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NCT Number: NCT06695741

Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat 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 the reduced dose of cyclophosphamide (hypo-CASH) for severe aplastic anemia.

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Key information

Conditions

Severe Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Cyclophosphamide Reduced-dose Hematologic Diseases Hemic and Lymphatic Diseases Immunosuppressive Therapy

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Red Blood Cell Disorders Center and Regenerative Medicine Center

Tianjin, Tianjin Municipality, 301617, China

Location status: Recruiting

Location contact

Hong Pan, MD

CONTACT

[email protected]

‭15822458611‬

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Subject has a diagnosis of naïve severe or very severe aplastic anemia
  • Male or female age ≥ 12 years
  • Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.
  • ECOG performance status ≤2
  • 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 anti-lymphocyte globulin, cyclosporine, herombopag or cyclophosphamide.
  • Uncontrolled fungal, bacterial, or viral infection. Hepatitis-associated aplastic anemia is allowed; hepatitis B or C infection is permitted unless it causes severe liver failure.
  • Tested positive for HIV or syphilis
  • Presence of severe liver, kidney, or heart failure, or other life-threatening comorbidities.
  • History of radiotherapy and chemotherapy for malignant solid tumors in recent 5 years
  • 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

Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy

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. Cyclophosphamide (20mg/kg) will be administered on days 15-16.

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. Superior response rate

    Time frame: With in 6 months

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

  2. Overall response rate

    Time frame: With in 6 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).

  3. 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

  4. Time to achieve robust superior response

    Time frame: Within 6 months

  5. First time to response

    Time frame: Within 6 months

Study contacts

Contact information is provided by the study sponsor or research team.

Hong Pan, MD

CONTACT

[email protected]

‭15822458611‬

Jingyu Zhao, MPH

CONTACT

[email protected]

13752253515

Sponsors and collaborators

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China

Other

Registry information

Official study title

Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy as Front-line Therapy in Patients With Severe Aplastic Anemia

Acronym: hypo-CASH

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Nov 19, 2024
Registry last updated
Nov 19, 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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