## Protocol Section
### Identification Module
**NCT ID:** NCT05531279

**Brief Title:** A Study of PEG-rhG-CSF and rhG-CSF Used for Aplastic Anemia Granulocyte Deficiency

**Official Title:** A Randomized,Open-label Dose-discovery Study of PEG-rhG-CSF and rhG-CSF in the Adjuvant Therapy of Aplastic Anemia Granulocyte Deficiency

#### Organization Study ID Info
**ID:** IIT2021022-EC-1
#### Organization
**Class:** OTHER
**Full Name:** Institute of Hematology & Blood Diseases Hospital, China
### Status Module
#### Completion Date
**Date:** 2026-01-05

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2022-09-26

**Type:** ACTUAL

**Last Update Submit Date:** 2022-09-22

**Overall Status:** RECRUITING

#### Primary Completion Date
**Date:** 2025-06-05

**Type:** ESTIMATED

#### Start Date
**Date:** 2022-06-05

**Type:** ACTUAL

**Status Verified Date:** 2022-09

#### Study First Post Date
**Date:** 2022-09-07
**Type:** ACTUAL
**Study First Submit Date:** 2022-09-02
**Study First Submit QC Date:** 2022-09-02
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** OTHER

**Name:** Institute of Hematology & Blood Diseases Hospital, China

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** False

### Description Module

**Brief Summary:** This study was a single-center,open-label,randomized,dose-exploring prospective study.Patients with granulocytotic aplastic anemia who received cytokine treatment with PEG-rhG-CSF or rhG-CSF were enrolled.Clinical demographic data,disease characteristics of aplastic anemia,clinical diagnosis and treatment,laboratory data and adverse events were collected to explore the dose and safety of PEG-rhG-CSF and rhG-CSF in patients with severe aplastic anemia.

**Detailed Description:** This study was a single-center,open-label,randomized,dose-exploring prospective study.Patients with granulocytotic aplastic anemia who received cytokine treatment with PEG-rhG-CSF or rhG-CSF were enrolled.Clinical demographic data,disease characteristics of aplastic anemia,clinical diagnosis and treatment,laboratory data and adverse events were collected to explore the dose and safety of PEG-rhG-CSF and rhG-CSF in patients with severe aplastic anemia.Research objectives: To explore the reasonable injection frequency of long-acting PEG-rhG-CSF in the adjuvant treatment of aplastic anemia patients with granulocytosis through a single center prospective clinical study.Disease classification of aplastic anemia: a total of 45 cases of SAA/VSAA with ANC\<0.5×109/L were stratified and randomized into three groups according to the radio of 1:1:1(15 cases in each group).PEG-rhG-CSF group A(q7d):6mg d1,8,subcutaneously injected;PEG-rhG-CSF group B(q10d):6mg d1,11,subcutaneously injected;RhG-CSF group(short-acting ): 480ug d1-14(daily for 14days),subcutaneously injected.Dose/protocol adjustment: after monitoring ANC\>0.5×109/L,the drug was stopped,and then ANC\<0.5×109/L was temporarily supplemented with one dose of the original group of drugs.

### Conditions Module

**Conditions:**

- Severe Aplastic Anemia

**Keywords:**

- G-CSF

### Design Module

#### Design Info

**Allocation:** RANDOMIZED

**Intervention Model:** PARALLEL

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 45

**Type:** ESTIMATED

**Phases:**

- NA

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** PEG-rhG-CSF group A(q7d):6mg d1,8,subcutaneously injected,after monitoring ANC\>0.5×109/L,the drug was stopped,and then ANC\<0.5×109/L was temporarily supplemented with one dose of the same drug.

**Intervention Names:**

- Drug: PEG-rhG-CSF

**Label:** PEG-rhG-CSF group A

**Type:** EXPERIMENTAL

#### Arm Group 2

**Description:** PEG-rhG-CSF group B(q10d):6mg d1,11,subcutaneously injected,after monitoring ANC\>0.5×109/L,the drug was stopped,and then ANC\<0.5×109/L was temporarily supplemented with one dose of the same drug.

**Intervention Names:**

- Drug: PEG-rhG-CSF

**Label:** PEG-rhG-CSF group B

**Type:** EXPERIMENTAL

#### Arm Group 3

**Description:** rhG-CSF group(short-acting ): 480ug d1-14(daily for 14days),subcutaneously injected.After monitoring ANC\>0.5×109/L,the drug was stopped,and then ANC\<0.5×109/L was temporarily supplemented with one dose of the same drug.

**Intervention Names:**

- Drug: PEG-rhG-CSF

**Label:** rhG-CSF group(short-acting )

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- PEG-rhG-CSF group A

- PEG-rhG-CSF group B

- rhG-CSF group(short-acting )

**Description:** PEG-rhG-CSF group A(q7d):6mg d1,8,subcutaneously injected; PEG-rhG-CSF group B(q10d):6mg d1,11,subcutaneously injected; RhG-CSF group(short-acting ): 480ug d1-14(daily for 14days),subcutaneously injected.

