Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis

August 13, 2026 updated by: CSL Behring

A Phase 2, Multicenter, Randomized, Multiple-Dose, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis

This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to evaluate the safety and tolerability of CSL889 in study participants, and to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD.

Study Overview

Status

Status

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

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.

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 (Estimated)

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.
70

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

Contacts and Locations

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

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Turkey (Türkiye)
      • Adana, Turkey (Türkiye), 01120
        • Recruiting
        • Baskent University School of Medicine
      • Ankara, Turkey (Türkiye), 06230
        • Not yet recruiting
        • Hacettepe Universitesi
        • Contact:
          • Central Contact
      • Istanbul, Turkey (Türkiye), 34093
        • Not yet recruiting
        • Istanbul Universitesi
        • Contact:
          • Central Contact
      • Mezitli-Mersin, Turkey (Türkiye), 33200
        • Recruiting
        • Medical Park Mersin Hospital
      • Seyhan, Turkey (Türkiye), 01130
        • Not yet recruiting
        • Özel Acibadem Adana Hastanesi
        • Contact:
          • Central Contact
      • Seyhan/Adana, Turkey (Türkiye), 01130
        • Recruiting
        • Özel Acibadem Adana Hastanesi
  • United Kingdom
      • London, United Kingdom, SE1 9RT
        • Recruiting
        • Guy's Hospital
  • United States
    • California
      • Oakland, California, United States, 94609
        • Recruiting
        • Univ. of California, San Francisco Health Care
      • Orange, California, United States, 92868
        • Not yet recruiting
        • University of California Irvine
        • Contact:
          • Use Central Contact
    • Florida
      • Fort Myers, Florida, United States, 33908
        • Recruiting
        • Golisano Children's Hospital
        • Contact:
          • Use Central Contact
      • Hollywood, Florida, United States, 33023-6703
        • Recruiting
        • The Foundation for Sickle Cell Disease
        • Contact:
          • Use Central Contact
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Recruiting
        • Arthur M. Blank Hospital-Children's Healthcare of Atlanta
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Recruiting
        • University of Illinois at Chicago
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • Recruiting
        • University of Louisville Hospital
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • Recruiting
        • University of Maryland
        • Contact:
          • Use Central Contact
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Recruiting
        • Henry Ford Health System
        • Contact:
          • Use Central Contact
      • Detroit, Michigan, United States, 48201
        • Recruiting
        • Detroit Medical Center
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Recruiting
        • St. Louis Children's Hospital
    • New York
      • New York, New York, United States, 10029
        • Recruiting
        • Mount Sinai Medical Center
        • Contact:
          • Use Central Contact
      • The Bronx, New York, United States, 10461
        • Recruiting
        • Jacobi Medical Center
        • Contact:
          • Central Contact
    • North Carolina
      • Greenville, North Carolina, United States, 27834
        • Recruiting
        • East Carolina University
    • Ohio
      • Cincinnati, Ohio, United States, 45267
        • Recruiting
        • University of Cincinnati
        • Contact:
          • Use Central Contact
      • Columbus, Ohio, United States, 43085
        • Recruiting
        • The Ohio State University
        • Contact:
          • Use Central Contact
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15232
        • Recruiting
        • Hillman Cancer Center
      • Pittsburgh, Pennsylvania, United States, 15224
        • Recruiting
        • University of Pittsburgh
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Recruiting
        • Virginia Commonwealth University

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

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • At the time of informed consent:

    • 18 years of age (adults); or
    • 12 to less than (<) 18 years of age (adolescents, where approved and when enrollment for adolescents has been opened by the sponsor, with the endorsement of the Independent Data Monitoring Committee [IDMC])
  • Diagnosed with SCD (any genotype).
  • Presented at the study site with a new acute VOC necessitating treatment with parenteral opioids.

Exclusion Criteria:

  • VOC pain onset greater than (>) 72 hours before administration of first parenteral opioid.
  • Must not have a history of > 5 VOCs requiring hospital admission in the past 6 months; or signs and / or symptoms of ACS; or new neurological symptoms suggestive of acute stroke or transient ischemic attack; or any stage (acute kidney injury) AKI; or been discharged from inpatient hospital admission for VOC or other vaso-occlusive event within 14 days before the current presentation.
  • Serum hemoglobin < 6 g/dL, serum ferritin ≥ 2000 ng/mL, receiving an approved medication for SCD that has not been on a stable, well-tolerated regimen, currently taking methadone or buprenorphine.

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: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

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: CSL889 Regimen 1
Participants in this arm will receive CSL889 as per regimen 1.
Biological: CSL889
CSL889 is a solution for infusion to be administered by the IV route.
Other Names:
  • Hemopexin, human
Experimental: CSL889 Regimen 2
Participants in this arm will receive CSL889 as per regimen 2.
Biological: CSL889
CSL889 is a solution for infusion to be administered by the IV route.
Other Names:
  • Hemopexin, human
Placebo Comparator: Placebo
Participants in this arm will receive placebo matching to CSL889 regimen.
Drug: Placebo
Volume and regimen matched to CSL889 will be administered.

