What is the mechanism of action of this treatment?

Common treatments for Sickle Cell Disease (SCD) include hydroxyurea, which increases fetal hemoglobin (Hb F) production, reducing the sickling of red blood cells and vaso-occlusive events. Chronic transfusions are used to lower the proportion of sickle hemoglobin, thereby reducing complications. P-selectin inhibitors like Inclacumab prevent the adhesion of sickle cells to the endothelium, reducing inflammation and vaso-occlusion. These treatments are crucial as they address the underlying pathophysiology of SCD, improving patient outcomes by reducing pain episodes, hospitalizations, and long-term complications.

What side effects are associated with this type of intervention?

Hydroxyurea, a common treatment for Sickle Cell Disease, can cause side effects such as bone marrow suppression, leading to decreased white blood cells and platelets, gastrointestinal disturbances, and potential long-term risks like secondary malignancies. Red blood cell transfusions, another treatment option, carry risks of iron overload, alloimmunization, and transfusion reactions. P-selectin inhibitors, like inclacumab, are being studied for their potential to reduce vaso-occlusive episodes, but their long-term safety profile is still under investigation. Common side effects reported in similar therapies include mild to moderate infusion reactions and potential immunogenicity.

What prior FDA approvals exist?

Hydroxyurea is the most established FDA-approved treatment for Sickle Cell Disease, known for its efficacy in reducing vaso-occlusive pain and other complications. It has the longest track record for improving survival and reducing the need for blood transfusions. While the context does not provide specific information on FDA-approved P-selectin inhibitors like Inclacumab, it does mention the importance of new medications that modify the pathogenesis of SCD-associated vaso-occlusion. Canakinumab, an IL-1beta inhibitor, has also shown promise in reducing hospitalization and vaso-occlusive episodes, although it is not specifically a P-selectin inhibitor.

What other types of research exist?

Promising novel alternatives to Inclacumab for Sickle Cell Disease patients include Crizanlizumab, which is a monoclonal antibody that reduces vaso-occlusive crises by inhibiting P-selectin, and Sevuparin, a heparin-derived polysaccharide that reduces vaso-occlusion by inhibiting the adhesion of sickle red blood cells to endothelial cells. Hydroxyurea remains a well-established option with extensive safety and efficacy data.

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Monoclonal Antibodies

Inclacumab for Sickle Cell Disease

Phase 3

Waitlist Available

Research Sponsored by Global Blood Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or female participant with SCD who participated and received study drug in a GBT-Sponsored inclacumab clinical study

If sexually active, female participants of childbearing potential must consistently use highly effective methods of contraception consistently throughout the study and for at least 165 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 165 days after the last dose of study drug

Must not have

Female participant who is breastfeeding or pregnant

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 1 through week 48

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial aims to check if extended use of inclacumab, a medication given through an infusion, is safe for people with sickle cell disease who have already used it. The medication is given every few months to help manage the disease.

Who is the study for?

This trial is for individuals with Sickle Cell Disease who previously participated in an inclacumab study. They must have finished the prior study within 30 days, not be pregnant or breastfeeding, and agree to use effective contraception. Those with conditions that could affect safety assessments or compliance are excluded.

What is being tested?

The trial is assessing the long-term safety of inclacumab in patients with Sickle Cell Disease. It's an open-label study, meaning everyone knows they're getting inclacumab, and it's given to those who completed a previous inclacumab trial.

What are the potential side effects?

While specific side effects aren't listed here, participants from the original study who had infusion-related reactions to inclacumab are excluded, suggesting such reactions may occur.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have SCD and was part of a GBT inclacumab study.

Select...

I am using or willing to use effective birth control during and for 165 days after the study.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am currently pregnant or breastfeeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 1 through week 48

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 1 through week 48

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 through week 48 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Other study objectives

Plasma pharmacokinetic (PK) of inclacumab as assessed by population PK analysis using nonlinear mixed-effects modeling.

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Inclacumab 30 mg/kgExperimental Treatment1 Intervention

Inclacumab 30 mg/kg administered intravenously (IV)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Inclacumab

2021

Completed Phase 3

~250

0 / 3Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Sickle Cell Disease (SCD) include hydroxyurea, which increases fetal hemoglobin (Hb F) production, reducing the sickling of red blood cells and vaso-occlusive events. Chronic transfusions are used to lower the proportion of sickle hemoglobin, thereby reducing complications. P-selectin inhibitors like Inclacumab prevent the adhesion of sickle cells to the endothelium, reducing inflammation and vaso-occlusion. These treatments are crucial as they address the underlying pathophysiology of SCD, improving patient outcomes by reducing pain episodes, hospitalizations, and long-term complications.

Sickle-cell disease.