Crizanlizumab for Pediatric Sickle Cell Disease

Recruiting in Palo Alto (17 mi)

+73 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Novartis Pharmaceuticals

No Placebo Group

Prior Safety Data

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

The purpose of the Phase 2 CSEG101B2201 study is to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 6 months to \<18 years with a history of VOC with or without HU/HC, receiving crizanlizumab for 2 years. The efficacy and safety of crizanlizumab was already demonstrated in adults with sickle cell disease. The approach is to extrapolate from the PK/pharmacodynamics (PD) already established in the adult population. The study is designed as a Phase II, multicenter, open-label study.

Eligibility Criteria

This trial is for pediatric patients aged 6 months to <18 years with sickle cell disease who've had at least one pain crisis in the past year. They must have been stable on certain medications if used, and have proper organ function. Those with recent blood transfusions, bleeding disorders, or severe illnesses are excluded.

Inclusion Criteria

I have been on the same dose of my medication for at least 6 months and plan to keep it the same during the trial.

I have been diagnosed with Sickle Cell Disease through blood tests.

My kidney and liver functions are within the required range.

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Exclusion Criteria

I have had a stem cell transplant.

I have not received any blood products in the last 30 days.

I have an active Hepatitis B infection.

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Treatment Details

Interventions

Crizanlizumab (Monoclonal Antibodies)

Trial OverviewThe study tests Crizanlizumab's dosing and safety in children with sickle cell disease over two years. It builds on adult trials by adjusting doses based on kids' responses. This open-label Phase II trial does not use a placebo group.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: CrizanlizumabExperimental Treatment1 Intervention

SEG101 (crizanlizumab) administered on Week 1 Day 1, Week3 Day 1 and Day 1 of every 4-week cycle

Crizanlizumab is already approved in United States for the following indications:

🇺🇸 Approved in United States as Adakveo for: