Inclacumab for Sickle Cell Disease

Not currently recruiting at 86 trial locations

Overseen By Pfizer CT.gov Call Center

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Global Blood Therapeutics

Must be taking: Inclacumab

Disqualifiers: Pregnancy, Breastfeeding, IRR, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: 4

Duration: 48 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the long-term safety of inclacumab in individuals with sickle cell disease (SCD), a condition that alters the shape of red blood cells and causes pain and other health issues. The study is open-label, so both doctors and participants know the treatment being administered. Participants who previously joined an inclacumab study and continued the medication without major issues are ideal candidates for this trial. As a Phase 3 trial, it represents the final step before FDA approval, allowing participants to contribute to the potential availability of a new treatment for SCD.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

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

Research has shown that inclacumab has undergone safety testing in several studies. One study focused on individuals with sickle cell disease and found that inclacumab was generally well-tolerated, with no unexpected safety issues.

Another study tested different doses of inclacumab in healthy volunteers. Even at higher doses, the treatment was tolerated without serious side effects, boosting confidence in its safety for potential trial participants.

Inclacumab is currently in Phase 3 trials, indicating that earlier studies have already assessed its safety in smaller groups. While inclacumab appears safe so far, monitoring for any side effects remains important. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for sickle cell disease?

Unlike the standard treatments for sickle cell disease, which often focus on managing symptoms or preventing complications, inclacumab works by targeting P-selectin. This is a protein that plays a crucial role in the painful episodes known as vaso-occlusive crises that are common in sickle cell patients. By blocking P-selectin, inclacumab may reduce the frequency and severity of these crises, offering a new mechanism of action that could improve the quality of life for patients. Researchers are excited because this targeted approach has the potential to address the underlying causes of these painful episodes more effectively than current options.

What evidence suggests that inclacumab might be an effective treatment for sickle cell disease?

Research shows that inclacumab is under study for its potential to reduce painful episodes called vaso-occlusive crises (VOCs) in people with sickle cell disease. These crises occur when blood flow is blocked. Inclacumab blocks a protein called P-selectin, which contributes to these blockages. Some studies found it did not significantly reduce the number of VOCs compared to a placebo, but it effectively prevents cells from sticking together, helping to avoid these crises. Overall, inclacumab appears promising, but further research is needed to confirm its effectiveness. ^{2 3 4 6 7}

Who Is on the Research Team?

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

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

I am a woman who can have children and have a negative pregnancy test.

Participant has provided written informed consent/assent. For underage participants, both the consent of the participant's legal representative or legal guardian and the participant's assent (where applicable) must be obtained based on local requirement

Exclusion Criteria

Participant has any medical, psychological, safety, or behavioral conditions that, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent

Participant withdrew consent from the originating inclacumab clinical study

Participant was lost to follow-up from the originating inclacumab clinical study

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive inclacumab 30 mg/kg administered intravenously every 12 weeks

48 weeks

4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive inclacumab for long-term safety evaluation

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Inclacumab

Trial Overview 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.

How Is the Trial Designed?

Group I: Inclacumab 30 mg/kg Experimental Treatment 1 Intervention

Inclacumab 30 mg/kg administered intravenously (IV)

Who Is Running the Clinical Trial?

Global Blood Therapeutics

Lead Sponsor

Trials

36

Recruited

3,200+

Pfizer

Lead Sponsor

Trials

4,712

Recruited

50,980,000+

Known For

Vaccine Innovations

Top Products

Viagra, Zoloft, Lipitor, Prevnar 13

Albert Bourla

Pfizer

Chief Executive Officer since 2019

PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki

Patrizia Cavazzoni

Pfizer

Chief Medical Officer

MD from McGill University

Published Research Related to This Trial

Sickle cell disease (SCD) affects millions globally, with a significantly shorter life expectancy for patients compared to the general population, particularly in resource-rich countries where treatment options are limited.

Recent approvals of multiple drugs for SCD, including hydroxyurea, L-glutamine, crizanlizumab, and voxelotor, offer new treatment possibilities, but raise important questions about drug choice, combination therapies, and affordability, especially for patients in low- and middle-income countries.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drug Therapies for the Management of Sickle Cell Disease. Rai, P., Ataga, KI.[2023]

A Phase I study involving 28 patients with sickle cell disease found that ICA-17043 was well tolerated with no dose-limiting adverse events, indicating a good safety profile for this medication.

The pharmacokinetics showed that the total systemic exposure to ICA-17043 increased with higher doses, and the drug has a long half-life of 12.8 days, suggesting that once-daily dosing could effectively maintain therapeutic levels.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dose-escalation study of ICA-17043 in patients with sickle cell disease. Ataga, KI., Orringer, EP., Styles, L., et al.[2022]

Citations

1. pfizer.com pfizer.com/news/announcements/pfizer-provides-update-phase-3-inclacumab-study-treatment-people-sickle-cell

Pfizer Provides Update on Phase 3 Inclacumab Study for ... Inclacumab was generally well tolerated in THRIVE-131. The most commonly reported treatment-emergent adverse events in either group were anemia, ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04935879

NCT04935879 | A Study to Assess the Safety and Efficacy ... This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs)

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10112492/

5572697 TRIALS IN PROGRESS - PubMed Central - NIH The THRIVE-131 and THRIVE-132 phase 3 studies will examine the efficacy of inclacumab in reducing the frequency of VOCs and readmissions due to VOCs.

4. empr.com empr.com/news/inclacumab-misses-endpoint-in-sickle-cell-disease-trial/

Inclacumab Misses Endpoint in Sickle Cell Disease Trial Findings showed inclacumab did not statistically significantly reduce the rate of VOCs compared with placebo after 48 weeks. Topline data were ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S3050598425000253

crizanlizumab and inclacumab are equally potent inhibitors ... crizanlizumab and inclacumab are equally potent inhibitors of cell adhesion in the blood of patients with sickle cell disease. - ScienceDirect.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10427511/

A phase 1 study in healthy participants to characterize the ... A study to assess the safety and efficacy of inclacumab in participants with sickle cell disease experiencing vaso-occlusive crises.

7. ashpublications.org ashpublications.org/blood/article/138/Supplement%201/977/480913/Preliminary-Results-of-a-Phase-1-Study-in-Healthy

Preliminary Results of a Phase 1 Study in Healthy Subjects ... The current Phase 1 study was initiated to evaluate the safety and pharmacology of inclacumab at doses of 20 mg/kg and 40 mg/kg in healthy ...