Poor Graft FunctionPhase 2ELTIONNCT03718533
Study of Efficacy and Safety of Eltrombopag in Patients With Poor Graft Function
A Phase 2 study testing Eltrombopag in people with Poor Graft Function, run by Novartis Pharmaceuticals at 7 sites in Spain.
Trial summary
The purpose of this study was to evaluate the efficacy of eltrombopag for poor graft function (PGF) on overall hematologic response (partial and complete), as determined by platelet, hemoglobin and neutrophil counts by 16 weeks after the initiation of eltrombopag in patients with poor graft function after allogeneic… Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Poor Graft Function
- What is being tested?
- Eltrombopag
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 7 sitesSpain
- Study dates
- 17 Dec 2018to 3 Nov 2020
- 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 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient must be able to understand and communicate with the investigator and comply with the requirements of the study and must provide written, signed and dated informed consent form before any study assessment is performed
- Male of female patients ≥ 18 years of age
- Patients diagnosed with primary or secondary poor graft function (PGF) defined as two or more cytopenias after day +30 post-transplant (re-tested in a peripheral blood analysis at screening):
- Platelet count <20,000/ µL (mandatory)
- Absolute neutrophil count (ANC) <1,000/µL
- Hemoglobin <100 g/L
- Presence of donor chimerism >90% in screening visit
- Karnofsky status ≥90% (Karnofsky assessment must be performed within 7 days prior to Day 1)
Who cannot join
- Pregnant or nursing (lactating women).
- Evidence of active acute or chronic graft versus host disease (GVHD).
- Evidence of any active malignancy.
- Subjects who are human immune deficiency virus (HIV), hepatitis C virus (HCV), hepatitis B surface antigen (HBsAg) positive in screening visit.
- Cytogenetic abnormality in chromosome 7 present before the allo-HSC.
- Evidence of any clonal abnormality on cytogenetics (in bone marrow analysis).
Show all 28Show fewer
- A local post-transplant conventional cytogenetic assessment should be available within 8 weeks before Day 1.
- If the cytogenetics is not valuable, i.e, it does not show metaphases, a FISH for MDS-related most frequent abnormalities including chromosome 7 is accepted.
- As a consequence, patients with dry tap bone marrow aspiration are NOT eligible.
- Evidence of bone marrow involvement or progression of the underlying disease assessed by the applicable methods in each case.
- Evidence of thrombotic microangiopathy.
- Evidence of possible causes of cytopenia other than PGF (active infections, myelotoxic drugs, hypersplenism…).
- Prior use of any thrombopoietin receptor (TPO-R) agonists for PGF.
- AST or ALT levels >3 x ULN.
- Creatinine level ≥1.5 x ULN.
- Total bilirubin level ≥1.5 x ULN.
- Previous thromboembolic event (other than line-related upper extremity thrombosis)
- Hypersensitivity to eltrombopag or its components.
- Clinically significant ECG abnormality history or current diagnosis of cardiac disease indicating significant risk of safety for subjects participating in the study such as uncontrolled or significant cardiac disease or impaired cardiac function including any of the following:
- . Corrected QTc > 450 msec (male subjects), > 460 msec (female subjects) using Fredericia correction (QTcF) on the screening ECG
- . Myocardial infarction
- . Uncontrolled congestive heart failure
- . Unstable angina
- . Congenital long QT syndrome.
- Administration of an investigational drug within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study treatment.
- Patient with liver cirrhosis.
- Risk factors for Torsade de Pointes including uncorrected hypokalemia or hypomagnesemia.
- Subjects with any serious and/ or unstable pre-existing medical, psychiatric disorder or other conditions that could interfere with patient´s safety, obtaining informed consent or compliance with the study procedures as per investigator discretion.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
Spain7 sites
Novartis Investigative Site
Barcelona, 08041
Novartis Investigative Site
Donostia / San Sebastian, Basque Country, 20080
Novartis Investigative Site
Málaga, Andalusia, 29010
Novartis Investigative Site
Palma de Mallorca, Balearic Islands, 07120
Novartis Investigative Site
Salamanca, Castille and León, 37007
Novartis Investigative Site
Valencia, Valencia, 46010
Novartis Investigative Site
Vigo, Pontevedra, 36212
About this study
Official title: The ELTION Study - A Multicenter Open-label Interventional Study of Eltrombopag in Patients With Poor Graft Function After Allogeneic Hematopoietic Stem Cell Transplantation
The purpose of this study was to evaluate the efficacy of eltrombopag for poor graft function (PGF) on overall hematologic response (partial and complete), as determined by platelet, hemoglobin and neutrophil counts by 16 weeks after the initiation of eltrombopag in patients with poor graft function after allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Read the detailed description
This was an open-label, single-arm phase II study, in which participants diagnosed with PGF after allo-HSCT were treated with eltrombopag up to week 36 or until the participant's premature withdrawal.
