Severe Aplastic Anemia (SAA)Phase 2NCT05323617
Efficacy of Romiplostim in Treatment of SAA in Adults Previously Untreated With or Refractory to Immunosuppressive Therapy
A Phase 2 study testing Romiplostim, Antithymocyte Globulin and Cyclosporine A in people with Severe Aplastic Anemia (SAA), run by Amgen.
Trial summary
Romiplostim has been used in clinical trials for the treatment of severe and very severe aplastic anemia (SAA/vSAA) in Asian participants who are either previously untreated with immunosuppressive therapy (IST) or refractory to IST. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Severe Aplastic Anemia (SAA)
- What is being tested?
- Romiplostim, Antithymocyte Globulin and Cyclosporine A
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 31 Aug 2023to 25 Feb 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Age ≥ 18 years at time of enrollment
- Diagnosis of SAA/vSAA confirmed by blood, bone marrow, and cytogenetic studies
- An Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 1 at screening
- Arm 1 only: participant requires initial treatment for SAA/vSAA, no matched related donor is available for allogenic hematopoietic cell transplantation (HCT) and will begin IST with antithymocyte globulin and CsA
- Arm 2 only: refractory to at least one course of immunosuppressive therapy including horse or rabbit ATG; or ineligible for ATG treatment and refractory to CsA
Who cannot join
- Diagnosed as having congenital aplastic anemia (AA) (Fanconi anemia, congenital dyskeratosis, etc)
- History of other malignancy within the past 5 years, with exceptions.
- Aplastic anemia with hemolytic paroxysmal nocturnal hemoglobinuria (PNH) (hemolytic predominant is defined as lactate dehydrogenase (LDH) > 1.5 x the upper limit of site normal
- Arm 1 only: Previously treated with ATG, CsA, or Alemtuzumab
- Previously treated with PEGylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin protein (TPO), romiplostim and other TPO-receptor agonist (eltrombopag, etc)
- Patients who are eligible for allogenic HCT and have an available matched related donor
About this study
Official title: Two Arm Bridging Study to Evaluate the Efficacy of Romiplostim in the Treatment of Adult Severe Aplastic Anemia Participants Who Are Either Previously Untreated With IST or Refractory to IST
Romiplostim has been used in clinical trials for the treatment of severe and very severe aplastic anemia (SAA/vSAA) in Asian participants who are either previously untreated with immunosuppressive therapy (IST) or refractory to IST. This study will evaluate the efficacy of romiplostim in the treatment of participants with SAA/vSAA.
The primary objectives of this study are to:
Arm 1: Evaluate the efficacy of romiplostim and IST in adult SAA/vSAA participants who are previously untreated with IST (1L)
Arm 2: Evaluate the efficacy of romiplostim treatment in adult SAA/vSAA participants who are refractory to IST (2L+)
- Conditions
- Severe Aplastic Anemia (SAA)
- Being tested
- Romiplostim, Antithymocyte Globulin, Cyclosporine A
- Keywords
- Severe Aplastic Anemia, SAA, Very Severe Aplastic Anemia, vSAA, Romiplostim
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimDrug
Also called Nplate®
Administered as a subcutaneous injection.
Antithymocyte GlobulinDrug
Horse or rabbit antithymocyte globulin administered as an intravenous infusion.
Cyclosporine ADrug
Administered orally.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1: Previously Untreated IST | Experimental | Participants with SAA/vSAA that are previously untreated with IST. Receives: Drug: Romiplostim; Drug: Antithymocyte Globulin; Drug: Cyclosporine A |
| Arm 2: Refractory IST | Experimental | Participants with SAA/vSAA that are refractory to IST. Receives: Drug: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Arms 1 and 2: proportion of participants achieving any hematologic response at week 14
Proportion of participants achieving any hematologic response at week 14 based on response criteria:
- Platelet response
- Erythroid response
- Red blood cell count
- Hemoglobin concentration
- Neutrophil response
Measured: Week 14
Secondary outcomes (12)
Arm 1: number of participants who achieve a complete response (CR) or partial response (PR) at week 14
Measured: Week 14
Arms 1 and 2: number of participants who have a decrease in frequency of platelet and/or red blood cell (RBC) transfusions, or become platelet and/or RBC transfusion independent at week 14
Measured: Week 14
Arms 1 and 2: number of participants with serious adverse events
Measured: 24 Weeks
Arms 1 and 2: number of participants with clinically significant changes in laboratory values
Measured: 24 Weeks
Arms 1 and 2: change from baseline in Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto (GIMEMA) bleeding scale at week 14
The Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto is as follows:
0: No bleeding
- Petecjoae or mucosal or retinal bleeding that did not require red-cell transfusion
- Melena, hematemesis, hematuria, or hemoptysis
- Any bleeding that required red-cell transfusion
- Retinal bleeding accompanied by visual impairment
- Nonfatal cerebral bleeding
- Fatal cerebral bleeding
- Fatal noncerebral bleeding
Measured: Baseline and Week 14
Arms 1 and 2: serum romiplostim trough concentrations
Measured: Prior to romiplostim administration on Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: maximum serum concentration (Cmax) of romiplostim
Measured: Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: area under the curve (AUC) of romiplostim
Measured: Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: time to reach maximum concentration (tmax) of romiplostim
Measured: Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: half-life (t1/2) of romiplostim
Measured: Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: number of participant with anti-romiplostim antibodies
Measured: Prior to romiplostim administration on Weeks 1 and 13
Arms 1 and 2: number of participants with antibodies to thrombopoietin
Measured: Prior to romiplostim administration on Weeks 1 and 13
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Apr 2022
- Study start31 Aug 2023 (estimated)
- Primary completion25 Feb 2025 (estimated)
- Study completion25 Feb 2025 (estimated)
Why it stopped: Operational
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Aplastic Anemia (SAA)Studies of the same condition, open and under way.
