Ignite Creation Date:
2024-05-06 @ 7:11 PM
Last Modification Date:
2024-10-26 @ 3:02 PM
Study NCT ID:
NCT05924100
Status:
RECRUITING
Last Update Posted:
2023-06-29
First Post:
2023-06-08
Brief Title:
Efficacy and Safety of Luspatercept for the Treatment of Anemia Due to MDS With del5q RefractoryResistantIntolerant to Prior Treatments RBC-TD
Sponsor:
Associazione Qol-one
Organization:
Associazione Qol-one
Study Overview
Official Title:
Efficacy and Safety of Luspatercept for the Treatment of Anemia Due to Myelodysplastic Syndromes With del5q RefractoryResistantIntolerant to Prior Treatments Who Require Red Blood Cell Transfusions
Status:
RECRUITING
Status Verified Date:
2023-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
Phoenix
Brief Summary:
Myelodysplastic syndromes primarily affecting older adults are a heterogeneous group of clonal disorders of hematopoietic stem cells characterized by ineffective hematopoiesis that manifest clinically as anemia neutropenia andor thrombocytopenia of variable severity these often result in RBC- transfusion dependent TD anemia increased risk of infection andor hemorrhage as well as a potential to progress to acute myeloid leukemia AML
Lenalidomide is approved for red blood cell transfusion-dependent RBC TD anemia due to low-risk myelodysplastic syndromes MDS with a chromosome 5q deletion del5q with or without additional cytogenetic abnormalities About one third of patients are refractoryresistantintolerant and will require further treatment options
Luspatercept ACE-536 an erythroid maturation agent is a recombinant fusion protein consisting of a modified form of the extracellular domain ECD of the human activin receptor type IIB ActRIIB linked to the Fc portion of human immunoglobulin G1 IgG1-Fc Luspatercept acts on endogenous inhibitors of late-stage erythropoiesis eg growth differentiation factor 11 GDF11 to increase release of mature erythrocytes into circulation Nonclinical data have demonstrated that luspatercept binds to negative regulators governing late-stage erythroid development to inhibit their action thereby promoting the maturation of erythrocytes in the bone marrow
Luspatercept is indicated for the treatment of adult patients with transfusion-dependent anaemia associated with beta-thalassaemia and due to very low low and intermediate-risk MDS with ring sideroblasts who had an unsatisfactory response to or are ineligible for erythropoietin-based-therapy It is not indicated for other MDS subtypes Unfortunately patients with MDS with del5q refractoryresistantintolerant to lenalidomide are excluded from clinical trials that evaluate novel treatments for the anemia of RBC TD lower risk MDS Therefore treatment of anemia in such patients is an unmet need
QOL-ONE Phoenix is a Phase 2 multicenter single arm prospective study The primary objective of the study is to evaluate the effect of luspatercept on RBC TI in subjects with MDS with del5q with IPSS-R very low low or intermediate risk and 5 bone marrow blasts resistantrefractoryintolerant to lenalidomide and who require RBC transfusions
The study is divided into a Screening Period a 2-year Treatment Period and a 3-year Follow-up Period
Primary objective is to evaluate the effect of luspatercept on RBC TI lack of transfusions for 8 consecutive weeks within the first 24 weeks in subjects with MDS with del5q with IPSS-R very low low or intermediate risk and 5 bone marrow blasts resistantrefractoryintolerant to lenalidomide and RBC TD
Lenalidomide is approved for red blood cell transfusion-dependent RBC TD anemia due to low-risk myelodysplastic syndromes MDS with a chromosome 5q deletion del5q with or without additional cytogenetic abnormalities About one third of patients are refractoryresistantintolerant and will require further treatment options
Luspatercept ACE-536 an erythroid maturation agent is a recombinant fusion protein consisting of a modified form of the extracellular domain ECD of the human activin receptor type IIB ActRIIB linked to the Fc portion of human immunoglobulin G1 IgG1-Fc Luspatercept acts on endogenous inhibitors of late-stage erythropoiesis eg growth differentiation factor 11 GDF11 to increase release of mature erythrocytes into circulation Nonclinical data have demonstrated that luspatercept binds to negative regulators governing late-stage erythroid development to inhibit their action thereby promoting the maturation of erythrocytes in the bone marrow
Luspatercept is indicated for the treatment of adult patients with transfusion-dependent anaemia associated with beta-thalassaemia and due to very low low and intermediate-risk MDS with ring sideroblasts who had an unsatisfactory response to or are ineligible for erythropoietin-based-therapy It is not indicated for other MDS subtypes Unfortunately patients with MDS with del5q refractoryresistantintolerant to lenalidomide are excluded from clinical trials that evaluate novel treatments for the anemia of RBC TD lower risk MDS Therefore treatment of anemia in such patients is an unmet need
QOL-ONE Phoenix is a Phase 2 multicenter single arm prospective study The primary objective of the study is to evaluate the effect of luspatercept on RBC TI in subjects with MDS with del5q with IPSS-R very low low or intermediate risk and 5 bone marrow blasts resistantrefractoryintolerant to lenalidomide and who require RBC transfusions
The study is divided into a Screening Period a 2-year Treatment Period and a 3-year Follow-up Period
Primary objective is to evaluate the effect of luspatercept on RBC TI lack of transfusions for 8 consecutive weeks within the first 24 weeks in subjects with MDS with del5q with IPSS-R very low low or intermediate risk and 5 bone marrow blasts resistantrefractoryintolerant to lenalidomide and RBC TD
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None