CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA | NCT07558434

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

NCT07558434

Sponsor

Peking Union Medical College Hospital

Status

Not yet recruiting

Last Update Posted

Apr 30, 2026Actual

Enrollment

43Estimated

Phase

Phase 2

Conditions

Severe Aplastic Anemia (SAA)
Transfusion-dependent Non-severe Aplastic Anemia

Interventions

Cyclosporine (CsA)
thrombopoietin receptor agonist (TPO-RA)
Romiplostim N01

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Apr 2026

Overall Recruitment Status or Expanded Access Status

Not yet recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Apr 2026Estimated

Primary Completion Date

Dec 2028Estimated

Completion Date

Dec 2029Estimated

First Submitted Date

Apr 23, 2026

First Submission Date that Met QC Criteria

Apr 23, 2026

First Posted Date

Apr 30, 2026Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 23, 2026

Last Update Posted Date

Apr 30, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

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)

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

43Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
CsA+EPAG/HPAG+Romiplostim N01	Experimental	CsA 3-5mg/kg/d EPAG 50mg/d or HPAG 7.5mg/d Romiplostim N01 20µg/kg, subcutaneously, once a week	Drug: Cyclosporine (CsA) Drug: thrombopoietin receptor agonist (TPO-RA) Drug: Romiplostim N01

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Cyclosporine (CsA)	Drug	CsA 3-5mg/kg/d, trough concentration 100-200ng/ml	CsA+EPAG/HPAG+Romiplostim N01

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Overall response rate (ORR)	ORR=CRR+PRR	6-month

Secondary Outcomes

Measure	Description	Time Frame
ORR	ORR=PRR+CRR	3-month, 12-month
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

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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;
With baseline liver and kidney functions <2 ULN;
ECOG score ≤ 2;
Signed the informed consent;

Exclusion Criteria:

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;
Previously used immunosuppressive treatments such as ATG, CsA, TPO receptor agonists (TPO-RAs);
Allergic or intolerant to romiplostim N01, eltrombopag, hetrombopag, or CsA;
Pregnant or lactating patients;
Severe bleeding or infection that cannot be controlled by standard treatment;
History of arterial or venous thrombosis;
Complicated with malignant tumors;
Participated in other clinical trials within 3 months;
Patients considered not suitable to participate in this study by the investigator.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

ID	Term
D000740	Anemia
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases
D000080983	Bone Marrow Failure Disorders

ID	Term
D003524	Cyclosporins
D010456	Peptides, Cyclic
D047028	Macrocyclic Compounds
D011083	Polycyclic Compounds

ID	Term
D016572	Cyclosporine
C520809	eltrombopag
C000614661	hetrombopag