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Severe Aplastic AnemiaPhase 2SOARNCT02998645

Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia

A Phase 2 study testing eltrombopag and Cyclosporine in people with Severe Aplastic Anemia, run by Novartis Pharmaceuticals at 20 sites in 9 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

The purpose of this study was to evaluate the efficacy and safety of eltrombopag in combination with cyclosporine alone as first-line therapy on overall hematologic response Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Severe Aplastic Anemia
What is being tested?
eltrombopag and Cyclosporine
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
54 participants
Where
20 sitesin 9 countries
Study dates
11 May 2017to 30 May 2022
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patient had signed the Informed Consent (ICF) prior to any screening procedures
  • Patient was male/female ≥18 years old at the time of informed consent and able to swallow a tablet.
  • Patient had SAA characterized by:
  • Bone marrow cellularity <30% (excluding lymphocytes) and
  • At least two of the following (peripheral blood):
  • Absolute neutrophil count <500/µL
Show all 11Show fewer
  • Platelet count <20,000/µL
  • Absolute reticulocyte count <60,000/µL
  • Normal ECG defined as the following as determined via the mean of a triplicate ECG
  • Resting heart rate: 50-90 bpm
  • QTcF at screening <450 msec (for male patients), ≤460 msec (for female patients)

Who cannot join

  • Diagnosis of Fanconi anemia.
  • Evidence of a clonal hematologic bone marrow disorder on cytogenetics by central review
  • Prior immunosuppressive therapy with cyclosporine, alemtuzumab, rabbit or horse ATG and thrombopoietin receptor (TPO-R) agonists.
  • Hypersensitivity to eltrombopag or cyclosporine or their components.
  • AST or ALT >3 x ULN.
  • Serum creatinine, total bilirubin, or alkaline phosphatase >1.5 x ULN.
Show all 19Show fewer
  • Patient with liver cirrhosis.
  • Patients who were human immune deficiency virus (HIV), hepatitis C virus or hepatitis B surface antigen (HBsAg) positive. HCV-RNA negative patients were allowed to be enrolled.
  • Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient's ability to consent, be compliant with study procedures, tolerate protocol therapy, or that death within 30 days is likely.
  • Patients with cancer who were not considered cure, were on active chemotherapeutic treatment or who took drugs with hematological effects.
  • Administration of an investigational drug within 30 days or 5 half-lives, whichever was longer, preceding the first dose of study treatment.
  • Pregnancy statements and contraception requirements:
  • Pregnancy or nursing (lactating) women Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant (or female partners of male patients), unless they were using highly effective methods of contraception during dosing and for 3 months after stopping medication.
  • Not able to understand the investigation nature of the study or to give informed consent.
  • Clinically significant ECG abnormality including cardiac arrhythmias (e. g. ventricular tachycardia) complete left bundle branch block, high grade atrioventricular block, or inability to determine the QTcF interval on the ECG.
  • Presence of cardiac disease, or family history of idiopathic sudden death or congenital long QT syndrome.
  • Risk factors for Torsades de Pointe including uncorrected hypokalemia or hypomagnesemia, or use of concomitant medication(s) with a known risk to prolong the QT interval that could not be discontinued or replaced by safe alternative medication per www.qtdrugs.org.
  • ECOG performance status of ≥2.
  • Patients under the age of 40 must be referred for consideration of allogeneic bone marrow transplantation (HSCT) if (human leukocyte antigen) HLA matching had been done and a suitable matched sibling donor was available and the patient was willing to undergo transplantation (i.e. patients who did not have a HLA match or were not medically fit, not willing or unable to undergo transplantation were considered for enrollment).

Trial locations and contacts

20 sites in 9 countries.

