Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants with Sickle Cell Disease - PHOENIX

Study identifier:ALXN1820-SCD-201

ClinicalTrials.gov identifier:NCT05565092

EudraCT identifier:2022-001615-74

CTIS identifier:N/A

Terminated/Withdrawn

Official Title

A Phase 2a, Randomized, Open-Label Study to Evaluate Multiple Dosing Regimens of Subcutaneous ALXN1820 in Adult Participants with Sickle Cell Disease

Medical condition

Sickle Cell Disease (SCD)

Phase

Phase 2

Healthy volunteers

Study drug

ALXN1820

Sex

All

Actual Enrollment

Study type

Interventional

Age

18 Years - n/a

Date

Study Start Date: 22 Feb 2023

Primary Completion Date: 09 Jan 2024

Study Completion Date: 09 Jan 2024

Study design

Allocation: Randomized
Endpoint Classification: -
Intervention Model: Parallel Assignment
Masking: -
Primary Purpose: Treatment

Verification:

Verified 01 Jul 2024 by Alexion Pharmaceuticals, Inc.

Collaborators

Inclusion and exclusion 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.

No locations available

Arms	Assigned Interventions
Experimental: ALXN1820 300 mg once weekly Participants will receive 300 milligrams (mg) once weekly (QW).	Drug: ALXN1820 ALXN1820 will be administered subcutaneously.
Experimental: ALXN1820 600 mg once every 4 weeks Participants will receive 600 mg once every 4 weeks (Q4W).	Drug: ALXN1820 ALXN1820 will be administered subcutaneously.
Experimental: ALXN1820 300 mg once every 2 weeks (Optional cohort) Participants will receive 300 mg once every 2 weeks (Q2W).	Drug: ALXN1820 ALXN1820 will be administered subcutaneously.

Contacts

Contact

Alexion Pharmaceuticals, Inc. (Sponsor)

1-855-752-2356

[email protected]