Clinical trial NCT05531279: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Severe Aplastic Anemia
Keywords: G-CSF
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2022-09-07
Enrollment: 45
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: total number of effective (ANC>0.5×109/L) days
timeFrame: 2 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
state: Tianjin
Study officials:
affiliation: Chinese Academy of Medical Sciences
name: Liping Jing, Doctor
role: STUDY_DIRECTOR
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.