nlm.nih.gov/36625343