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.

NCT01520909 | Completed Phase 3 Results DMC

• 2 other identifiers: 1154502011-002184-17
• Study Type: interventional
• Target: 92
• Locations: 13 countries
• Sites: 51

Brief 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.

Conditions

Idiopathic Thrombocytopenic Purpura

Keywords

immune thrombocytopenic purpura; pediatrics; chronic ITP; idiopathic thrombocytopenic purpura; eltrombopag; Chronic Immune Thrombocytopenia; platelet disorder; thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• 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

  Participants who achieved a platelet count \>=50 Gi/L for at least 6 out of 8 weeks, between Weeks 5 and 12 of Part 1, were reported.

  From Week 5 up to Week 12 of Part 1

Secondary Outcomes (32)

• Percentage of Responders

  From Week 1 up to Week 12 of Part 1

• Number of Participants Achieving a Platelet Count >=50 Gi/L at Any Time During the First 12 Weeks of Part 1

  From Baseline up to Week 12 of Part 1

• Number of Participants Achieving a Platelet Count >=50 Gi/L at Any Time During the First 6 Weeks of Part 1

  From Baseline up to Week 6 of Part 1

• Weighted Mean Platelet Count

  Baseline and Week 12 of Part 1

• Maximum Duration for Which a Participant Continuously Maintained a Platelet Count of >=50 Gi/L During the First 12 Weeks of Part 1

  From Baseline up to Week 12 of Part 1

Study Arms (3)

Eltrombopag plus standard of care

EXPERIMENTAL

Part 1, double-blind treatment group

Drug: Eltrombopag

Placebo plus standard of care

PLACEBO COMPARATOR

Part 1, double-blind treatment group

Drug: Placebo

Eltrombopag plus standard of care (Part 2 open-label)

EXPERIMENTAL

Part 2, open-label

Drug: Eltrombopag

Interventions

Eltrombopag DRUG

Thrombopoietin receptor agonist

Eltrombopag plus standard of care; Eltrombopag plus standard of care (Part 2 open-label)

Placebo DRUG

Placebo with no active pharmaceutical ingredient

Placebo plus standard of care

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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 \<18 years of age at Day 1
• Patients will have a confirmed diagnosis of chronic ITP for at least 1 year, at screening, according to the guidelines published in the International Working Group Report
• A peripheral blood smear or bone marrow examination will support the diagnosis of ITP with no evidence of other causes of thrombocytopenia.
• Patients must be refractory or have relapsed after at least one prior ITP therapy, or patients must be unable, for a medical reason, to continue other ITP treatments.
• Patients must have a Day 1 (or within 48 hours prior) platelet count \<30 Gi/L.
• Previous therapy for ITP with immunoglobulins (IVIg and anti-D) must have been completed at least 2 weeks prior to Day 1, or these therapies must have been completed at least 1 week prior to Day 1 and have been clearly ineffective.
• Previous treatment for ITP with splenectomy, rituximab and cyclophosphamide must have been completed at least 4 weeks prior to Day 1.
• Patients treated with concomitant ITP medication (e.g. corticosteroids or azathioprine) must be receiving a dose that has been stable for at least 4 weeks prior to Day 1.
• Patients must have a complete blood count (CBC) not suggestive of another hematological disorder.
• Patients must have the following laboratory results:
• prothrombin time international normalized ratio (INR) and activated partial thromboplastin time (aPTT) within 80 to 120% of the normal range.
• clinical chemistries that do NOT exceed the upper limit of normal reference range by more than 20% for the following: creatinine, ALT, AST, total bilirubin, and alkaline phosphatase.
• total albumin that is not below the lower limit of normal by more than 10%.
• Female patients of child-bearing potential (after menarche) must:

You may not qualify if:

• Patients with any clinically relevant abnormality, other than ITP, identified on the screening examination or any other medical condition or circumstance, which in the opinion of the investigator makes the patient unsuitable for participation in the study or suggests another primary diagnosis (e.g. Thrombocytopenia is secondary to another disease).
• Patients with concurrent or past malignant disease, including myeloproliferative disorder.
• Patients expected not to be suitable for continuation of their current therapy for at least 13 additional weeks.
• Patients with a history of platelet agglutination abnormality that prevents reliable measurement of platelet counts.
• Patients with a diagnosis of secondary immune thrombocytopenia, including those with laboratory or clinical evidence of HIV infection, anti-phospholipid antibody syndrome, chronic hepatitis B infection, hepatitis c virus infection, or any evidence of active hepatitis at the time of subject screening.
• Patients with Evans syndrome (autoimmune thrombocytopenia and autoimmune hemolysis).
• Patients with known inherited thrombocytopenia (e.g. MYH9 disorders).
• Patients treated with any medication that affects platelet function (including but not limited to aspirin, clopidogrel and/or NSAIDS) or anti-coagulants for \>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.

