Improvement of MRI assessed cerebral Perfusion and oxygenation by luspatercept-induced Anemia Correction in non-transfusion dependent Thalassemia

Amsterdam UMC0 sites12 target enrollmentAugust 8, 2023

Overview

No summary available.

Registry

who.int

Start Date

August 8, 2023

End Date

TBD

Last Updated

last year

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

Amsterdam UMC

•NTDT beta\-thalassemia, Baseline Hb level of \= 10\.0 based on the mean Hb level 24 weeks before initiation of luspatercept g/dL, \= 18 years of age, \= 5 RBC units transfused in the 24 weeks prior to inclusion in the study, Participants, who if female and of childbearing potential, are using highly effective methods of contraception from study start to 30 days after the last dose of study drug, and who if male are willing to use barrier methods of contraception, from study start to 30 days after the last dose of study drug., Participant has provided documented informed consent or assent (the informed consent form \[ICF] must be reviewed and signed by each participant; the participant’s legal representative or legal guardian, and the participant’s assent must be obtained).

•No informed consent has been given., Contra\-indication for MRI or acetazolamide, Female who is breast feeding or pregnant., Hepatic dysfunction characterized by alanine aminotransferase (ALT) \> 4 × ULN., Severe renal dysfunction (estimated glomerular filtration rate \<30mL/min)., History of malignancy within the past 2 years prior to participation requiring chemotherapy and/or radiation (with the exception of local therapy for non\-melanoma skin malignancy)., History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent, including but not limited to the following: 1\. Unstable angina pectoris or myocardial infarction or elective coronary intervention. 2\. Congestive heart failure requiring hospitalization. 3\. Uncontrolled clinically significant arrhythmias., Participated in another clinical trial of an investigational agent (or medical device) within 30 days or 5 half\-lives of date of informed consent, whichever is longer, or is currently participating in another trial of an investigational agent (or medical device)., Medical, psychological, or behavioral conditions, which, in the opinion of the Investigator, may preclude safe participation, confound study interpretation, interfere with compliance, or preclude informed consent.