Cyclophosphamide Added to Standard Immunosuppressive Therapy With Herombopag as Front-line Therapy in Patients With Severe Aplastic Anemia

NCT05975996 | Active Not Recruiting Phase 2

• 1 other identifier: IIT2023038
• Study Type: interventional
• Target: 43
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective, single-center, single-arm, phase 2 study. This study aims to evaluate the efficacy and safety of Anti-lymphocyte globulin plus herombopag in combination with moderate-dose cyclophosphamide for severe aplastic anemia.

Conditions

Severe Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• Overall response rate

  Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), good partial response(GPR) and partial response(PR).

  Within 3 months

Secondary Outcomes (4)

• Robust response

  Within 3 months

• All-cause mortality

  Within 3 months

• Time to achieve robust hematological response

  Within 6 months

• Incidence of the adverse event

  Within 6 months

Study Arms (1)

Experiemental

EXPERIMENTAL

ALG/CsA(Cyclosporine) and herombopag and moderate-dose cyclophosphamide

Drug: Cyclophosphamide added to standard immunosuppressive therapy with herombopag

Interventions

Cyclophosphamide added to standard immunosuppressive therapy with herombopag DRUG

Severe aplastic anemia patients will receive a daily dose of Anti-lymphocyte globulin (25mg/kg) for the initial five days at the beginning of the treatment. Cyclosporine will be administered daily at a dosage of 3-5mg/kg. Herombopag will be administered daily at a dosage of 15mg starting from the first day of treatment and continuing for a duration of six months. The moderate-dose cyclophosphamide (20mg/kg) will be administered on days 29-30 and days 43-44.

Experiemental

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female age ≥ 12 years
• Subject has a diagnosis of naïve severe or very severe aplastic anemia
• ECOG performance status ≤2
• Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.
• Willing and able to comply with the requirements for this study and written informed consent.

You may not qualify if:

• Previously received immunosuppressive therapy \> 4 weeks
• Previously treated with TPO-RA \> 4 weeks
• Have an allergy or intolerance to either herombopag or cyclophosphamide.
• Have an allergy to ALG
• Uncontrolled systemic fungal, bacterial, or viral infection
• Poorly controlled hypertension (≥140/90mmHg) or diabetes (a fasting plasma glucose concentration ≥7.0mmol/L or a random venous plasma glucose concentration ≥11.1mmol/L)
• Abnormal liver or kidney function: ALT or AST \>3 ULN, or serum creatinine (sCr)≥ 2.5 ULN.
• History of radiotherapy and chemotherapy for malignant solid tumors
• Combined with other serious disorders
• Pregnant or breast-feeding patients
• Patients considered to be ineligible for the study by the investigator for reasons other than the above.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Regenerative Medicine Center

Tianjin, Tianjin Municipality, 300131, China

Location

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Liwei Fang, MD

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Chief Physician of Regenerative Medical Center

Study Record Dates

• First Submitted: July 27, 2023
• First Posted: August 4, 2023
• Study Start: July 10, 2023
• Primary Completion: December 31, 2024
• Study Completion: December 31, 2025
• Last Updated: September 25, 2024

Record last verified: 2024-09

Locations

CN (1)