Immune Thrombocytopenia (ITP)Phase 2XPAG-ITPNCT04346654
A Study to Assess Efficacy and Safety of Eltrombopag in Combination With a Short Course of Dexamethasone in Patients With Newly Diagnosed ITP
A Phase 2 study testing Eltrombopag and Dexamethasone in people with Immune Thrombocytopenia (ITP), run by Novartis Pharmaceuticals at 10 sites in Germany.
Trial summary
The purpose of this study was to compare the ability of eltrombopag in combination with a short course of high-dose dexamethasone to induce sustained response off treatment in patients with newly-diagnosed ITP versus 1-3 cycles of dexamethasone monotherapy. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Immune Thrombocytopenia (ITP)
- What is being tested?
- Eltrombopag and Dexamethasone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 10 sitesGermany
- Study dates
- 9 Oct 2020to 22 Sep 2023
- 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 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed informed consent must be obtained prior to participation in the study.
- Men and women ≥ 18 years of age
- Newly diagnosed with primary ITP (time from diagnosis within 3 months)
- Platelet count < 30 × 109/L at screening and a need for treatment (per physician's discretion) Note: If pre-treatment is necessary, platelet count data performed directly before pre-treatment (can be used for study inclusion (screening value). Treatment-naïve patients will be included based on their platelet counts performed at screening
Who cannot join
- Previous history of treatment for ITP, except any ITP-directed therapy for a maximum of 3 days within 7 days before randomization
- Patients with diagnosis of secondary thrombocytopenia
- Patients who have life threatening bleeding complications per physician´s discretion
- Patients with a history of thromboembolic events in the 6 months preceding enrollment or known risk factors for thromboembolism
- Serum creatinine > 1.5 mg/dL
- Total bilirubin (TBIL) > 1.5 × upper limit of normal (ULN)
Show all 17Show fewer
- Aspartate transaminase (AST) > 3.0 × ULN
- Alanine transaminase (ALT) > 3.0 × ULN
- Patients who are human immune deficiency virus (HIV),hepatitis C virus (HCV) or hepatitis B surface antigen (HBsAg) positive
- Patients with hepatic impairment (Child-Pugh score > 5)
- Patients with known active or uncontrolled infections not responding to appropriate therapy
- History of current diagnosis of cardiac disease or impaired cardiac function denoted
- Patients who have active malignancy
- Patients with evidence of current alcohol/drug abuse
- 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
- 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
- Women of child-bearing potential and males unwilling to use adequate contraception during the study
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
Germany10 sites
Novartis Investigative Site
Aachen, 52074
Novartis Investigative Site
Aschaffenburg, Bavaria, 63739
Novartis Investigative Site
Chemnitz, 09113
Novartis Investigative Site
Donauwörth, 86609
Novartis Investigative Site
Dortmund, 44263
Novartis Investigative Site
Dresden, 01307
Novartis Investigative Site
Frankfurt, 60590
Novartis Investigative Site
Jena, 07740
Novartis Investigative Site
Kiel, 24116
Novartis Investigative Site
Kronach, 96317
About this study
Official title: A Phase II, Randomized (1:1) Open Label Study to Assess the Efficacy and Safety of Eltrombopag in Combination With Dexamethasone Compared to Dexamethasone, as First-line Treatment in Adult Patients With Newly Diagnosed Immune Thrombocytopenia
The purpose of this study was to compare the ability of eltrombopag in combination with a short course of high-dose dexamethasone to induce sustained response off treatment in patients with newly-diagnosed ITP versus 1-3 cycles of dexamethasone monotherapy.
The unmet clinical need and the potential for eltrombopag when added to steroids to improve the treatment outcome and the potential to induce sustained response off treatment serve as the basis for clinical investigation of eltrombopag in first-line ITP.
Read the detailed description
This is a Phase II, multicenter, 1:1 randomized, open-label study that compared the efficacy and safety of eltrombopag in combination with a short course of high-dose dexamethasone to 1-3 cycles of high-dose dexamethasone monotherapy, as first-line treatment in adult patients with newly diagnosed ITP.
Adult patients with newly diagnosed ITP who had platelet counts < 30 × 10^9/L and required treatment were screened, and if eligible, were randomized to either Arm A (eltrombopag in combination with a short course of dexamethasone) or Arm B (1-3 cycles of dexamethasone monotherapy).
The study was conducted in the following periods:
Screening Period: Patients were screened for 14 days based on the inclusion and exclusion criteria.