Sponsors & Collaborators

• GlaxoSmithKline: lead

Study Sites (51)

GSK Investigational Site

Los Angeles, California, 90027, United States

Location

GSK Investigational Site

St. Petersburg, Florida, 33701, United States

Location

GSK Investigational Site

Brooklyn, New York, 11219, United States

Location

GSK Investigational Site

Salt Lake City, Utah, 84113, United States

Location

GSK Investigational Site

Capital Federal, Buenos Aires, 1425, Argentina

Location

GSK Investigational Site

Brno, 613 00, Czechia

Location

GSK Investigational Site

Olomouc, 775 20, Czechia

Location

GSK Investigational Site

Ostrava, 708 52, Czechia

Location

GSK Investigational Site

Prague, 154 00, Czechia

Location

GSK Investigational Site

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

Location

GSK Investigational Site

Mainz, Rhineland-Palatinate, 55131, Germany

Location

GSK Investigational Site

Berlin, State of Berlin, 13353, Germany

Location

GSK Investigational Site

Pokfulam, Hong Kong

Location

GSK Investigational Site

Shatin, Hong Kong

Location

GSK Investigational Site

Beersheba, 84101, Israel

Location

GSK Investigational Site

Haifa, 31048, Israel

Location

GSK Investigational Site

Petah Tikva, 49202, Israel

Location

GSK Investigational Site

Ramat Gan, 52621, Israel

Location

GSK Investigational Site

Rehovot, 76100, Israel

Location

GSK Investigational Site

Tel Aviv, 64239, Israel

Location

GSK Investigational Site

Bologna, Emilia-Romagna, 40138, Italy

Location

GSK Investigational Site

Rome, Lazio, 00165, Italy

Location

GSK Investigational Site

Monza, Lombardy, 20900, Italy

Location

GSK Investigational Site

Turin, Piedmont, 10126, Italy

Location

GSK Investigational Site

Bydgoszcz, Poland

Location

GSK Investigational Site

Gdansk, 80-952, Poland

Location

GSK Investigational Site

Lodz, 91-738, Poland

Location

GSK Investigational Site

Lublin, 20-093, Poland

Location

GSK Investigational Site

Krasnodar, 350007, Russia

Location

GSK Investigational Site

Moscow, 105077, Russia

Location

GSK Investigational Site

Moscow, 117198, Russia

Location

GSK Investigational Site

Saint Petersburg, 198205, Russia

Location

GSK Investigational Site

Barcelona, 08035, Spain

Location

GSK Investigational Site

Madrid, 28007, Spain

Location

GSK Investigational Site

Madrid, Spain

Location

GSK Investigational Site

Málaga, 29011, Spain

Location

GSK Investigational Site

Murcia (El Palmar), 30120, Spain

Location

GSK Investigational Site

Pamplona, 31008, Spain

Location

GSK Investigational Site

Valencia, 46026, Spain

Location

GSK Investigational Site

Tainan, 704, Taiwan

Location

GSK Investigational Site

Taoyuan District, 333, Taiwan

Location

GSK Investigational Site

Bangkok, 10330, Thailand

Location

GSK Investigational Site

Bangkok, Thailand

Location

GSK Investigational Site

Khon Kaen, 40002, Thailand

Location

GSK Investigational Site

Songkhla, 90110, Thailand

Location

GSK Investigational Site

Birmingham, B4 6NH, United Kingdom

Location

GSK Investigational Site

Cardiff, CF14 4XW, United Kingdom

Location

GSK Investigational Site

London, SW17 0QT, United Kingdom

Location

GSK Investigational Site

Manchester, M13 9WL, United Kingdom

Location

GSK Investigational Site

Romford, RM7 0AG, United Kingdom

Location

GSK Investigational Site

Sheffield, S10 2TH, United Kingdom

Location

Related Publications (2)

• Wire MB, Li X, Zhang J, Sallas W, Aslanis V, Ouatas T. Modeling and Simulation Support Eltrombopag Dosing in Pediatric Patients With Immune Thrombocytopenia. Clin Pharmacol Ther. 2018 Dec;104(6):1199-1207. doi: 10.1002/cpt.1066. Epub 2018 Apr 17.

  PMID: 29536526 DERIVED

• Grainger JD, Locatelli F, Chotsampancharoen T, Donyush E, Pongtanakul B, Komvilaisak P, Sosothikul D, Drelichman G, Sirachainan N, Holzhauer S, Lebedev V, Lemons R, Pospisilova D, Ramenghi U, Bussel JB, Bakshi KK, Iyengar M, Chan GW, Chagin KD, Theodore D, Marcello LM, Bailey CK. Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial. Lancet. 2015 Oct 24;386(10004):1649-58. doi: 10.1016/S0140-6736(15)61107-2. Epub 2015 Jul 28.

  PMID: 26231455 DERIVED

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Blood Platelet Disorders; Thrombocytopenia

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: GSK Response Center
• Organization: GlaxoSmithKline

866-435-7343

Study Officials

• GSK Clinical Trials

  GlaxoSmithKline

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2011
• First Posted: January 30, 2012
• Study Start: March 1, 2012
• Primary Completion: January 1, 2014
• Study Completion: January 1, 2014
• Last Updated: March 10, 2015
• Results First Posted: September 29, 2014

Record last verified: 2015-02

Locations

CZ (4); RU (4); ES (7); TW (2); TH (4); GB (6); US (4); AR (1); DE (3); HK (2); IT (4); PL (4); IL (6)