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

NCT06699849 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: CSL889_20012024-513440-29-00
• Study Type: interventional
• Target: 70
• Locations: 2 countries
• Sites: 17

Brief Summary

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.

Conditions

Sickle Cell Disease Vaso-occlusive Crisis

Keywords

Sickle cell disease; Acute kidney injury; Pharmacokinetics; Acute chest syndrome; Vaso-occlusive crisis; Hemopexin

Outcome Measures

Primary Outcomes (5)

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

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

• Percentage of participants with TEAEs

  Up to Day 28 (EOS Visit)

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

  Up to Day 28 (EOS Visit)

• Percentage of participants with detectable TE anti-CSL889 antibodies

  Up to Day 28 (EOS Visit)

• Time to resolution of VOC (time to discontinuation of parenteral opioids)

  Up to Day 28 (EOS Visit)

Secondary Outcomes (21)

• Hospital admission rate

  Up to Day 28 (EOS Visit)

• Length of hospital stay (If hospitalized)

  Up to Day 28 (EOS Visit)

• Percentage of participants experiencing acute chest syndrome (ACS), acute kidney injury (AKI), or stroke

  From the start of investigational product (IP) administration up to Day 8

• Length of acute care stay

  Up to Day 28 (EOS Visit)

• Total Length of acute care and hospital stay

  Up to Day 28 (EOS Visit)

Study Arms (3)

CSL889 Regimen 1

EXPERIMENTAL

Participants in this arm will receive CSL889 as per regimen 1.

Biological: CSL889

CSL889 Regimen 2

EXPERIMENTAL

Participants in this arm will receive CSL889 as per regimen 2.

Biological: CSL889

Placebo

PLACEBO COMPARATOR

Participants in this arm will receive placebo matching to CSL889 regimen.

Drug: Placebo

Interventions

CSL889 BIOLOGICAL

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

Also known as: Hemopexin, human

CSL889 Regimen 1; CSL889 Regimen 2

Placebo DRUG

Volume and regimen matched to CSL889 will be administered.

Placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• At the time of informed consent:
• years of age (adults); or
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• CSL Behring: lead

Study Sites (17)

Univ. of California, San Francisco Health Care

Oakland, California, 94609, United States

RECRUITING

University of California Irvine

Orange, California, 92868, United States

NOT YET RECRUITING

Golisano Children's Hospital

Fort Myers, Florida, 33908, United States

RECRUITING

The Foundation for Sickle Cell Disease

Hollywood, Florida, 33023-6703, United States

RECRUITING

University of Maryland

Baltimore, Maryland, 21201, United States

RECRUITING

Detroit Medical Center

Detroit, Michigan, 48201, United States

RECRUITING

Henry Ford Health System

Detroit, Michigan, 48202, United States

RECRUITING

Mount Sinai Medical Center

New York, New York, 10029, United States

RECRUITING

Jacobi Medical Center

The Bronx, New York, 10461, United States

RECRUITING

University of Cincinnati

Cincinnati, Ohio, 45267, United States

RECRUITING

The Ohio State University

Columbus, Ohio, 43085, United States

RECRUITING

University of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

RECRUITING

Hillman Cancer Center

Pittsburgh, Pennsylvania, 15232, United States

RECRUITING

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

RECRUITING

Hacettepe Universitesi

Ankara, 06230, Turkey (Türkiye)

NOT YET RECRUITING

Istanbul Universitesi

Istanbul, 34093, Turkey (Türkiye)

NOT YET RECRUITING

Özel Acibadem Adana Hastanesi

Seyhan, 01130, Turkey (Türkiye)

NOT YET RECRUITING

MeSH Terms

Conditions

Anemia, Sickle Cell; Acute Kidney Injury; Acute Chest Syndrome; Vaso-Occlusive Crises

Interventions

Hemopexin

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Lung Diseases; Respiratory Tract Diseases; Respiration Disorders

Intervention Hierarchy (Ancestors)

Glycoproteins; Glycoconjugates; Carbohydrates; Acute-Phase Proteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Beta-Globulins; Serum Globulins; Globulins

Study Officials

• Study Director

  CSL Behring

  STUDY DIRECTOR

Central Study Contacts

Trial Registration Coordinator

CONTACT

+1 610-878-4697; clinicaltrials@cslbehring.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 19, 2024
• First Posted: November 21, 2024
• Study Start: August 7, 2025
• Primary Completion (Estimated): October 22, 2027
• Study Completion (Estimated): October 22, 2027
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• 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
• 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.

Locations

US (14); TU (3)