Treatment Period: Arm A: Patients were treated for 26 weeks during the treatment period. Patients who reached platelet counts ≥ 30 × 10^9/L and maintained counts ≥ 30 × 10^9/L during the tapering phase were eligible for treatment discontinuation. Duration of tapering before treatment discontinuation at Week 26 was 6 weeks. Arm B: Patients were treated up to 12 weeks during the treatment period. Patients who reached platelet counts ≥ 30 × 10^9/L and maintained counts ≥ 30 × 10^9/L after 1-3 cycles of dexamethasone treatment were eligible for treatment discontinuation. Patients with platelet counts < 30 × 10^9/L after 3 cycles of dexamethasone treatment were offered a course of eltrombopag treatment within the study and were discontinued from study at week 52.
Observation period: After completion of the treatment period, all patients were observed for sustained response off treatment until week 52. Only patients with sustained response at week 52 were followed for another 26 weeks.
- Conditions
- Immune Thrombocytopenia (ITP)
- Being tested
- Eltrombopag, Dexamethasone
- Keywords
- ITP, Eltrombopag, Dexamethasone, Sustained response off treatment, TFR, Adult, ETB115, Platelets, Thrombocytopenic, Thrombopoietin receptor Agonist, Newly-diagnosed, Blood bleeding disorder
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
Also called ETB115
Eltrombopag is for oral use and comes in 25, 50 and 75 mg tablets. Prescribed dose is taken once daily.
DexamethasoneDrug
Dexamethasone is for oral use and comes in 8 mg tablets. Prescribed dose is taken once daily.
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag + Dexamethasone | Experimental | Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment. Receives: Drug: Eltrombopag; Drug: Dexamethasone |
| Dexamethasone | Active comparator | Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment. Receives: Drug: Dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Patients With Sustained Response Off Treatment at 52 Weeks
Sustained response off treatment at 52 weeks is defined as maintenance of platelet count ≥ 30 × 10^9/L after treatment discontinuation until Week 52 in the absence of bleeding events ≥ Grade II or use of any rescue medication at all visits until Week 52. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding.
Measured: Study treatment discontinuation until week 52
Secondary outcomes (12)
Percentage of Patients With Overall Response at Week 52
Overall response after treatment at week 52 was defined as maintenance of platelet count ≥ 30 x 109/L and ≥ 2-fold increase of screening platelet count after treatment discontinuation in the absence of bleeding event ≥ Grade II and no rescue therapy at all visits until Week 52. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding.
Measured: Study treatment discontinuation until week 52
Duration of Sustained Response Off Treatment
Duration of sustained response off treatment is defined as time of treatment discontinuation until platelet count < 30 × 109/L or bleeding events ≥ Grade II or use of any rescue therapy. If sustained response remains, the interval was censored with the date of the last platelet assessment. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding.
Measured: from last dose of study treatment until loss of response, approx. 52 weeks
Overall Response by Week 4
Overall response by week 4 is defined as platelet count ≥ 30 × 109/L and ≥ 2 fold increase of screening platelet count and absence of bleeding and no rescue therapy within the first 4 weeks
Measured: By Week 4
Complete Response by Week 4
Complete Response by week 4 is defined as platelet count ≥ 100 × 109/L and absence of bleeding and no rescue therapy until week 4.
Measured: By Week 4
Absolute Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points
Absolute change in platelet count from pre-treatment or screening to baseline (week 1) and from pre-treatment / screening to 2, 4, 13, 27 and 53 weeks. If pre-treatment was necessary before inclusion, platelet count data performed directly before pre-treatment were used for study inclusion (screening value to be used for inclusion/exclusion check and for analysis as a covariate).
Measured: Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53
Relative Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points
Relative change in platelet count from pre-treatment or screening to baseline (week 1) and from pre-treatment or screening to 2, 4, 13, 27, and 53 weeks. If pre-treatment was necessary before inclusion, platelet count data performed directly before pre-treatment were used for study inclusion (screening value to be used for inclusion/exclusion check and for analysis as a covariate).
Measured: Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53
Time to Overall Response (TOR)
Time to overall response is defined as time from starting study treatment to time of achievement of overall response. Overall response is defined as a platelet count ≥ 30 × 10^9/L and ≥ 2 fold increase of baseline platelet count and absence of bleeding and no rescue therapy censored with the last visit date for patients not achieving overall response. Results of TOR are reported per Kaplan-Meier estimates.
