Testing Luspatercept against a placebo for anemia
Official title: A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia
A Phase 2, Study for the Treatment of Anemia With Alpha (α)-Thalassemia to Determine the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) in Adults and Evaluate the Safety and Pharmacokinetics in Adolescents
- Phase 2
- 4 groups
- One site, in Halifax
- Recruiting
Interventions
- Biological therapy
Luspatercept
Specified dose on specified days
- Medication
Placebo
Specified dose on specified days
Canadian Sites (1)
1 listed, none recruiting
- Withdrawn
Local Institution - 0008
Halifax, Nova Scotia
Eligibility Criteria
See who this study is looking for16 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +iii) Participant has Karnofsky (age ≥16 years) or Lansky (age \< 16 years) performance status score ≥ 50 at screeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
- +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 randomizationRandomisedWhich group you go into is decided by chance, not by you or your doctor.Read more →. 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 baselineBaselineYour starting measurements, taken before treatment begins.Read more → 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 enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more → 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.
Exclusion
- −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 randomizationRandomisedWhich group you go into is decided by chance, not by you or your doctor.Read more →. 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 protocolProtocolThe detailed plan a study must follow.Read more → is considered: blood pressure value corresponding to ≤Grade 1 according to NCI CTCAE Version 5.0. with or without pharmacological treatment.
- −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 enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more → for NTD participants and ≤ 24 weeks for TD participants.
- −Participant who has extramedullary hematopoiesis (EMH) complications requiring treatment to controlControl groupThe group a new treatment is measured against.Read more → the growth of EMH mass(es) during the screening periodScreeningThe checks done before joining, to see whether a study fits.Read more →.
- −Other protocol-defined inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more →/exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → apply.
- −Reproductive Status: Women who are pregnant, plan to get pregnant during the study, or who are breastfeeding.
- −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 investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more → would put the participant at unacceptable risk of participating in the study or that could affect interpretability of data.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more → — a middle-stage study in a few hundred people at most, looking at what the treatment does and watching for side effectsSide effectAn unwanted effect thought to be caused by the treatment itself.Read more →.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 4 groups in this study.
Groups A and C receive Luspatercept.
Registry label: A: Transfusion Dependent (TD): Luspatercept + Best supportive care (BSC) · C: Non-transfusion Dependent (NTD): Luspatercept + BSC
Groups B and D, the placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group, receive Placebo.
Registry label: B: Adult TD Cohort: Placebo + BSC · D: Adult NTD Cohort: Placebo + BSC
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
One group receives a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → — a dummy treatment with no active medicine in it.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists a minimum age of 12 years, with no upper limit given.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 189 people.
The study is currently expected to finish around August 2034.
The main measurement is taken over: Up to Week 48.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Number of participants with ≥ 50% reduction from baselineBaselineYour starting measurements, taken before treatment begins.Read more → in RBC transfusion burden with a reduction of at least 2 units during any continuous 12 weeks during Week 13-48 compared to 12-week interval immediately prior to date of first dose — measured over Up to Week 48.
Number of participants with an increase from baseline of ≥ 1.0 grams (g)/decilitre (dL) in mean hemoglobin (Hb) values over the continuous 12-week interval from Week 13 to Week 24 in the absence of RBC transfusion — measured over Up to Week 24.
Dose-limiting toxicities (DLTs) defined as observance of ≥ Grade 3-related hemolytic crises or ≥ Grade 3-related event outside of the known safety profile occurring within the 21 days from their first dose of study therapy — measured over Up to Week 3.
Number of participants with adverse eventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → (AEs) — measured over Up to 5 years.
The study lists 1 further main measurements.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT05664737 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About LuspaterceptBiological
Specified dose on specified days
From the trial registry — its own words, unedited.
What a biological is here: A treatment made from or by living systems — such as antibodies, vaccines, or cell-based therapies.
Read the full explanation → · in clinical review
About PlaceboDrug
Specified dose on specified days
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: a minimum age of 12 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
Yes. This study includes a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group — a dummy treatment with no active medicine in it.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists a minimum age of 12 years, with no upper limit given.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: Up to Week 48.
The study as a whole is currently expected to finish around 2034-08-14.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 189 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists one location: Halifax, Nova Scotia, Canada.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
The purpose of the study is to evaluate the efficacy and safety of luspatercept plus best supportive care (BSC) vs placebo plus BSC on anemia in adult participants with α-thalassemia hemoglobin H (HbH) disease and determine the safety and drug levels in adolescent participants.
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Complete a quick intake form and we will match you with this and other relevant trials based on your medical profile.
See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.