Immune ThrombocytopeniaPhase 2NCT01129024
An Open-label Safety Study of Lusutrombopag (S-888711) in Adults With Chronic Immune Thrombocytopenia (ITP)
A Phase 2 study testing Lusutrombopag in people with Immune Thrombocytopenia, run by Shionogi at 19 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Lusutrombopag
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 19 sitesUnited States
- Study dates
- 29 Apr 2010to 30 Jun 2011
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects who previously participated in Study 0913M0621 (NCT01054443) and who completed treatment or discontinued treatment due to a platelet count > 400,000/μL and continued to meet all inclusion criteria of the previous study, listed below, including platelet counts < 50,000/μL were eligible for study participation. For the purpose of this study, initial screening visit and all prestudy time period refer to Study 0913M0621.
- 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
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- 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
Who cannot join
- 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;
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- 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
- Total bilirubin > 1.5 x upper limit of normal
- Alanine aminotransferase (ALT) > 1.5 x upper limit of normal
- Aspartate aminotransferase (AST) > 1.5 x upper limit of normal
- Creatinine > 1.5 x upper limit of normal
- Human immunodeficiency virus positive
- Hepatitis A IgM antibody positive, hepatitis B surface antigen or hepatitis C antibody positive
- Exposure to previous thrombopoietin (TPO) mimetics/agonists (e.g., eltrombopag,romiplostim, E5501 [AKR-501] or LGD-4665) within 4 weeks prior to Initial Screening
- Subjects unresponsive to previous TPO mimetics/agonists (e.g., eltrombopag, romiplostim, E5501 [AKR-501] or LGD-4665)
- Exposure to an investigative medication within 4 weeks prior to the initial Screening Visit
Trial locations and contacts
19 sites in 1 country.
19 sites in 1 country
United States19 sites
Investigator
Anaheim, California, 92801
Investigator
Atlanta, Georgia, 30341
Investigator
Bethesda, Maryland, 20817
Investigator
Boston, Massachusetts, 02114
Investigator
Boynton Beach, Florida, 33426
Investigator
Cleveland, Ohio, 44106
Investigator
Jacksonville, Florida, 32207
Investigator
Jefferson City, Missouri, 65109
Investigator
Kansas City, Missouri, 64131
Investigator
Los Angeles, California, 90272
Investigator
Metairie, Louisiana, 70006
Investigator
New Brunswick, New Jersey, 08903
Investigator
New York, New York, 10021
Investigator
New York, New York, 10029
Investigator
Riverdale, Georgia, 30274
Investigator
Salt Lake City, Utah, 84132
Investigator
San Antonio, Texas, 78229
Investigator
Seattle, Washington, 98109
Investigator
Washington D.C., District of Columbia, 20007
About this study
Official title: An Open-label Safety Study of S-888711 in Adult Subjects With Relapsed Persistent or Chronic Immune Thrombocytopenia With or Without Prior Splenectomy
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.
Read the detailed description
This was an open-label, long-term safety study of lusutrombopag in the treatment of adults with relapsed persistent or chronic ITP with or without prior splenectomy. Patients who participate in this study must have completed the Phase 2 study 0913M0621 (NCT01054443), a double-blind, placebo controlled, parallel group study that evaluated the efficacy and safety lusutrombopag during which they either completed treatment or discontinued treatment due to a platelet count > 400,000/μL.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Lusutrombopag
- Keywords
- Splenectomy, Low Platelet Count, Thrombopoiesis, Thrombocytopaenia, Idiopathic Thrombocytopenic Purpura, Immune Thrombocytopenia (ITP), Thrombotic Thrombocytopenic Purpura (ITP), Hematologic Disease, Auto-immune Thrombocytopenic Purpura, S-888711, Blood Platelet Disorders, Relapsed Persistent or Chronic ITP, ITP
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Thrombocytopenia, Purpura, Thrombotic Thrombocytopenic, Hematologic Diseases, Blood Platelet Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LusutrombopagDrug
Also called MULPLETA®, S-888711
tablet
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Lusutrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Duration of response was defined as the percentage of the cumulative time a platelet count was ≥ 50,000 cells/µL during the extension study.
