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New drug could cut transfusions for rare blood disorder

NCT ID NCT05664737

Disease control Sponsor: Bristol-Myers Squibb Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Sep 11, 2026 · Updated 5 times

Summary

This phase 2 trial tests luspatercept (Reblozyl) in adults and adolescents with alpha-thalassemia, a genetic blood disorder that causes anemia. The goal is to see if the drug can reduce the need for blood transfusions and raise hemoglobin levels. About 189 participants will receive either luspatercept or a placebo, plus standard care, to compare safety and effectiveness.

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 and improve hemoglobin levels in people with alpha-thalassemia.
What could go wrong
This is a mid-stage trial, so results are not yet proven. The drug may not work for everyone, and side effects like hemolytic crises are possible.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 189 people

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

Started

Dec 2022

Expected to finish

Aug 2034

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

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

Ages

12 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: * Adult participant≥ 18 years with documented diagnosis of A-Thal HbH disease with Transfusion dependence defined as:. i) TD participant: ≥ 6 RBC units during the 24 weeks prior to randomization. ii) NTD participant:\< 6 RBC units during the 24 weeks prior to randomization(transfusion due to conditions other than A-Thal will not be considered)and, RBC transfusion-free during at least 8 weeks prior to randomization(unless transfusion was required to treat an acute medical condition other than A-Thal) and, mean baseline Hb ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to randomization; hemoglobin values within 21 days post-transfusion will be excluded. * Adult participant has Eastern Cooperative Oncology Group (ECOG) 34 score of 0 or 1. * Adolescent participant 12 years to \< 18 years with documented diagnosis of A-Thal HbH disease with transfusion dependence defined as:. i) TD participant: ≥ 4 RBC events during the 24 weeks prior to enrollment and, no transfusion-free period for \> 56 days during the 24 weeks prior to enrollment. Participants must have a history of regular transfusions for at least 2 years. ii) NTD participant:\< 4 RBC events during the 24 weeks prior to enrollment and RBC transfusion-free during at least 8 weeks prior to enrollment and, mean baseline Hb ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to enrollment, hemoglobin values within 21 days post-transfusion will be excluded. iii) Participant has Karnofsky (age ≥16 years) or Lansky (age \< 16 years) performance status score ≥ 50 at screening. Exclusion Criteria: * Medical Conditions: Diagnosis of A-ThalTrait, Hb Bart hydrops, ATRx A-Thal, hemoglobin S/β-thalassemia, myelodysplasia subtype anemia, or with HbE homozygous beta gene mutation. Anemia related to nutritional deficiency, anemia of chronic disease, autoimmune hemolytic anemia, or any other hemolytic anemias. Undergone episodes of hemolysis not related to A-Thal within the 8 weeks prior to randomization. Applicable for the EU only: Bleeding disorders manifested by frequent bleeding episodes (eg, menorrhagia, epistaxis, clotting disorders). * Participant has deep vein thrombosis (DVT), stroke or other thromboembolic event(s) (except clogged indwelling catheter) requiring medical intervention ≤ 24weeks prior to randomization. * Participant has uncontrolled hypertension. Controlled hypertension for this protocol is considered: blood pressure value corresponding to ≤Grade 1 according to NCI CTCAE Version 5.0. with or without pharmacological treatment. * Reproductive Status: Women who are pregnant, plan to get pregnant during the study, or who are breastfeeding. * Prior/Concomitant: Undergone HSCTs or gene therapy (candidates for HSCT or gene therapy with waiting period of ≥ 12 months are eligible). * Use of hydroxyurea treatment ≤ 12 weeks prior to enrollment for NTD participants and ≤ 24 weeks for TD participants. * Participant who has extramedullary hematopoiesis (EMH) complications requiring treatment to control the growth of EMH mass(es) during the screening period. * Any medical or psychiatric condition (including active infections, recent surgery, sequelae of diseases or interventions, clinically significant laboratory abnormalities or concurrent treatment) that in the opinion of the investigator would put the participant at unacceptable risk of participating in the study or that could affect interpretability of data. * Other protocol-defined inclusion/exclusion criteria apply.

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Conditions

The condition(s) this trial relates to.

alpha thalassemia spectrum anemia hemoglobin H disease

As listed by the trial registrant

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

Anemia

Contacts and locations

Locations

  • Local Institution - 0001

    Bangkok Noi, Bangkok, 10700, Thailand

  • Local Institution - 0002

    Haikou, 570311, China

  • Local Institution - 0003

    Taichung, TXG, 40447, Taiwan

  • Local Institution - 0004

    Nan Gang Qu, TPE, 10002, Taiwan

  • Local Institution - 0006

    Athens, 115 27, Greece

  • Local Institution - 0007

    Larissa, 26504, Greece

  • Local Institution - 0009

    Goudi, 11527, Greece

  • Local Institution - 0010

    Kaohsiung City, KHH, 807, Taiwan

  • Local Institution - 0011

    Haikou, Hainan, 570203, China

  • Local Institution - 0012

    Kunming, Yunnan, 650032, China

  • Local Institution - 0013

    Foshan, Guangdong, 528000, China

  • Local Institution - 0014

    Nanning, GX, 530021, China

  • Local Institution - 0015

    Guangzhou, GD, 510515, China

  • Local Institution - 0016

    Nanning, 530021, China

  • Local Institution - 0017

    Maoming Shi, Guangdong, 525000, China

  • Local Institution - 0018

    Rio, G, 265 04, Greece

  • Local Institution - 0019

    Naples, 80138, Italy

  • Local Institution - 0020

    Orbassano, TO, 10043, Italy

  • Local Institution - 0021

    Topkapı, 34093, Turkey (Türkiye)

  • Local Institution - 0023

    Liuzhou, Guangxi, 545006, China

  • Local Institution - 0025

    Hong Kong, HK, Hong Kong

  • Local Institution - 0026

    Genova, GE, 16128, Italy

  • Local Institution - 0027

    Altındağ, 06230, Turkey (Türkiye)

  • Local Institution - 0028

    Naples, 80131, Italy

  • Local Institution - 0029

    Guangzhou, GD, 510120, China

  • Local Institution - 0030

    Shenzhen Shi, Guangdong, 518025, China

  • Local Institution - 0031

    Mueang Phitsanulok, 65000, Thailand

  • Local Institution - 0032

    Kuala Lumpur, WP, 50586, Malaysia

  • Local Institution - 0033

    Johor Bahru, 80100, Malaysia

  • Local Institution - 0034

    Riyadh, 11411, Saudi Arabia

  • Local Institution - 0036

    Singapore, 229899, Singapore

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