safety, efficacy, pharmacokinetic, and pharmacodynamic study of alxn1820 in adult participants with sickle cell disease

Conditions: Sickle Cell Disease (SCD)

Summary: the primary objective of this study is to assess the safety and tolerability of alxn1820 sc (subcutaneous) in participants with scd (sickle cell disease).


Eligibility criteria:
inclusion criteria: * confirmed diagnosis of scd (hbss, or hbsβ0-thalassemia). * body weight ≥ 40 kg (inclusive) at screening. * must follow protocol-specified contraception guidance while on treatment and for up to 6 months after last dose. * hemoglobin between 5.5 and 10 g/dl at screening * have had 1 to 10 vocs in the past 12 months. * patients receiving hydroxyurea must have been on a stable dose for ≥ 3 months prior to providing informed consent, with no anticipated need for dose adjustment during the study. * patients will be vaccinated with mcv4 and serogroup b meningococcal vaccinations at least 14 days before dosing, if not already vaccinated within 3 years before the first dose. * haemophilus influenzae type b (hib) and streptococcus pneumoniae vaccination are up to date according to current national/local vaccination guidelines for patients with scd. exclusion criteria: * planned initiation, termination, or dose alteration of hydroxyurea during the study. * receiving voxelotor (oxbryta) or crizanlizumab (adakveo) within 60 days of providing informed consent. * receiving treatment with recombinant human erythropoetins (eg, epoetin alfa). * treated with complement inhibitors within 6 months prior to the first dose. * patients who are on chronic transfusion or receive a transfusion within 60 days of first dose. * any significant disease or disorder which, in the opinion of the investigator, may put the participant at risk. * hepatitis b (positive hepatitis surface antigen \[hbsag\] or positive core antibody (anti-hbc) with negative surface antibody \[anti-hbs\]) or hepatitis c viral infection (hepatitis c virus \[hcv\] antibody positive, except for patients with documented successful treatment and documented sustained virologic response) at screening. * active systemic bacterial, viral, or fungal infection within 14 days prior to dosing. * participation (ie, last protocol-required study visit) in a clinical study within 90 days or 5 half-lives of the investigational agent, whichever is longer, before initiation of dosing on day 1. * participation in more than 1 clinical study of a monoclonal antibody (mab), or participation in a clinical study of a mab within the 6 months or 5 half-lives of the mab, whichever is longer, prior to screening, during which the participant was exposed to the active study drug. * severe renal impairment (estimated glomerular filtration rate \[egfr\] \< 30 ml/min/1.73 m2 ) or on chronic dialysis. * history of allergy or hypersensitivity to excipients of alxn1820 (eg, polysorbate 80). * history of complement deficiency. * history of n meningitidis, s pneumoniae, or h influenzae infection. * history of malignancy with the exception of a nonmelanoma skin cancer or carcinoma in situ of the cervix that has been treated with no evidence of recurrence within 5 years. * participants who are pregnant or breastfeeding.

Keywords: sickle cell disease alxn1820 scd