- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03524612
A Study to Assess the Ability of Eltrombopag to Induce Sustained Response Off Treatment in Subjects With ITP (TAPER)
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
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Protocol Amendment 01 added a follow-up period of 12 months for patients with sustained response off treatment at month 12 to obtain further data on response duration.
The starting dose was eltrombopag 50 mg daily (25 mg daily for Asian patients and 12.5 mg daily for Japanese patients in Japan). The starting dose for this study was consistent with the dosing guidelines approved for eltrombopag use in ITP. An increase of eltrombopag dose up to 75 mg was allowed for patients who did not respond to standard-dosage treatment and to reduce the risk of bleeding. The rationale for this increased dose was to use the minimal efficacious dosage of eltrombopag in order to achieve a platelet count ≥ 100×10^9/L and maintain it around 100×10^9/L (no counts below 70×10^9/L) for 2 months in order to allow patients to start the eltrombopag tapering and withdrawal process.
Patients who reached a platelet count of >= 100 × 10^9/L and maintained counts around 100×10^9/L for 2 months with no platelet count < 70 × 10^9/L were eligible for tapering-off and treatment discontinuation, which occurred via 25 mg reduction every 2 weeks up to 25 mg on alternate days for 2 weeks until treatment discontinuation.
Patients who successfully discontinue eltrombopag and maintained platelet count >= 30 × 10^9/L in the absence of bleeding or use of rescue therapy were followed to month 12. If a relapse (defined as platelet count <30 × 10^9/L) occurred during the 12-month treatment period, they were offered a new course of eltrombopag treatment within this timeframe at the appropriate starting dose. If a patient relapsed in the post 12-month follow-up period, no further attempts for tapering and achieving sustained response off treatment were made.
The taper-off scheme followed recommendations within the current established dosing regimen for when to consider dose adjustment.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Linz, Austria, 4010
- Novartis Investigative Site
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BA
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Salvador, BA, Brazil, 41253-190
- Novartis Investigative Site
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RJ
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Rio de Janeiro, RJ, Brazil, 20211-030
- Novartis Investigative Site
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SP
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Sao Paulo, SP, Brazil, 05319-000
- Novartis Investigative Site
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Araucania
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Temuco, Araucania, Chile, 4800827
- Novartis Investigative Site
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Valparaiso
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Vina del Mar, Valparaiso, Chile, 2540364
- Novartis Investigative Site
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Caen Cedex, France, 14033
- Novartis Investigative Site
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Pessac Cedex, France, 33604
- Novartis Investigative Site
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Athens, Greece, 115 27
- Novartis Investigative Site
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Patras, Greece, 265 00
- Novartis Investigative Site
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BO
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Bologna, BO, Italy, 40138
- Novartis Investigative Site
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TS
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Trieste, TS, Italy, 34129
- Novartis Investigative Site
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Aichi
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Nagoya-city, Aichi, Japan, 466-8650
- Novartis Investigative Site
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Ciudad de Mexico, Mexico, 14000
- Novartis Investigative Site
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Jalisco
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Guadalajara, Jalisco, Mexico, 44160
- Novartis Investigative Site
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Muscat, Oman, 123
- Novartis Investigative Site
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Moscow, Russian Federation, 125167
- Novartis Investigative Site
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St Petersburg, Russian Federation, 191024
- Novartis Investigative Site
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Madrid, Spain, 28046
- Novartis Investigative Site
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Madrid, Spain, 28009
- Novartis Investigative Site
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Malaga, Spain, 29010
- Novartis Investigative Site
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Murcia, Spain, 30008
- Novartis Investigative Site
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Castilla Y Leon
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Salamanca, Castilla Y Leon, Spain, 37007
- Novartis Investigative Site
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Catalunya
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Barcelona, Catalunya, Spain, 08035
- Novartis Investigative Site
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Madrid
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Majadahonda, Madrid, Spain, 28222
- Novartis Investigative Site
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Bern, Switzerland, 3010
- Novartis Investigative Site
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Aydin, Turkey, 09100
- Novartis Investigative Site
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Edirne, Turkey, 22030
- Novartis Investigative Site
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Kocaeli, Turkey, 41380
- Novartis Investigative Site
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London, United Kingdom, W12 0HS
- Novartis Investigative Site
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New York
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Nyack, New York, United States, 10960
- Hematology Oncology Association of Rockland Drug Shipment
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Ohio
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Cleveland, Ohio, United States, 44106-5000
- Case Western Reserve SC - 2
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Signed informed consent must be obtained prior to participation in the study
- Patients ≥ 18 years old
- Patients 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 < 30×10^9/L and assessed as needing treatment (per physician's discretion
Exclusion Criteria:
- ITP patients previously treated with any ITP second-line therapies, thrombopoietin receptor (TPO-R) agonists for ITP, except steroids / IVIG
- Patients who relapsed more than one year after the end of first-line full course of steroid therapy
- Patients with a diagnosis of secondary thrombocytopenia
- Patients who have life threatening bleeding complications per investigator discretion
- Patients 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
- Patients who are human immune deficiency virus (HIV), hepatitis C virus (HCV), hepatitis B surface antigen (HBsAg) positive
- Patients with hepatic impairment (Child-Pugh score > 5)
- Patients who have active malignancy
- Patients 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 Patients participating in the study
- Patients with known active or uncontrolled infections not responding to appropriate therapy
- Patients 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 Patients 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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Other: eltrombopag
Participants were treated with eltrombopag to induce sustained response off treatment to reach a target platelet count of >=100×10^9/L (CR), after 1st line steroids had failed.
