Severe Aplastic Anemia (SAA)Phase 2REACTSNCT04328727
Combination of Eltrombopag With Immunosuppressive Therapy in East-Asian Patients With Severe Aplastic Anemia
A Phase 2 study testing eltrombopag, rabbit anti-thymocyte globulin (r-ATG) and cyclosporine A (CsA) in people with Severe Aplastic Anemia (SAA), run by Novartis Pharmaceuticals at 12 sites in 4 countries.
Trial summary
This study was designed to evaluate the efficacy and safety of eltrombopag when added to r-ATG and CsA in treatment naive East-Asian adult and pediatric patients with severe aplastic anemia (SAA). Read more about this study
- Who is it for?
- Ages 6 to 100 years
- Conditions the study is for
- Severe Aplastic Anemia (SAA)
- What is being tested?
- eltrombopag, rabbit anti-thymocyte globulin (r-ATG) and cyclosporine A (CsA)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 12 sitesin 4 countries
- Study dates
- 4 Nov 2020to 6 Dec 2024
- 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 6 to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Written study informed consent and (where applicable) assent from the subject, parent, or guardian must be obtained prior to participation in the study.
- Subjects of East Asian ethnicity aged ≥ 6 years old at the time of written informed consent and assent form (if applicable).
- SAA characterized by:
- Bone marrow cellularity < 25%, or 25-50% with < 30% residual hematopoietic cells and pancytopenia, with at least two of the following parameters in peripheral blood:
- Absolute neutrophil count < 0.5×109/L
- Platelet count < 20×109/L
- Absolute reticulocyte count < 20×109/L
- HSCT not suitable or available as a treatment option (determined as per local practices or national guidelines), or has been refused by subject.
Who cannot join
- Prior IST with any ATG/ALG , alemtuzumab, high dose cyclophosphamide (≥ 45 mg/kg/day), CsA within 6 months, or prior thrombopoietin receptor agonists.
- Eastern Cooperative Oncology Group (ECOG) performance status (age ≥ 16 years) >2, or Lansky performance status (age < 16 years) <50.
- Prior and/or active medical history of:
- Known underlying congenital/inherited bone marrow failure or aplastic anemia (e.g.,such as but not limited to Fanconi anemia, congenital dyskeratosis, congenital amegakaryocytic thrombocytopenia, or Shwachman-Diamond Syndrome)
- Symptomatic paroxysmal nocturnal hemoglobinuria (PNH) and/or PNH clones >50% of polymorphonuclear neutrophil (PMN) or RBC at time of enrollment
- Myelodysplastic syndrome (MDS)
Show all 19Show fewer
- Any cytogenetic abnormalities on karyotyping or FISH within 30 days of study enrollment (an evaluable karyotyping with at least 10 metaphases is mandatory for eligibility)
- Other known or suspected underlying primary immunodeficiency
- Any concomitant malignancies that have not fully recovered from treatment or have not been disease-free for 5 years
- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3 times the upper limit of normal (ULN).
- Creatinine ≥ 2.5×local ULN
- Past medical history of thromboembolism within 6 months, and/or prior or current antiphospholipid antibody syndrome (APS).
- Presence of clinically active uncontrolled significant (of such severity that it would preclude the subject's ability to consent, be compliant with study procedures, tolerate protocol therapy) infection, including bacterial, fungal, mycobacterial, parasitic or viral infection, or any concurrent condition that, in the Investigator's opinion, would jeopardize the safety of the subject or compliance with the protocol
- Any severe and/or uncontrolled medical conditions which could cause unacceptable safety risks or compromise compliance with the protocol, such as:
- Known hepatocellular disease (e.g. active hepatitis or cirrhosis)
- Impairment of gastrointestinal (GI) function or gastrointestinal disease that may significantly alter the absorption of study treatment (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome)
- Active skin, mucosa, ocular or GI disorders of Grade > 1
- Presence of hepatitis B surface antigen (HBsAg) or positive hepatitis C antibody test result at screening. A positive serology for Hepatitis B (HB) is considered as a positive test for HBsAg. In addition, if serology is negative for HBsAg but hepatitis B core antibody (HBcAb) is positive (regardless of hepatitis B surface antibody (HBsAb) status), a hepatitis B virus (HBV) DNA test will be performed and if positive the patient will be excluded.
