A Study to Assess the Ability of Eltrombopag to Induce Sustained Remission in Subjects With ITP (TAPER)
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| ClinicalTrials.gov Identifier: NCT03524612 |
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Recruitment Status :
Recruiting
First Posted : May 15, 2018
Last Update Posted : October 19, 2020
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Sponsor:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
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Brief Summary:
This study will assess the ability of eltrombopag to induce sustained treatment-free remission in ITP subjects who relapsed or failed to respond to an initial treatment with steroids.
There is limited, mainly retrospective evidence that earlier use of eltrombopag after ITP diagnosis, will allow a larger proportion of subjects to achieve sustained remission after tapering off drug. Clinically there is a need for a less toxic regimen that will provide responses and sustained remission with a shorter treatment interval. This trial is designed to assess this.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Immune Thrombocytopenic Purpura (ITP) | Drug: eltrombopag | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 101 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase II, Open-label, Prospective, Single-arm, Study to Assess Ability of Eltrombopag to Induce Sustained Remission in Subjects With ITP Who Are Refractory or Relapsed After First-line Steroids |
| Actual Study Start Date : | November 2, 2018 |
| Estimated Primary Completion Date : | November 30, 2022 |
| Estimated Study Completion Date : | November 30, 2022 |
Resource links provided by the National Library of Medicine
Drug Information available for:
Eltrombopag
| Arm | Intervention/treatment |
|---|---|
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eltrombopag
Participants will be treated with eltrombopagto to induce sustained remission to reach a target platelet count of ≥ 100×109/L (CR), after 1st line steroids have failed.
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Drug: eltrombopag
eltrombopag is for oral use and comes in 12.5, 25, 50 and 75 mg tablets; prescribed dose is taken once daily
Other Name: ETB115 |
Primary Outcome Measures :
- Percentage of participants with sustained remission (R) by 12 months [ Time Frame: 12 months ]Sustained remission is defined as reach platelet count ≥ 100×109/L (complete response [CR]) and then maintain platelet counts around 100×109/L for 2 months (no counts below 70×109/L) AND then taper off the drug until treatment discontinuation while, maintain platelet count ≥ 30×109/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy until month 12.
Secondary Outcome Measures :
- Median duration of sustained remission [ Time Frame: Last dose of eltrombopag to 12 months ]Duration of sustained remission (in weeks) counted from last dose of eltrombopag to month 12 for participants with sustained remission (R).
- Percentage of participants with platelet count ≥ 50×109/L [ Time Frame: By 1 month ]Percentage of subjects who reach platelet count ≥ 50×109/L at least once within the first month (month 1) without bleeding events and no rescue therapy
- Percentage of participants with at least one platelet count ≥ 30×109/L after eltrombopag is re-introduced without bleeding and no rescue medication [ Time Frame: 12 months ]Percentage of subjects with at least one platelet count ≥ 30×109/L after eltrombopag is re-introduced, in case of loss of response (< 30×109/L and/or bleeding event) without bleeding events and no rescue therapy
- Absolute and relative change in platelet count from baseline to various time points [ Time Frame: Baseline 3, 6, 9, 12 months ]Quantify platelet count from baseline to different time points; box plots for absolute and/or relative change in platelet counts from baseline to different time points
- Percentage of participants who maintain a platelet count ≥ 30×109/L without bleeding and no rescue medication [ Time Frame: From first time of reaching the level to 3, 6, 9, 12 months ]Ability of eltrombopag to maintain platelet count ≥ 30×109/L within 12 months.
- Change from baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) questionaire [ Time Frame: Baseline to 3, 6, 9, 12 months ]The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) instrument is a 13-item validated tool used to measure an individual's level of fatigue during usual daily activities over the past 7 days (Cella 2002, Webster 2003). FACIT-Fatigue is a subscale of the FACIT measurement system. FACIT-fatigue is scored using a 4-point Likert scale (4=not at all fatigued to 0=very much fatigued) where the total possible score ranges from 0-5; higher scores represent better HRQoL.