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12.5, 25, 50 and 75 mg tablets for oral use once daily
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Percentage of Participants With Sustained Response Off Treatment (SRoT) by 12 Months
Time Frame: Month 12
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Sustained response off treatment (SRoT) was defined as: reaching platelet count >= 100×10^9/L (complete response [CR]) and then maintaining platelet counts around 100×10^9/L for 2 months (no counts below 70×10^9/L), AND then tapering off the drug until treatment discontinuation while maintaining platelet count >= 30×10^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy until month 12.
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Month 12
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Median Duration of Sustained Response Off Treatment (SRoT) After Treatment Discontinuation for Participants With Sustained Response Off Treatment
Time Frame: From last dose of eltrombopag to month 12
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Sustained response off treatment (SRoT) was defined as: reaching platelet count >= 100×10^9/L (complete response [CR]) and then maintaining platelet counts around 100×10^9/L for 2 months (no counts below 70×10^9/L), AND then tapering off the drug until treatment discontinuation while maintaining platelet count >= 30×10^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy until month 12.
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From last dose of eltrombopag to month 12
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Estimated Median Duration of Sustained Response Off Treatment (SRoT) for Participants With Sustained Response Off Treatment at Month 12 and Who Enter 12 Months Follow-up Period
Time Frame: From last dose of eltrombopag to relapse, assessed up to month 24
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Sustained response off treatment (SRoT) was defined as: reaching platelet count >= 100×10^9/L (complete response [CR]) and then maintaining platelet counts around 100×10^9/L for 2 months (no counts below 70×10^9/L), AND then tapering off the drug until treatment discontinuation while maintaining platelet count >= 30×10^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy. Patients with SRoT until month 12 who entered follow-up and did not relapse by cut-off date/Month 24 were censored at the earliest of discontinuation date/death date/Month 24 platelet assessment date/cutoff date. Patients with SRoT until Month 12 patients who did not enter or do not yet have data in follow-up phase are censored at their Month 12 platelet assessment date. |
From last dose of eltrombopag to relapse, assessed up to month 24
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Estimated Median Duration of Sustained Response Off Treatment (SRot) for All Patients
Time Frame: From last dose of eltrombopag to month 24
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Patients who tapered and discontinued successfully, or did not relapse/die by cutoff date /month 24 were censored at the earliest of discontinuation date/death date/month 24 platelet assessment date/cutoff date.
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From last dose of eltrombopag to month 24
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Percentage of Participants With Sustained Response Off Treatment Until Month 24
Time Frame: Month 15, 18, 21 and 24
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Sustained response off treatment is defined as reach platelet count ≥ 100×10^9/L (complete response [CR]) and then maintain platelet counts around 100×10^9/L for 2 months (no counts below 70×10^9/L) AND then taper off the drug until treatment discontinuation while, maintain platelet count ≥ 30×10^9/L in the absence of bleeding (no bleeding AEs) or use of any rescue therapy
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Month 15, 18, 21 and 24
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Percentage of Participants With Early Response Within First Month
Time Frame: By 1 month
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Early response is defined as reaching a platelet count >= 50×10^9/L at least once within the first month (month 1) without bleeding events and no rescue therapy.