- Cardiac disorder (subjects with congestive heart disease of New York Heart Association (NYHA) functional classification Grade II/III/IV (for pediatric subjects, refer to the Grade II/III/IV of Modified ross heart failure classification for Children) should not be enrolled; subjects with NYHA Grade II due to cardiac disorder should not be enrolled but those with NYHA Grade II due to aplastic anemia (AA) may be enrolled.), arrhythmia with a risk of thrombosis (e.g. atrial fibrillation), pulmonary hypertension, or uncontrolled hypertension (>180/100 mmHg).
Trial locations and contacts
12 sites in 4 countries.
12 sites in 4 countries
China6 sites
Novartis Investigative Site
Changchun, Jilin, 130021
Novartis Investigative Site
Guangzhou, Guangdong, 510000
Novartis Investigative Site
Nanchang, Jiangxi, 330006
Novartis Investigative Site
Tianjin, 300020
Novartis Investigative Site
Tianjin, 300052
Novartis Investigative Site
Zhengzhou, Henan, 450052
Japan3 sites
Novartis Investigative Site
Chuo Ku, Tokyo, 104 8560
Novartis Investigative Site
Fukuoka, Fukuoka, 812-8582
Novartis Investigative Site
Nagoya, Aichi-ken, 466 8560
South Korea2 sites
Novartis Investigative Site
Seoul, 03080
Novartis Investigative Site
Seoul, 06351
About this study
Official title: A Non-randomized, Open Label, Multi-center, Phase II Study to Assess the Safety and Efficacy of Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin (r-ATG) and Cyclosporine A (CsA) in East-Asian Patients With Treatment Naive Severe Aplastic Anemia (REACTS)
This study was designed to evaluate the efficacy and safety of eltrombopag when added to r-ATG and CsA in treatment naive East-Asian adult and pediatric patients with severe aplastic anemia (SAA).
Read the detailed description
This was a non-randomized, open label, single arm, multi-center, Phase II study to evaluate the efficacy and safety of eltrombopag in combination with immunosuppressive therapy (IST) regimen of r-ATG + CsA in East-Asian patients with severe aplatsic anemia who had not received prior IST.
Eligible participants were enrolled into the study and received eltrombopag (from Day 1 to Week 26) concomitantly with r-ATG (on Days 1-5) and CsA (from Day 1 to Week 26) in the core treatment part.
Participants who were assessed as responders (meeting complete (CR) or partial (PR) response criteria) at Week 26 were eligible for the extension part of the study and continued treatment with eltrombopag and CsA after Week 26 up to Week 52. During the extension part, eltrombopag treatment was provided up to Week 52. CsA was maintained or tapered at the investigator's discretion according to local practice, with a total duration of at least 2 years (18 months after Week 26). There was a 30 days after end of treatment safety follow-up at the end of the extension part.
All participants were offered to enter the long term follow-up after the discontinuation of eltrombopag, with yearly efficacy and clonal evolution assessments up to Year 3 (Week 156).
- Conditions
- Severe Aplastic Anemia (SAA)
- Being tested
- eltrombopag, rabbit anti-thymocyte globulin (r-ATG), cyclosporine A (CsA)
- Keywords
- Treatment naïve severe aplastic anemia, SAA, eltrombopag, ETB115, immunosuppressive therapy, rabbit anti-thymocyte globulin, r-ATG, cyclosporine A, CsA, East-Asian patients
- 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
Tablet 25mg and 12.5mg
rabbit anti-thymocyte globulin (r-ATG)Drug
Also called r-ATG
r-ATG 25 mg sterile lyophilized powder in 10 mL vials for IV use
cyclosporine A (CsA)Drug
Also called CsA
CsA 25mg Capsule or CsA 5.0g/50mL solution for oral use
| Study group | Role | What they receive |
|---|---|---|
| eltrombopag | Experimental | Participants received eltrombopag in combination with r-ATG and CsA.
