Trial Outcomes & Findings for Sunitinib Malate to Treat Advanced Eye Disease in Patients With Von Hippel-Lindau Syndrome (NCT NCT00673816)
NCT ID: NCT00673816
Last Updated: 2024-01-05
Results Overview
Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
2 participants
Primary outcome timeframe
Baseline and 36 Weeks
Results posted on
2024-01-05
Participant Flow
The recruitment goal was to enroll five participants; however, the study was terminated after only two participants had been enrolled as a result of slow recruitment and adverse events. Date of enrollment of the first participant was December 10, 2008, and the study was terminated on December 1, 2010.
Participant milestones
| Measure |
Sunitinib Malate
Participants were expected to receive 9 months of sunitinib malate therapy administered in 6 cycles. Each cycle consisted of a daily oral dose of 50 mg sunitinib malate for 4 weeks followed by a 2-week rest period). Only one participant remained in the study for the Week 36 measures.
|
|---|---|
|
Overall Study
STARTED
|
2
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Sunitinib Malate
Participants were expected to receive 9 months of sunitinib malate therapy administered in 6 cycles. Each cycle consisted of a daily oral dose of 50 mg sunitinib malate for 4 weeks followed by a 2-week rest period). Only one participant remained in the study for the Week 36 measures.
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
Sunitinib Malate to Treat Advanced Eye Disease in Patients With Von Hippel-Lindau Syndrome
Baseline characteristics by cohort
| Measure |
Sunitinib Malate
n=2 Participants
Participants were expected to receive 9 months of sunitinib malate therapy administered in 6 cycles. Each cycle consisted of a daily oral dose of 50 mg sunitinib malate for 4 weeks followed by a 2-week rest period).
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
48.5 years
FULL_RANGE 4.95 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and 36 WeeksVisual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20.
Outcome measures
| Measure |
Left Eye
n=1 Participants
|
Right Eye
n=1 Participants
|
|---|---|---|
|
Change in Best Corrected Visual Acuity (BCVA) From Baseline to Week 36
|
1 ETDRS Letters
|
-5 ETDRS Letters
|
SECONDARY outcome
Timeframe: Baseline and 36 WeeksRetinal thickness was assessed by spectral-domain optical coherence tomography (Cirrus HD-OCT; Carl Zeiss Meditec, Dublin, CA), a non-invasive imaging technique that uses long-wavelength light to capture micrometer-resolution cross-sectional images from biological tissue.
Outcome measures
| Measure |
Left Eye
n=1 Participants
|
Right Eye
n=1 Participants
|
|---|---|---|
|
Change in Retinal Thickness From Baseline to Week 36
|
116 µm
|
1 µm
|
SECONDARY outcome
Timeframe: Baseline and 36 WeeksNumber of participants with improvement in leakage intensity.
Outcome measures
| Measure |
Left Eye
n=2 Participants
|
Right Eye
|
|---|---|---|
|
Reduction in Leakage Intensity as Measured by Fluorescein Angiography From Baseline to Week 36
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: Baseline and 36 WeeksPopulation: Microperimetry was not accurate for any participants due to fixation issues so no data was collected. As such, zero participants were analyzed for this outcome.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Duration of studyCount of participants with new non-ocular lesions present since baseline at any follow-up visit.
Outcome measures
| Measure |
Left Eye
n=2 Participants
|
Right Eye
|
|---|---|---|
|
Changes in Non-ocular VHL Lesion Status
Renal · New Lesions Since Baseline
|
0 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Renal · No New Lesions Since Baseline
|
1 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Renal · N/A - Lesions Present at Baseline
|
1 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Pancreatic · New Lesions Since Baseline
|
0 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Pancreatic · No New Lesions Since Baseline
|
1 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Pancreatic · N/A - Lesions Present at Baseline
|
1 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Central Nervous System Hemangioblastoma · New Lesions Since Baseline
|
0 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Central Nervous System Hemangioblastoma · No New Lesions Since Baseline
|
1 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Central Nervous System Hemangioblastoma · N/A - Lesions Present at Baseline
|
1 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Pheochromocytomas · New Lesions Since Baseline
|
0 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Pheochromocytomas · No New Lesions Since Baseline
|
2 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Pheochromocytomas · N/A - Lesions Present at Baseline
|
0 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Endolymphatic Sac Tumors · New Lesions Since Baseline
|
0 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Endolymphatic Sac Tumors · No New Lesions Since Baseline
|
2 Participants
|
—
|
|
Changes in Non-ocular VHL Lesion Status
Endolymphatic Sac Tumors · N/A - Lesions Present at Baseline
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Baseline and 36 WeeksNumber of participants with improvement in leakage area.
