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PNHPhase 1/2NCT02598583

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

A Phase 1/2 study testing ALXN1210 in people with PNH, run by Alexion Pharmaceuticals, Inc. at 8 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
PNH
What is being tested?
ALXN1210
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
13 participants
Where
8 sitesin 2 countries
Study dates
12 Nov 2015to 11 Mar 2021
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or female ≥18 years of age
  • PNH diagnosis confirmed by documented high-sensitivity flow cytometry
  • Documented meningococcal vaccination not more than 3 years prior to dosing
  • 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
  • Willing and able to give written informed consent and comply with the study visit schedule

Who cannot join

  • Treatment with a complement inhibitor at any time
  • Females who were pregnant, breastfeeding or who had a positive pregnancy test at screening or Day 1
  • 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
  • History of allergy to excipients of ALXN1210 or known allergy to Chinese hamster ovary cell proteins
  • Inability to comply with study requirements
  • History of any clinically significant cardiac, hepatic, immunologic, pulmonary, or rheumatoid disease that, in the Investigator's judgment, would preclude participation
  • Other unspecified reasons that, in the opinion of the Investigator or Sponsor, made the participant unsuitable for enrollment

Trial locations and contacts

8 sites in 2 countries.

8 sites in 2 countries

South Korea6 sites

  • Clinical Trial Site

    Seoul, 03080

  • Clinical Trial Site

    Seoul, 03722

  • Clinical Trial Site

    Seoul, 05505

  • Clinical Trial Site

    Seoul, 06351

  • Clinical Trial Site

    Seoul, 06951

  • Clinical Trial Site

    Ulsan, 44033

Other trials in South Korea

Australia2 sites

  • Clinical Trial Site

    Liverpool, New South Wales, 2170

  • Clinical Trial Site

    Woolloongabba, Queensland, 4102

Other trials in Australia

About this study

Official title: 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.

Read the 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.

Conditions
PNH
Being tested
ALXN1210
Keywords
Paroxysmal Nocturnal Hemoglobinuria, PNH, complement inhibitor
Medical subject headings
Hemoglobinuria, Paroxysmal

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ALXN1210Biological

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

Study groups in NCT02598583
Study groupRoleWhat they receive
Cohort 1ExperimentalParticipants were administered ALXN1210 900 mg. In the Extension period participants continued at the same dose and frequency as the Primary Evaluation Period.
Receives: Biological: ALXN1210
Cohort 2ExperimentalParticipants were administered ALXN1210 1800 mg. In the Extension period participants continued at the same dose and frequency as the Primary Evaluation Period.
Receives: Biological: ALXN1210

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percent Change In Lactate Dehydrogenase (LDH) Levels From Baseline To Day 169

    Baseline was defined as the average of all available assessments prior to first ALXN1210 infusion.

    Measured: Baseline, Day 169

Secondary outcomes (18)

  1. Percent Change In Free Hemoglobin Levels From Baseline To Day 169 And Day 1821

    Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.

    Measured: Baseline, Day 169, Day 1821

  2. Percent Change In Haptoglobin Levels From Baseline To Day 169 And Day 1821

    Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.

    Measured: Baseline, Day 169, Day 1821

  3. Percent Change In Reticulocyte/Erythrocyte Count From Baseline To Day 169 And Day 1821

    Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.

    Measured: Baseline, Day 169, Day 1821

  4. Percent Change In Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clones From Baseline To Day 169 And Day 1933

    Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.

    Measured: Baseline, Day 169, Day 1933

  5. Percent Change In D-dimer Levels From Baseline To Day 169 And Day 1821

    Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.

    Measured: Baseline, Day 169, Day 1821

  6. Change In Clinical Manifestations Of PNH From Baseline To Day 169 And 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.

    Measured: Baseline, Day 169, Day 1821

  7. Area Under The Serum Concentration-versus-time-curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) At Day 1

    AUCt reported in hours*microgram/milliliter (h*ug/mL).

    Measured: Day 1

  8. AUCt/ Dose-normalized (D) At Day 1

    Measured: Day 1

  9. Area Under The Serum Concentration-versus-time-curve From Time 0 (Dosing) To The End Of The Dosing Interval (AUCtau) At Day 141

    Measured: Day 141

  10. AUCtau/D At Day 141

    Measured: Day 141

  11. Maximum Observed Serum Concentration (Cmax) At Day 1 And Day 141

    Measured: Day 1 and Day 141

  12. Cmax/D At Day 1 And Day 141

    Measured: Day 1 and Day 141

  13. Concentration At The End Of The Dosage Interval (Ctrough) At Day 1 And At Day 141

    Measured: Day 1 and Day 141

  14. Time To Maximum Observed Serum Concentration (Tmax) At Day 1 And Day 141

    Measured: Day 1 and Day 141

  15. Percent Change In Chicken Red Blood Cell (cRBC) Hemolysis From Baseline To Day 1709

    Measured: Baseline, Day 1709

  16. Percent Change In Free Complement Component 5 (C5) Concentration From Baseline To Day 1709

    Measured: Baseline, Day 1709

  17. Percent Change In Total C5 Concentration From Baseline To Day 1709

    Measured: Baseline, Day 1709

  18. Participants Experiencing Antidrug Antibodies (ADAs)

    Measured: Day 1821

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Nov 2015 (estimated)
  2. Study start12 Nov 2015
  3. Primary completion14 Jul 2016
  4. Study completion11 Mar 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
13 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02598583
Sponsor's protocol ID
ALXN1210-PNH-103
Status last verified
Apr 2022
First submitted
2 Nov 2015
Last updated
16 May 2022
Data monitoring committee
Yes
Individual data sharing
Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Publications

  • Roth A, Rottinghaus ST, Hill A, Bachman ES, Kim JS, Schrezenmeier H, Terriou L, Urbano-Ispizua A, Wells RA, Jang JH, Kulasekararaj AG, Szer J, Aguzzi R, Damokosh AI, Shafner L, Lee JW. Ravulizumab (ALXN1210) in patients with paroxysmal nocturnal hemoglobinuria: results of 2 phase 1b/2 studies. Blood Adv. 2018 Sep 11;2(17):2176-2185. doi: 10.1182/bloodadvances.2018020644.

    PubMed 30171081

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing ALXN1210 in people with PNH, run by Alexion Pharmaceuticals, Inc. at 8 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 16 May 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in South Korea, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 30 Jan 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percent Change In Lactate Dehydrogenase (LDH) Levels From Baseline To Day 169 (time frame: Baseline, Day 169). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02598583, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 May 2022.

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