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

NCT05565092 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: ALXN1820-SCD-2012022-001615-74
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 2

Brief 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).

Conditions

Sickle Cell Disease (SCD)

Keywords

Sickle Cell Disease; ALXN1820; SCD

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-Emergent Adverse Events and Serious Adverse Events

  Baseline through Day 211 (Cohorts 1 and 2) and through Day 169 (Optional Cohort 3)

Secondary Outcomes (8)

• Pharmacokinetics: Serum ALXN1820 Concentration

  Baseline through Day 211 (Cohorts 1 and 2) and through Day 169 (Optional Cohort 3)

• Change From Baseline in Serum Concentration of Total and Free Properdin Through Day 211 (Cohorts 1 and 2) and Day 169 (Optional Cohort 3)

  Baseline through Day 211 (Cohorts 1 and 2) and Day 169 (Optional Cohort 3)

• Change From Baseline Complement Alternative Pathway Activity Through Day 211 (Cohorts 1 and 2) and Day 169 (Optional Cohort 3)

  Baseline through Day 211 (Cohorts 1 and 2) and Day 169 (Optional Cohort 3)

• Change From Baseline in Complement Biomarkers Through Week 12 (Cohorts 1 and 2)

  Baseline, Week 12

• Change From Baseline in Hemoglobin Level at Week 12 (Cohorts 1 and 2)

  Baseline, Week 12

Study Arms (3)

ALXN1820 300 mg once weekly

EXPERIMENTAL

Participants will receive 300 milligrams (mg) once weekly (QW).

Drug: ALXN1820

ALXN1820 600 mg once every 4 weeks

EXPERIMENTAL

Participants will receive 600 mg once every 4 weeks (Q4W).

Drug: ALXN1820

ALXN1820 300 mg once every 2 weeks (Optional cohort)

EXPERIMENTAL

Participants will receive 300 mg once every 2 weeks (Q2W).

Drug: ALXN1820

Interventions

ALXN1820 DRUG

ALXN1820 will be administered subcutaneously.

ALXN1820 300 mg once every 2 weeks (Optional cohort); ALXN1820 300 mg once weekly; ALXN1820 600 mg once every 4 weeks

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (2)

Research Site

Hollywood, Florida, 33023, United States

Location

Research Site

Indianapolis, Indiana, 46260, United States

Location

Related Links

• Related Info
• Related Info

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Limitations and Caveats

Per Sponsor decision, the study was terminated. Only 2 participants were enrolled. Due to concerns regarding participant confidentiality, no data are reported.

Results Point of Contact

• Title: Alexion Pharmaceuticals, Inc.
• Organization: Alexion Pharmaceuticals, Inc.

clinicaltrials@alexion.com

+1.855.752.2356

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 15, 2022
• First Posted: October 4, 2022
• Study Start: February 22, 2023
• Primary Completion: January 9, 2024
• Study Completion: January 9, 2024
• Last Updated: January 13, 2025
• Results First Posted: January 13, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR

Locations

US (2)