* Eltrombopag was administered orally once daily with initial doses of 75mg/day for ≥ 12 years old and 37.5 mg/day for participants between ≥ 6 and < 12 years. Doses could be adjusted based on platelet count
* r-ATG was administered intravenously at a dose of 2.5 to 3.5 mg/kg/day on Days 1-5
* CsA was administered orally every 12 h at a starting dose of 3-6 mg/kg/day Receives: Drug: eltrombopag; Drug: rabbit anti-thymocyte globulin (r-ATG); Drug: cyclosporine A (CsA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Response (CR) Rate at Week 26
Complete response rate was defined as percentage of patients achieving complete response (CR).
Complete response was defined as subjects meeting all the three criteria on two consecutive serial blood count measurements at least one week apart but not more than four weeks apart:
- Absolute neutrophil count > 1.0 ×10^9/L
- Platelet count > 100 ×10^9/L
- Hemoglobin > 100 g/L
The participants who discontinued from the trial before Week 26 and those that received blood products prior to response assessment (7 days prior for platelet transfusions, 14 days for RBC transfusion and 21 days for growth factors) were treated as non-responders.
Measured: Week 26 (6 months after starting study treatment)
Secondary outcomes (16)
Complete Response (CR) Rate
Complete response rate was defined as percentage of patients achieving complete response (CR).
Complete response was defined as subjects meeting all the three criteria on two consecutive serial blood count measurements at least one week apart but not more than four weeks apart:
- Absolute neutrophil count > 1.0 ×10^9/L
- Platelet count > 100 ×10^9/L
- Hemoglobin > 100 g/L
The participants who discontinued from the trial before Week 26 and those that received blood products prior to response assessment (7 days prior for platelet transfusions, 14 days for RBC transfusion and 21 days for growth factors) were treated as non-responders.
Measured: Week 13 (3 months), Week 52 (12 months) and yearly after up to 3 years
Overall Response (ORR) Rate
Overall response rate was defined as percentage of patients achieving complete response (CR) or partial response (PR).
Partial response (PR) was defined as blood counts no longer meeting the standard criteria for severe pancytopenia in SAA, equivalent to at least 2 of the 3 criteria below, but not sufficient for a CR:
- Absolute neutrophil count ≥ 0.5 × 10^9/L
- Platelet count ≥ 20 × 10^9/L
- Reticulocyte count ≥ 20 × 10^9/L
Complete response (CR) was defined as subjects meeting all the three criteria on two consecutive serial blood count measurements at least one week apart but not more than four weeks apart:
- Absolute neutrophil count > 1.0 ×10^9/L
- Platelet count > 100 ×10^9/L
- Hemoglobin > 100 g/L
Measured: Week 13 (3 months), 26 weeks (6 months), 52 weeks and yearly after up to 3 years
Duration of Complete Response
Duration of response was derived as the time from first documented and confirmed complete response (CR) until the time of relapse or death, whichever occurred first. Duration of response was estimated using Kaplan-Meier method.
Clinical relapse was considered as the occurrence of any of the following events in a participant who had achieved a hematological response (CR) but had subsequently lost response (not explained by any other independent concomitant medical conditions) in one blood count measurements:
- Meeting again the criteria for SAA
- Requirement for transfusion again for subjects who had been transfusion independent
- Decrease in any of the peripheral blood counts to absolute neutrophil count < 0.5 x10^9/L or platelets < 20 x10^9/L.
Measured: Up to aproximately 3 years
Duration of Overall Response
Duration of response was derived as the time from first documented and confirmed response (either CR or PR) until the time of relapse or death, whichever occurred first. Duration of response was estimated using Kaplan-Meier method.