The study consisted of the following periods:
- Screening Period (baseline): the participant screening criteria was reviewed, and the procedures established in the evaluation schedule were performed.
- Treatment Period: from administration of the first dose of eltrombopag until time when participant permanently stopped taking study treatment for any reason or until 36 weeks had passed. Eltrombopag was initiated on Day 1 at a dose of 150 mg once daily (75 mg daily in Asian ancestry participants). During the Treatment Period, participants were evaluated weekly during the first month, every 2 weeks during the next 2 months and subsequently every 4 weeks until week 24. For the last 3 months, participants were followed every 6 weeks Only participants who had a partial or complete response at Week 16 continued to receive eltrombopag up to Week 36 or until loss of response (defined as a decrease in blood counts to levels that did not continue to meet the criteria for response established in the protocol), unacceptable toxicity, or discontinuation for any other reason.
Participants who discontinued eltrombopag because efficacy, continued in the study and attended the scheduled visits of Treatment Period as per protocol. If loss of response occurred, eltrombopag was reintroduced at the last effective dose.
- Final Visit (or Early Withdrawal): this visit took place 30 days after completion of the Treatment Period, or premature participant withdrawal.
- Follow-up for Survival: all participants who discontinued from the study, regardless the reason of discontinuation, were followed for survival for 24 and 36 weeks, unless they withdrew their consent, died or were lost-to follow-up, in which case study visits were no longer carried out.
- Conditions
- Poor Graft Function
- Being tested
- Eltrombopag
- Keywords
- Eltrombopag, Hematological response, Poor graft function, Stem cell transplantation, Adult, ELTION, Phase II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
Eltrombopag was provided as 50 mg or 25 mg film-coated tablets for oral use administration
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag | Experimental | Patients received eltrombopag orally once daily up to 36 weeks. Receives: Drug: Eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hematologic Response Rate by 16 Weeks After the Initiation of Eltrombopag
Hematologic response rate was defined as the percentage of participants who met the criteria of either complete response (CR) or partial response (PR) by Week 16. PR was defined when any of the following: Platelet count ≥ 20000/microliter(μL) (with platelet transfusion independence), absolute neutrophil count (ANC) ≥1000/μL (when pretreatment ANC was <1000/μL) and/or hemoglobin (Hb) ≥100 gram(g)/ liter(L) (when pretreatment Hb was <100g/L) (with red blood cells transfusion independence), confirmed in two blood tests separated a minimum of 7 days. CR was defined when all of the following: platelet count ≥100000/μL, ANC ≥1500/μL (when pretreatment ANC was <1000/μL) and Hb ≥110 g/L (when pretreatment Hb was <100g/L), confirmed in two blood tests separated a minimum of 7 days. Participants who discontinued before Week 16 were considered as responders if, in the last evaluation, they had PR or CR. The 95% Confidence Interval (CI) was the binomial exact CI based on Clopper-Pearson method.
Measured: Baseline up to Week 16
Secondary outcomes (11)
Percentage of Participants Who Had a Response in the Neutrophil Lineage
Percentage of participants who had a response (partial or complete) in the neutrophil lineage. A partial response in the neutrophil lineage was defined as absolute neutrophil count (ANC) ≥1000/ µL (when pretreatment ANC was <1000/μL) confirmed in two consecutive blood tests separated a minimum of 7 days. A complete response in the neutrophil lineage was defined as ANC ≥ 1500/ µL (when pretreatment ANC was <1000/μL) confirmed in two consecutive blood tests separated a minimum of 7 days. The 95% CI was the binomial exact CI based on Clopper-Pearson method
Measured: Week 16, 20, 24, 30 and 36
Percentage of Participants Who Had a Response in the Platelet Lineage
Percentage of participants who had a response (partial or complete) in the platelet lineage. A partial response in the platelet lineage was defined as platelet count ≥20000/µL (with platelet transfusion independence) confirmed in two consecutive blood tests separated a minimum of 7 days. A complete response in the platelet lineage was defined as platelet count ≥ 100000/µL confirmed in two consecutive blood tests separated a minimum of 7 days. The 95% CI was the binomial exact CI based on Clopper-Pearson method.
