Von Hippel-Lindau DiseasePhase 2VHLSUTNCT01168440
Study of Sunitinib in Patients With Von Hippel-Lindau (VHL) Disease
A Phase 2 study testing Sunitinib in people with Von Hippel-Lindau Disease, run by Association Pour La Recherche des Thérapeutiques Innovantes en Cancérologie at 3 sites in France.
Trial summary
VHL patients may benefit from sunitinib. This study will investigate the following objectives : PRIMARY OBJECTIVE To determine the objective response rate according to RECIST criteria, in VHL patients with advanced tumors or tumors untreatable by other means, and treated with sunitinib. Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Von Hippel-Lindau Disease
- What is being tested?
- Sunitinib
- How many people
- 5 participants
- Where
- 3 sitesFrance
- Study dates
- Mar 2010to Feb 2011
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 90 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must have genetically or clinically confirmed VHL disease and have symptoms from VHL that are no longer controllable by conventional approaches.
- Patients must have at least one of the following lesions :
- Eye : retinal hemangioblastoma that can no longer be treated by laser therapy or cryotherapy and resulting in progressive loss of vision;
- CNS : cerebellar, bulbar, spinal, or cerebellopontine angle haemangioblastoma or endolymphatic sac tumor causing neurological symptoms that are not amenable to further surgery, or have recurred after a first surgery;
- Kidney: multiple or bilateral tumors not accessible to conservative surgery, or tumors having recurred after surgery and/or radiofrequency ablation or advanced/metastatic RCC;
- Pancreas: inextirpable or advanced neuroendocrine tumors.
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- Patients previously treated for VHL with surgery, chemotherapy or radiotherapy are considered eligible for this study under the condition that these treatments were completed more than 4 weeks prior starting the study treatment. Previously radiated lesions will be considered as target lesions only if they demonstrate unequivocal evidence of growth upon imagery.
- Male or female, at least 18 year-old.
- Performance status ECOG 0-2
- Life expectancy = 3 months
- Biological/clinical values within the following limits:
- Total serum bilirubin = 1.5 x ULN (patients with Gilbert's disease are not eligible)
- Serum transaminases and alkaline phosphatases = 2.5 x ULN, or in case of underlying malignancy (hepatic metastasis) = 5x ULN
- Serum creatinine = 1.5 x ULN, creatinine clearance = 80 ml/min
- Absolute neutrophil count = 1500/mm3
- Platelets = 100,000/mm3
- Hemoglobin = 9.0 g/dL
- QTc interval = 450 msec
- Left ventricular ejection fraction (LVEF) = lower limit of institutional normal as assessed by multigated acquisition (MUGA) scan or echocardiogram
- Eligibility of patients receiving any medications or substances which may alter the activity or pharmacokinetics of sunitinib (CYP3A4 inhibitors or inducers among which ketoconazole, theophylline, phenobarbital, coumadin/warfarin) will be decided after review by the principal investigator of possibility to interrupt or switch to other medications. Otherwise, patient is not eligible. Anticoagulants drugs (among which coumadin/warfarin) may be, either switched to low-molecular-weight heparin, or be subject to individual dose adaptation in order to maintain INR in the target range with regard to patient's history, all along his participation in the study.
- Signed and dated informed consent document stating that the patient, or legally representative, has been informed of all the aspects of the trial prior to enrollment
- Willingness and ability to comply with all protocol assessments, schedule of visits, and procedures that are or could be requested as part of this study.
- Affiliated to French social security system
Who cannot join
- Chemotherapy, radiotherapy, radiofrequency or surgery within 4 weeks prior to entering the study or not complete recovering from adverse events due to drugs administered more than 4 weeks earlier.
- Patients receiving any other investigational agent or having participated in a clinical trial in the last 30 days.
- History of allergic reaction attributed to compounds of similar chemical or biological composition to sunitinib.
- Previous treatment with sunitinib
- NCI-CTCAE grade = 3 hemorrhage within 4 weeks prior to study entry.
- History of known or suspected brain metastases, spinal cord compression, carcinomatous meningitis, evidence of leptomeningeal disease (excepted leptomeningeal hemangioblastoma, according to the neurologist) on screening CT scan or MRI.
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- Any of the following within the 6 months prior to study drug administration: symptomatic congestive heart failure, myocardial infarction or coronary artery bypass, pulmonary embolism, ongoing severe or unstable angina pectoris, NCI-CTCAE grade = 2 cardiac dysrhythmia, cerebrovascular accident or transient ischemic attack.