Measured: Time from starting study treatment to achievement of complete response (up to 52 weeks)
Time to Complete Response
Time to complete response is defined as time from starting study treatment to time of achievement of complete response. Complete response is defined as a platelet count ≥ 100 × 109/L and absence of bleeding and no rescue therapy. Results of time to complete response are reported per Kaplan-Meier estimates.
Measured: Time from starting study treatment to achievement of complete response (up to 52 weeks)
Duration of Overall Response (OR) and Complete Response (CR)
Duration of overall or complete response (CR) is defined as time of achievement of overall or CR until loss of overall or CR. The duration of CR was calculated from the date of onset of CR until platelet count < 100 x 109/L, or bleeding events ≥ Grade II (assessed by the modified World Health Organization (WHO) Bleeding Scale) or use of any rescue therapy, whatever was earlier. Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding. Results of duration of overall and complete response are reported per Kaplan-Meier estimates.
Measured: Achievement of overall or complete response until loss of response (up to 52 weeks)
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionnaire
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. Items are scored on a 0-4 response scale (4=not at all to 0=very much) where the total possible score ranges from 0-52 (all items are summed up to create the total score); A score of less than 30 indicates severe fatigue. The higher scores represent better HRQoL.
Measured: Baseline (Week 1), Week 2, 3, 5, 13, 27 and 53
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM))
SF36 questionnaire is a tool to measure health-related QoL. SF36 questionnaires (physical and mental score) were answered throughout the study and is a validated instrument with 36 questions to measure general physical and mental health status 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. The SF36 is scored using norm-based scoring procedures and scores ranging from 0-100; higher values indicate less impairment, a higher QoL. In addition to this SAP-planned scoring score, an alternative scoring for both the physical SF36 score and the mental SF36 were performed by QualityMetric (QM) Incorporated, an IQVIA business.
Measured: Baseline (Week 1), Week 2, 3, 5, 13, 27 and 53
Incidence and Severity of Bleeding Events
Incidence and severity of bleeding assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding).
Incidence of bleeding: participants had at least one bleeding event. Severity of bleeding: bleeding event is from grade 2 and higher
Measured: Baseline up to 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2020
- Study start9 Oct 2020
- Primary completion22 Sep 2023
- Study completion22 Sep 2023
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 Thrombocytopenia (ITP)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaacueticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04346654
- Sponsor's protocol ID
- CETB115JDE01
- Other IDs
- 2019-002658-21, 2019
- Acronym
- XPAG-ITP
- Status last verified
- May 2025
- First submitted
- 12 Apr 2020
- Last updated
- 16 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Randomized (1:1) Open Label Study to Assess the Efficacy and Safety of Eltrombopag in Combination With Dexamethasone Compared to Dexamethasone, as First-line Treatment in Adult Patients With Newly Diagnosed Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT04346654
- Acronym / sponsor ID
- XPAG-ITP / CETB115JDE01
- Conditions
- Immune Thrombocytopenia (ITP)
- Keywords
- ITP, Eltrombopag, Dexamethasone, Sustained response off treatment, TFR, Adult, ETB115, Platelets
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 9 Oct 2020 (actual)
- Primary completion
- 22 Sep 2023 (actual)
- Study completion
- 22 Sep 2023 (actual)
- First posted
- 15 Apr 2020 (actual)
- Results
- Posted 17 Mar 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study was to compare the ability of eltrombopag in combination with a short course of high-dose dexamethasone to induce sustained response off treatment in patients with newly-diagnosed ITP versus 1-3 cycles of dexamethasone monotherapy.
The unmet clinical need and the potential for eltrombopag when added to steroids to improve the treatment outcome and the potential to induce sustained response off treatment serve as the basis for clinical investigation of eltrombopag in first-line ITP.
Detailed description
This is a Phase II, multicenter, 1:1 randomized, open-label study that compared the efficacy and safety of eltrombopag in combination with a short course of high-dose dexamethasone to 1-3 cycles of high-dose dexamethasone monotherapy, as first-line treatment in adult patients with newly diagnosed ITP.
Adult patients with newly diagnosed ITP who had platelet counts < 30 × 10^9/L and required treatment were screened, and if eligible, were randomized to either Arm A (eltrombopag in combination with a short course of dexamethasone) or Arm B (1-3 cycles of dexamethasone monotherapy).
The study was conducted in the following periods:
Screening Period: Patients were screened for 14 days based on the inclusion and exclusion criteria.
