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Monoclonal Antibodies

Crizanlizumab for Pediatric Sickle Cell Disease

Phase 2

Waitlist Available

Research Sponsored by Novartis Pharmaceuticals

Eligibility Criteria Checklist

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

Must have

Received standard age-appropriate care for SCD, including penicillin prophylaxis, pneumococcal immunization, and parental education

Female of non-childbearing potential or with negative serum pregnancy test on Screening and a negative urine pregnancy test (dipstick) prior to dosing on Day 1

Must not have

History of stem cell transplant

Received any blood products within 30 days prior to Week 1 Day 1 dosing

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 months, 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is studying whether a drug already shown to be safe and effective in adults with sickle cell disease is also safe and effective in children 6 months to 17 years old.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects for this age group aren't listed, adults taking Crizanlizumab may experience joint pain, backache, fever, and stomach ache. Children might face similar issues; monitoring during the trial will clarify these effects.

Eligibility Criteria

Inclusion Criteria

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

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I have received all recommended treatments and vaccines for sickle cell disease.

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I am not able to have children or have tested negative for pregnancy.

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My kidney and liver functions are within the required range.

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I can do most activities but may need help, regardless of my age.

Exclusion Criteria

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

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I have had a stem cell transplant.

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

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I have an active Hepatitis B infection.

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I have had serious heart issues or abnormal heart rhythms recently.

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I have a bleeding disorder.

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I am not allergic to the study drug or similar medications.

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I have not had antibody or immunoglobulin therapy in the last 6 months, nor have I had a bad reaction to such treatments before.

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I was in the hospital within the last week before starting the treatment.

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I plan to have a major surgery during the study period.

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I have not taken voxelotor in the last 30 days and do not plan to take it during the study.

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I am not currently institutionalized by court or administrative order.

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I have not taken crizanlizumab or similar medications.

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I have a history of Hepatitis C.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months, 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months, 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months, 2 years for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Frequency of any adverse events (AEs) as a measure of safety and tolerability

PD (AUCd15) after 1st dose

PD (AUCtau) after multiple dose

+4 more

Secondary study objectives

Absolute change from baseline in hemoglobin

Annualized rate Vaso Occlusive Crisis (VOC) events leading to healthcare visit in clinic/ER/hospital

Annualized rate Vaso Occlusive Crisis (VOC) events treated at home (based on documentation by health care provider following phone contact with the patient)

+10 more