Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Anemia

NCT06695741 | Recruiting Phase 2

• 1 other identifier: Hypo-CASH-2024
• Study Type: interventional
• Target: 75
• 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 the reduced dose of cyclophosphamide (hypo-CASH) for severe aplastic anemia.

Conditions

Severe Aplastic Anemia; Cyclophosphamide Reduced-dose; Immunosuppressive Therapy

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 (5)

• Superior response rate

  With in 6 months

• Overall response rate

  With in 6 months

• Incidence of the adverse event

  Within 6 months

• Time to achieve robust superior response

  Within 6 months

• First time to response

  Within 6 months

Study Arms (1)

hypo-CASH

EXPERIMENTAL

ALG/CsA and herombopag and reduced dose of cyclophosphamide

Drug: Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy

Interventions

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.

hypo-CASH

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:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

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

Study Sites (1)

Red Blood Cell Disorders Center and Regenerative Medicine Center

Tianjin, Tianjin Municipality, 301617, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

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

Central Study Contacts

Hong Pan, MD

CONTACT

‭15822458611‬; panhong@ihcams.ac.cn

Jingyu Zhao, MPH

CONTACT

13752253515; zhaojingyu@ihcams.ac.cn

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: Director of the Red Blood Cell Disorders Center & Director of the Regenerative Medicine Clinic

Study Record Dates

• First Submitted: November 16, 2024
• First Posted: November 19, 2024
• Study Start: November 1, 2024
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: November 19, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)