Luspatercept in Patients Affected With Rare Inherited Anemias

NCT07331818 | Not Yet Recruiting Phase 2

• 1 other identifier: LUSPARA - EuroBloodNet
• Study Type: interventional
• Target: 45
• Locations: 2 countries
• Sites: 6

Brief Summary

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

Conditions

Hereditary Red Blood Cell Disorder (Disorder)

Outcome Measures

Primary Outcomes (2)

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

  the proportion of patients who achieve an erythroid response, defined as a reduction in the transfusion burden of at least 33% from baseline (the 12-week period before the first dose of luspatercept) during 12 weeks plus a reduction of at least 2 red cell units over this 12-week interval.

  Up to 52 weeks

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

  the proportion of patients with a mean hemoglobin concentration increase of 1.0 g/dL or higher from baseline over a continuous 12-week interval in the absence of red blood cell transfusions

  Up to 52 weeks

Secondary Outcomes (8)

• Proportion of Transfusion dependent patients with a reduction in the transfusion burden (33 % / week 13 - 24)

  UP to 52 weeks

• Proportion of Transfusion dependent patients with a reduction in the transfusion burden (50% / week 13 - 24)

  UP to 52 weeks

• Proportion of Transfusion dependent patients with a reduction in the transfusion burden (33 % / week 37 - 48)

  UP to 52 weeks

• Proportion of Transfusion dependent patients with a reduction in the transfusion burden (50 % / week 37 - 48)

  UP to 52 weeks

• mean change from baseline in the transfusion burden

  UP to 52 weeks

Study Arms (1)

LUSPATERCEPT

EXPERIMENTAL

All eligible subjects will receive a starting dose of luspatercept of 1 mg/kg on day 1 of each 21 day cycle (every three weeks) In transfused patients, the first dose will be done at D8 from previous transfusion Responders at any dose will continue at the same dose until week 52 if they tolerate the drug (a follow up study will be envisaged)

Drug: Reblozyl

Interventions

Reblozyl DRUG

Reblozyl 25 mg powder for solution for injection / Reblozyl 75 mg powder for solution for injection

LUSPATERCEPT

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient affected with a rare constitutional anemia including. :
• constitutional non syndromic sideroblastic anemia (CSA) including those due to germline mutation in ALAS2, SLC25A38, SLC19A2, GLRX5, HSPA9 and also more rare cases with other mutations. . Patients without genetic diagnosis (currently up to 30% of CSa patients may be included after approval of PI and geneticists
• constitutional dyserythropïetic anemias CDA (type I and II)
• 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:
• 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:
• Have had 2 negative pregnancy tests as verified by the investigator prior to starting the Investigational Product (IP) (unless the screening pregnancy test was done within 72 hours of Cycle 1 Day 1). She must have had agreed to ongoing a monthly pregnancy testing during the course of the study and after EOT
• If sexually active, agreed to have used, and been able to comply with, highly effective contraception\*\* without interruption, 5 weeks prior to starting IP, during treatment with IP (including dose interruptions), and for 12 weeks after discontinuation of IP. \*\* Highly effective contraception was defined in this protocol as the following (information also appeared in the ICF): Hormonal contraception (eg, birth control pills, injection, implant, transdermal patch, vaginal ring), intrauterine device, tubal ligation (tying your tubes), or a partner with a vasectomy
• 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

You may not qualify if:

• 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
• Severe infection or any other uncontrolled severe condition.
• Uncontrolled hypertension
• Significant cardiac disease - NYHA Class III or IV or having suffered a myocardial infarction in the last 6 months.
• Use of investigational agents within 30 days or any anticancer therapy (including IMiD) within 2 weeks before the study entry. The patient must have recovered at least a grade 1 from all acute toxicity from any previous therapy.
• Use of EPO within 4 weeks of study entry
• Active cancer or cancer during the year prior to trial entry other than basal cell carcinoma, or carcinoma in situ of the cervix or breast.
• Patient already enrolled in another therapeutic trial of an investigational drug.
• Known HIV infection or active hepatitis B or C.
• Women who are or could become pregnant or who are currently breastfeeding.
• Any medical or psychiatric contraindication that would prevent the patient from understanding and signing the informed consent form.
• Patient eligible at short or medium term for allogeneic stem cell transplantation.
• Known allergies to luspatercept or any of its excipients.
• No affiliation to a health insurance system

Sponsors & Collaborators

• EuroBloodNet Association: lead

Study Sites (6)

GCS Groupement des Hôpitaux de l'Institut Catholique de Lille

Lille, 59800, France

Location

Centre Hospitalier Universitaire de Montpellier

Montpellier, 34295, France

Location

Assistance Publique Hôpitaux de Paris - Hôpital Saint-Louis

Paris, 75010, France

Location

Assistance Publique Hôpitaux de Paris - Hôpital Necker

Paris, France

Location

CHU Bordeaux-Hôpital Haut-Lévêque

Pessac, 33600, France

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano

Milan, Italy

Location

MeSH Terms

Interventions

luspatercept

Study Officials

• Thierry Leblanc, Phd

  Assistance Publique Hôpitaux de Paris - Hôpital Robert-Debré

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Adeline Gladieux

CONTACT

0171207059; adeline.gladieux@eurobloodnetassociation.com

Fatiha Chermat, Phd

CONTACT

0171207059; fatiha.chermat-ext@aphp.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 5, 2025
• First Posted: January 12, 2026
• Study Start: January 15, 2026
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): September 30, 2029
• Last Updated: January 12, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

FR (5); IT (1)