- Hypertension >140/90 mmHg that cannot be controlled despite optimal antihypertensive therapy.
- Pre-existing thyroid abnormality with thyroid function that cannot be maintained in the normal range despite medication.
- Other severe acute or chronic medical condition including (but not limited to), ongoing infection, unstable or uncompensated respiratory, cardiac, hepatic or renal disease, psychiatric condition, laboratory abnormality that would impart, in the judgment of the investigator, excess risk associated with study participation or study drug administration, or which would make the patient inappropriate for entry into the trial.
- Any medical condition (gastric or small intestine pathology, malabsorption syndrome) that might interfere with oral medication absorption.
- Known HIV-positive patients treated with antiretroviral therapy (potential pharmacokinetic interactions with sunitinib).
- Pregnancy or breastfeeding. Patients must agree to use effective contraception during the study, including oral contraceptives, intrauterine devices, or being unable to procreate.
- Any other malignancy within the last 3 years excepted basal cell carcinoma, in situ cervical carcinoma, squamous cell skin cancer, pT1/a bladder cancer with no evidence of recurrence during the last 12 months.
Trial locations and contacts
3 sites in 1 country.
France3 sites
Hospital Saint André - Service de cancérologie
Bordeaux, 33075
Hopital Kremlin-Bicêtre
Le Kremlin-Bicêtre, 94275
Hôpital Européen Georges Pompidou - Service d'Oncologie Médicale
Paris, 75015
About this study
Official title: A Single-arm, Phase II Study of Sunitinib in Patients With Von Hippel-Lindau (VHL) Disease
VHL patients may benefit from sunitinib. This study will investigate the following objectives :
PRIMARY OBJECTIVE
- To determine the objective response rate according to RECIST criteria, in VHL patients with advanced tumors or tumors untreatable by other means, and treated with sunitinib.
SECONDARY OBJECTIVES
- To evaluate the safety and tolerability of sunitinib in VHL patients according to the NCI-CTC criteria Version 3.0.
- To determine the following time-to-event endpoints: overall survival, time to disease progression, progression free survival, time to response and duration of response.
- To evaluate quality of life in VHL patients receiving sunitinib.
Read the detailed description
Treatment with sunitinib, 50 mg PO daily, 6 weeks courses with schedule 4/2 (4 weeks of treatment followed by 2 weeks of rest).
Treatment until disease progression or unacceptable toxicity.
Dose reduction depending on type and severity of toxicity. At the end of treatment period (after 8 courses), responding and well tolerating patients will be allowed to receive sunitinib upon investigator's opinion.
Follow-up for up to 24 months from inclusion.
- Conditions
- Von Hippel-Lindau Disease
- Being tested
- Sunitinib
- Keywords
- von Hippel-Lindau, VHL, sunitinib
- Medical subject headings
- von Hippel-Lindau Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SunitinibDrug
Sunitinib, 50 mg PO daily, 6 weeks courses with schedule 4/2 (4 weeks of treatment followed by 2 weeks of rest).
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective response rate (RECIST criteria)
Course: 4 weeks sunitinib / 2weeks rest. Response assessment: after 4 weeks and 8 weeks of sunitinib (1 extra assessment by contrast-enhanced US after 2 weeks for kidney tumors). Then every 6 weeks for eye and every 12 weeks for all other tumors.
Measured: Every 6 weeks
Secondary outcomes (3)
Safety and tolerability (NCI-CTC criteria Version 3.0).
Measured: Every 6 weeks
Time-to-event endpoints: overall survival, time to disease progression, progression free survival, time to response and duration of response.
Measured: Every 6 weeks
Quality of life in VHL patients receiving sunitinib.
