A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease (Hestia2)

AstraZeneca

Status and phase

Completed

Phase 2

Conditions

Sickle Cell Disease

Treatments

Drug: Ticagrelor

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT02482298

D5136C00008

Details and patient eligibility

About

The purpose of this study is to determine whether ticagrelor is effective in reducing the number of days of pain, intensity of pain, and reducing the use of analgesics due to sickle cell disease

Full description

This is a randomised, double-blind, double-dummy, parallel-group, placebo-controlled, study evaluating 2 doses of ticagrelor in 90 patients aged 18 to 30 years, with sickle cell disease (SCD). Patients will be randomised to double-blind double-dummy treatment period in a 1:1:1 ratio (30 to each treatment group) to receive ticagrelor 10 mg twice daily (bid), or ticagrelor 45 mg bid, or placebo bid to determine the frequency of days with pain using an electronic diary (eDiary) every day. Approximately 180 patients will be enrolled. Patient will be followed for safety assessment during and after 2 weeks of treatment completion.

During the 16 week treatment period, patients will complete a daily eDiary concerning daily pain intensity, pain location, use of analgesics and absence from school or work. At the end of the study patients will be asked to rate the change in their sickle cell pain compared to the start of treatment. Platelet aggregation will be measured and reported as P2Y12 reaction units (PRU) pre-dose and 2 hours post-dose at week 4 and week 5 after treatment start. Pharmacokinetic (PK) parameters will be measured at 2 hours post-dose at week 4, and pre-dose and at 2 hours post-dose at week 5. Biomarkers will be assessed pre-dose at week 4, week 5 and week 8. During the study, patients will be evaluated for adverse events (AEs) including bleeding and vaso-occlusive crisis (VOC).

Enrollment

87 patients

Sex

All

Ages

18 to 30 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Confirmed medical history or diagnosis of homozygous sickle cell (HbSS) or sickle beta-zero-thalassaemia (HbS/β0) by HPLC
If treated with hydroxyurea, the dose must have been stable for 3 months

Exclusion criteria

History of transient ischaemic attack or clinically overt cerebrovascular accident
Moderate or severe hepatic impairment
Treatment with chronic red blood cell transfusion therapy
Pre-dominate cause of pain is not sickle cell disease related
Chronic treatment with anticoagulants or antiplatelet drugs.