Not Yet RecruitingPhase 2

Luspatercept in Patients Affected With Rare Inherited Anemias

France, Italy45 participantsStarted 2026-01-15

Plain-language summary

This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias

Who can participate

Age range

18 Years – 99 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Patient affected with a rare constitutional anemia including. :
. For diseases of the three subtypes (CSA, CDA, and DBA-NTD), diagnosis must be supported genetically by presence of ACMG class 4 or 5 variant(s).
. Age ≥18 years at the first screening
. For CSA and CDA, both Transfusion dependent (TD) patients and Non Transfusion dependent (TD) patients may be included:

Exclusion criteria

. Adequate renal function, defined by creatinine less than 1.5 times the upper limit of normal, creatinine clearance ≥ 30 mL/min (MDRD formula).
. Adequate liver function, defined by transaminases and gamma-glutamyl transferase less than 1.5 times the upper limit of normal.
. ECOG performance status 0-2 at the time of screening.
. Be willing and able to give written informed consent and to comply to all study procedures for the duration of the study.
. A FCBP (female of childbearing potential) for this study was defined as a sexually mature woman who: (1) had not undergone a hysterectomy or bilateral oophorectomy; or (2) had not been naturally postmenopausal (amenorrhea following cancer therapy did not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months). A FCBP participating in the study must:

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Evaluating Luspatercept in patients affected with rare inherited anemias - Transfusion dependent patients

Timeframe: Up to 52 weeks

Evaluating Luspatercept in patients affected with rare inherited anemias - Non Transfusion dependent patients

Timeframe: Up to 52 weeks

Trial details

NCT IDNCT07331818

SponsorEuroBloodNet Association

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2028-09

Contact for this trial

Adeline Gladieux

0171207059 adeline.gladieux@eurobloodnetassociation.com

View on ClinicalTrials.gov More Hereditary Red Blood Cell Disorder (Disorder) trials