Clinical relapse was considered as the occurrence of any of the following events in a participant who had achieved a hematological response (CR or PR) but had subsequently lost response (not explained by any other independent concomitant medical conditions) in one blood count measurements:
- Meeting again the criteria for SAA
- Requirement for transfusion again for subjects who had been transfusion independent
- Decrease in any of the peripheral blood counts to absolute neutrophil count < 0.5 x10^9/L or platelets < 20 x10^9/L.
Measured: Up to aproximately 3 years
Overall Survival (OS)
OS was defined as the time from the date of the first dose of study treatment to the date of death due to any cause. If a subject was not known to have died, survival was censored at the date of last contact. The distribution function of OS was estimated using the Kaplan- Meier method.
Measured: Up to approximately 3 years
Overall Survival (OS) Rate
OS was defined as the time from the date of the first dose of study treatment to the date of death due to any cause. The OS rate is the estimated probability that a patient will remain event-free up to the specified time point and was obtained from the Kaplan-Meier survival estimates. If a subject was not known to have died, survival was censored at the date of last contact.
Measured: Week 26, Week 52 and yearly after up to 3 years
Red Blood Cells (RBC) and Platelet Transfusion-free Interval Before Week 13 and 26
Transfusion-free interval was defined as the time from most recent RBC/platelet transfusion preceding response assessment to the date of response assessment.
Measured: Week 13, 26
Percentage of Participants Who Become RBC Transfusion Independent
Transfusion independent participants were defined as those participants who were transfusion dependent at baseline but became transfusion free for a period of ≥ 8 weeks post-baseline for RBCs.
Measured: From date of first dose to approximately 3 years
Percentage of Participants Who Become Platelet Transfusion Independent
Transfusion independent participants were defined as those participants who were transfusion dependent at baseline but became transfusion free for a period of ≥ 4 weeks post-baseline for platelets.
Measured: From date of first dose to approximately 3 years
Time From the Date of First Dose of Investigational Treatment to the Date of First Occurrence of Any Clonal Evolution Events
Clonal evolution events were assessed by karyotyping (G-banding) and FISH (Fluorescence in situ hybridization) targeting abnormalities including, but not restricted to chromosome 3q del,5q del, monosomy 7, trisomy 8 and those associated with SAA (Severe aplastic anemia), MDS (Myelodysplastic syndrome), AML (Acute myeloid leukemia). Time to clonal evolution was to be estimated using the Kaplan-Meier method.
Measured: From date of first dose to approximately 3 years
Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: AUClast
AUClast is the area under the curve from time zero to the last measurable concentration sampling time.
Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach.
Measured: Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose
Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: AUCtau
AUCtau is area under the curve calculated to the end of a dosing interval (tau) at steady-state
Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach.
Measured: Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose
Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: Ctrough
Cthrough is the pre-dose concentration at the end of dose interval.
Blood samples were collected to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach.
Eltrombopag was administered orally once daily with initial doses of 75mg/day for ≥ 12 years old and 37.5 mg/day for participants between ≥ 6 and < 12 years.
Doses could be adjusted based on platelet count every 2 weeks by decreasing it by 25 mg/day (12.5 mg/day, for participants below 12 years old) if the platelet count was above 200×10^9/L. or interrupted if platelet count rose above 400×10^9/L. In partial response participants dose could be restarted or increased to that before the decrease if platelet counts < 30 x10^9/L, Hb< 90 g/L, ANC< 0.5 x 10^9/L or participant needed transfusion. In complete response participants dose could be restarted or increased to that before decrease if blood counts dropped to not meet CR criteria.
Measured: Pre-dose on day 15 after initation of eltrombopag and and pre-dose on the 15th day after each new dose up to week 26
Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: Cmax
Cmax is the The maximum (peak) observed plasma drug concentration after dose administration.
Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach.
Measured: Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose
Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: Tmax
Tmax is the time to reach maximum (peak) plasma drug concentration after dose administration.
Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach.
Measured: Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose
Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: CLss/F
CLss/F is Apparent systemic (or total body) clearance at steady state from plasma.
Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach.
