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Tyrosine Kinase Inhibitor

Pazopanib for Von Hippel-Lindau Disease

Phase 2

Waitlist Available

Led By Eric Jonasch

Research Sponsored by M.D. Anderson Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

At least one measurable VHL related lesion, which is undergoing surveillance, and patient is not at immediate risk of needing intervention for this or other lesions; biopsy is not required given the known likely etiology and natural history in the setting of a positive genetic test

Female subjects must meet specific criteria related to childbearing potential and contraception

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 24 weeks

Awards & highlights

Study Summary

This trial looks at the side effects and effectiveness of pazopanib hydrochloride for patients with von Hippel-Lindau syndrome. Pazopanib hydrochloride may stop cancer cell growth by blocking some enzymes needed for cell growth.

Who is the study for?

This trial is for individuals with Von Hippel-Lindau syndrome who have at least one measurable related lesion not requiring immediate intervention. Participants must be willing to follow the study's procedures, use contraception if applicable, and have certain blood parameters within specific ranges. Those with prior malignancies, significant gastrointestinal issues affecting drug absorption, recent surgeries or trauma, unstable medical conditions, or prohibited medication use are excluded.Check my eligibility

What is being tested?

The trial is testing Pazopanib Hydrochloride to see how well it works in treating patients with Von Hippel-Lindau syndrome by potentially blocking enzymes needed for cancer cell growth. It includes laboratory biomarker analysis to monitor effects.See study design

What are the potential side effects?

Possible side effects of Pazopanib Hydrochloride may include liver problems, high blood pressure, diarrhea, hair color changes, nausea or vomiting. Side effects can vary from person to person.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a VHL-related lesion that doesn't currently need intervention.

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I am following the required birth control guidelines due to my potential to conceive.

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I haven't taken any VHL-related treatments in the last 14 days or five half-lives of the drug before starting pazopanib.

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I have a genetic diagnosis of VHL or signs of VHL disease.

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I can take care of myself and am up and about more than half of my waking hours.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 24 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 24 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 24 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Drug discontinuation due to toxicity

Overall response rate (complete response + partial response)