Measured: Week 16, 20, 24, 30 and 36
Percentage of Participants Who Had a Response in the Hemoglobin Lineage
Percentage of participants who had a response (partial or complete) in the hemoglobin (Hb) lineage. A partial response in the Hb lineage was defined as Hb ≥100 g/L (when pretreatment Hb was <100g/L) (with RBC transfusion independence), confirmed in two consecutive blood tests separated a minimum of 7 days. A complete response in the Hb lineage was defined as Hb≥ 110 g/L (when pretreatment Hb was <100g/L) confirmed in two consecutive blood tests separated a minimum of 7 days. The 95% CI was the binomial exact CI based on Clopper-Pearson method.
Measured: Week 16, 20, 24, 30 and 36
Hematologic Response Rate at Week 24 and 36
Hematologic response rate was defined as the percentage of participants who met the criteria of either complete response (CR) or partial response (PR) at 24 weeks and 36 weeks after the initiation of eltrombopag. PR was defined when any of the following: Platelet count ≥20000/microliter (μL) (with platelet transfusion independence), absolute neutrophil count (ANC) ≥1000/μL (when pretreatment ANC was <1000/μL) and/or hemoglobin (Hb) ≥100 gram (g)/ liter (L) (when pretreatment Hb was <100g/L) (with red blood cells transfusion independence), confirmed in two blood tests separated a minimum of 7 days. CR was defined when all three of the following: platelet count ≥100000/μL, ANC ≥1500/μL (when pretreatment ANC was <1000/μL) and Hb ≥110 g/L (when pretreatment Hb was <100g/L), confirmed in two blood tests separated a minimum of 7 days. The 95% CI was the binomial exact CI based on Clopper-Pearson method.
Measured: Week 24 and 36
Percentage of Participants Who Were Previously Platelet Transfusion-dependent and Did no Longer Require Platelet Transfusions After the Initiation of Eltrombopag
Percentage of participants who received at least one platelet transfusion before starting treatment and who did no longer require platelet transfusion before and after the first 16 weeks of treatment. The 95% CI was the binomial exact CI based on Clopper-Pearson method.
Measured: From start of treatment to end of treatment, assessed up to 36 weeks
Percentage of Participants Who Were Previously Red Blood Cells Transfusion-dependent and Did no Longer Require Red Blood Cells Transfusions
Percentage of participants who received at least one red blood cells transfusion before starting treatment and who did no longer require red blood cells transfusion before and after the first 16 weeks of treatment. The 95% CI was the binomial exact CI based on Clopper-Pearson method.
Measured: From start of treatment to end of treatment, assessed up to 36 weeks
Duration of Transfusion Independence
Duration of transfusion independence defined as the period of time where participants did not receive any platelet or red blood cells transfusions during the treatment period
Measured: From start of treatment to end of treatment, assessed up to 36 weeks
Percentage of Participants Who Discontinued or Reduced the Use of Concomitant Erythropoietin (EPO) Therapy
Percentage of participants who discontinued or reduced by ≥50% from baseline the use of concomitant EPO therapy while receiving eltrombopag. The 95% CI was the binomial exact CI based on Clopper-Pearson method.
Measured: From start of treatment to end of treatment, assessed up to Week 36
Percentage of Participants Who Discontinued or Reduced the Use of Concomitant Granulocyte Colony-stimulating Factor (G-CSF) Therapy
Percentage of participants who discontinued or reduced by ≥50% from baseline the use of concomitant G-CSF therapy while receiving eltrombopag. The 95% CI was the binomial exact CI based on Clopper-Pearson method.
