Reduced dose of cyclophosphamide with immunosuppressive therapy for severe aplastic anemia
Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy as Front-line Therapy in Patients With Severe Aplastic Anemia
This study is testing a lower dose of cyclophosphamide combined with other treatments to see if it can help people with severe aplastic anemia feel better and improve their health.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 75 (estimated) |
| Ages | 12 Years and up |
| Sex | All |
| Sponsor | Institute of Hematology & Blood Diseases Hospital, China Academic / other |
| Drugs / interventions | chemotherapy, cyclophosphamide |
| Locations | 1 site (Tianjin, Tianjin Municipality) |
| Trial ID | NCT06695741 on ClinicalTrials.gov |
What this trial studies
This phase 2 clinical trial evaluates the efficacy and safety of a reduced dose of cyclophosphamide combined with anti-lymphocyte globulin and herombopag in treating patients with severe aplastic anemia. It is a prospective, single-center, single-arm study designed to assess how this combination therapy can improve patient outcomes. Participants will be closely monitored for both efficacy and safety throughout the treatment period.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals aged 12 years and older diagnosed with naïve severe or very severe aplastic anemia who are unable or unwilling to receive allogeneic hematopoietic stem cell transplantation.
Not a fit: Patients who have previously received immunosuppressive therapy within the last four weeks or have severe comorbidities may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could provide a safer and effective treatment option for patients with severe aplastic anemia who cannot undergo stem cell transplantation.
How similar studies have performed: While this approach is novel in its specific combination, similar studies have shown promise in using reduced doses of immunosuppressive therapies for treating aplastic anemia.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Tianjin, Tianjin Municipality
- Red Blood Cell Disorders Center and Regenerative Medicine Center — Tianjin, Tianjin Municipality, China (Recruiting)
Study contacts
- Study coordinator: Hong Pan, MD
- Email: panhong@ihcams.ac.cn
- Phone: 15822458611
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.