20 sites in 9 countries

Italy3 sites

  • Novartis Investigative Site

    Bologna, BO, 40138

  • Novartis Investigative Site

    Brescia, BR, 25123

  • Novartis Investigative Site

    Milan, MI, 20122

Other trials in Italy

Mexico3 sites

  • Novartis Investigative Site

    Mexico D F, Mexico City, 06726

  • Novartis Investigative Site

    Monterrey, Nuevo León, 64460

  • Novartis Investigative Site

    Puebla City, 72000

Other trials in Mexico

Thailand3 sites

  • Novartis Investigative Site

    Bangkok, 10330

  • Novartis Investigative Site

    Bangkok, 10700

  • Novartis Investigative Site

    Chiang Mai, 50200

Other trials in Thailand

Brazil2 sites

  • Novartis Investigative Site

    Ribeirão Preto, São Paulo, 14048-900

  • Novartis Investigative Site

    São Paulo, São Paulo, 01323-900

Other trials in Brazil

India2 sites

  • Novartis Investigative Site

    Hyderabad, Telangana, 500082

  • Novartis Investigative Site

    Vellore

Other trials in India

South Korea2 sites

  • Novartis Investigative Site

    Seoul, 03722

  • Novartis Investigative Site

    Seoul, 06351

Other trials in South Korea

Spain2 sites

  • Novartis Investigative Site

    Donostia / San Sebastian, Basque Country, 20080

  • Novartis Investigative Site

    Madrid, 28041

Other trials in Spain

Turkey (Türkiye)2 sites

  • Novartis Investigative Site

    Istanbul, 34890

  • Novartis Investigative Site

    Samsun, 55139

Other trials in Turkey (Türkiye)

Hong Kong1 site

  • Novartis Investigative Site

    Hong Kong

Other trials in Hong Kong

About this study

Official title: SOAR, Interventional Phase II Single-arm Study to Assess Efficacy and Safety of Eltrombopag Combined With Cyclosporine as First Line Therapy in Adult Patients With Severe Acquired Aplastic Anemia

The purpose of this study was to evaluate the efficacy and safety of eltrombopag in combination with cyclosporine alone as first-line therapy on overall hematologic response

Read the detailed description

This was an interventional phase II, single-arm, multicenter, open-label, study to investigate the efficacy and safety of the combination of eltrombopag and cyclosporine in treatment-naive, adult subjects with severe aplastic anemia (SAA) as first line therapy.

Eligible subjects received eltrombopag and cyclosporine for up to 6 months. Participants who achieved hematologic response any time on or before 6 months were considered as responders; else they were considered as non-responders.

Responders at Month 6 discontinued eltrombopag and started to taper cyclosporine until relapse or Month 24, whichever was early. Responders who relapsed prior to 6 months and non-responders discontinued the treatment at 6 months and were followed-up for 30 days.

Responders who started to taper cyclosporine and relapsed prior to 24 months discontinued cyclosporine and were followed-up for 30 days.

Being tested
eltrombopag, Cyclosporine
Keywords
severe acquired aplastic anemia, SAA, first line, eltrombopag, cyclosporine, h-ATG, severe aplastic anemia, aplastic anemia
Medical subject headings
Anemia, Aplastic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • eltrombopagDrug

    Also called ETB115

    Film-coated tablets (12.5 mg, 25 mg, 50 mg and 75 mg) administered orally, once daily for up to 6 months. East and Southeast Asian participants were treated with 100 mg once daily, to adjust for the lower apparent clearance of eltrombopag. All other participants were treated with 150 mg once daily.

  • CyclosporineDrug

    Supplied as oral soft gel capsules. The starting dose was based on body weight at 10.0 mg/kg/day (acceptable rounding range was from 9.5 to 10.5 mg/kg/day) in divided doses every 12 hours. After Day 1, dosing was titrated individually according to therapeutic trough level between 200 and 400 μg/L for 6 months. After 6 months (only for responders at Month 6), tapering of cyclosporine was done as follows:

    • 6-9 months: at the 6 months visit, the dose was reduced by 25% for 3 months
    • 9-12 months: at the 9 months visit, the dose was further reduced by 25% for another 3 months
    • 12-24 months: dose was maintained
Study groups in NCT02998645
Study groupRoleWhat they receive
Eltrombopag + cyclosporineExperimentalParticipants received eltrombopag (orally, 150 mg once daily for non-Asian participants / 100 mg once daily for participants of Asian ancestry) in combination with cyclosporine (orally, 10.0 mg/kg/day in divided doses every 12 hours) for up to 6 months. Thereafter, responders at Month 6 were treated with cyclosporine until Month 24
Receives: Drug: eltrombopag; Drug: Cyclosporine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Hematologic Response Rate by 6 Months

    Overall hematologic response rate by 6 months was defined as the percentage of participants with complete response (CR) or partial response (PR) any time on or before 6 months.

    PR was defined as any two of the following parameters at two consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:

    • Absolute neutrophil count (ANC) >500/μL
    • Platelet count >20 000/μL
    • Reticulocyte count >60 000/μL

    CR was defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:

    • ANC > 1 000/μL
    • Platelet count >100 000/μL
    • Hemoglobin >10 g/L

    Measured: Up to 6 months

Secondary outcomes (17)

  1. Overall Hematologic Response Rate by 3 Months

    Overall hematologic response rate by 3 months was defined as the percentage of participants with CR or PR any time on or before 3 months.

