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Tyrosine Kinase Inhibitor

Evaluation of Pazopanib on Bleeding in Subjects With Hereditary Haemorrhagic Telangiectasia

Phase 2

Waitlist Available

Research Sponsored by GlaxoSmithKline

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 1.5, week 3, week 4.5, week 6, week 7.5, week 9, week 10.5, week 12, week 16, week 20, week 24 and week 28

Awards & highlights

All Individual Drugs Already Approved

Approved for 5 Other Conditions

Summary

This study will investigate whether pazopanib can reduce epistaxis and improve anaemia in subjects with hereditary haemorrhagic telangiectasia (HHT) at a dose that is well tolerated. The study will have 2 parts. Part A will be an open label, dose-escalation study in which up to 4 cohorts of approximately 6 subjects each will receive increasing doses of pazopanib for a maximum of 12 weeks. The dose in the first cohort will be 50mg per day and the maximum dose in a cohort will be 400 mg per day. Dose escalation will not occur as planned if the predefined safety stopping criteria are met or at least 4 subjects in a cohort have demonstrated efficacy (as measured by epistaxis, haemoglobin, transfusion or iron infusion requirements). If efficacy is demonstrated in Part A with an acceptable safety profile, Part B will be initiated to further define the optimal dose(s) including dose duration/schedule and to provide further support for the proof of mechanism. Approximately 15 subjects will participate and will be randomised to active or placebo in a ratio of 3:2. This part of the study will be double-blind.

Eligible Conditions

Osler-Weber-Rendu Syndrome
Hereditary Hemorrhagic Telangiectasia (HHT)

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 1.5, week 3, week 4.5, week 6, week 7.5, week 9, week 10.5, week 12, week 16, week 20, week 24 and week 28

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 1.5, week 3, week 4.5, week 6, week 7.5, week 9, week 10.5, week 12, week 16, week 20, week 24 and week 28

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 1.5, week 3, week 4.5, week 6, week 7.5, week 9, week 10.5, week 12, week 16, week 20, week 24 and week 28 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change From Baseline in Epistaxis Severity Score at the Indicated Time Points

Hemoglobin

Change From Baseline in the Average of the Last 3 Hemoglobin Measures in the Dosing Period (Week 9, Week 10.5 and Week 12)

+6 more

Secondary study objectives

Change From Baseline in Ferritin at the Indicated Time Points

Change From Baseline in the Average of the Last 3 Ferritin Measures in the Dosing Period (Week 9, Week 10.5 and Week 12)

Graphical Exploration of PK/Pharmacodynamic (PD) Relationships Between Pazopanib Exposure and Selected PD

+8 more

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Part B-Dose Optimization phaseExperimental Treatment2 Interventions

If efficacy is demonstrated in Part A with an acceptable safety profile, Part B will be initiated to further define the optimal dose(s) including dose duration/schedule and to provide further support for the proof of mechanism. Approximately 15 subjects will participate and will be randomised to active or placebo in a ratio of 3:2. This part of the study will be double-blind

Group II: Part A- Dose Escalation phaseExperimental Treatment1 Intervention

Part A will be an open label, dose-escalation study in which 4 cohorts of approximately 6 subjects will receive increasing doses of pazopanib for a maximum of 12 weeks. The dose in the first cohort will be 50mg per day and the maximum dose in a cohort will be 400 mg per day. Dose escalation will not occur if the predefined safety stopping criteria are met or at least 4 subjects in a cohort have demonstrated efficacy. Cohort 4 receiving 400 mg dosing schedule may involve cycles of up to 3 weeks of active treatment, followed by up to 3 weeks wash-out (instead of 12 weeks continuous dosing). Decision will be based on safety data obtained from lower doses

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pazopanib

FDA approved

Placebo

1995

Completed Phase 3

~2670

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