Study of ALXN1820 in Healthy Adult Participants

NCT04631562 | Completed Phase 1 Results

• 2 other identifiers: ALXN1820-HV-1012021-002472-39
• Study Type: interventional
• Target: 66
• Locations: 2 countries
• Sites: 3

Brief Summary

This is a Phase 1, randomized, double-blind, placebo-controlled single and multiple ascending dose study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of ALXN1820 administered subcutaneously (SC) (ALXN1820 SC) and intravenously (IV) (ALXN1820 IV).

Conditions

Healthy

Keywords

ALXN1820; Properdin; Pharmacodynamics; Pharmacokinetics

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-related Adverse Events (TEAEs) For ALXN1820 SC And ALXN1820 IV

  An adverse event (AE) was defined as any unfavorable and unintended sign (for example, including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product or procedure, whether or not considered related to the medicinal product or procedure, which occurred during the course of the clinical study. TEAEs were defined as any AEs that commenced after the start of administration of study intervention. Serious AEs (SAEs) were defined as any untoward medical occurrence that met at least 1 of the following serious criteria: resulted in death, was life-threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, other medically important serious event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Reported AEs' Section.

  Cohorts 1 to 6: Baseline up to Day 127; Cohorts 8 to 9: Baseline up to Day 155

Secondary Outcomes (12)

• Area Under The Concentration-time Curve (AUC) From Time 0 (Dosing) To Time Infinity (AUC0-inf) And AUC During The Dosing Interval (AUCtau) of Serum ALXN1820 For Single Dose Cohorts

  Up to 126 days following the first day of dosing

• Area Under The Concentration-time Curve During The Dosing Interval (AUCtau) Of Serum ALXN1820 For Multiple Dose Cohorts

  Up to 154 days following the first day of dosing

• Maximum Observed Serum Concentration (Cmax) Of Serum ALXN1820 For Single Dose Cohorts

  Up to 126 days following the first day of dosing

• Maximum Observed Serum Concentration (Cmax) Of Serum ALXN1820 For Multiple Dose Cohorts

  Up to 154 days following the first day of dosing

• Change From Baseline in Serum Concentrations Of Total Properdin For ALXN1820 SC And ALXN1820 IV-Single Dose Cohorts

  Baseline, Day 127

Study Arms (2)

ALXN1820

EXPERIMENTAL

Participants will receive ALXN1820 SC or ALXN1820 IV according to their assigned cohort. ALXN1820 SC will be evaluated in single and multiple ascending doses while ALXN1820 IV will be evaluated in a single dose cohort only.

Drug: ALXN1820 SC; Drug: ALXN1820 IV

Placebo

PLACEBO COMPARATOR

Participants will receive Placebo SC or Placebo IV according to their assigned cohort.

Drug: Placebo SC; Drug: Placebo IV

Interventions

ALXN1820 SC DRUG

ALXN1820 SC will be administered as a manual SC push or SC infusion via a syringe pump. Doses will range from 12.5 milligrams (mg) to a maximum of 2250 mg. Multiple dosing duration will range from 3 to 5 weeks.

ALXN1820

ALXN1820 IV DRUG

ALXN1820 IV (450 mg) will be administered as an IV infusion.

ALXN1820

Placebo SC DRUG

Placebo SC will be administered as a manual SC push or SC infusion via a syringe pump.

Placebo

Placebo IV DRUG

Placebo IV will be administered as an IV infusion.

Placebo

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Body weight 50 to 100 kilograms (kg); body mass index 17 to 32 kg/meter squared.
• Cohort 9 only: Japanese participants (defined as those participants whose parents and grandparents are both Japanese and who have spent less than 5 years outside of Japan).
• Satisfactory medical assessment.
• Must follow protocol-specified contraception guidance while on treatment and for up to 6 months after last dose.
• Vaccination requirement:
• Vaccination with tetravalent meningococcal conjugate vaccine at least 56 days and not more than 2 years, 6 months prior to dosing;
• Vaccination with serogroup B meningococcal vaccine at least 56 days prior to dosing, with a booster at least 28 days prior to dosing, with at least 28 days between the first and second injections.

You may not qualify if:

• Current/recurrent diseases or relevant medical history.
• History of any Neisseria infection.
• Hepatitis B/C, human immunodeficiency virus.
• History of latent or active tuberculosis (TB), or positive TB test.
• Active systemic infection within 14 days of dosing.
• Risk of meningococcal infections due to living/working conditions.
• History of complement deficiency or complement activity below the reference range.
• Participation 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.
• Acquired complement deficiencies (for example, those receiving eculizumab).

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead
• Syneos Health: collaborator

Study Sites (3)

Research Site

Herston, 4006, Australia

Location

Research Site

London, SE1 1YR, United Kingdom

Location

Research Site

London, SE5 9RS, United Kingdom

Location

Related Publications (1)

• Sandhu A, Shen T, Herrero PM, Yuan CX, Qureshi S, Jiang X, Sheng Y, Gasteyger C, Dai Y. Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of ALXN1820 (Tarperprumig) in Healthy Adults: Results of a Phase I Study. Clin Transl Sci. 2025 Apr;18(4):e70190. doi: 10.1111/cts.70190.

  PMID: 40134111 DERIVED

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 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: BASIC SCIENCE
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 13, 2020
• First Posted: November 17, 2020
• Study Start: January 13, 2021
• Primary Completion: September 1, 2022
• Study Completion: September 1, 2022
• Last Updated: August 20, 2024
• Results First Posted: March 15, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

GB (2); AU (1)