    PR was defined as any two of the following parameters at two consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:

    • ANC >500/μL
    • Platelet count >20 000/μL
    • Reticulocyte count >60 000/μL

    CR was defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:

    • ANC > 1 000/μL
    • Platelet count >100 000/μL
    • Hemoglobin >10 g/L

    Measured: Up to 3 months

  2. Overall Hematologic Response Rate at 12 and 24 Months

    Overall hematologic response rate at 12 and 24 months was defined as the percentage of participants with CR or PR at 12 and 24 months respectively.

    PR was defined as any two of the following parameters at two consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:

    • ANC>500/μL
    • Platelet count >20 000/μL
    • Reticulocyte count >60 000/μL

    CR was defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:

    • ANC > 1 000/μL
    • Platelet count >100 000/μL
    • Hemoglobin >10 g/L

    Measured: 12 and 24 months

  3. Duration of First Hematologic Response

    Duration of first hematologic response is the time from the date of the start of first response to the date of first relapse. Relapse is defined as no longer meeting definition of PR or CR. Kaplan-Meier method was used for the analysis. If no relapse occurred, the participant was censored at the date of last contact. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. PR was defined as any 2 of the following parameters at 2 consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:

    • ANC >500/μL
    • Platelet count >20 000/μL
    • Reticulocyte count >60 000/μL.

    CR was defined as all 3 parameters meet the following criteria at 2 consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:

    • ANC >1 000/μL
    • Platelet count >100 000/μL
    • Hemoglobin >10 g/L

    Measured: Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6)

  4. Relapse Rate by 6 and 24 Months

    Relapse was defined as no longer meeting the definition of PR (and not CR). Relapse rate by 6 months and 24 was defined as the percentage of responders by 6 months who relapsed prior to 6 months or prior to 24 months respectively. Responders by 6 months were participants who achieved CR or PR any time on or before 6 months. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24.

    PR: any 2 of the following parameters at 2 consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:

    • ANC>500/μL
    • Platelet count>20 000/μL
    • Reticulocyte count>60 000/μL.

    CR: all 3 parameters meet the following criteria at 2 consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:

    • ANC>1 000/μL
    • Platelet count>100 000/μL
    • Hemoglobin>10 g/L

    Measured: Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6)

  5. Percentage of Participants With Evolution to Myelodysplasia, Paroxysmal Nocturnal Hemoglobinuria (PNH) and Leukemia

    The percentage of participants with evolution to myelodysplasia, PNH and acute leukemia occurring at any time during the study. Clonal evolution to myelodysplasia was defined as a new marrow cytogenic abnormality with or without characteristic dysplastic marrow findings. Clonal evolution to leukemia was defined as greater than 20% peripheral blood and/or marrow blasts. Clonal evolution to paroxysmal nocturnal hemoglobinuria (PNH) was defined as a clone at baseline < 10% that rose to greater than 50% on study.

    Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24

    Measured: Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6)

  6. Percentage of Participants Who Were Red Blood Cells (RBC) Transfusion Independent

    Percentage of participants who were RBC transfusion independent at least once by 6 months and by 24 months (responders only). Independence was defined as no RBC transfusion for at least 56 days.

    Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders.

    Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24.

    Measured: Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6)

  7. Percentage of Participants Who Were Platelet Transfusion Independent

    Percentage of participants who were platelet transfusion independent at least once by 6 months and by 24 months (responders only). Independence was defined as no platelet transfusion for at least 28 days.

    Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders.

    Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24.

    Measured: Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6)

  8. Longest Duration of Transfusion Independence (Platelet or RBC)

    Longest duration of transfusion independence (platelet or RBC) by 6 months and by 24 months (responders only). Transfusion independence was defined as no transfusions required in at least a 28-day period for platelet transfusion and at least 56-day period for RBC transfusion. The duration of the longest interval with transfusion independence was summarized using Kaplan-Meier analysis.

    Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders.

    Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24

    Measured: Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6)

  9. Change From Baseline in Scores of Functional Assessment of Cancer Therapy - General (FACT-G) Patient Reported Outcome

    The FACT-G consists of 27-items divided into 4 domains (physical well-being, social well-being, emotional and functional well-being). All items of the FACT-G use a 5 point scale ranging from 0 to 4 with a 0 rating being "not at all" and a 4 rating being "very much". Total FACT-G score is the sum of physical well-being score, social well-being score, emotional well-being score and functional well-being score. Score ranges from 0 to 108, with higher scores indicating better quality of life (QoL). A positive change from baseline indicates improvement in the QoL.

    Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders.

    Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24.