Outcome measures
| Measure |
Left Eye
n=2 Participants
|
Right Eye
|
|---|---|---|
|
Reduction in Leakage Area as Measured by Fluorescein Angiography at the Week 36 Visit
|
0 Participants
|
—
|
Adverse Events
Sunitinib Malate
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Sunitinib Malate
n=2 participants at risk
Participants were expected to receive 9 months of sunitinib malate therapy administered in 6 cycles. Each cycle consisted of a daily oral dose of 50 mg sunitinib malate for 4 weeks followed by a 2-week rest period). Only one participant remained in the study for the Week 36 measures.
|
|---|---|
|
Reproductive system and breast disorders
Vaginal haemorrhage
|
50.0%
1/2 • Number of events 2
|
|
Gastrointestinal disorders
Diarrhoea
|
50.0%
1/2 • Number of events 3
|
|
Nervous system disorders
Dizziness
|
50.0%
1/2 • Number of events 1
|
|
General disorders
Fatigue
|
100.0%
2/2 • Number of events 3
|
|
Gastrointestinal disorders
Sensitivity of teeth
|
50.0%
1/2 • Number of events 2
|
|
General disorders
Chills
|
100.0%
2/2 • Number of events 5
|
|
Gastrointestinal disorders
Stomatitis
|
50.0%
1/2 • Number of events 2
|
|
General disorders
Temperature intolerance
|
50.0%
1/2 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Nail disclouration
|
50.0%
1/2 • Number of events 1
|
|
Investigations
Urine analysis abnormal
|
50.0%
1/2 • Number of events 1
|
|
Investigations
Alanine aminotransferase increased
|
50.0%
1/2 • Number of events 1
|
|
Investigations
Blood creatine phosphokinase decreased
|
50.0%
1/2 • Number of events 1
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
50.0%
1/2 • Number of events 1
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
50.0%
1/2 • Number of events 1
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
100.0%
2/2 • Number of events 4
|
|
Vascular disorders
Hypertension
|
50.0%
1/2 • Number of events 2
|
|
Investigations
Blood bilirubin increased
|
50.0%
1/2 • Number of events 1
|
|
Endocrine disorders
Hypothyroidism
|
50.0%
1/2 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash
|
100.0%
2/2 • Number of events 2
|
|
Surgical and medical procedures
Tooth extraction
|
50.0%
1/2 • Number of events 1
|
|
Investigations
Platelet count decreased
|
100.0%
2/2 • Number of events 3
|
|
Investigations
Blood creatine decreased
|
50.0%
1/2 • Number of events 1
|
|
Infections and infestations
Urinary tract infection
|
50.0%
1/2 • Number of events 1
|
|
Eye disorders
Cataract
|
50.0%
1/2 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Onychoclasis
|
50.0%
1/2 • Number of events 1
|
|
Nervous system disorders
Hyperaesthesia
|
50.0%
1/2 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Hair disorder
|
50.0%
1/2 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
50.0%
1/2 • Number of events 1
|
|
Nervous system disorders
Dysgeusia
|
50.0%
1/2 • Number of events 3
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
50.0%
1/2 • Number of events 1
|
|
Investigations
Blood pressure increased
|
50.0%
1/2 • Number of events 1
|
|
Gastrointestinal disorders
Constipation
|
50.0%
1/2 • Number of events 1
|
|
Gastrointestinal disorders
Paraesthesia oral
|
50.0%
1/2 • Number of events 1
|
|
General disorders
Asthenia
|
50.0%
1/2 • Number of events 1
|
|
Investigations
Blood alkaline phosphatase increased
|
50.0%
1/2 • Number of events 1
|
|
Investigations
Blood lactate dehydrogenase increased
|
50.0%
1/2 • Number of events 1
|
|
Investigations
Weight decreased
|
50.0%
1/2 • Number of events 1
|
|
Ear and labyrinth disorders
Vertigo
|
50.0%
1/2 • Number of events 1
|
|
Gastrointestinal disorders
Dyspepsia
|
50.0%
1/2 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
50.0%
1/2 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
50.0%
1/2 • Number of events 2
|
|
Gastrointestinal disorders
Glossodynia
|
50.0%
1/2 • Number of events 2
|
|
Gastrointestinal disorders
Lip pain
|
50.0%
1/2 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
50.0%
1/2 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Skin discomfort
|
50.0%
1/2 • Number of events 2
|
|
Psychiatric disorders
Sleep disorder
|
50.0%
1/2 • Number of events 1
|
|
Gastrointestinal disorders
Oral discomfort
|
50.0%
1/2 • Number of events 1
|
|
Gastrointestinal disorders
Glossitis
|
50.0%
1/2 • Number of events 1
|
|
Metabolism and nutrition disorders
Decreased appetite
|
50.0%
1/2 • Number of events 1
|
|
Investigations
Blood thyroid stimulating hormone increased
|
50.0%
1/2 • Number of events 1
|
|
Investigations
Red blood cell count decreased
|
50.0%
1/2 • Number of events 1
|
|
Investigations
White blood cell count decreased
|
50.0%
1/2 • Number of events 1
|
|
Nervous system disorders
Headache
|
100.0%
2/2 • Number of events 5
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place