Inclacumab for Sickle Cell Disease

Recruiting at 78 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

147 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

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.

What data supports the effectiveness of the drug Inclacumab for treating sickle cell disease?

Inclacumab is a drug that targets P-selectin, a molecule involved in the blockage of blood vessels, which is a problem in sickle cell disease. It was initially studied for other conditions like peripheral arterial disease and coronary artery disease, suggesting it might help reduce the frequency of painful episodes in sickle cell disease by preventing blood vessel blockages. ^{1 2 3 4 5}

Research Team

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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]

References

1. Germany pubmed.ncbi.nlm.nih.gov

A phase 1 study in healthy participants to characterize the safety and pharmacology of inclacumab, a fully human anti-P-selectin antibody, in development for treatment of sickle cell disease. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Analytical comparability demonstrated for an IgG4 molecule, inclacumab, following transfer of manufacturing responsibility from Roche to Global Blood Therapeutics. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Dose-escalation study of ICA-17043 in patients with sickle cell disease. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Drug Therapies for the Management of Sickle Cell Disease. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Survival among children and adults with sickle cell disease in Belgium: Benefit from hydroxyurea treatment. [2022]