- Change from baseline in Functinal Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) questionnaire [ Time Frame: Baseline to 3, 6, 9, 12 months ]FACT-Th6 instrument is used to measure worry/concern about bleeding and bruising, and the impact of this worry/concern on physical and social activity (Cella 2006). FACT-Th6 is a 6-item subset of the more detailed FACT-Th, which is an 18-item subscale of the validated FACT that specifically measures concerns related to thrombocytopenia in the past 7 days. The FACT-Th6 is scored using a 5-level Likert scale (0=not at all to 4=very much) and is calculated by summing scores for the 6-items; therefore, scores can range from 0-24, with higher scores representing better HRQoL
- Change from baseline in Short Form 36 Health Survey (SF-36v2) questionnaire [ Time Frame: Baseline to 3, 6, 9, 12 months ]SF-36v2 is a validated instrument used to measure general physical and mental health status (Ware 2000) via assessment of 8 domains-Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional, and Mental Health-over the past 4 weeks or 7 days. The SF-36 is scored using norm-based scoring procedures and scores ranging from 0-100; higher scores represent better HRQoL
- Overall change from baseline of the overall impact of side effects on treatment via Functional Assessment of Cancer Therapy-G (GP5) [ Time Frame: Baseline, 12 months or end of study ]The GP5 is a single question used to assess the overall bothersomeness of treatment side effects. The GP5 is scored using a 5-point rating scale (0 = not at all; 1 = a little bit; 2 = somewhat; 3 = quite a bit; and 4 = very much), where lower scores reflect less bothersomeness from treatment side effects.
- Overall change of treatment satisfaction using Treatment Satisfaction Questionnaire (TSQM-9) [ Time Frame: Baseline, 12 months or end of study ]TSQM-9 will be used to assess treatment satisfaction with medication. The three scales of the TSQM-9 include the effectiveness scale, convenience scale, and global satisfaction scale
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Signed informed consent must be obtained prior to participation in the study
- Subjects ≥ 18 years old
- Subjects with a confirmed diagnosis of primary ITP, who are not responsive or in relapse after a first line of steroid therapy ± intravenous immunoglobulin (IVIG) (used as a rescue therapy)
- Platelet count < 30×109/L and assessed as needing treatment (per physician's discretion
Exclusion Criteria:
- ITP subjects previously treated with any ITP second-line therapies, thrombopoietin receptor (TPO-R) agonists for ITP, except steroids / IVIG
- Subjects who relapsed more than one year after the end of first-line full course of steroid therapy
- Subjects with a diagnosis of secondary thrombocytopenia
- Subjects who have life threatening bleeding complications per investigator discretion
- Subjects who had a deep vein thrombosis or arterial thrombosis in the 6 months preceding enrollment
- Serum creatinine ≥ 1.5 mg/dL
- Total bilirubin > 1.5 × upper limit of normal (ULN)
- Aspartate transaminase (AST) > 3.0 × ULN
- Alanine transaminase (ALT) > 3.0 × ULN
- Subjects who are human immune deficiency virus (HIV), hepatitis C virus (HCV), hepatitis B surface antigen (HBsAg) positive
- Subjects with hepatic impairment (Child-Pugh score > 5)
- Subjects who have active malignancy
- Subjects with any serious and/or unstable pre-existing medical, psychiatric disorder or other conditions that could interfere with subject's safety, obtaining informed consent or compliance with the study procedures per investigator discretion
- History or current diagnosis of cardiac disease indicating significant risk of safety for subjects participating in the study
- Subjects with known active or uncontrolled infections not responding to appropriate therapy
- Subjects with evidence of current alcohol/drug abuse
- Women of child-bearing potential and sexually active males unwilling to use adequate contraception during the study
- Female subjects who are nursing or pregnant (positive serum or urine B-human chorionic gonadotrophin (B-hCG) pregnancy test) at screening or pre-dose on Day 1
Other protocol-defined inclusion/exclusion criteria may apply.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03524612
Contacts
| Contact: Novartis Pharmaceuticals | 1-888-669-6682 | novartis.email@novartis.com | |
| Contact: Novartis Pharmaceuticals | +41613241111 |
Locations
Show 49 study locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
| Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
| Responsible Party: | Novartis Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT03524612 |
| Other Study ID Numbers: |
CETB115J2411 2018-000452-18 ( EudraCT Number ) |
| First Posted: | May 15, 2018 Key Record Dates |
| Last Update Posted: | October 19, 2020 |
| Last Verified: | October 2020 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
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Thrombocytopenic Immune Platelets Bleeding ITP |
eltrombopag ETB115 induce sustained remission refractory or relapsed first-line steroids |
Additional relevant MeSH terms:
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Thrombocytopenia Immune System Diseases Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Blood Coagulation Disorders Hematologic Diseases |
Hemorrhage Pathologic Processes Skin Manifestations Thrombotic Microangiopathies Blood Platelet Disorders Hemorrhagic Disorders Autoimmune Diseases |