Hereditary Red Blood Cell Disorder (Disorder)Phase 2LUSPARANCT07331818
Luspatercept in Patients Affected With Rare Inherited Anemias
A Phase 2 study testing Reblozyl in people with Hereditary Red Blood Cell Disorder (Disorder), run by EuroBloodNet Association at 6 sites in 2 countries.
Trial summary
This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Hereditary Red Blood Cell Disorder (Disorder)
- What is being tested?
- Reblozyl
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 15 Jan 2026to 30 Sep 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
- Diamond-Blackfan anemia not requiring regular transfusion support (NTD-DBA) (therapeutic independence or with continuous steroid therapy); 2 subgroups should be considered: RPS19 versus other genetic subgroups (mainly RPL5, RPL11 and RPS26 variants). Inclusions will be considered in order to have at least 3 patients in each subgroup before to expand inclusions
- 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
Show all 17Show fewer
- For CSA and CDA, both Transfusion dependent (TD) patients and Non Transfusion dependent (TD) patients may be included:
- TD patients: transfusion-dependency definition is: 6 to 20 units of packed red cells within previous 24 weeks with no transfusion-free period of > 56 days (except for DBA patients for whom transfusion dependency is a factor of exclusion)
- NTD patients: patients must have significant anemia e.g. hemoglobin < 10.5 gr/dl (average of at least 2 Hb measurements separated by a minimum of 7 days during screening period) occasional transfusion aloowed if ≤ 5 red-cell units per 24 weeks and red blood cell transfusion free > 8 weeks before inclusion
- 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
Who cannot join
- 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.
Show all 18Show fewer
- 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
- For men and women of reproductive potential: unwillingness to be abstinent or use double anticonception during the trial period.
- Persons deprived of liberty by judicial or administrative decision
- Persons subject to a legal protection measure (guardianship, curatorship, safeguard of justice)
Trial locations and contacts
6 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
6 sites in 2 countries
France5 sites
GCS Groupement des Hôpitaux de l'Institut Catholique de Lille
Lille, 59800
Benjamin Carpentier, Phd, Carpentier.Benjamin@ghicl.net
Centre Hospitalier Universitaire de Montpellier
Montpellier, 34295
Patricia Aguilar Martinez, Phd, 04 67 33 54 75 ext. 33, p-martinez@chu-montpellier.fr
Assistance Publique Hôpitaux de Paris - Hôpital Saint-Louis
Paris, 75010
Pierre Fenaux, MD/PhD, 0171207022 ext. 33, pierre.fenaux@aphp.fr
Assistance Publique Hôpitaux de Paris - Hôpital Necker
Paris
Olivier Hermine, MD/Phd, 01 44 49 52 87 ext. 33, olivier.hermine@aphp.fr
CHU Bordeaux-Hôpital Haut-Lévêque
Pessac, 33600
Chloé Dhunputh, 05 57 65 65 11 ext. 33, chloe.dhunputh@chu-bordeaux.fr
Italy1 site
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano
Milan
Bruno Fattizzo, 0255033345 ext. 39, bruno.fattizzo@policlinico.mi.it
About this study
This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias
Read the detailed description
This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias including : ✔ CSA group: constitutional non syndromic sideroblastic anemia () due to germline mutation including those with ALAS2, SLC25A38, SLC19A2, GLRX5, HSPA9. and other gene mutations ✔ CDA group: constitutional dyserythropïetic anemias ( (type I and II) ✔ NTD-DBA group: Diamond-Blackfan anemia (DBA) not requiring regular transfusion support (NTD-DBA) with or without continuous steroid therapy); (therapeutic independence or with continuous steroid therapy); 2 subgroups will be considered: RPS19 versus other genetic subgroups (RPL5, RPL11 and RPS26 mutations) ; to note these 4 genotypes account for the vast majority of patients
Patients will be recruited from centers of expertise within the European Union (France and Italy). In total, 45 patients are will be recruited.
- Being tested
- Reblozyl
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ReblozylDrug
Reblozyl 25 mg powder for solution for injection / Reblozyl 75 mg powder for solution for injection
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: Drug: Reblozyl |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: Up to 52 weeks
Secondary outcomes (8)
Proportion of Transfusion dependent patients with a reduction in the transfusion burden (33 % / week 13 - 24)
Proportion of patients with a reduction in the transfusion burden of at least 33% from baseline during weeks 13 through 24 plus a reduction of at least 2 red-cell units over this 12-week interval
Measured: UP to 52 weeks
Proportion of Transfusion dependent patients with a reduction in the transfusion burden (50% / week 13 - 24)
Proportion of patients with a reduction in the transfusion burden of at least 50% from baseline during weeks 13 through 24 plus a reduction of at least 2 red-cell units over this 12-week interval
Measured: UP to 52 weeks
Proportion of Transfusion dependent patients with a reduction in the transfusion burden (33 % / week 37 - 48)
Proportion of patients with a reduction in the transfusion burden of at least 33% from baseline during weeks 37 through 48 plus a reduction of at least 2 red-cell units over this 12-week interval,
Measured: UP to 52 weeks
Proportion of Transfusion dependent patients with a reduction in the transfusion burden (50 % / week 37 - 48)
Proportion of patients with a reduction in the transfusion burden of at least 50% from baseline during weeks 37 through 48 plus a reduction of at least 2 red-cell units over this 12-week interval
Measured: UP to 52 weeks
mean change from baseline in the transfusion burden
mean change from baseline in the transfusion burden during weeks 13 through 24 and during weeks 37 through 48.
