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NCT Number: NCT05975996

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

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Severe Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Regenerative Medicine Center

Tianjin, Tianjin Municipality, 300131, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

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.

Primary outcomes

  1. Overall response rate

    Time frame: Within 3 months

    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).

Secondary outcomes

  1. Robust response

    Time frame: Within 3 months

    Percentage of patients with robust response, including CR, near CR, VGPR and GPR.

  2. All-cause mortality

    Time frame: Within 3 months

  3. Time to achieve robust hematological response

    Time frame: Within 6 months

  4. Incidence of the adverse event

    Time frame: Within 6 months

    Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event

Sponsors and collaborators

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China

Other

Registry information

Important dates

Study start
2023
Primary completion
2024
Study completion
2025
First posted
Aug 4, 2023
Registry last updated
Sep 25, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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