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By 1 month
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Percentage of Participants With Recovery Response in Case of Loss of Response During or After Tapering of Eltrombopag Until Month 24
Time Frame: Up to month 24
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Recovery response is defined as platelet count >=30×10^9/L after eltrombopag is re-introduced, in case of loss of response (< 30×10^9/L and/or bleeding event) without bleeding events and no rescue therapy.
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Up to month 24
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Relative Change From Baseline in Platelet Count Over Time
Time Frame: Baseline, month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Relative change (%) is the absolute change divided by the platelet counts at baseline and multiplied by 100.
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Baseline, month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Percentage of Participants Who Maintain Platelet Counts Level Within 12 Months and Every 3 Months Until Month 24
Time Frame: From first time of reaching the level to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Platelet counts level is defined as having platelet counts >=30×10^9/L without bleeding events and no rescue therapy.
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From first time of reaching the level to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionaire
Time Frame: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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The Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue©) is a 13- item questionnaire that assesses self-reported fatigue and its impact upon daily activities over the past 7 days.
FACIT-fatigue is scored using a 4-point Likert scale.
Items are scored as follows: 4 = Not At All; 3 = A Little Bit; 2 = Somewhat; 1 = Quite A Bit; 0 = Very Much, EXCEPT items #7 and #8 which are reversed scored.
Score range from 0-52.
A score of less than 30 indicates severe fatigue.
The higher the score, the better the quality of life (less fatigue).
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Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Change From Baseline in Functional Assessment of Cancer Therapy- Thrombocytopenia (FACT-Th6) Questionnaire
Time Frame: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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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
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Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Bodily Pain (BP) Score
Time Frame: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life.
It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS).
The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100.
Higher scores indicative of better HRQoL.
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Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: General Health (GH) Score
Time Frame: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life.
It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS).
The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100.
Higher scores indicative of better HRQoL.
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Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Health (MH) Score
Time Frame: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life.
It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS).
The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100.
Higher scores indicative of better HRQoL.
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Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Functioning (PF) Score
Time Frame: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life.
It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS).
The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100.
Higher scores indicative of better HRQoL.
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Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Emotional (RE) Score
Time Frame: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life.
It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS).
The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100.
Higher scores indicative of better HRQoL.
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Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Role Physical (RP) Score
Time Frame: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life.
It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS).
The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100.
Higher scores indicative of better HRQoL.
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Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Social Functioning (SF) Score
Time Frame: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life.
It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS).
The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100.
Higher scores indicative of better HRQoL.
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Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Vitality (VT) Score
Time Frame: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life.
It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS).
The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100.
Higher scores indicative of better HRQoL.
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Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Physical Component Summary (PCS) Score
Time Frame: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life.
It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS).
The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100.
Higher scores indicative of better HRQoL.
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Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire: Mental Component Summary (MCS) Score
Time Frame: Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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The SF-36 Scale is a 36-item, patient-reported survey which measures overall quality of life.
It consists of 8 subscales (bodily pain (BP), general health (GH), mental health (MH), physical functioning (PF), role emotional (SE), role physical (RP), social role functioning (SF) and vitality (VT)) which can be aggregated to derive a physical-component summary (PCS) score and a mental-component score (MCS).
The SF-36 is scored using norm-based scoring procedures: each sub-scale score ranges from 0 to 10, and the composite score ranges from 0 to 100.
Higher scores indicative of better HRQoL.
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Baseline to month 3, 6, 9, 12 (End of Treatment Visit for non-responders), 15, 18, 21 and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Percentage of Participants With Worst Post-baseline Value in Functional Assessment of Cancer Therapy-G (GP5)
Time Frame: Baseline to end of treatment visit, assessed up to 12 months for non-responders and up to 24 months for responders
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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.
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Baseline to end of treatment visit, assessed up to 12 months for non-responders and up to 24 months for responders
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Overall Change of Treatment Satisfaction Using Treatment Satisfaction Questionnaire (TSQM-9)
Time Frame: Baseline to month 12 (End of Treatment Visit for non-responders) and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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The Treatment Satisfaction Questionnaire for Medication (TSQM-9) is a psychometric measure of a patient's satisfaction with medication.
It consists of 3 subscales: effectiveness, convenience and global satisfaction.
The scores were computed by adding items for each domain, i.e. 1 to 3 for effectiveness, 4 - 6 for convenience and 7 to 9 for global satisfaction.
