Zoledronic Acid Treatment in Patients With Congenital Dyserythropoietic Anemia
Recruiting
Phase 1
Phase 2
Interventional Study
Congenital Dyserythropoietic Anemia (CDA)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 and older
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 2 (estimated)
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China · Other
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About This Trial
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, …
Trial Locations
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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 …
Contacts