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CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA

ConditionsSevere Aplastic Anemia (SAA)Transfusion-dependent Non-severe Aplastic Anemia
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorPeking Union Medical College Hospital

About this trial

This study aimed to explore the efficacy and safety of cyclosporine (CsA)

+eltrombopag (EPAG)/hetrombopag (HPAG)+romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe aplastic anemia (SAA)

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age ≥ 18 years old;

Diagnosed with aplastic anemia (AA) through routine blood tests, bone marrow puncture, bone marrow biopsy, and exclusion tests, and determined as transfusion-dependent non-severe aplastic anemia (TD-NSAA) or severe aplastic anemia (SAA) according to the Camitta criteria; Platelet < 30×10^9/L;

Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT);

Not suitable for ATG, due to reasons such as age, complications, and the patient's own wishes;

Disqualifiers

Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;

With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;

PNH clone ≥ 50%;

Received HSCT before enrollment;

Trial design

Design model

Single group

Treatments tested in this trial

  • Cyclosporine (CsA)

    Drug

    CsA 3-5mg/kg/d, trough concentration 100-200ng/ml

  • thrombopoietin receptor agonist (TPO-RA)

    Drug

    Eltrombopag 50mg/d, increased by 25mg every two weeks Hetrombopag 7.5mg/d, increased by 2.5mg every two weeks

  • Romiplostim N01

    Drug

    Romiplostim N01 20µg/kg, subcutaneously, once a week

Treatment groups

43 Participants
are divided into 1 treatment group
Group A: CsA+EPAG/HPAG+Romiplostim N01Experimental treatment 3 interventions

Trial outcomes

Primary outcomes

1

Overall response rate (ORR)

ORR=CRR+PRR

Time frame
6-month

Secondary outcomes

1

ORR

ORR=PRR+CRR

Time frame
3-month, 12-month
2

red blood cell (RBC)/platelet (PLT) transfusion independent rate

Proportion of patients who achieve red blood cell (RBC)/platelet (PLT) transfusion independence for 8 weeks or longer

Time frame
3-month, 6-month, 12-month
3

AE rate

proportion of patients with adverse events, according to CTCAE

Time frame
through study completion, an average of 1 year

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Peking Union Medical College Hospital

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: