Phase 3
N=92
Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body.
Idiopathic Thrombocytopenic Purpura
Bottom Line
View on ClinicalTrials.gov: NCT01520909 ↗Enrolled (actual)
92
Serious AEs
10.1%
Results posted
Sep 2014
Primary outcome: Primary: Number of Participants Achieving a Platelet Count >=50 Giga Cells Per Liter (Gi/L) for at Least 6 Out of 8 Weeks, Between Weeks 5 and 12 of Part 1 — 1; 25 Participants — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Eltrombopag (Drug); Placebo (Drug)
- Age
- Pediatric · 1+ yrs
- Sex
- All
- Sponsor
- GlaxoSmithKline
- Primary completion
- Jan 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Achieving a Platelet Count >=50 Giga Cells Per Liter (Gi/L) for at Least 6 Out of 8 Weeks, Between Weeks 5 and 12 of Part 1 |
1; 25 | <0.001 sig |
| SECONDARY Percentage of Responders |
0; 15.9; 3.4; 23.8; 0; 31.7 | <0.001 sig |
| SECONDARY Number of Participants Achieving a Platelet Count >=50 Gi/L at Any Time During the First 12 Weeks of Part 1 |
6; 47 | — |
| SECONDARY Number of Participants Achieving a Platelet Count >=50 Gi/L at Any Time During the First 6 Weeks of Part 1 |
5; 39 | — |
| SECONDARY Weighted Mean Platelet Count |
14.2; 14.0; 23.7; 63.9 | — |
| SECONDARY Maximum Duration for Which a Participant Continuously Maintained a Platelet Count of >=50 Gi/L During the First 12 Weeks of Part 1 |
0.4; 3.3 | — |
| SECONDARY Number of Participants Who Required a Protocol-defined Rescue Treatment During Part 1 |
7; 12 | — |
| SECONDARY Number of Participants With Any Bleeding and Significant Bleeding as Assessed Using the World Health Organization (WHO) Bleeding Scale During Part 1 |
20; 45; 6; 16; 19; 41 | — |
| SECONDARY Number of Participants Who Achieved a Platelet Count >=50 Gi/L at Any Time During Part 2 |
70 | — |
| SECONDARY Number of Weeks in Which Participants Achieved a Platelet Count >=50 Gi/L, Between Weeks 4 and 24 of Part 2 |
10 | — |
| SECONDARY Maximum Duration for Which a Participant Continuously Maintained a Platelet Count of >=50 Gi/L During Part 2 |
8.6 | — |
| SECONDARY Number of Participants Who Reduced or Discontinued Baseline Concomitant ITP Medications During Part 2 Without Requiring Subsequent Rescue Therapy |
8 | — |
| SECONDARY Number of Participants Who Required a Protocol-defined Rescue Treatment During Part 2 |
11 | — |
| SECONDARY Number of Participants With Any Bleeding and Significant Bleeding as Assessed Using the WHO Bleeding Scale During Part 2 |
55; 17; 29; 3; 22; 2 | — |
| SECONDARY Number of Participants With Any Adverse Event (AE) or Serious Adverse Event (SAE) During Part 1 |
21; 51; 4; 5 | — |
| SECONDARY Number of Participants With Any Adverse Event (AE) or Serious Adverse Event (SAE) During Part 2 |
69; 9 | — |
| SECONDARY Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Clinical Chemistry Parameters at Any Time Post-Baseline During Part 1 |
26; 50; 3; 12; 0; 1 | — |
| SECONDARY Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Clinical Chemistry Parameters at Any Time Post-Baseline During Part 2 |
63; 21; 1; 2; 0; 67 | — |
| SECONDARY Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Hematology Parameters at Any Time Post-Baseline During Part 1 |
23; 50; 6; 11; 0; 0 | — |
| SECONDARY Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Hematology Parameters at Any Time Post-Baseline During Part 2 |
59; 23; 3; 2; 0; 63 | — |
| SECONDARY Number of Participants With Vital Sign Data Falling Outside the Reference Ranges (RR) at the Indicated Visit During Part 1 |
6; 8; 2; 5; 3; 10 | — |
| SECONDARY Number of Participants With Vital Sign Data Falling Outside the Reference Ranges (RR) at the Indicated Visit During Part 2 |
14; 8; 14; 8; 13; 11 | — |
| SECONDARY Number of Participants With a Change in Visual Acuity Since Baseline at Week 12 of Part 1 |
22; 47; 4; 8; 1; 2 | — |
| SECONDARY Number of Participants With a Change in Visual Acuity Since Baseline at Week 24 of Part 2 |
58; 13; 3; 6; 7 | — |
| SECONDARY Number of Participants With a Change in Visual Acuity Since Baseline at Follow-Up Week 24 |
63; 12; 9; 2; 1 | — |
| SECONDARY Number of Participants With Worsening Visual Acuity Due to Cataracts at Week 12 of Part 1 |
0; 1 | — |
| SECONDARY Number of Participants With Worsening Visual Acuity Due to Cataracts at Week 24 of Part 2 |
1; 5 | — |
| SECONDARY Number of Participants With Worsening Visual Acuity Due to Cataracts at Follow-Up Week 24 |
0; 2 | — |
| SECONDARY Pharmacokinetic (PK) Assessments for Eltrombopag for AUC (0-t) |
104; 171; 184 | — |
| SECONDARY Pharmacokinetic (PK) Assessments for Eltrombopag for Cmax |
6.94; 11.2; 12.5 | — |
| SECONDARY Pharmacokinetic (PK) Assessments for Eltrombopag for Apparent Oral Clearance (CL/F) and Apparent Intercompartmental Clearance (Q/F) |
0.48; 0.29; 0.19; 0.61; 0.38; 0.24 | — |
| SECONDARY PK Assessments for Eltrombopag for Apparent Central Volume (Vc/F) and Apparent Peripheral Volume (Vp/F) |
2.46; 1.57; 0.90; 19.2; 11.8; 7.17 | — |
| SECONDARY Population PK Model Point Estimate for Eltrombopag for Absorption Rate-constant (Ka) |
0.189; 0.189; 0.189 | — |
Summary
The purpose of this study is to investigate the efficacy, safety and tolerability of eltrombopag in children with previously treated chronic immune thrombocytopenia who are between 1 and 17 years of age. This is a 2 part study. In part 1, patients will be randomized to receive either eltrombopag or placebo for 13 weeks. All patients who complete part 1 will enter part 2. In part 2, all patients will receive 24 weeks of eltrombopag.
Eligibility Criteria
Inclusion Criteria
- Written informed consent must be obtained from the patient's guardian and accompanying informed assent from the patient (for children over 6 years old)
- Patients must be between 1 year and 3 consecutive days within 2 weeks of Day 1.
- Patients who have received treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding Day 1.
- Patients who have previously received eltrombopag or any other thrombopoietin receptor agonist.
- Any patient considered to be a child in care, defined as one who has been placed under the control or protection of an agency, organization, institution or entity by the courts, the government or a government body, acting in accordance with powers conferred on them by law or regulation. This can include a child cared for by foster parents or living in a care home or institution, provided that the arrangement falls within the definition above. The definition of a child in care does not include a child who is adopted or who has an appointed legal guardian.
- Patients who have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to eltrombopag or excipients that contraindicates their participation.
- Any serious and/or unstable pre-existing medical, psychiatric disorder, or other conditions that could interfere with the patient's safety or compliance to the study procedures.
Data sourced from ClinicalTrials.gov (NCT01520909). 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.