The lowest possible score (1 for each item and 3 for all 3 subscales) was subtracted from the composite score and divided by the greatest possible score range.
The greatest range was (7-1) X 3 items = 18 for the effectiveness and convenience, and (5-1) x 3 items = 12 for global satisfaction.
This provided a transformed score between 0 and 1 that was then multiplied by 100.
A positive change from baseline indicates improvement.
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Baseline to month 12 (End of Treatment Visit for non-responders) and 24 (End of Treatment Visit for responders), assessed up to 12 months for non-responders and up to 24 months for responders
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Collaborators and Investigators
Sponsor
Investigators
- Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Pathologic Processes
- Immune System Diseases
- Autoimmune Diseases
- Hematologic Diseases
- Hemorrhage
- Hemorrhagic Disorders
- Blood Coagulation Disorders
- Skin Manifestations
- Thrombocytopenia
- Blood Platelet Disorders
- Thrombotic Microangiopathies
- Purpura
- Purpura, Thrombocytopenic
- Purpura, Thrombocytopenic, Idiopathic
Other Study ID Numbers
- CETB115J2411
- 2018-000452-18 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Immune Thrombocytopenic Purpura (ITP)
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Neufeld, Ellis J, MD, PhDGenentech, Inc.; Biogen; Glaser Pediatric Research Network; Terrana ITP Research...CompletedImmune Thrombocytopenic Purpura (ITP) | Idiopathic Thrombocytopenic Purpura (ITP)United States
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AmgenCompletedThrombocytopenia | Immune Thrombocytopenia | Idiopathic Thrombocytopenic Purpura | Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) | Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) | Thrombocytopenic PurpuraUnited States, Canada, Australia
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AmgenCompletedIdiopathic Thrombocytopenic Purpura | Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) | Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
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Gruppo Italiano Malattie EMatologiche dell'AdultoNot yet recruitingITP - Immune Thrombocytopenia | Chronic ITP | Refractory ITP
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Momenta Pharmaceuticals, Inc.TerminatedImmune Thrombocytopenic Purpura (ITP)United States, Poland, Hungary, Netherlands, Italy, Spain, Belgium
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National Institute of Blood and Marrow Transplant...NovartisUnknownImmune Thrombocytopenic Purpura (ITP)Pakistan
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Kyowa Kirin Co., Ltd.CompletedImmune (Idiopathic) Thrombocytopenic Purpura (ITP)Japan
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Genentech, Inc.CompletedHIV Infections | Immune Thrombocytopenic Purpura ( ITP )United States
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Protalex, Inc.TerminatedPhase I Safety and Tolerability Study of Staphylococcal Protein A in Adult Patients With Chronic ITPIdiopathic Thrombocytopenic Purpura (ITP)Australia, New Zealand
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CSL LimitedCompletedIdiopathic Thrombocytopenic Purpura (ITP)Australia
Clinical Trials on eltrombopag
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Novartis PharmaceuticalsCompletedPurpura, Thrombocytopenic, IdiopathicRussian Federation
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Fondazione Progetto EmatologiaCompletedChronic Lymphocytic Leukemia | Purpura, Thrombocytopenic, Idiopathic | Non Hodgkin's Lymphoma | Autoimmune Thrombocytopenia | Autoimmune Thrombocytopenic PurpuraItaly
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Weill Medical College of Cornell UniversityNovartisTerminatedImmune ThrombocytopeniaUnited States
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Institute of Hematology & Blood Diseases Hospital...Xijing Hospital; Xi'an Central Hospital; The Second Hospital of Hebei Medical... and other collaboratorsActive, not recruitingPreviously Treated Primary Immune ThrombocytopeniaChina
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Tel-Aviv Sourasky Medical CenterNovartisRecruitingB Cell Lymphoma | CART TreatmentIsrael
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Gruppo Italiano Malattie EMatologiche dell'AdultoCompletedPrimacy Immune ThrombocytopeniaItaly
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Novartis PharmaceuticalsCompletedPurpura, Thrombocytopaenic, Idiopathic
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Nanfang Hospital of Southern Medical UniversityGuangzhou First People's Hospital; Second Affiliated Hospital, Sun Yat-Sen... and other collaboratorsUnknownAcute Myeloid Leukemia | Thrombocytopenia | EltrombopagChina
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IRCCS Policlinico S. MatteoCompleted