Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-11-12

Study Completion Date

2021-03-11

Brief Summary

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This study evaluated the safety, tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of multiple intravenous (IV) doses of ALXN1210 administered to participants with PNH who have not previously been treated with complement inhibitor.

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Detailed Description

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The data presented is up to the Primary Completion date of the study and is for the 24-week Primary Evaluation period. The study also includes an Extension Period of up to 5 years.

Conditions

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PNH

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

Participants were administered ALXN1210 900 mg.

In the Extension period participants continued at the same dose and frequency as the Primary Evaluation Period.

Group Type EXPERIMENTAL

ALXN1210

Intervention Type BIOLOGICAL

Participants were administered ravulizumab as an IV infusion every 4 weeks.

Cohort 2

Participants were administered ALXN1210 1800 mg.

In the Extension period participants continued at the same dose and frequency as the Primary Evaluation Period.

Group Type EXPERIMENTAL

ALXN1210

Intervention Type BIOLOGICAL

Participants were administered ravulizumab as an IV infusion every 4 weeks.

Interventions

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ALXN1210

Participants were administered ravulizumab as an IV infusion every 4 weeks.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

1. Male or female ≥18 years of age
2. PNH diagnosis confirmed by documented high-sensitivity flow cytometry
3. Documented meningococcal vaccination not more than 3 years prior to dosing
4. Female participants of childbearing potential used highly effective contraception starting at screening and continuing until at least 24-weeks after the last dose of ALXN1210
5. Willing and able to give written informed consent and comply with the study visit schedule

Exclusion Criteria

1. Treatment with a complement inhibitor at any time
2. Females who were pregnant, breastfeeding or who had a positive pregnancy test at screening or Day 1
3. Participation in an interventional clinical study within 30 days before initiation of dosing on Day 1, or use of any experimental therapy within 30 days prior to dosing on Day 1, or within 5 half-lives of the product, whichever is greater
4. History of allergy to excipients of ALXN1210 or known allergy to Chinese hamster ovary cell proteins
5. Inability to comply with study requirements
6. History of any clinically significant cardiac, hepatic, immunologic, pulmonary, or rheumatoid disease that, in the Investigator's judgment, would preclude participation
7. Other unspecified reasons that, in the opinion of the Investigator or Sponsor, made the participant unsuitable for enrollment

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No