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NCT Number: NCT06699849

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.

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Key information

Conditions

Sickle Cell Disease Vaso-occlusive Crisis Acute Chest Syndrome Acute Kidney Injury Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Kidney Diseases Lung Diseases Male Urogenital Diseases Renal Insufficiency Respiration Disorders Respiratory Tract Diseases Urogenital Diseases Urologic Diseases Vaso-Occlusive Crises

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hacettepe Universitesi, Ankara, Turkey (Türkiye)

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

CSL889

Biological

CSL889 is a solution for infusion to be administered by the IV route.

Other names: Hemopexin, human

Placebo

Drug

Volume and regimen matched to CSL889 will be administered.

Primary outcomes

  1. Number of participants with treatment-emergent adverse events (TEAEs)

    Time frame: Up to Day 28 (End of study [EOS] Visit)

  2. Percentage of participants with TEAEs

    Time frame: Up to Day 28 (EOS Visit)

  3. Number of participants with detectable treatment emergent (TE) anti-CSL889 antibodies

    Time frame: Up to Day 28 (EOS Visit)

  4. Percentage of participants with detectable TE anti-CSL889 antibodies

    Time frame: Up to Day 28 (EOS Visit)

  5. Time to resolution of VOC (time to discontinuation of parenteral opioids)

    Time frame: Up to Day 28 (EOS Visit)

Secondary outcomes

  1. 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.

  2. Length of hospital stay (If hospitalized)

    Time frame: Up to Day 28 (EOS Visit)

  3. 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

  4. Length of acute care stay

    Time frame: Up to Day 28 (EOS Visit)

  5. Total Length of acute care and hospital stay

    Time frame: Up to Day 28 (EOS Visit)

  6. Re-presentation rate to an acute care facility for VOC or ACS after discharge

    Time frame: From discharge up to Day 28

  7. Hospital admission rate for VOC or ACS after discharge

    Time frame: From discharge up to Day 28

  8. 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.

  9. 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.

  10. 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.

  11. 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

  12. 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

  13. 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

  14. Trough concentration (Ctrough) after each dose of CSL889

    Time frame: Up to Day 5

  15. 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

  16. 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

  17. 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

  18. 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.

  19. 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.

  20. 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.

  21. 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Trial Registration Coordinator

CONTACT

[email protected]

+1 610-878-4697

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
Nov 21, 2024
Registry last updated
May 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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