Measured: Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2020
- Study start4 Nov 2020
- Primary completion10 Jun 2022
- Study completion6 Dec 2024
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 Severe Aplastic Anemia (SAA)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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04328727
- Sponsor's protocol ID
- CETB115G2201
- Other IDs
- 2024-000602-14
- Acronym
- REACTS
- Status last verified
- Dec 2025
- First submitted
- 9 Mar 2020
- Last updated
- 13 Jan 2026
- 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 expert 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 is currently available according to the process described on www.clinicalstudydatarequest.com.
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Non-randomized, Open Label, Multi-center, Phase II Study to Assess the Safety and Efficacy of Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin (r-ATG) and Cyclosporine A (CsA) in East-Asian Patients With Treatment Naive Severe Aplastic Anemia (REACTS)
- ClinicalTrials.gov ID
- NCT04328727
- Acronym / sponsor ID
- REACTS / CETB115G2201
- Conditions
- Severe Aplastic Anemia (SAA)
- Keywords
- Treatment naïve severe aplastic anemia, SAA, eltrombopag, ETB115, immunosuppressive therapy, rabbit anti-thymocyte globulin, r-ATG, cyclosporine A
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 4 Nov 2020 (actual)
- Primary completion
- 10 Jun 2022 (actual)
- Study completion
- 6 Dec 2024 (actual)
- First posted
- 31 Mar 2020 (actual)
- Results
- Posted 17 Jul 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study was designed to evaluate the efficacy and safety of eltrombopag when added to r-ATG and CsA in treatment naive East-Asian adult and pediatric patients with severe aplastic anemia (SAA).
Detailed description
This was a non-randomized, open label, single arm, multi-center, Phase II study to evaluate the efficacy and safety of eltrombopag in combination with immunosuppressive therapy (IST) regimen of r-ATG + CsA in East-Asian patients with severe aplatsic anemia who had not received prior IST.
Eligible participants were enrolled into the study and received eltrombopag (from Day 1 to Week 26) concomitantly with r-ATG (on Days 1-5) and CsA (from Day 1 to Week 26) in the core treatment part.
Participants who were assessed as responders (meeting complete (CR) or partial (PR) response criteria) at Week 26 were eligible for the extension part of the study and continued treatment with eltrombopag and CsA after Week 26 up to Week 52. During the extension part, eltrombopag treatment was provided up to Week 52. CsA was maintained or tapered at the investigator's discretion according to local practice, with a total duration of at least 2 years (18 months after Week 26). There was a 30 days after end of treatment safety follow-up at the end of the extension part.
All participants were offered to enter the long term follow-up after the discontinuation of eltrombopag, with yearly efficacy and clonal evolution assessments up to Year 3 (Week 156).
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: eltrombopag, Drug: rabbit anti-thymocyte globulin (r-ATG), Drug: cyclosporine A (CsA)Participants received eltrombopag in combination with r-ATG and CsA. * Eltrombopag was administered orally once daily with initial doses of 75mg/day for ≥ 12 years old and 37.5 mg/day for participants between ≥ 6 and < 12 years. Doses could be adjusted based on platelet count * r-ATG was administered intravenously at a dose of 2.5 to 3.5 mg/kg/day on Days 1-5 * CsA was administered orally every 12 h at a starting dose of 3-6 mg/kg/day |
Who may qualify
- Ages
- 6 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Written study informed consent and (where applicable) assent from the subject, parent, or guardian must be obtained prior to participation in the study.
- Subjects of East Asian ethnicity aged ≥ 6 years old at the time of written informed consent and assent form (if applicable).
- SAA characterized by:
- Bone marrow cellularity < 25%, or 25-50% with < 30% residual hematopoietic cells and pancytopenia, with at least two of the following parameters in peripheral blood:
- Absolute neutrophil count < 0.5×109/L
Key exclusion criteria (19 listed)
- Prior IST with any ATG/ALG , alemtuzumab, high dose cyclophosphamide (≥ 45 mg/kg/day), CsA within 6 months, or prior thrombopoietin receptor agonists.
- Eastern Cooperative Oncology Group (ECOG) performance status (age ≥ 16 years) >2, or Lansky performance status (age < 16 years) <50.
