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

April 22, 2022 updated by: Alexion Pharmaceuticals

An Open-Label, Intrapatient, Dose-Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
13

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Liverpool, New South Wales, Australia, 2170
        • Clinical Trial Site
    • Queensland
      • Woolloongabba, Queensland, Australia, 4102
        • Clinical Trial Site
  • Korea, Republic of
      • Seoul, Korea, Republic of, 03080
        • Clinical Trial Site
      • Seoul, Korea, Republic of, 03722
        • Clinical Trial Site
      • Seoul, Korea, Republic of, 05505
        • Clinical Trial Site
      • Seoul, Korea, Republic of, 06351
        • Clinical Trial Site
      • Seoul, Korea, Republic of, 06951
        • Clinical Trial Site
      • Ulsan, Korea, Republic of, 44033
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
EXPERIMENTAL: 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.
Biological: ALXN1210
Participants were administered ravulizumab as an IV infusion every 4 weeks.
EXPERIMENTAL: 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.
Biological: ALXN1210
Participants were administered ravulizumab as an IV infusion every 4 weeks.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Percent Change In Lactate Dehydrogenase (LDH) Levels From Baseline To Day 169
Time Frame: Baseline, Day 169
Baseline was defined as the average of all available assessments prior to first ALXN1210 infusion.
Baseline, Day 169

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percent Change In Free Hemoglobin Levels From Baseline To Day 169 And Day 1821
Time Frame: Baseline, Day 169, Day 1821
Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
Baseline, Day 169, Day 1821
Percent Change In Haptoglobin Levels From Baseline To Day 169 And Day 1821
Time Frame: Baseline, Day 169, Day 1821
Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
Baseline, Day 169, Day 1821
Percent Change In Reticulocyte/Erythrocyte Count From Baseline To Day 169 And Day 1821
Time Frame: Baseline, Day 169, Day 1821
Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
Baseline, Day 169, Day 1821
Percent Change In Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clones From Baseline To Day 169 And Day 1933
Time Frame: Baseline, Day 169, Day 1933
Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
Baseline, Day 169, Day 1933
Percent Change In D-dimer Levels From Baseline To Day 169 And Day 1821
Time Frame: Baseline, Day 169, Day 1821
Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
Baseline, Day 169, Day 1821
Change In Clinical Manifestations Of PNH From Baseline To Day 169 And Day 1821
Time Frame: Baseline, Day 169, Day 1821
Clinical manifestations are defined as fatigue, abdominal pain, dyspnea, dysphagia, chest pain, and erectile dysfunction (ED) by cohort. Improvement is defined as present at baseline and absent at Day 169 endpoint. Worsening is defined as absent at Baseline and present at Day 169 endpoint.
Baseline, Day 169, Day 1821
Area Under The Serum Concentration-versus-time-curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) At Day 1
Time Frame: Day 1
AUCt reported in hours*microgram/milliliter (h*ug/mL).
Day 1
AUCt/ Dose-normalized (D) At Day 1
Time Frame: Day 1
Day 1
Area Under The Serum Concentration-versus-time-curve From Time 0 (Dosing) To The End Of The Dosing Interval (AUCtau) At Day 141
Time Frame: Day 141
Day 141
AUCtau/D At Day 141
Time Frame: Day 141
Day 141
Maximum Observed Serum Concentration (Cmax) At Day 1 And Day 141
Time Frame: Day 1 and Day 141
Day 1 and Day 141
Cmax/D At Day 1 And Day 141
Time Frame: Day 1 and Day 141
Day 1 and Day 141
Concentration At The End Of The Dosage Interval (Ctrough) At Day 1 And At Day 141
Time Frame: Day 1 and Day 141
Day 1 and Day 141
Time To Maximum Observed Serum Concentration (Tmax) At Day 1 And Day 141
Time Frame: Day 1 and Day 141
Day 1 and Day 141
Percent Change In Chicken Red Blood Cell (cRBC) Hemolysis From Baseline To Day 1709
Time Frame: Baseline, Day 1709
Baseline, Day 1709
Percent Change In Free Complement Component 5 (C5) Concentration From Baseline To Day 1709
Time Frame: Baseline, Day 1709
Baseline, Day 1709
Percent Change In Total C5 Concentration From Baseline To Day 1709
Time Frame: Baseline, Day 1709
Baseline, Day 1709
Participants Experiencing Antidrug Antibodies (ADAs)
Time Frame: Day 1821
Day 1821

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alexion Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Alexion Pharmaceuticals, Inc., Alexion Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 12, 2015

Primary Completion (ACTUAL)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 14, 2016

Study Completion (ACTUAL)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 11, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 2, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 4, 2015

First Posted (ESTIMATE)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 6, 2015

Study Record Updates

Last Update Posted (ACTUAL)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 16, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 22, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ALXN1210-PNH-103

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • CSR

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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