Not Yet RecruitingPhase 2

Luspatercept in Patients Affected With Rare Inherited Anemias

France45 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 range18 Years – 99 Years

SexALL

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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:
✕0. Male subjects must: Have agreed to use a condom, defined as a male latex condom or nonlatex condom NOT made out of natural (animal) membrane (eg, polyurethane), during sexual contact with a pregnant female or a FCBP while participating in the study, during dose interruptions, and for at least 12 weeks following IP discontinuation, even if he had undergone a successful vasectomy
✕. DBA patients with transfusion dependency or DBA patients with non RPS19, RPS26, RPL5 or RPL11 genotype or without gene identification
✕. For patients with CSA and no established genetic diagnosis, acquired sideroblastic anemia and SF3B1 variant should be excluded with non RPS19, RPS26, RPL5 or RPL11 genotype or without gene identification

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