Measured: From start of treatment to end of treatment, assessed up to Week 36
Overall Survival (OS)
OS defined as the time from the date of inclusion until the date of death due to any cause was calculated using Kaplan-Meier estimated. All patients who discontinued from the study, regardless the reason of discontinuation, were followed for survival, unless they withdrew their consent, died or were lost-to follow-up, in which case were censored at the last contact.
Measured: From start of treatment until the date of death, assessed up to 40 weeks
Overall Survival Rate at 24 and 36 Weeks
Overall survival rate defined as the rate estimate of the percentage of participants who were alive at 24 and 36 weeks. All patients who discontinued from the study, regardless the reason of discontinuation, were followed for survival at Week 24 and 36, unless they withdrew their consent, died or were lost-to follow-up, in which case were censored at the last contact.
Measured: Week 24 and 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2018
- Study start17 Dec 2018
- Primary completion3 Nov 2020
- Study completion3 Nov 2020
Why it stopped: In the context of COVID-19 pandemic, the benefit / risk ratio of the participation of a patient with pancytopenia could be compromised. The decision of this premature termination was not the consequence of any safety reason inherent to the drug.
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 Poor Graft FunctionStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03718533
- Sponsor's protocol ID
- CETB115EES03
- Other IDs
- 2018-001129-15, 2018
- Acronym
- ELTION
- Status last verified
- Feb 2024
- First submitted
- 23 Oct 2018
- Last updated
- 29 Feb 2024
- 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 29 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The ELTION Study - A Multicenter Open-label Interventional Study of Eltrombopag in Patients With Poor Graft Function After Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT03718533
- Acronym / sponsor ID
- ELTION / CETB115EES03
- Conditions
- Poor Graft Function
- Keywords
- Eltrombopag, Hematological response, Poor graft function, Stem cell transplantation, Adult, ELTION, Phase II
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: In the context of COVID-19 pandemic, the benefit / risk ratio of the participation of a patient with pancytopenia could be compromised. The decision of this premature termination was not the consequence of any safety reason inherent to the drug.
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 17 Dec 2018 (actual)
- Primary completion
- 3 Nov 2020 (actual)
- Study completion
- 3 Nov 2020 (actual)
- First posted
- 24 Oct 2018 (actual)
- Results
- Posted 5 Feb 2024
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 evaluate the efficacy of eltrombopag for poor graft function (PGF) on overall hematologic response (partial and complete), as determined by platelet, hemoglobin and neutrophil counts by 16 weeks after the initiation of eltrombopag in patients with poor graft function after allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Detailed description
This was an open-label, single-arm phase II study, in which participants diagnosed with PGF after allo-HSCT were treated with eltrombopag up to week 36 or until the participant's premature withdrawal.
The study consisted of the following periods:
- Screening Period (baseline): the participant screening criteria was reviewed, and the procedures established in the evaluation schedule were performed.
- Treatment Period: from administration of the first dose of eltrombopag until time when participant permanently stopped taking study treatment for any reason or until 36 weeks had passed. Eltrombopag was initiated on Day 1 at a dose of 150 mg once daily (75 mg daily in Asian ancestry participants). During the Treatment Period, participants were evaluated weekly during the first month, every 2 weeks during the next 2 months and subsequently every 4 weeks until week 24. For the last 3 months, participants were followed every 6 weeks Only participants who had a partial or complete response at Week 16 continued to receive eltrombopag up to Week 36 or until loss of response (defined as a decrease in blood counts to levels that did not continue to meet the criteria for response established in the protocol), unacceptable toxicity, or discontinuation for any other reason.
Participants who discontinued eltrombopag because efficacy, continued in the study and attended the scheduled visits of Treatment Period as per protocol. If loss of response occurred, eltrombopag was reintroduced at the last effective dose.
- Final Visit (or Early Withdrawal): this visit took place 30 days after completion of the Treatment Period, or premature participant withdrawal.