- More trials run by AmgenThe 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05323617
- Sponsor's protocol ID
- 20210112
- Status last verified
- Sep 2023
- First submitted
- 5 Apr 2022
- Last updated
- 29 Sep 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Two Arm Bridging Study to Evaluate the Efficacy of Romiplostim in the Treatment of Adult Severe Aplastic Anemia Participants Who Are Either Previously Untreated With IST or Refractory to IST
- ClinicalTrials.gov ID
- NCT05323617
- Acronym / sponsor ID
- 20210112
- Conditions
- Severe Aplastic Anemia (SAA)
- Keywords
- Severe Aplastic Anemia, SAA, Very Severe Aplastic Anemia, vSAA, Romiplostim
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Operational
- Enrolment
- 0 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- 31 Aug 2023 (estimated)
- Primary completion
- 25 Feb 2025 (estimated)
- Study completion
- 25 Feb 2025 (estimated)
- First posted
- 12 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Romiplostim has been used in clinical trials for the treatment of severe and very severe aplastic anemia (SAA/vSAA) in Asian participants who are either previously untreated with immunosuppressive therapy (IST) or refractory to IST. This study will evaluate the efficacy of romiplostim in the treatment of participants with SAA/vSAA.
The primary objectives of this study are to:
Arm 1: Evaluate the efficacy of romiplostim and IST in adult SAA/vSAA participants who are previously untreated with IST (1L)
Arm 2: Evaluate the efficacy of romiplostim treatment in adult SAA/vSAA participants who are refractory to IST (2L+)
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm 1: Previously Untreated IST | Experimental | Drug: Romiplostim, Drug: Antithymocyte Globulin, Drug: Cyclosporine AParticipants with SAA/vSAA that are previously untreated with IST. |
| Arm 2: Refractory IST | Experimental | Drug: RomiplostimParticipants with SAA/vSAA that are refractory to IST. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥ 18 years at time of enrollment
- Diagnosis of SAA/vSAA confirmed by blood, bone marrow, and cytogenetic studies
- An Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 1 at screening
- Arm 1 only: participant requires initial treatment for SAA/vSAA, no matched related donor is available for allogenic hematopoietic cell transplantation (HCT) and will begin IST with antithymocyte globulin and CsA
- Arm 2 only: refractory to at least one course of immunosuppressive therapy including horse or rabbit ATG; or ineligible for ATG treatment and refractory to CsA
Key exclusion criteria (6 listed)
- Diagnosed as having congenital aplastic anemia (AA) (Fanconi anemia, congenital dyskeratosis, etc)
- History of other malignancy within the past 5 years, with exceptions.
- Aplastic anemia with hemolytic paroxysmal nocturnal hemoglobinuria (PNH) (hemolytic predominant is defined as lactate dehydrogenase (LDH) > 1.5 x the upper limit of site normal
- Arm 1 only: Previously treated with ATG, CsA, or Alemtuzumab
- Previously treated with PEGylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin protein (TPO), romiplostim and other TPO-receptor agonist (eltrombopag, etc)
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 |
|---|---|---|
| RomiplostimAlso: Nplate®Administered as a subcutaneous injection. | Drug | Arm 1: Previously Untreated IST, Arm 2: Refractory IST |
| Antithymocyte GlobulinHorse or rabbit antithymocyte globulin administered as an intravenous infusion. | Drug | Arm 1: Previously Untreated IST |
| Cyclosporine AAdministered orally. | Drug | Arm 1: Previously Untreated IST |
Primary outcome
Arms 1 and 2: proportion of participants achieving any hematologic response at week 14
Time frame: Week 14
Proportion of participants achieving any hematologic response at week 14 based on response criteria: * Platelet response * Erythroid response * Red blood cell count * Hemoglobin concentration * Neutrophil response
12 secondary outcomes
Arm 1: number of participants who achieve a complete response (CR) or partial response (PR) at week 14
Time frame: Week 14
Arms 1 and 2: number of participants who have a decrease in frequency of platelet and/or red blood cell (RBC) transfusions, or become platelet and/or RBC transfusion independent at week 14
Time frame: Week 14
Arms 1 and 2: number of participants with serious adverse events
Time frame: 24 Weeks
Arms 1 and 2: number of participants with clinically significant changes in laboratory values
Time frame: 24 Weeks
Arms 1 and 2: change from baseline in Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto (GIMEMA) bleeding scale at week 14
Time frame: Baseline and Week 14
Arms 1 and 2: serum romiplostim trough concentrations
Time frame: Prior to romiplostim administration on Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: maximum serum concentration (Cmax) of romiplostim
Time frame: Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: area under the curve (AUC) of romiplostim
Time frame: Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: time to reach maximum concentration (tmax) of romiplostim
Time frame: Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: half-life (t1/2) of romiplostim
Time frame: Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: number of participant with anti-romiplostim antibodies
Time frame: Prior to romiplostim administration on Weeks 1 and 13
Arms 1 and 2: number of participants with antibodies to thrombopoietin
Time frame: Prior to romiplostim administration on Weeks 1 and 13
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2023.
- AmgenTrials clinical trials websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.
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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.
MD study director
Amgen
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Romiplostim, Antithymocyte Globulin and Cyclosporine A in people with Severe Aplastic Anemia (SAA), run by Amgen.
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 5 inclusion and 6 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: withdrawn, as of the sponsor's last update on 29 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Arms 1 and 2: proportion of participants achieving any hematologic response at week 14 (time frame: Week 14). 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 Sep 2023.