Crizanlizumab for Pediatric Sickle Cell Disease

Recruiting at 73 trial locations

Overseen By Novartis Pharmaceuticals

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Novartis Pharmaceuticals

Must be taking: HU/HC, L-glutamine

Must not be taking: Monoclonal antibodies, Voxelotor

Disqualifiers: Stem cell transplant, Bleeding disorders, HIV, Hepatitis B, others

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

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 107

Duration: 2 years

118 Participants Needed

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.

Research Team

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

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.

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.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive crizanlizumab with or without hydroxyurea/hydroxycarbamide for 2 years

2 years

Visits on Week 1 Day 1, Week 3 Day 1, and Day 1 of every 4-week cycle

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months

Post-treatment follow-up (last infusion +105 days)

Treatment Details

Interventions

• Crizanlizumab

Trial Overview The 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

Group I: Crizanlizumab Experimental Treatment 1 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:

• Prevention of recurrent vaso-occlusive crises in sickle cell disease patients aged 16 years and older

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD