CSL889 for Sickle Cell Disease During Pain Crisis

Not currently recruiting at 21 trial locations

Overseen By Trial Registration Coordinator

Age: Any Age

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: CSL Behring

Must not be taking: Methadone, Buprenorphine

Disqualifiers: Frequent VOCs, ACS, Stroke, AKI, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 28 days

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called CSL889, administered through an IV, to assist people with sickle cell disease (SCD) during painful episodes known as vaso-occlusive crises (VOC). The trial aims to determine if CSL889 can speed up recovery from these pain crises and assess its safety. Participants will receive either the treatment or a placebo (a substance with no active drug). Individuals with SCD who are currently experiencing a new VOC that requires opioid treatment may be suitable for this trial. As a Phase 2 trial, this research measures how well CSL889 works in an initial, smaller group, offering participants a chance to contribute to important advancements in SCD treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are currently taking methadone or buprenorphine.

Is there any evidence suggesting that CSL889 is likely to be safe for humans?

Research has shown that CSL889 has generally been well-tolerated in past studies, which focused on safety and the treatment's mechanism in the body. Results indicated that most patients did not experience serious side effects with CSL889. Some mild side effects, similar to those from many medications, were noted but were not severe.

The current study is in Phase 2/3, indicating that earlier phases have already assessed safety in humans. These early phases are crucial for identifying any major safety issues. Reaching this phase suggests that CSL889 has demonstrated sufficient safety to allow testing in larger groups.

In summary, initial data suggest CSL889 is safe for many people, with mostly mild side effects. Ongoing research aims to confirm these findings and ensure the treatment's safety for those with sickle cell disease during pain crises. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for sickle cell disease?

Researchers are excited about CSL889 for sickle cell disease because it offers a novel approach during pain crises. Unlike most treatments that focus on managing symptoms like pain and inflammation, CSL889 targets the root cause by potentially stabilizing red blood cells to reduce sickling events. This new mechanism could lead to fewer pain episodes and a better quality of life for patients. Additionally, if CSL889 proves effective, it could offer a faster onset of relief compared to existing therapies, which often require longer periods to show benefits.

What evidence suggests that CSL889 could be an effective treatment for sickle cell disease during a pain crisis?

Research has shown that CSL889, a type of human hemopexin, may help treat sickle cell disease (SCD) during pain crises. Early studies suggest that CSL889 can shorten the duration of these painful episodes, known as vaso-occlusive crises (VOC), which occur when blood flow is blocked. CSL889 works by attaching to and removing free heme, a substance that can cause inflammation and block blood vessels in people with SCD. Initial results indicate that CSL889 is safe and well-tolerated in adults with SCD. This trial will administer different regimens of CSL889 or a placebo to confirm these benefits and assess its effectiveness for SCD patients in everyday situations. ^{1 6 7 8 9}

Who Is on the Research Team?

Study Director

Principal Investigator

CSL Behring

Are You a Good Fit for This Trial?

This trial is for adults and adolescents with Sickle Cell Disease (SCD) who are experiencing a pain crisis known as vaso-occlusive crisis (VOC). Participants must be at least 12 years old, diagnosed with any genotype of SCD, and require treatment with opioids for VOC.

Inclusion Criteria

I have been diagnosed with sickle cell disease.

I am at the hospital for a sudden pain crisis needing strong painkillers.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive CSL889 or placebo intravenously during vaso-occlusive crises

Up to 28 days

Multiple visits for IV administration

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 28 days

End of study visit (EOS)

What Are the Treatments Tested in This Trial?

Interventions

• CSL889

Trial Overview The study tests CSL889, given intravenously to see if it can shorten the time it takes for VOC to resolve in patients. It's compared against a placebo in a two-part study that includes assessing its safety and how the body processes it.

How Is the Trial Designed?

Group I: Part B: CSL889 Experimental Treatment 1 Intervention

Participants in this arm will receive CSL889, as per regimen selected after Part A.

Group II: Part A: CSL889 Regimen A3 Experimental Treatment 1 Intervention

Participants in this arm will receive CSL889 as per regimen A3.

Group III: Part A: CSL889 Regimen A2 Experimental Treatment 1 Intervention

Participants in this arm will receive CSL889 as per regimen A2.

Group IV: Part A: CSL889 Regimen A1 Experimental Treatment 1 Intervention

Participants in this arm will receive CSL889 as per regimen A1.

Group V: Part B: Placebo Placebo Group 1 Intervention

Participants in this arm will receive placebo matching the selected CSL889 regimen after Part A.

Group VI: Part A: Placebo Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

CSL Behring

Lead Sponsor

Trials

204

Recruited

1,207,000+

Dr. Paul McKenzie

CSL Behring

Chief Executive Officer since 2023

PhD in Chemical Engineering from Carnegie Mellon University, B.S. in Chemical Engineering from the University of Pennsylvania

Dr. Bill Mezzanotte

CSL Behring

Chief Medical Officer since 2021

MD from Duke University

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06699849

Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults ... The main objectives of the study are to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD, and to evaluate the safety and ...

2. academic.oup.com academic.oup.com/jscd/article/1/Supplement_1/yoae002.003/7686703

Phase 1 Study of the Safety and Pharmacokinetics of CSL889 ... We report results of a phase 1, first-in-human study evaluating the safety, tolerability and pharmacokinetics (PK) of CSL889 in adults with SCD.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11971234/

A review on disease modifying pharmacologic therapies for ... Novel therapeutic strategies for SCD and hydroxyurea being the sole medication that is shown to reduce vaso-occlusive events and mortality for almost 20 years.

4. researchgate.net researchgate.net/publication/381290270_Phase_1_Study_of_the_Safety_and_Pharmacokinetics_of_CSL889_Hemopexin_in_Adults_with_SCD

Phase 1 Study of the Safety and Pharmacokinetics ... Seventy treatment emergent adverse events (TEAEs) were reported in 22 subjects (51 mild, 18 moderate and 1 severe). Number or severity of TEAEs ...

5. trials-api.montefioreeinstein.org trials-api.montefioreeinstein.org/node/413661

Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults ... It is a multicenter study designed to evaluate the safety, effectiveness, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10112651/

5610845 A PHASE 1 STUDY OF CSL888 (HEMOPEXIN ... We describe the design of a Phase 1, first in human, multicenter, open-label study to evaluate the safety, tolerability, and pharmacokinetics (PK) of CSL889 in ...

7. academic.oup.com academic.oup.com/jscd/article/2/Supplement_1/yoaf013.015/8156970

Design of the RHEMEDY Study: A Phase 2/3 Safety, Efficacy ... Design of the RHEMEDY Study: A Phase 2/3 Safety, Efficacy and Pharmacokinetics Study of CSL889 in Adults and Adolescents With Sickle Cell ...

8. ctv.veeva.com ctv.veeva.com/study/safety-efficacy-and-pharmacokinetics-of-csl889-in-adults-and-adolescents-with-sickle-cell-disease

Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults ... It is a multicenter study designed to evaluate the safety, effectiveness, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) ...

9. onderzoekmetmensen.nl onderzoekmetmensen.nl/en/node/52183/pdf

A 2-Part, Phase 1, Multicenter, Single Dose, Open-Label ... The study drug CSL889 is being developed for the treatment of vaso-occlusive crisis (also known as pain crisis) that occurs in patients with ...