Could a bone drug fix a rare blood disorder?

NCT ID NCT07471516

Disease control Sponsor: Institute of Hematology & Blood Diseases Hospital, China Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early study tests whether zoledronic acid, a drug usually used for bone health, can help people with a rare inherited anemia called congenital dyserythropoietic anemia (CDA). Only 2 adults with CDA who have low hemoglobin or need regular transfusions will receive 4 doses of the drug. Researchers will check if it raises hemoglobin levels and reduces transfusion needs over 12 to 24 weeks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: zoledronic acid
What this could lead to: If it works, this could point toward a new treatment option for people with congenital dyserythropoietic anemia, potentially reducing their need for blood transfusions.
What could go wrong: This is a very early, tiny study with only 2 participants, so results may not apply to others. Zoledronic acid can cause kidney problems and low calcium, and it's unclear if it will improve anemia at all.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 2 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2026

Expected to finish

Aug 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

congenital dyserythropoietic anemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Congenital dyserythropoietic anemia (CDA)

How to take part

Only the study team decides who joins. These are the ways to reach them.

The places running it

1 site. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

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A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Could a bone drug fix a rare blood disorder?

What the study statuses mean