Phase 2
Completed N=46
A Pharmacokinetic (PK) and Pharmacodynamic (PD) Dose-ranging Phase II Study of Ticagrelor in Paediatric Patients With Sickle Cell Disease
Investigation of Platelet Aggregation in Paediatric Patients With Sickle Cell Disease
Source: ClinicalTrials.gov NCT02214121 ↗
Enrolled (actual)
46
Serious AEs
14.7%
Results posted
May 2018
Primary outcomePrimary: P2Y12 Reaction Units (PRU) - Part A — 301.6; 268.0; 295.4; 287.7 P2Y12 reaction units
Summary
The purpose of this Phase II dose-ranging study is to investigate pharmacokinetic (PK) and pharmacodynamic (PD) properties of various doses of ticagrelor followed by 4 weeks of twice-daily treatment in paediatric patients with sickle cell disease
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY P2Y12 Reaction Units (PRU) - Part A |
301.6; 268.0; 295.4; 287.7; 283.7; 277.7 | — |
| PRIMARY P2Y12 Reaction Units (PRU) - Part B |
282.8; 313.3; 259.6; 217.3 | — |
| PRIMARY Maximum Plasma Concentration (Cmax) - Part A |
15.240; 162.961; 52.069; 96.031; 269.174; 566.550 | — |
| PRIMARY Maximum Plasma Concentration (Cmax) - Part B |
16.394 | — |
| PRIMARY Area Under the Plasma Concentration Time Curve (AUC) - Part A |
161.9; 1151.9; 437.5; 879.3; 1638.7; 2850.9 | — |
| PRIMARY Area Under the Plasma Concentration Time Curve (AUC) - Part B |
160.6 | — |
| SECONDARY Assessment of Ticagrelor Concentration - Part A |
2.170; 31.937; 28.066; 12.713; 104.243; 34.069 | — |
| SECONDARY Assessment of Ticagrelor Concentration - Part B |
2.478; 9.677; 14.144; 9.605 | — |
| SECONDARY Assessment of AR-C124910XX Concentration - Part A |
1.250; 17.380; 20.359; 1.782; 16.302; 4.577 | — |
| SECONDARY Assessment of AR-C124910XX Concentration - Part B |
1.810; 2.865; 4.160; 3.953 | — |
| SECONDARY Oral Clearance (CL/F) - Part A |
22.50 | — |
| SECONDARY Oral Clearance (CL/F) - Part B |
19.15 | — |
| SECONDARY Number of Vaso-occlusive Crises - Part B |
1.0; 0.6 | — |
| SECONDARY Number of Vaso-occlusive Crises Requiring Hospitalization or Emergency Department Visits - Part B |
0.2; 0.1 | — |
| SECONDARY Percentage of Days Hospitalized for Vaso-occlusice Crisis or Other Complications of Sickle Cell Disease - Part B |
4.52; 1.34 | — |
| SECONDARY Percentage of Days With Pain (Age >=4) - Part B |
27.01; 31.78 | — |
| SECONDARY Mean Intensity of Pain (Age >=4) - Part B |
1.40; 0.87; 1.64; 1.36; 1.11; 0.38 | — |
| SECONDARY Percentage of Days of Analgesic Use (Age >= 4) - Part B |
16.79; 18.56 | — |
| SECONDARY Percentage of Days of Opioid Analgesic Use (Age >=4) - Part B |
12.46; 0.54 | — |
| SECONDARY Percentage of Days of Absence From School or Work (Age >=6) - Part B |
4.87; 5.95 | — |
Eligibility Criteria
Inclusion criteria
- Children aged ≥2 to <18 years of age
- Diagnosed with homozygous sickle cell (HbSS) or sickle beta-zero-thalassaemia (HbS/β0)
Exclusion criteria
- At risk for haemorrhagic or bradycardic events
- Significant hepatic impairment
- Renal failure requiring dialysis
- Concomitant oral or intravenous therapy with strong CYP3A4 (cytochrome) inhibitors, CYP3A4 substrates with narrow therapeutic indices, or strong CYP3A4 inducers.
- Surgical procedure planned to occur during the study.
- Patients who are currently pregnant or breastfeeding or planning to become pregnant during the study.
- Patients who have known hypersensitivity or contraindication to ticagrelor.
Data sourced from ClinicalTrials.gov (NCT02214121). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.