NCT07471516 Zoledronic Acid Treatment in Patients With Congenital Dyserythropoietic Anemia
| NCT ID | NCT07471516 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Institute of Hematology & Blood Diseases Hospital, China |
| Condition | Congenital Dyserythropoietic Anemia (CDA) |
| Study Type | INTERVENTIONAL |
| Enrollment | 2 participants |
| Start Date | 2026-02-25 |
| Primary Completion | 2027-02-25 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 2 participants in total. It began in 2026-02-25 with a primary completion date of 2027-02-25.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
Background: Congenital Dyserythropoietic Anemia (CDA) is a group of rare hereditary blood disorders characterized by ineffective erythropoiesis, leading to chronic anemia and organ damage. Current treatment options are very limited, mainly relying on regular blood transfusions, which can cause severe complications over time. Our laboratory research and animal models suggest that Zoledronic acid, a medication commonly used for bone health, may improve ineffective erythropoiesis. Purpose: The purpose of this exploratory study is to evaluate the efficacy and safety of Zoledronic acid in adult patients with CDA who do not have other effective treatment options. The primary goal is to see if this treatment can increase hemoglobin levels and reduce the need for blood transfusions. Study Design: This is a prospective, single-center, single-arm study. Participants will receive an initial intravenous dose (4 mg) of Zoledronic acid. After a 4-week observation period to ensure safety, participants will receive additional doses every 4 weeks for a total of 4 doses. Researchers will monitor hemoglobin levels, transfusion frequency, spleen size, and overall quality of life over a period of 12 to 24 weeks.
Eligibility Criteria
Inclusion Criteria: * Age ≥ 18 years, regardless of gender. * Diagnosis of Congenital Dyserythropoietic Anemia (CDA) based on clinical presentation, laboratory tests, and family investigation, with the presence of RBM28 mutation and/or increased vacuolization within nucleated red blood cells under light microscopy of bone marrow. * Presence of anemia (Hemoglobin \< 100 g/L at screening) or transfusion dependence (defined as an average transfusion interval of \< 8 weeks within the past 3 months). * Performance status is acceptable (ECOG score 0-2). * Normal renal function (estimated glomerular filtration rate, eGFR ≥ 60 mL/min/1.73m²). * Serum calcium levels within the normal range. * Female patients of childbearing potential must have a negative pregnancy test before enrollment and agree to use effective contraception during the study and for 3 months after completion. * The patient and/or guardian voluntarily sign the informed consent form. Exclusion Criteria: * Known hypersensitivity to bisphosphonates or any of their excipients. * Severe periodontal disease or a recent history (within 6 months) of osteonecrosis of the jaw. * Hypocalcemia. * Pregnant or lactating women. * Currently receiving other experimental drug treatments that may affect erythropoiesis (e.g., Luspatercept). * Active, uncontrolled systemic infection. * Severe cardiac, pulmonary, or hepatic dysfunction, as judged by the investigator to be unsuitable for participation in the study
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07471516 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Congenital Dyserythropoietic Anemia (CDA). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07471516 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT07471516 currently recruiting?
Yes, NCT07471516 is actively recruiting participants. Contact the research team at zhaoxin@ihcams.ac.cn for enrollment information.
Where is the NCT07471516 trial being conducted?
This trial is being conducted at Tianjin, China.
Who is sponsoring the NCT07471516 clinical trial?
NCT07471516 is sponsored by Institute of Hematology & Blood Diseases Hospital, China. The trial plans to enroll 2 participants.