NCT01129024

Brief Summary

The primary objective of this study was to assess the long-term safety of lusutrombopag in the treatment of adults with relapsed persistent or chronic ITP with or without prior splenectomy.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
19

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Apr 2010

Shorter than P25 for phase_2

Geographic Reach
1 country

19 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 29, 2010

Completed
8 days until next milestone

First Submitted

Initial submission to the registry

May 7, 2010

Completed
17 days until next milestone

First Posted

Study publicly available on registry

May 24, 2010

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2011

Completed
9.7 years until next milestone

Results Posted

Study results publicly available

February 26, 2021

Completed
Last Updated

February 26, 2021

Status Verified

February 1, 2021

Enrollment Period

1.2 years

First QC Date

May 7, 2010

Results QC Date

February 5, 2021

Last Update Submit

February 24, 2021

Conditions

Keywords

SplenectomyLow Platelet CountThrombopoiesisThrombocytopaeniaIdiopathic Thrombocytopenic PurpuraImmune Thrombocytopenia (ITP)Thrombotic Thrombocytopenic Purpura (ITP)Hematologic DiseaseAuto-immune Thrombocytopenic PurpuraS-888711Blood Platelet DisordersRelapsed Persistent or Chronic ITPITP

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Adverse Events

    An AE is defined as any untoward medical occurrence in a subject administered a pharmaceutical product during the course of a clinical investigation, including any unfavorable and unintended sign, symptom, or disease temporally associated with the use of an investigational product (IP), whether or not thought to be related to the IP. AEs reported after initial study drug administration were considered treatment-emergent. A serious adverse event is defined as any AE that resulted in death, was life-threatening, hospitalization or prolongation of existing hospitalization, a persistent or significant disability/incapacity, or a congenital anomaly/birth defect or an important medical event that, based upon medical judgment, may jeopardize the participant or require medical or surgical intervention to prevent one of the outcomes listed above. A treatment-related AE is any AE determined by the investigator to be possibly related, probably related, or definitely related to study drug.

    From first dose of study drug in the extension study up to 6 weeks after last dose; median (range) time on study treatment was 148 (10-387) days.

Secondary Outcomes (5)

  • Duration of Response

    From first dose of study drug in the extension study up to 6 weeks after last dose; median (range) time on treatment was 148 (10 - 387) days.

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

    Bleeding assessments were performed at Weeks 1, 2, 3, 4, 5, and 6, Months 1, 2, 3, 6, 9, and 12, and every 3 months thereafter until end of treatment; median (range) time on treatment was 148 (10-387) days.

  • Percentage of Participants Who Achieved a Platelet Count of < 50,000 Cells/μL, Between 50,000 to 400,000 Cells/μL, and ≥ 400,000 Cells/μL

    Platelets were assessed at Weeks 1, 2, 3, 4, 5, and 6, Months 1, 2, 3, 6, 9, and 12, and every 3 months thereafter, until end of treatment; median (range) time on treatment was 148 (10-387) days.

  • Percentage of Participants Who Achieved a Platelet Count of < 50,000 Cells/μL, Between 50,000 to 400,000 Cells/μL, and ≥ 400,000 Cells/μL Without Rescue Medication

    Platelets were assessed at Weeks 1, 2, 3, 4, 5, and 6, Months 1, 2, 3, 6, 9, and 12, and every 3 months thereafter, until end of treatment; median (range) time on treatment was 148 (10-387) days.

  • Change From Baseline in Platelet Counts at the Final Visit

    Baseline and the final visit (If a subject had multiple platelet count measurements for the specific dose level due to dose adjustments, the final platelet count was used)

Study Arms (1)

Lusutrombopag

EXPERIMENTAL

Participants received lusutrombopag 0.5 mg administered orally once a day for up to 3 years or until study termination. The dose was adjusted based on platelet counts. If a subject's platelet count remained \< 50,000/μL, the dose could have been increased by 0.25 mg up to a maximum dose of 2.0 mg.

Drug: Lusutrombopag

Interventions

tablet

Also known as: MULPLETA®, S-888711
Lusutrombopag

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (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
  • Subjects receiving steroid therapy must be on a stable dose for at least 2 weeks prior to Screening
  • Prothrombin time (PT) and activated partial thromboplastin time (aPTT) within 20% of the upper limit of normal (ULN) at Screening
  • Subjects receiving stable dosages of cyclosporine A, mycophenolate mofetil, azathioprine, or danazol are allowed

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 1 week - Rho(D) immune globulin or intravenous immunoglobulin;
  • Within 2 weeks - plasmaphoresis treatment;
  • Within 4 weeks - use of anti-platelet or anti-coagulant drugs;
  • Within 8 weeks - rituximab;
  • Within 12 weeks - alemtuzumab, multi-drug systemic chemotherapy, stem cell therapy;
  • History of clinically significant cardiovascular or thromboembolic disease within 26 weeks prior to Initial Screening
  • Splenectomy within 4 weeks prior to Initial Screening
  • Clinically significant laboratory abnormalities
  • Hemoglobin \< 10.0 g/dL for men or women, not clearly related to ITP
  • Absolute neutrophil count \< 1000/mm3
  • Abnormal peripheral blood smear with evidence of fibrosis confirmed by bonemarrow biopsy
  • +9 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

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, IdiopathicThrombocytopeniaPurpura, Thrombotic ThrombocytopenicHematologic DiseasesBlood Platelet Disorders

Interventions

lusutrombopag

Condition Hierarchy (Ancestors)

Purpura, ThrombocytopenicPurpuraBlood Coagulation DisordersHemic and Lymphatic DiseasesThrombotic MicroangiopathiesCytopeniaHemorrhagic DisordersAutoimmune DiseasesImmune System DiseasesHemorrhagePathologic ProcessesPathological Conditions, Signs and SymptomsSkin ManifestationsSigns and SymptomsThrombophilia

Results Point of Contact

Title
Shionogi Clinical Trials Administrator
Organization
Shionogi Inc.

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
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 7, 2010

First Posted

May 24, 2010

Study Start

April 29, 2010

Primary Completion

June 30, 2011

Study Completion

June 30, 2011

Last Updated

February 26, 2021

Results First Posted

February 26, 2021

Record last verified: 2021-02

Locations