**Name:** PEG-rhG-CSF

**Other Names:**

- rhG-CSF

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** total number of effective (ANC\>0.5×109/L) days during 2 weeks of treatment(cases×days).

**Measure:** total number of effective (ANC>0.5×109/L) days

**Time Frame:** 2 weeks

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

1. Age 18-70 years old, male or female, or weight≥50kg.
2. Patients with severe or very severe aplastic anemia of absolute neutrophil value\< 0.5×109/L
3. ECOG score ≤ 2 points.
4. Normal renal function.

Exclusion Criteria:

1. Patients with clonal chromosomal abnormalities.
2. Patients with previous malignant tumors.
3. Patients with severe or uncontrolled infectious diseases and /or bleeding.
4. Patients with AIDS or syphilis positive.
5. Severe organ dysfunction.
6. Patients used GM/G-CSF,PEG-rhG-CSF,interleukin-11 within 2 weeks before admission.
7. Allergic to G-CSF or PEG-rhG-CSF related components.
8. Participated in other clinical trials within 6 months.

**Maximum Age:** 70 Years

**Minimum Age:** 18 Years

**Sex:** ALL

**Standard Ages:**

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** fkzhang@ihcams.ac.cn

**Name:** Fengkui Zhang, Doctor

**Phone:** +862223909229

**Role:** CONTACT

**Contact 2:**

**Email:** fanhuihui@ihcams.ac.cn

**Name:** Huihui Fan, Doctor

**Phone:** +862223909223

**Role:** CONTACT

#### Locations

**Location 1:**

**City:** Tianjin

**Contacts:**

***Contact 1:***

- **Email:** fkzhang@ihcams.ac.cn

- **Name:** Fengkui Zhang, Doctor

- **Phone:** 8602223909229

- **Role:** CONTACT

***Contact 2:***

- **Email:** fanhuihui@ihcams.ac.cn

- **Name:** Huihui Fan, Doctor

- **Phone:** 8602223909223

- **Role:** CONTACT

**Country:** China

**Facility:** Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

**State:** Tianjin

**Status:** RECRUITING

**Zip:** 300020

#### Overall Officials

**Official 1:**

**Affiliation:** Institute of Hematology ＆ Blood Diseases Hospital，Chinese Academy of Medical Sciences

**Name:** Liping Jing, Doctor

**Role:** STUDY_DIRECTOR

### IPD Sharing Statement Module

**Access Criteria:** After the paper is written and published

**Description:** We can share the plan after completing the experiment

**Info Types:**

- STUDY_PROTOCOL

- ICF

- CSR

**IPD Sharing:** YES

**Time Frame:** Follow-up and publication of the paper are planned for December 2025

### References Module

#### References

**Citation:** Tichelli A, Schrezenmeier H, Socie G, Marsh J, Bacigalupo A, Duhrsen U, Franzke A, Hallek M, Thiel E, Wilhelm M, Hochsmann B, Barrois A, Champion K, Passweg JR. A randomized controlled study in patients with newly diagnosed severe aplastic anemia receiving antithymocyte globulin (ATG), cyclosporine, with or without G-CSF: a study of the SAA Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2011 Apr 28;117(17):4434-41. doi: 10.1182/blood-2010-08-304071. Epub 2011 Jan 13.

**PMID:** 21233311

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000006402

- Term: Hematologic Diseases

- ID: D000080983

- Term: Bone Marrow Failure Disorders

- ID: D000001855

- Term: Bone Marrow Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC15

- Name: Blood and Lymph Conditions

- Abbrev: All

- Name: All Conditions

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M4071

- Name: Anemia, Aplastic

- Relevance: HIGH

- As Found: Aplastic Anemia

- ID: M4070

- Name: Anemia

- Relevance: HIGH

- As Found: Anemia

- ID: M9490

- Name: Hematologic Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M2241

- Name: Bone Marrow Failure Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M13118

- Name: Pancytopenia

- Relevance: LOW

- As Found: Unknown

- ID: M5134

- Name: Bone Marrow Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T460

- Name: Aplastic Anemia

- Relevance: HIGH

- As Found: Aplastic Anemia

### Condition Browse Module - Meshes

- ID: D000000740

- Term: Anemia

- ID: D000000741

- Term: Anemia, Aplastic

### Intervention Browse Module - Browse Branches

- Abbrev: Micro

- Name: Micronutrients

- Abbrev: All

- Name: All Drugs and Chemicals

- Abbrev: Ot

- Name: Other Dietary Supplements

### Intervention Browse Module - Browse Leaves

- ID: M271049

- Name: Coenzyme Q10

- Relevance: LOW

- As Found: Unknown

- ID: M17201

- Name: Ubiquinone

- Relevance: LOW

- As Found: Unknown

- ID: T383

- Name: Coenzyme Q10

- Relevance: LOW

- As Found: Unknown

### Misc Info Module

- Version Holder: 2024-05-24