- Follow-up for Survival: all participants who discontinued from the study, regardless the reason of discontinuation, were followed for survival for 24 and 36 weeks, unless they withdrew their consent, died or were lost-to follow-up, in which case study visits were no longer carried out.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eltrombopag | Experimental | Drug: EltrombopagPatients received eltrombopag orally once daily up to 36 weeks. |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Patient must be able to understand and communicate with the investigator and comply with the requirements of the study and must provide written, signed and dated informed consent form before any study assessment is performed
- Male of female patients ≥ 18 years of age
- Patients diagnosed with primary or secondary poor graft function (PGF) defined as two or more cytopenias after day +30 post-transplant (re-tested in a peripheral blood analysis at screening):
- Presence of donor chimerism >90% in screening visit
- Karnofsky status ≥90% (Karnofsky assessment must be performed within 7 days prior to Day 1)
Key exclusion criteria (28 listed)
- Pregnant or nursing (lactating women).
- Evidence of active acute or chronic graft versus host disease (GVHD).
- Evidence of any active malignancy.
- Subjects who are human immune deficiency virus (HIV), hepatitis C virus (HCV), hepatitis B surface antigen (HBsAg) positive in screening visit.
- Cytogenetic abnormality in chromosome 7 present before the allo-HSC.
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 |
|---|---|---|
| EltrombopagEltrombopag was provided as 50 mg or 25 mg film-coated tablets for oral use administration | Drug | Eltrombopag |
Primary outcome
Hematologic Response Rate by 16 Weeks After the Initiation of Eltrombopag
Time frame: Baseline up to Week 16
Hematologic response rate was defined as the percentage of participants who met the criteria of either complete response (CR) or partial response (PR) by Week 16. PR was defined when any of the following: Platelet count ≥ 20000/microliter(μL) (with platelet transfusion independence), absolute neutrophil count (ANC) ≥1000/μL (when pretreatment ANC was <1000/μL) and/or hemoglobin (Hb) ≥100 gram(g)/ liter(L) (when pretreatment Hb was <100g/L) (with red blood cells transfusion independence), confirmed in two blood tests separated a minimum of 7 days. CR was defined when all of the following: platelet count ≥100000/μL, ANC ≥1500/μL (when pretreatment ANC was <1000/μL) and Hb ≥110 g/L (when pretreatment Hb was <100g/L), confirmed in two blood tests separated a minimum of 7 days. Participants who discontinued before Week 16 were considered as responders if, in the last evaluation, they had PR or CR. The 95% Confidence Interval (CI) was the binomial exact CI based on Clopper-Pearson method.
11 secondary outcomes
Percentage of Participants Who Had a Response in the Neutrophil Lineage
Time frame: Week 16, 20, 24, 30 and 36
Percentage of Participants Who Had a Response in the Platelet Lineage
Time frame: Week 16, 20, 24, 30 and 36
Percentage of Participants Who Had a Response in the Hemoglobin Lineage
Time frame: Week 16, 20, 24, 30 and 36
Hematologic Response Rate at Week 24 and 36
Time frame: Week 24 and 36
Percentage of Participants Who Were Previously Platelet Transfusion-dependent and Did no Longer Require Platelet Transfusions After the Initiation of Eltrombopag
Time frame: From start of treatment to end of treatment, assessed up to 36 weeks
Percentage of Participants Who Were Previously Red Blood Cells Transfusion-dependent and Did no Longer Require Red Blood Cells Transfusions
Time frame: From start of treatment to end of treatment, assessed up to 36 weeks
Duration of Transfusion Independence
Time frame: From start of treatment to end of treatment, assessed up to 36 weeks
Percentage of Participants Who Discontinued or Reduced the Use of Concomitant Erythropoietin (EPO) Therapy
Time frame: From start of treatment to end of treatment, assessed up to Week 36
Percentage of Participants Who Discontinued or Reduced the Use of Concomitant Granulocyte Colony-stimulating Factor (G-CSF) Therapy
Time frame: From start of treatment to end of treatment, assessed up to Week 36
Overall Survival (OS)
Time frame: From start of treatment until the date of death, assessed up to 40 weeks
Overall Survival Rate at 24 and 36 Weeks
Time frame: Week 24 and 36
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Feb 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Plain Language Trial SummaryLink 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.
The record lists no contact. Use the official record to reach the sponsor.
7 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 in people with Poor Graft Function, run by Novartis Pharmaceuticals at 7 sites in Spain.
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 8 inclusion and 28 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 29 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Spain. 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 5 Feb 2024). 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?
Hematologic Response Rate by 16 Weeks After the Initiation of Eltrombopag (time frame: Baseline up to Week 16). 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 29 Feb 2024.