Treatment Period: Arm A: Patients were treated for 26 weeks during the treatment period. Patients who reached platelet counts ≥ 30 × 10^9/L and maintained counts ≥ 30 × 10^9/L during the tapering phase were eligible for treatment discontinuation. Duration of tapering before treatment discontinuation at Week 26 was 6 weeks. Arm B: Patients were treated up to 12 weeks during the treatment period. Patients who reached platelet counts ≥ 30 × 10^9/L and maintained counts ≥ 30 × 10^9/L after 1-3 cycles of dexamethasone treatment were eligible for treatment discontinuation. Patients with platelet counts < 30 × 10^9/L after 3 cycles of dexamethasone treatment were offered a course of eltrombopag treatment within the study and were discontinued from study at week 52.
Observation period: After completion of the treatment period, all patients were observed for sustained response off treatment until week 52. Only patients with sustained response at week 52 were followed for another 26 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Eltrombopag + Dexamethasone | Experimental | Drug: Eltrombopag, Drug: DexamethasonePatients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment. |
| Dexamethasone | Active comparator | Drug: DexamethasonePatients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Signed informed consent must be obtained prior to participation in the study.
- Men and women ≥ 18 years of age
- Newly diagnosed with primary ITP (time from diagnosis within 3 months)
- Platelet count < 30 × 109/L at screening and a need for treatment (per physician's discretion) Note: If pre-treatment is necessary, platelet count data performed directly before pre-treatment (can be used for study inclusion (screening value). Treatment-naïve patients will be included based on their platelet counts performed at screening
Key exclusion criteria (17 listed)
- Previous history of treatment for ITP, except any ITP-directed therapy for a maximum of 3 days within 7 days before randomization
- Patients with diagnosis of secondary thrombocytopenia
- Patients who have life threatening bleeding complications per physician´s discretion
- Patients with a history of thromboembolic events in the 6 months preceding enrollment or known risk factors for thromboembolism
- Serum creatinine > 1.5 mg/dL
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 |
|---|---|---|
| EltrombopagAlso: ETB115Eltrombopag is for oral use and comes in 25, 50 and 75 mg tablets. Prescribed dose is taken once daily. | Drug | Eltrombopag + Dexamethasone |
| DexamethasoneDexamethasone is for oral use and comes in 8 mg tablets. Prescribed dose is taken once daily. | Drug | Dexamethasone, Eltrombopag + Dexamethasone |
Primary outcome
Percentage of Patients With Sustained Response Off Treatment at 52 Weeks
Time frame: Study treatment discontinuation until week 52
Sustained response off treatment at 52 weeks is defined as maintenance of platelet count ≥ 30 × 10^9/L after treatment discontinuation until Week 52 in the absence of bleeding events ≥ Grade II or use of any rescue medication at all visits until Week 52. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding.
12 secondary outcomes
Percentage of Patients With Overall Response at Week 52
Time frame: Study treatment discontinuation until week 52
Duration of Sustained Response Off Treatment
Time frame: from last dose of study treatment until loss of response, approx. 52 weeks
Overall Response by Week 4
Time frame: By Week 4
Complete Response by Week 4
Time frame: By Week 4
Absolute Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points
Time frame: Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53
Relative Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points
Time frame: Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53
Time to Overall Response (TOR)
Time frame: Time from starting study treatment to achievement of complete response (up to 52 weeks)
Time to Complete Response
Time frame: Time from starting study treatment to achievement of complete response (up to 52 weeks)
Duration of Overall Response (OR) and Complete Response (CR)
Time frame: Achievement of overall or complete response until loss of response (up to 52 weeks)
Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionnaire
Time frame: Baseline (Week 1), Week 2, 3, 5, 13, 27 and 53
Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM))
Time frame: Baseline (Week 1), Week 2, 3, 5, 13, 27 and 53
Incidence and Severity of Bleeding Events
Time frame: Baseline up to 52 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- A Plain Language Trial Summary is available on www.novctrd.comLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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
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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.
Novartis Pharmaacueticals study director
Novartis Pharmaceuticals
10 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 Eltrombopag and Dexamethasone in people with Immune Thrombocytopenia (ITP), run by Novartis Pharmaceuticals at 10 sites in Germany.
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 4 inclusion and 17 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: finished, as of the sponsor's last update on 16 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Germany. 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 17 Mar 2025). 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?
Percentage of Patients With Sustained Response Off Treatment at 52 Weeks (time frame: Study treatment discontinuation until week 52). 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 16 May 2025.