New hope for rare anemias: drug may cut transfusions

NCT ID NCT07331818

Not yet recruiting Disease control Sponsor: EuroBloodNet Association Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This Phase 2 trial tests the drug luspatercept (Reblozyl) in 45 people with rare inherited anemias that affect red blood cells. The goal is to see if it can reduce the need for blood transfusions in those who depend on them, or raise hemoglobin levels in those who don't. Participants will receive injections over several weeks, and researchers will monitor their response.

What this could mean

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

Active substance: Luspatercept (Reblozyl)
What this could lead to: If successful, this could offer a new treatment option to reduce blood transfusions or increase hemoglobin levels for people with rare inherited anemias.
What could go wrong: This is an early Phase 2 trial with only 45 participants, so results may not apply to all patients. The drug may not work for every type of anemia, and side effects are still being studied.

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Conditions

The condition(s) this trial relates to.

congenital dyserythropoietic anemia type 1 congenital dyserythropoietic anemia type 2 Diamond-Blackfan anemia erythrocyte disorder inherited sideroblastic anemia

As listed by the trial registrant

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

HEREDITARY RED BLOOD CELL DISORDER (DISORDER)

Contacts and locations

Locations

Assistance Publique Hôpitaux de Paris - Hôpital Necker

Paris, France
Assistance Publique Hôpitaux de Paris - Hôpital Saint-Louis

Paris, 75010, France
CHU Bordeaux-Hôpital Haut-Lévêque

Pessac, 33600, France
Centre Hospitalier Universitaire de Montpellier

Montpellier, 34295, France
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano

Milan, Italy
GCS Groupement des Hôpitaux de l'Institut Catholique de Lille

Lille, 59800, France

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