Measured: Every 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2010 (estimated)
- Study startMar 2010
- Primary completionFeb 2011
- Study completionFeb 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Hippel-Lindau DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Association Pour La Recherche des Thérapeutiques Innovantes en Cancérologie
- Sponsor type
- University, hospital or other
- Investigators
- Stephane RICHARD, MD, PhD, Principal investigator, Hôpital Kremlin-Bicêtre (France)Reza T ELAIDI, PhD, Study director, ARTIC (Hopital Européen Georges Pompidou, FRANCE)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01168440
- Sponsor's protocol ID
- VHLSUT09
- Acronym
- VHLSUT
- Status last verified
- Sep 2012
- First submitted
- 18 Mar 2010
- Last updated
- 13 Sep 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm, Phase II Study of Sunitinib in Patients With Von Hippel-Lindau (VHL) Disease
- ClinicalTrials.gov ID
- NCT01168440
- Acronym / sponsor ID
- VHLSUT / VHLSUT09
- Conditions
- Von Hippel-Lindau Disease
- Keywords
- von Hippel-Lindau, VHL, sunitinib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Association Pour La Recherche des Thérapeutiques Innovantes en CancérologieUniversity, hospital or other
- Study start
- Mar 2010
- Primary completion
- Feb 2011 (actual)
- Study completion
- Feb 2011 (actual)
- First posted
- 23 Jul 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
VHL patients may benefit from sunitinib. This study will investigate the following objectives :
PRIMARY OBJECTIVE
- To determine the objective response rate according to RECIST criteria, in VHL patients with advanced tumors or tumors untreatable by other means, and treated with sunitinib.
SECONDARY OBJECTIVES
- To evaluate the safety and tolerability of sunitinib in VHL patients according to the NCI-CTC criteria Version 3.0.
- To determine the following time-to-event endpoints: overall survival, time to disease progression, progression free survival, time to response and duration of response.
- To evaluate quality of life in VHL patients receiving sunitinib.
Detailed description
Treatment with sunitinib, 50 mg PO daily, 6 weeks courses with schedule 4/2 (4 weeks of treatment followed by 2 weeks of rest).
Treatment until disease progression or unacceptable toxicity.
Dose reduction depending on type and severity of toxicity. At the end of treatment period (after 8 courses), responding and well tolerating patients will be allowed to receive sunitinib upon investigator's opinion.
Follow-up for up to 24 months from inclusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- Patients must have genetically or clinically confirmed VHL disease and have symptoms from VHL that are no longer controllable by conventional approaches.
- Patients must have at least one of the following lesions :
- Patients previously treated for VHL with surgery, chemotherapy or radiotherapy are considered eligible for this study under the condition that these treatments were completed more than 4 weeks prior starting the study treatment. Previously radiated lesions will be considered as target lesions only if they demonstrate unequivocal evidence of growth upon imagery.
- Male or female, at least 18 year-old.
- Performance status ECOG 0-2
Key exclusion criteria (14 listed)
- Chemotherapy, radiotherapy, radiofrequency or surgery within 4 weeks prior to entering the study or not complete recovering from adverse events due to drugs administered more than 4 weeks earlier.
- Patients receiving any other investigational agent or having participated in a clinical trial in the last 30 days.
- History of allergic reaction attributed to compounds of similar chemical or biological composition to sunitinib.
- Previous treatment with sunitinib
- NCI-CTCAE grade = 3 hemorrhage within 4 weeks prior to study entry.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SunitinibSunitinib, 50 mg PO daily, 6 weeks courses with schedule 4/2 (4 weeks of treatment followed by 2 weeks of rest). | Drug | – |
Primary outcome
Objective response rate (RECIST criteria)
Time frame: Every 6 weeks
Course: 4 weeks sunitinib / 2weeks rest. Response assessment: after 4 weeks and 8 weeks of sunitinib (1 extra assessment by contrast-enhanced US after 2 weeks for kidney tumors). Then every 6 weeks for eye and every 12 weeks for all other tumors.
3 secondary outcomes
Safety and tolerability (NCI-CTC criteria Version 3.0).
Time frame: Every 6 weeks
Time-to-event endpoints: overall survival, time to disease progression, progression free survival, time to response and duration of response.
Time frame: Every 6 weeks
Quality of life in VHL patients receiving sunitinib.
Time frame: Every 6 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2012.
Related trials for Von Hippel-Lindau Disease
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stephane RICHARD, MD, PhD principal investigator
Hôpital Kremlin-Bicêtre (France)
Reza T ELAIDI, PhD study director
ARTIC (Hopital Européen Georges Pompidou, FRANCE)
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sunitinib in people with Von Hippel-Lindau Disease, run by Association Pour La Recherche des Thérapeutiques Innovantes en Cancérologie at 3 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 23 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Sep 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective response rate (RECIST criteria) (time frame: Every 6 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Sep 2012.