A Study to Investigate the Efficacy and Safety of Lusutrombopag (S-888711) Tablets Administered to Adults With Immune Thrombocytopenia (ITP)

NCT01054443 | Terminated Phase 2 Results

• 1 other identifier: 0913M0621
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 19

Brief Summary

The primary objective of this study was to assess the efficacy of 3 dose levels of lusutrombopag (0.5 mg, 0.75 mg, and 1.0 mg) and placebo on platelet count.

Conditions

Immune Thrombocytopenia (ITP)

Keywords

Blood Platelet Disorders; Immune Thrombocytopenia (ITP); Low Platelet Count; Thrombocytopaenia; S-888711; Splenectomy; Thrombopoiesis; Hematologic Disease; Auto-immune thrombocytopenic Purpura; Relapsed Persistent or Chronic ITP; Idiopathic Thrombocytopenic Purpura; Thrombotic Thrombocytopenic Purpura (TTP)

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With a Response

  Responders were participants with one of the following: 1. achieved a platelet count of ≥ 50,000 cells/µL after 6 weeks of dosing; or 2. prematurely withdrawn due to a platelet count \> 400,000 cells/µL prior to Day 42. Participants were counted as non-responders if any of the following conditions held: * The above conditions were not satisfied; * They received rescue medications; * They satisfied the above conditions after receiving restricted medications during the treatment period; * They had achieved a platelet count of ≥ 50,000 cells/µL before Week 6 but not after Week 6; or * They withdrew for any reason other than a platelet count \> 400,000 cells/µL.

  Week 6

Secondary Outcomes (9)

• Change From Baseline in Platelet Count at Week 6

  Baseline and Week 6

• Duration of Response

  6 weeks

• Percentage of Participants Who Achieved a Platelet Count of ≥ 30,000 Cells/µL and Doubled the Baseline Platelet Count After 6 Weeks of Dosing

  Week 6

• Percentage of Participants Who Achieved a Platelet Count of ≥ 50,000 Cells/µL and Doubled the Baseline Platelet Count After 6 Weeks of Dosing

  Week 6

• Number of Participants With Worst Severity of Bleeding Associated With ITP During the Treatment Period,

  6 weeks

Study Arms (4)

Placebo

PLACEBO COMPARATOR

Participants received placebo tablets orally once a day for 42 days.

Drug: Placebo

Lusutrombopag 0.5 mg

EXPERIMENTAL

Participants received 0.5 mg lusutrombopag orally once a day for 42 days.

Drug: Lusutrombopag

Lusutrombopag 0.75 mg

EXPERIMENTAL

Participants received 0.75 mg lusutrombopag orally once a day for 42 days.

Drug: Lusutrombopag

Lusutrombopag 1.0 mg

EXPERIMENTAL

Participants received 1.0 mg lusutrombopag orally once a day for 42 days.

Drug: Lusutrombopag

Interventions

Placebo DRUG

Tablet

Placebo

Lusutrombopag DRUG

Tablet

Also known as: MULPLETA®, S-888711

Lusutrombopag 0.5 mg; Lusutrombopag 0.75 mg; Lusutrombopag 1.0 mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• A signed and dated written informed consent
• Males and females ≥ 18 years of age
• All subjects must agree to use barrier contraception
• Diagnosis of ITP
• Subjects \> 60 years must have had a diagnostic bone marrow aspiration
• Relapsed persistent or chronic ITP status, with or without prior splenectomy (exception: in Hungary only splenectomized subjects will be enrolled), after having failed at least 1 prior ITP therapy (excluding TPO agonists) and have a platelet count \< 30,000/μL if not taking medications or \< 50,000/μL despite concomitant steroids or other ITP therapies, such as danazol or immunosuppressive drugs
• Subjects receiving steroid therapy must be on a stable dose
• Prothrombin time (PT) and activated partial thromboplastin time (aPTT) within 20% of the upper limit of normal (ULN)
• Subjects receiving stable dosages of cyclosporine A, mycophenolate mofetil, azathioprine, or danazol are allowed. The dosages of all these medications must be stable for at least 4 weeks prior to Visit 1 (Day 1)

You may not qualify if:

• History of clinically important hemorrhagic clotting disorder
• Females who are pregnant, lactating, or taking oral contraceptives
• History of alcohol/drug abuse or dependence within 1 year
• Use of the following drugs or treatment prior to Visit 1 (Day 1):
• Within 12 weeks - alemtuzumab, multi-drug systemic chemotherapy, stem cell therapy;
• Within 8 weeks - rituximab
• Within 2 weeks - platelet transfusions or plasmapheresis treatment
• Within 4 weeks - use of anti-platelet or anti-coagulant drugs
• Within 1 week - Rho(D) immune globulin or intravenous immunoglobulin
• History of clinically significant cardiovascular or thromboembolic disease within 26 weeks prior to Screening
• Splenectomy within 4 weeks prior to Screening
• Clinically significant laboratory abnormalities
• Hemoglobin \< 10.0 g/dL for men or women, not clearly related to ITP
• Absolute neutrophil count \< 1000/mm\^3
• Abnormal peripheral blood smear

Sponsors & Collaborators

• Shionogi: lead

Study Sites (19)

Investigator

Anaheim, California, 92801, United States

Location

Investigator

Los Angeles, California, 90272, United States

Location

Investigator

Washington D.C., District of Columbia, 20007, United States

Location

Investigator

Boynton Beach, Florida, 33426, United States

Location

Investigator

Jacksonville, Florida, 32207, United States

Location

Investigator

Atlanta, Georgia, 30341, United States

Location

Investigator

Riverdale, Georgia, 30274, United States

Location

Investigator

Metairie, Louisiana, 70006, United States

Location

Investigator

Bethesda, Maryland, 20817, United States

Location

Investigator

Boston, Massachusetts, 02114, United States

Location

Investigator

Jefferson City, Missouri, 65109, United States

Location

Investigator

Kansas City, Missouri, 64131, United States

Location

Investigator

New Brunswick, New Jersey, 08903, United States

Location

Investigator

New York, New York, 10021, United States

Location

Investigator

New York, New York, 10029, United States

Location

Investigator

Cleveland, Ohio, 44106, United States

Location

Investigator

San Antonio, Texas, 78229, United States

Location

Investigator

Salt Lake City, Utah, 84132, United States

Location

Investigator

Seattle, Washington, 98109, United States

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Blood Platelet Disorders; Thrombocytopenia; Hematologic Diseases; Purpura, Thrombotic Thrombocytopenic

Interventions

lusutrombopag

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; 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; Thrombophilia

Results Point of Contact

• Title: Shionogi Clinical Trials Administrator
• Organization: Shionogi Inc.

shionogiclintrials-admin@shionogi.co.jp

800-849-9707

Study Officials

• Shionogi Clinical Trials Administrator Clinical Support Help Line

  Shionogi

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• 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: January 20, 2010
• First Posted: January 22, 2010
• Study Start: March 18, 2010
• Primary Completion: November 24, 2010
• Study Completion: November 24, 2010
• Last Updated: March 18, 2021
• Results First Posted: February 24, 2021

Record last verified: 2021-02

Locations

US (19)