Measured: 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 by the Investigator according to the World Health Organization (WHO) criteria bleeding scale:
Grade 0: no bleeding; Grade 1: petechial bleeding; Grade 2: mild blood loss (clinically significant); Grade 3: gross blood loss, requires transfusion (severe); Grade 4: debilitating blood loss, retinal or cerebral associated with fatality.
For each participant, the most severe WHO bleeding grade observed during the treatment period is reported.
Measured: 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
This analysis includes platelet counts measured during the treatment period, including while participants were taking rescue medications.
Measured: 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
This analysis excludes platelet counts measured while the participant was taking rescue medications and during the 4 weeks after rescue medication.
Measured: 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
Measured: 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)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2010 (estimated)
- Study start29 Apr 2010
- Primary completion30 Jun 2011
- Study completion30 Jun 2011
Why it stopped: The study was terminated due to cessation of clinical development of S-888711 for chronic ITP based on a business decision by the Sponsor.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by ShionogiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shionogi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Shionogi Clinical Trials Administrator Clinical Support Help Line, Study director, Shionogi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01129024
- Sponsor's protocol ID
- 0914M0622
- Status last verified
- Feb 2021
- First submitted
- 7 May 2010
- Last updated
- 26 Feb 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Safety Study of S-888711 in Adult Subjects With Relapsed Persistent or Chronic Immune Thrombocytopenia With or Without Prior Splenectomy
- ClinicalTrials.gov ID
- NCT01129024
- Acronym / sponsor ID
- 0914M0622
- Conditions
- Immune Thrombocytopenia
- Keywords
- Splenectomy, Low Platelet Count, Thrombopoiesis, Thrombocytopaenia, Idiopathic Thrombocytopenic Purpura, Immune Thrombocytopenia (ITP), Thrombotic Thrombocytopenic Purpura (ITP), Hematologic Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was terminated due to cessation of clinical development of S-888711 for chronic ITP based on a business decision by the Sponsor.
- Enrolment
- 19 participants (actual)
- Lead sponsor
- ShionogiIndustry (pharma, biotech, devices)
- Study start
- 29 Apr 2010 (actual)
- Primary completion
- 30 Jun 2011 (actual)
- Study completion
- 30 Jun 2011 (actual)
- First posted
- 24 May 2010 (estimated)
- Results
- Posted 26 Feb 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This was an open-label, long-term safety study of lusutrombopag in the treatment of adults with relapsed persistent or chronic ITP with or without prior splenectomy. Patients who participate in this study must have completed the Phase 2 study 0913M0621 (NCT01054443), a double-blind, placebo controlled, parallel group study that evaluated the efficacy and safety lusutrombopag during which they either completed treatment or discontinued treatment due to a platelet count > 400,000/μL.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Lusutrombopag | Experimental | Drug: LusutrombopagParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Subjects who previously participated in Study 0913M0621 (NCT01054443) and who completed treatment or discontinued treatment due to a platelet count > 400,000/μL and continued to meet all inclusion criteria of the previous study, listed below, including platelet counts < 50,000/μL were eligible for study participation. For the purpose of this study, initial screening visit and all prestudy time period refer to Study 0913M0621.
- 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
Key exclusion criteria (24 listed)
- 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):
- History of clinically significant cardiovascular or thromboembolic disease within 26 weeks prior to Initial Screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LusutrombopagAlso: MULPLETA®, S-888711tablet | Drug | Lusutrombopag |
Primary outcome
Number of Participants With Adverse Events
Time frame: 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.
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.
5 secondary outcomes
Duration of Response
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Feb 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shionogi Clinical Trials Administrator Clinical Support Help Line study director
Shionogi
19 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Lusutrombopag in people with Immune Thrombocytopenia, run by Shionogi at 19 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 26 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Feb 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (time frame: 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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Feb 2021.