a study of shp655 (radamts13) in sickle cell disease

Conditions: Sickle Cell Disease

Summary: tak-755 (previously known as shp655) is a medicine used to treat sickle cell disease (scd). the main aim of the study is to measure the safety and tolerability of tak-755 in scd participants. study participants will receive tak-755 or placebo on day 1. their scd will be treated by their doctor according to their doctor's usual clinical practice. during the study, participants will be asked to follow-up on 13 days following shp655 or placebo administration for safety assessment. maximum duration of participation is expected to be about 2 months.


Eligibility criteria:
inclusion criteria: * age 18 to 65 years at the time of signing the informed consent. * an understanding, ability, and willingness to fully comply with study procedures and requirements. * ability to voluntarily provide written, signed, and dated (personally or via a legally authorized representative) informed consent to participate in the study. * male or female with a documented history of hbss or hbsβo thalassemia (based on clinical record of genetic, electrophoresis, or high-performance liquid chromatography testing). * participant currently taking hydroxyurea must be on a stable dosing for 3 months at screening. exclusion criteria: * the participant was diagnosed with acute voc in the 21 days before dosing on day 1. * the participant has undergone blood transfusion within the last 30 days or blood transfusion on greater than or equal to (\>=) 2 occasions in the last 90 days, at screening visit. * the participant has a history of acquired or congenital thrombotic thrombocytopenic purpura. * the participant has serum creatinine level greater than (\>) 1.2 milligrams per deciliter (mg/dl). * the participant has alanine transaminase \>3\* upper limit of normal (based on clinical laboratory normal range), direct bilirubin level \>2 mg/dl, or indirect bilirubin level \>5 mg/dl at the screening visit. * the participant has a hemoglobin level \<5 grams per deciliter (g/dl) at the screening visit. * the participant has a platelet count of \<100 000/cubic millimeter (mm\^3) at the screening visit. * signs or symptoms of infection requiring treatment with iv antibiotics during the screening period. * the participant has fever with body temperature of \>=38.5 degree celsius (ºc) (101.3 degree fahrenheit \[ºf\]) at the screening visit or before dosing on day 1. * the participant has acute chest syndrome (acs), diagnosed or strongly suspected, as evidenced by a new infiltrate on chest radiograph, and one or more of the following criteria: 1. fever with body temperature \>39°c (102.2°f) 2. hypoxia (confirmed by arterial blood gases with partial pressure of arterial oxygen (pao2) \<70 millimeter of mercury \[mmhg\]) 3. chest pain 4. suspicious findings on physical examination (tachypnea, intercostal retraction, wheezing, and/or rales) * the participant has recently (within the past 28 days, from screening visit) undergone major surgery, requires hospitalization, documented serious bacterial infection requiring antibiotic treatment, or significant bleeding. * the participant has had a recent (within the past 90 days, from screening visit) episode of stroke, transient ischemic attack, symptomatic pulmonary hypertension, or seizure. * any history of hemorrhagic stroke or bleeding diathesis. * the participant has received any of the following protocol-restricted medicines: a) systemic steroid therapy within 48 hours before dosing, or there is the expectation that such therapy may be given during the study (inhaled or topical steroids are allowed); b) anticoagulant or antiplatelet therapy within the past 3 weeks before dosing; c) crizanlizumab within the past 30 days before dosing; d) voxelotor within the past 14 days before dosing. * for participants receiving chronic or long-acting opioids, a change in dose or pain requiring medical attention in the past 14 days before dosing. * the participant has a medical or psychiatric condition that, in the opinion of the investigator, may pose a risk to the participant for participation or interfere with the conduct or results of the study. * the participant has received or plans to receive any other investigational agent within the 4 weeks prior to the study screening visit or during the course of the study. * there is the expectation that the participant will not be able to be followed for the duration of the study. * the participant is pregnant or lactating or a female of childbearing potential or male unable or unwilling to comply with birth control methods or abstinence until the end of study visit. * the participant with active use of illicit drugs (excluding marijuana) and/or alcohol dependence, as determined by the investigator. * the participant has been administered shp655 previously. * known life-threatening hypersensitivity reaction, including anaphylaxis, to the parent molecule adamts-13, hamster protein, or other constituents of shp655. * the participant has a positive test result for hepatitis b surface antigen, or hepatitis c antibody, or human immunodeficiency virus (hiv) antigen/antibody, at the screening visit. however, a participant with a hepatitis c antibody and a negative hepatitis c virus ribonucleic acid (rna) polymerase chain reaction test is not excluded.