Romiplostim N01 in the Treatment of Refractory Chemoradio... | NCT07532408

Studies
Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced AA

NCT07532408

Sponsor

Peking Union Medical College Hospital

Status

Withdrawn

Last Update Posted

Jun 4, 2026Actual

Enrollment

0Actual

Phase

Phase 2

Conditions

Aplastic Anemia

Interventions

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

Jun 2026

Overall Recruitment Status or Expanded Access Status

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

The registration of this research plan was incorrect. A new registration has been done.

Expanded Access Info

Start Date

Jun 2026Estimated

Primary Completion Date

Dec 2028Estimated

Completion Date

Dec 2029Estimated

First Submitted Date

Apr 8, 2026

First Submission Date that Met QC Criteria

Apr 8, 2026

First Posted Date

Apr 15, 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

Jun 2, 2026

Last Update Posted Date

Jun 4, 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 romiplostim N01 in the treatment of relapsed/refractory chemoradiotherapy-induced aplastic anemia

Detailed Description

Currently, there are few studies on the use of TPO-RAs after tumor radiotherapy and chemotherapy. Our center has published a study on the efficacy and safety of avatrombopag (AVA) in AA secondary to radiotherapy and chemotherapy. The study demonstrated that the ORR at 1, 3, and 6 months were 32.4%, 55.9%, and 58.8%, respectively. In a phase II/III study for refractory AA, romiplostim monotherapy achieved an ORR of 84% at week 27, which showed that romiplostim seemed to be effective and safe in patients with refractory aplastic anemia, with a recommended starting dose of 20 μg/kg once a week.

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

0Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Romiplostim N01	Experimental	Romiplostim N01 20 µg/kg subcutaneously once a week for at least 3 months	Drug: Romiplostim N01

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Romiplostim N01	Drug	Romiplostim N01 20 µg/kg subcutaneously once a week for at least 3 months	Romiplostim N01

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
ORR	Overall response rate (ORR) = PRR+CRR	3-month, 6-month

Secondary Outcomes

Measure	Description	Time Frame
CRR	Complete response rate	3-month, 6-month, 12-month
AE rate	Proportion of patients with adverse events, according to CTCAE	through study completion, an average of 1 year

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Age ≥ 18 years old;
Diagnosed with relapsed/refractory chemoradiotherapy-induced aplastic anemia (AA). Relapsed/ refractory was defined as patients who had no response to at least 3 months of cyclosporine or thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag;
At least one of the following conditions must be met: hemoglobin < 90 g/L, platelet count < 30×109/L, neutrophil count < 1.0×109/L;
With baseline liver and kidney functions <2 ULN;
Without active infection that cannot be controlled by standard treatment;
Signed the informed consent;
ECOG score ≤ 2;

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;
Received ATG treatment before enrollment;
Severe bleeding or infection that cannot be controlled by standard treatment;
Allergic or intolerant to romiplostim N01;
Active infections of HIV, HCV, or HBV, liver cirrhosis, or portal hypertension
History of malignant tumors within 5 years;
History of arterial or venous thrombosis;
Pregnant or lactating patients;
Participated in other clinical trials within 3 months.

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

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

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