Progressive disease rate

+1 more

Other outcome measures

Genetic mutations, discrete markers, and continuous biomarkers

Side effects data

From 2016 Phase 2 trial • 22 Patients • NCT01247571

79%

Nausea

74%

Fatigue

68%

Diarrhea

53%

Neutrophil Count Decreased

53%

White Blood Cell Decreased

53%

Hypertension

47%

Anemia

42%

Vomiting

42%

Peripheral Sensory Neuropathy

37%

Constipation

37%

Abdominal Pain

32%

Anorexia

32%

Headache

32%

Dyspnea

26%

Cough

21%

Weight Loss

21%

Alopecia

21%

Alanine Aminotransferase Increased

21%

Hyponatremia

21%

Hypoalbuminemia

21%

Pain In Extremity

21%

Depression

21%

Aspartate Aminotransferase Increased

16%

Blurred Vision

16%

Dysphagia

16%

Blood Bilirubin Increased

16%

Dry Mouth

16%

Alkaline Phosphatase Increased

16%

Hypomagnesemia

16%

Hypocalcemia

16%

Arthralgia

16%

Memory Impairment

16%

Insomnia

16%

Epistaxis

11%

Creatinine Increased

11%

Rectal Hemorrhage

11%

Hearing Impaired

11%

Platelet Count Decreased

11%

Gastroesophageal Reflux Disease

11%

Tinnitus

11%

Hypokalemia

11%

Hypoglycemia

11%

Hyperglycemia

11%

Neck Pain

11%

Generalized Muscle Weakness

11%

Dizziness

11%

Anxiety

11%

Vaginal Hemorrhage

11%

Vaginal Discharge

11%

Rash Maculo-Papular

11%

Skin Hypopigmentation

11%

Urinary Frequency

Ear Pain

Ascites

Lymphocyte Count Decreased

Dyspepsia

Bloating

Oral Pain

Fever

Bruising

Gastrointestinal Pain

Sinus Bradycardia

Sepsis

Sinusitis

Flatulence

Pain

Investigations - Other

Proctitis

Death Nos

Sinus Tachycardia

Reproductive System And Breast Disorders - Other

Mucositis Oral

Edema Trunk

Edema Limbs

Urinary Tract Infection

Dehydration

Back Pain

Arthritis

Paresthesia

Dysgeusia

Urinary Retention

Pelvic Pain

Allergic Rhinitis

Skin And Subcutaneous Tissue Disorders - Other

Nail Discoloration

Dry Skin

Proteinuria

100%

80%

60%

40%

20%

Study treatment Arm

Pazopanib

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (pazopanib hydrochloride)Experimental Treatment2 Interventions

Patients receive pazopanib hydrochloride PO QD on days 1-28. Treatment repeats every 4 weeks for up to 24 weeks in the absence of disease progression or unacceptable toxicity. Patients benefitting from treatment may continue pazopanib hydrochloride in the absence of disease progression.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pazopanib Hydrochloride

2009

Completed Phase 2

~820

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

M.D. Anderson Cancer CenterLead Sponsor

2,971 Previous Clinical Trials

1,787,205 Total Patients Enrolled

4 Trials studying Von Hippel-Lindau Disease

150 Patients Enrolled for Von Hippel-Lindau Disease

National Cancer Institute (NCI)NIH

13,658 Previous Clinical Trials

40,924,457 Total Patients Enrolled

9 Trials studying Von Hippel-Lindau Disease

7,277 Patients Enrolled for Von Hippel-Lindau Disease

Eric JonaschPrincipal InvestigatorM.D. Anderson Cancer Center

4 Previous Clinical Trials

219 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many participants have been enrolled in this trial thus far?

"Unfortunately, this medical study is not presently accepting patients. It was initially posted on January 17th 2012 and revised most recently on October 11th 2022. If you are seeking alternative trials to participate in, there exist 1386 studies for von hippel-lindau disease as well as 24 related to Pazopanib Hydrochloride which are currently enrolling participants."

Answered by AI

Has Pazopanib Hydrochloride been authorized for release by the FDA?

"The safety of Pazopanib Hydrochloride is estimated to be a 2 on the scale of 1-3. This is because Phase 2 trials have not generated any confirmations in regards to efficacy, but there are some indications that it can provide safety benefits."

Answered by AI

Is the enrollment period for this clinical trial still open?

"This medical trial is no longer accepting patients. The first posting of the study was on January 17th 2012 and it was last modified on October 11th 2022. However, for those interested in other studies related to von hippel-lindau disease or Pazopanib Hydrochloride, there are currently 1386 trials recruiting participants and 24 respective clinical studies that remain active."

Answered by AI

Has this medical research been conducted before?

"At present, 24 studies for Pazopanib Hydrochloride are still active and can be found in 23 countries across 608 cities. The initial trial was launched by Novartis in 2011 and it successfully passed Phase 2 drug approval with 180 participants involved. Since then, another 726 trials have been conducted on the medication."

Answered by AI

Recent research and studies

The Lancet OncologyJournal

Pazopanib in Patients with Von Hippel-lindau Disease: A Single-arm, Single-centre, Phase 2 Trial

Jonasch, Eric, Ian E McCutcheon, Dan S Gombos, Kamran Ahrar, Nancy D Perrier, Diane Liu, Christine C Robichaux, et al.. 2018. “Pazopanib in Patients with Von Hippel-lindau Disease: A Single-arm, Single-centre, Phase 2 Trial”. The Lancet Oncology. Elsevier BV. doi:10.1016/s1470-2045(18)30487-x.

clinicaltrials.govStudy

Pazopanib Hydrochloride in Treating Patients With Von Hippel-Lindau Syndrome

2012. "Pazopanib Hydrochloride in Treating Patients With Von Hippel-Lindau Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01436227.