Actively Recruiting
Zoledronic Acid Treatment in Patients With Congenital Dyserythropoietic Anemia
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2026-03-13
2
Participants Needed
1
Research Sites
76 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Zoledronic Acid Treatment in Patients With Congenital Dyserythropoietic Anemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older of any gender
- Diagnosed with Congenital Dyserythropoietic Anemia (CDA) confirmed by clinical presentation, lab tests, family history, RBM28 mutation, or bone marrow findings
- Have anemia with hemoglobin less than 100 g/L or require blood transfusions more frequently than every 8 weeks in the past 3 months
- Have an acceptable performance status (ECOG score 0-2)
- Normal kidney function with eGFR of 60 mL/min/1.73m² or higher
- Serum calcium levels within normal limits
- Female participants of childbearing potential must have a negative pregnancy test before joining and agree to use effective birth control during the study and for 3 months after
- Provide voluntary informed consent
You will not qualify if you...
- Known allergy or sensitivity to bisphosphonates or their ingredients
- Severe gum disease or recent (within 6 months) jaw bone damage (osteonecrosis)
- Low blood calcium levels (hypocalcemia)
- Pregnant or breastfeeding women
- Currently receiving other experimental treatments affecting red blood cell production, such as Luspatercept
- Active uncontrolled infections
- Severe heart, lung, or liver problems making participation unsafe as judged by the investigator
AI-Screening
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Trial Site Locations
Total: 1 location
1
Institute of Hematology & Blood Diseases Hospital
Tianjin, China
Actively Recruiting
Research Team
X
Xin Zhao, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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