    Measured: Baseline, every 2 weeks from Week 2 to Week 24 or End of Treatment Period 1 (for all participants); and at Week 38, Week 53, Month 24 or End of Treatment Period 2 (for responders at Month 6 only)

  10. Change From Baseline in Scores of FACT - Thrombocytopenia 18 (FACT-TH18) Patient Reported Outcome

    The FACT-TH18 is comprised of FACT-G and a thrombocytopenia specific questionnaire. FACT-G consists of 27-items divided into 4 domains (physical, social, emotional and functional well-being). FACT-TH18 has 18 additional items which asks the patient to rate degree of thrombocytopenia. All items of the FACT-TH18 use a 5 point scale ranging from 0 to 4, with 0 = "not at all" and 4 = "very much". Total FACT-TH18 score is the sum of physical, social, emotional and functional well-being scores, and thrombocytopenia subscale. Score ranges from 0 to 180, with higher scores indicating better QoL. A positive change from baseline indicates improvement in the QoL. Results are presented for responders and non-responders. If any participant achieved hematologic response (CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24.

    Measured: Baseline, every 2 weeks from Week 2 to Week 24 or End of Treatment Period 1 (for all participants); and at Week 38, Week 53, Month 24 or End of Treatment Period 2 (for responders at Month 6 only)

  11. Change From Baseline in Scores of Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT- Fatigue) Patient Reported Outcome

    The FACIT- Fatigue is a 13-item fatigue subscale that asks the patient to rate their degree of tiredness, weakness and fatigue. All items of the FACIT-Fatigue use a 5-point scale ranging from 0 to 4 with a 0 rating being "not at all" and a 4 rating being "very much". Total score ranges from 0 to 52. Negatively worded items were reverse scored before summing so that higher total scores indicate less fatigue. A positive change from baseline indicates improvement.

    Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders.

    Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24.

    Measured: Baseline, every 2 weeks from Week 2 to Week 24 or End of Treatment Period 1 (for all participants); and at Week 38, Week 53, Month 24 or End of Treatment Period 2 (for responders at Month 6 only)

  12. Pharmacokinetic Parameter- Cmax of Eltrombopag When Combined With Cyclosporine

    Cmax is the observed maximum plasma concentration following administration. The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 microgram/milliliter (ug/mL). Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day.

    Measured: Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose.

  13. Pharmacokinetic Parameter-AUClast of Eltrombopag When Combined With Cyclosporine

    AUClast is the area under the curve calculated to the last quantifiable concentration point (Tlast). The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day.

    Measured: Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose

  14. Pharmacokinetic Parameter- AUCtau of Eltrombopag When Combined With Cyclosporine

    AUCtau is the area under the curve calculated to the end of the dosing interval (tau). The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day.

    Measured: Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose

  15. Pharmacokinetic Parameter- Ctrough of Eltrombopag When Combined With Cyclosporine

    Ctrough is the pre-dose plasma concentration. The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day.

    Measured: Week 2 at pre-dose

  16. Pharmacokinetic Parameter- Tmax of Eltrombopag When Combined With Cyclosporine

    Tmax is the time to reach peak or maximum concentration. The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day.

    Measured: Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose

  17. Pharmacokinetic Parameter- CLss/F of Eltrombopag When Combined With Cyclosporine

    CLss/F is the apparent systemic (or total body) clearance at steady state from plasma. The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day.

    Measured: Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Dec 2016 (estimated)
  2. Study start11 May 2017
  3. Primary completion3 Nov 2020
  4. Study completion30 May 2022

Resources

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
54 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02998645
Sponsor's protocol ID
CETB115E2403
Other IDs
2016-002814-29, 2016
Acronym
SOAR
Status last verified
Feb 2023
First submitted
4 Nov 2016
Last updated
11 Dec 2023
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Scheinberg P, Finelli C, Montano-Figueroa EH, Vallejo C, Norasetthada L, Calado RT, Turgut M, Peffault de Latour R, Kriemler-Krahn U, Haenig J, Clark J, Jang J. Activity and safety of eltrombopag in combination with cyclosporin A as first-line treatment of adults with severe aplastic anaemia (SOAR): a phase 2, single-arm study. Lancet Haematol. 2024 Mar;11(3):e206-e215. doi: 10.1016/S2352-3026(23)00395-2. Epub 2024 Feb 6.

    PubMed 38335978

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing eltrombopag and Cyclosporine in people with Severe Aplastic Anemia, run by Novartis Pharmaceuticals at 20 sites in 9 countries.

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 11 inclusion and 19 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 11 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 20 sites in Italy, Mexico, Thailand, Brazil, India and 4 more countries. 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 11 Dec 2023). 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?

Overall Hematologic Response Rate by 6 Months (time frame: Up to 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02998645, which also lists 1 linked publication.

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 11 Dec 2023.

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