Clinical trial NCT02482298: A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease
Official title: A Randomised, Double-blind, Double-dummy, Parallel-group, Multicenter, Phase IIb Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing the Number of Days With Pain in Young Adults With Sickle Cell Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sickle Cell Disease
Keywords: Sickle cell disease
Young adults
Hestia2
Ticagrelor
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
First posted: 2015-06-26
Enrollment: 87
Sex: ALL
Minimum age (years): 18
Maximum age (years): 30
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

Brief summary: 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

Detailed 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).

Eligibility criteria: 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.

Primary outcomes:
measure: Change in Proportion of Days With Pain Due to Sickle Cell Disease as Measured by an eDiary
timeFrame: Baseline through Week 12
Secondary outcomes:
measure: Average of the Daily Worst Pain Values Reported Via eDiary
timeFrame: Baseline through Week 12
measure: Change in Proportion of Days With Analgesic Use Measured by an eDiary
timeFrame: Baseline through Week 12
Other outcomes:
measure: Number of Major Bleeding or Clinically Relevant Non-major Bleeding Events (Patients)
timeFrame: Baseline through Week 12
measure: Number of Major Bleeding or Clinically Relevant Non-major Bleeding Events (Events)
timeFrame: Baseline through Week 12
Locations:
city: Miami
country: United States
facility: Research Site
state: Florida
city: Bethesda
country: United States
facility: Research Site
state: Maryland
city: Charleston
country: United States
facility: Research Site
state: South Carolina
city: Alexandria
country: Egypt
facility: Research Site
city: Cairo
country: Egypt
facility: Research Site
city: Cairo
country: Egypt
facility: Research Site
city: Bordeaux Cedex
country: France
facility: Research Site
city: Strasbourg
country: France
facility: Research Site
city: Verona
country: Italy
facility: Research Site
city: Kikuyu
country: Kenya
facility: Research Site
city: Kisian
country: Kenya
facility: Research Site
city: Nairobi
country: Kenya
facility: Research Site
city: Beirut
country: Lebanon
facility: Research Site
city: Beirut
country: Lebanon
facility: Research Site
city: Adana
country: Turkey
facility: Research Site
city: Mersin
country: Turkey
facility: Research Site
city: Van
country: Turkey
facility: Research Site
city: Harrow
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
Study officials:
affiliation: Quintiles, Inc.
name: Maria Ignacia -Berraondo, MD
role: STUDY_DIRECTOR
References: