Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease

• Status: Not yet recruiting
• Trial end date: 2024-02-15
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals

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.