Measured: UP to 52 weeks
Proportion of Non Transfusion dependent patients - hemoglobin concentration
mean change from baseline in hemoglobin concentration over a continuous 12-week interval (weeks 13-24)
Measured: UP to 52 weeks
Proportion of Non Transfusion dependent patients - hemoglobin concentration increase
Proportion of Non Transfusion dependent patients with a mean hemoglobin concentration increase of 1.5 g/dL or higher from baseline over a continuous 12-week interval (weeks 13-24 and weeks 37-48) in the absence of red blood cell transfusions
Measured: UP to 52 weeks
Proportion of Non Transfusion dependent patients - duration of mean hemoglobin concentration
Proportion of Non Transfusion dependent patients - duration of mean hemoglobin concentration increase of 1.0 g/dL or higher from baseline"
Measured: UP to 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2026
- Study start15 Jan 2026 (estimated)
- Primary completionSep 2028 (estimated)
- Study completion30 Sep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Red Blood Cell Disorder (Disorder)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- EuroBloodNet Association
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Thierry Leblanc, Phd, Principal investigator, Assistance Publique Hôpitaux de Paris - Hôpital Robert-Debré
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07331818
- Sponsor's protocol ID
- LUSPARA - EuroBloodNet
- Acronym
- LUSPARA
- Status last verified
- Jan 2026
- First submitted
- 5 Dec 2025
- Last updated
- 12 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Luspatercept in Patients Affected With Rare Inherited Anemias
- ClinicalTrials.gov ID
- NCT07331818
- Acronym / sponsor ID
- LUSPARA / LUSPARA - EuroBloodNet
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- EuroBloodNet AssociationUniversity, hospital or other
- Study start
- 15 Jan 2026 (estimated)
- Primary completion
- Sep 2028 (estimated)
- Study completion
- 30 Sep 2029 (estimated)
- First posted
- 12 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias
Detailed description
This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias including : ✔ CSA group: constitutional non syndromic sideroblastic anemia () due to germline mutation including those with ALAS2, SLC25A38, SLC19A2, GLRX5, HSPA9. and other gene mutations ✔ CDA group: constitutional dyserythropïetic anemias ( (type I and II) ✔ NTD-DBA group: Diamond-Blackfan anemia (DBA) not requiring regular transfusion support (NTD-DBA) with or without continuous steroid therapy); (therapeutic independence or with continuous steroid therapy); 2 subgroups will be considered: RPS19 versus other genetic subgroups (RPL5, RPL11 and RPS26 mutations) ; to note these 4 genotypes account for the vast majority of patients
Patients will be recruited from centers of expertise within the European Union (France and Italy). In total, 45 patients are will be recruited.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LUSPATERCEPT | Experimental | Drug: ReblozylAll 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) |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- 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:
- Adequate renal function, defined by creatinine less than 1.5 times the upper limit of normal, creatinine clearance ≥ 30 mL/min (MDRD formula).
Key exclusion criteria (18 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ReblozylReblozyl 25 mg powder for solution for injection / Reblozyl 75 mg powder for solution for injection | Drug | LUSPATERCEPT |
Primary outcome
Evaluating Luspatercept in patients affected with rare inherited anemias - Transfusion dependent patients
Time frame: Up to 52 weeks
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.
Evaluating Luspatercept in patients affected with rare inherited anemias - Non Transfusion dependent patients
Time frame: Up to 52 weeks
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
8 secondary outcomes
Proportion of Transfusion dependent patients with a reduction in the transfusion burden (33 % / week 13 - 24)
Time frame: UP to 52 weeks
Proportion of Transfusion dependent patients with a reduction in the transfusion burden (50% / week 13 - 24)
Time frame: UP to 52 weeks
Proportion of Transfusion dependent patients with a reduction in the transfusion burden (33 % / week 37 - 48)
Time frame: UP to 52 weeks
Proportion of Transfusion dependent patients with a reduction in the transfusion burden (50 % / week 37 - 48)
Time frame: UP to 52 weeks
mean change from baseline in the transfusion burden
Time frame: UP to 52 weeks
Proportion of Non Transfusion dependent patients - hemoglobin concentration
Time frame: UP to 52 weeks
Proportion of Non Transfusion dependent patients - hemoglobin concentration increase
Time frame: UP to 52 weeks
Proportion of Non Transfusion dependent patients - duration of mean hemoglobin concentration
Time frame: UP to 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jan 2026.
- Individual participant dataSharing planned: No
Related trials for Hereditary Red Blood Cell Disorder (Disorder)
Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)
Diamond Blackfan Anemia
Not yet recruiting
Hypoxic Red Blood Cells in Sickle Cell Anemia
Sickle Cell Anaemia, Sickle Cell Anemia Crisis
RecruitingObservational study
The Correlation Between Red Cell Transfusion and Complications of Prematurity
Retinopathy of Prematurity (ROP), Bronchopulmonary Dysplasia (BPD)
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)
Sickle Cell Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Thierry Leblanc, Phd principal investigator
Assistance Publique Hôpitaux de Paris - Hôpital Robert-Debré
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Reblozyl in people with Hereditary Red Blood Cell Disorder (Disorder), run by EuroBloodNet Association at 6 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 12 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in France, Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Adeline Gladieux (0171207059 ext. 33, adeline.gladieux@eurobloodnetassociation.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluating Luspatercept in patients affected with rare inherited anemias - Transfusion dependent patients (time frame: Up to 52 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jan 2026.