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
Time Frame
Number of participants with treatment-emergent adverse events (TEAEs)
Time Frame: Up to Day 28 (End of study [EOS] Visit)
Up to Day 28 (End of study [EOS] Visit)
Percentage of participants with TEAEs
Time Frame: Up to Day 28 (EOS Visit)
Up to Day 28 (EOS Visit)
Number of participants with detectable treatment emergent (TE) anti-CSL889 antibodies
Time Frame: Up to Day 28 (EOS Visit)
Up to Day 28 (EOS Visit)
Percentage of participants with detectable TE anti-CSL889 antibodies
Time Frame: Up to Day 28 (EOS Visit)
Up to Day 28 (EOS Visit)
Time to resolution of VOC (time to discontinuation of parenteral opioids)
Time Frame: Up to Day 28 (EOS Visit)
Up to Day 28 (EOS Visit)

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
Length of hospital stay (If hospitalized)
Time Frame: Up to Day 28 (EOS Visit)
Up to Day 28 (EOS Visit)
Percentage of participants experiencing acute chest syndrome (ACS), acute kidney injury (AKI), or stroke
Time Frame: From the start of investigational product (IP) administration up to Day 8
From the start of investigational product (IP) administration up to Day 8
Length of acute care stay
Time Frame: Up to Day 28 (EOS Visit)
Up to Day 28 (EOS Visit)
Total Length of acute care and hospital stay
Time Frame: Up to Day 28 (EOS Visit)
Up to Day 28 (EOS Visit)
Opioid consumption
Time Frame: From the time of enrollment to discharge (up to Day 28 [EOS Visit])
Opioid consumption will be measured in morphine milligram equivalent units.
From the time of enrollment to discharge (up to Day 28 [EOS Visit])
Maximum observed concentration (Cmax) after Doses 1 and 3 of CSL889
Time Frame: Before dosing, and up to 12 hours after Doses 1 and 3
Before dosing, and up to 12 hours after Doses 1 and 3
Area under the concentration (AUCtau) after Doses 1 and 3 of CSL889
Time Frame: Before dosing, and up to 12 hours after Doses 1 and 3
Before dosing, and up to 12 hours after Doses 1 and 3
Time of maximum concentration (Tmax) after Doses 1 and 3 of CSL889
Time Frame: Before dosing, and up to 12 hours after Doses 1 and 3
Before dosing, and up to 12 hours after Doses 1 and 3
Trough concentration (Ctrough) after each dose of CSL889
Time Frame: Up to Day 5
Up to Day 5
Accumulation ratio (AR) for AUCtau of CSL889 (the ratio between the AUCtau of Doses 3 and 1)
Time Frame: Before dosing and at up to 12 hours after Doses 1 and 3
Before dosing and at up to 12 hours after Doses 1 and 3
AR for Cmax of CSL889 (the ratio between the Cmax of Doses 3 and 1)
Time Frame: Before dosing and at up to 12 hours after Doses 1 and 3
Before dosing and at up to 12 hours after Doses 1 and 3
AR for Ctrough of CSL889 (the ratio between the Ctrough of the last dose and Dose 1)
Time Frame: Before dosing and after Dose 1 and the last dose
Before dosing and after Dose 1 and the last dose
Hospital admission rate
Time Frame: Up to Day 28 (EOS Visit)
Hospital admission rate in participants with SCD presenting with VOC who received greater than or equal to (≥) 1 dose of CSL889 in the acute care setting.
Up to Day 28 (EOS Visit)
Re-presentation rate to an acute care facility for VOC or ACS after discharge
Time Frame: From discharge up to Day 28
From discharge up to Day 28
Hospital admission rate for VOC or ACS after discharge
Time Frame: From discharge up to Day 28
From discharge up to Day 28
Number of participants with ≥ 30% pain reduction by Numeric Rating Scale (NRS) score
Time Frame: Within 4 hours after the start of CSL889 infusion
The NRS for pain is a validated self-reported 11-point pain severity scale (where 0 means no pain and 10 means the most or worst possible pain) that can be used in adults, adolescents, and children ≥ 8 years of age with SCD.
Within 4 hours after the start of CSL889 infusion
Percentage of participants with ≥ 30% pain reduction by NRS score
Time Frame: Within 4 hours after the start of CSL889 infusion
The NRS for pain is a validated self-reported 11-point pain severity scale (where 0 means no pain and 10 means the most or worst possible pain) that can be used in adults, adolescents, and children ≥ 8 years of age with SCD.
Within 4 hours after the start of CSL889 infusion
Cmax after each dose in Sparse PK subset of CSL889
Time Frame: Up to Day 5
Sparse PK blood sampling will be performed in remainder of participants who are not in the Adult or Adolescent PK Subsets.
Up to Day 5
Ctrough after each dose in Sparse PK subset of CSL889
Time Frame: Up to Day 5
Sparse PK blood sampling will be performed in remainder of participants who are not in the Adult or Adolescent PK Subsets.
Up to Day 5
AR for Cmax in Sparse PK subset of CSL889
Time Frame: Up to Day 5
Sparse PK blood sampling will be performed in remainder of participants who are not in the Adult or Adolescent PK Subsets.
Up to Day 5
AR for Ctrough in Sparse PK subset of CSL889
Time Frame: Up to Day 5
Sparse PK blood sampling will be performed in remainder of participants who are not in the Adult or Adolescent PK Subsets.
Up to Day 5

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.
CSL Behring

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: Study Director, CSL Behring

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.
August 7, 2025

Primary Completion (Estimated)

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.
October 22, 2027

Study Completion (Estimated)

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.
October 22, 2027

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 19, 2024

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 19, 2024

First Posted (Actual)

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 21, 2024

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.
August 14, 2026

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.
August 13, 2026

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.
August 1, 2026

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.
  • CSL889_2001
  • 2024-513440-29-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.

IPD Sharing Time Frame

Requests for IPD will generally be considered once review by major regulatory authorities (i.e. FDA, EMA) is complete and the primary publication is available

IPD Sharing Access Criteria

Proposed research should seek to answer a previously unanswered important medical or scientific question.

Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.

If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

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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