- Prior and/or active medical history of:
- Known underlying congenital/inherited bone marrow failure or aplastic anemia (e.g.,such as but not limited to Fanconi anemia, congenital dyskeratosis, congenital amegakaryocytic thrombocytopenia, or Shwachman-Diamond Syndrome)
- Symptomatic paroxysmal nocturnal hemoglobinuria (PNH) and/or PNH clones >50% of polymorphonuclear neutrophil (PMN) or RBC at time of enrollment
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: ETB115Tablet 25mg and 12.5mg | Drug | eltrombopag |
| rabbit anti-thymocyte globulin (r-ATG)Also: r-ATGr-ATG 25 mg sterile lyophilized powder in 10 mL vials for IV use | Drug | eltrombopag |
| cyclosporine A (CsA)Also: CsACsA 25mg Capsule or CsA 5.0g/50mL solution for oral use | Drug | eltrombopag |
Primary outcome
Complete Response (CR) Rate at Week 26
Time frame: Week 26 (6 months after starting study treatment)
Complete response rate was defined as percentage of patients achieving complete response (CR). Complete response was defined as subjects meeting all the three criteria on two consecutive serial blood count measurements at least one week apart but not more than four weeks apart: * Absolute neutrophil count > 1.0 ×10^9/L * Platelet count > 100 ×10^9/L * Hemoglobin > 100 g/L The participants who discontinued from the trial before Week 26 and those that received blood products prior to response assessment (7 days prior for platelet transfusions, 14 days for RBC transfusion and 21 days for growth factors) were treated as non-responders.
16 secondary outcomes
Complete Response (CR) Rate
Time frame: Week 13 (3 months), Week 52 (12 months) and yearly after up to 3 years
Overall Response (ORR) Rate
Time frame: Week 13 (3 months), 26 weeks (6 months), 52 weeks and yearly after up to 3 years
Duration of Complete Response
Time frame: Up to aproximately 3 years
Duration of Overall Response
Time frame: Up to aproximately 3 years
Overall Survival (OS)
Time frame: Up to approximately 3 years
Overall Survival (OS) Rate
Time frame: Week 26, Week 52 and yearly after up to 3 years
Red Blood Cells (RBC) and Platelet Transfusion-free Interval Before Week 13 and 26
Time frame: Week 13, 26
Percentage of Participants Who Become RBC Transfusion Independent
Time frame: From date of first dose to approximately 3 years
Percentage of Participants Who Become Platelet Transfusion Independent
Time frame: From date of first dose to approximately 3 years
Time From the Date of First Dose of Investigational Treatment to the Date of First Occurrence of Any Clonal Evolution Events
Time frame: From date of first dose to approximately 3 years
Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: AUClast
Time frame: Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose
Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: AUCtau
Time frame: Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose
Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: Ctrough
Time frame: Pre-dose on day 15 after initation of eltrombopag and and pre-dose on the 15th day after each new dose up to week 26
Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: Cmax
Time frame: Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose
Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: Tmax
Time frame: Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose
Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: CLss/F
Time frame: Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jan 2026.
- 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.
- A Pediatric 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 expert 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 is currently available according to the process described on www.clinicalstudydatarequest.com.
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Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI
Acute Leukemia, Myelodysplastic Syndromes
Enrolling by invitationPhase 1/2
Phase I Clinical Study of Haplo-HSCT Combined With Hypoxic 3D-Cultured Umbilical Cord MSC for the Treatment of SAA
Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Refractory
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 Pharmaceuticals study director
Novartis Pharmaceuticals
12 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing eltrombopag, rabbit anti-thymocyte globulin (r-ATG) and cyclosporine A (CsA) in people with Severe Aplastic Anemia (SAA), run by Novartis Pharmaceuticals at 12 sites in 4 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 8 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 13 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in China, Japan, South Korea, Taiwan. 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 Jul 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?
Complete Response (CR) Rate at Week 26 (time frame: Week 26 (6 months after starting study treatment)). 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 13 Jan 2026; AzTrials last refreshed its copy on 8 Nov 2026.