Crizanlizumab for Anemia, Sickle Cell

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Anemia, Sickle Cell+1 More
Crizanlizumab - Drug
Eligibility
< 18
All Sexes
What conditions do you have?
Select

Study 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.

Eligible Conditions
  • Anemia, Sickle Cell

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

7 Primary · 16 Secondary · Reporting Duration: 6 months, 2 years

6 months, 2 years
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)
Annualized rate Vaso Occusive Crisis (VOC) events leading to healthcare visit in clinic/ER/hospital
Annualized rate Vaso Occusive Crisis (VOC) events treated at home (based on documentation by health care provider following phone contact with the patient)
Annualized rate days of ER/hospitalization (both overall and VOC-related)
Annualized rate each subcategory of VOC event (uncomplicated pain crisis, acute chest syndrome, hepatic sequestration, splenic sequestration, priapism)
Annualized rate hospitalizations and ER visits (both overall and VOC-related)
Annualized rate of dactylitis events
Frequency of any adverse events (AEs) as a measure of safety and tolerability
Number, seriousness, severity, and causality assessments of treatement emergent adverse events and other data as considered appropiate.
Number, seriousness, severity, and causality assessments of treatment emergent adverse events and other data as considered appropriate.
Year 2
Absolute change from baseline in hemoglobin
Day 15
PD (AUCd15) after 1st dose
PK (AUCd15) after 1st dose
Week 51
Growth and sexual maturation assessments (Tanner stage)
Week 51
Electrocardiogram (ECGs) at relevant PK time points
Week 15
PK (Cmax) after 1st dose and multiple dose
Week 19
PK pre-dose concentrations
Year 2
Immunogenicity: measurement of anti-drug antibodies (ADA) to crizanlizumab
Week 51
PK pre-dose concentrations prior to each study drug dose.
Percentage P-selectin inhibition prior to dosing
Week 15
PD (AUCtau) after multiple dose
PK (AUCtau) after multiple dose

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Side Effects for

Crizanlizumab
8%Chest pain
4%Dark and Infrequent Urination
4%Venous thrombembolism
4%Diarrhea
4%Altered mental status
4%Headache
This histogram enumerates side effects from a completed 2021 Phase 2 trial (NCT04435184) in the Crizanlizumab ARM group. Side effects include: Chest pain with 8%, Dark and Infrequent Urination with 4%, Venous thrombembolism with 4%, Diarrhea with 4%, Altered mental status with 4%.

Trial Design

1 Treatment Group

Crizanlizumab
1 of 1

Experimental Treatment

119 Total Participants · 1 Treatment Group

Primary Treatment: Crizanlizumab · No Placebo Group · Phase 2

Crizanlizumab
Drug
Experimental Group · 1 Intervention: Crizanlizumab · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Crizanlizumab
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 6 months, 2 years

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor
2,697 Previous Clinical Trials
3,524,022 Total Patients Enrolled
18 Trials studying Anemia, Sickle Cell
4,192 Patients Enrolled for Anemia, Sickle Cell

Eligibility Criteria

Age < 18 · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
Patients aged 2 to < 16 years with HbSS, HbSβ0-thalassemia, and HbSD disease are at low risk for stroke
You have experienced at least 1 VOC within the preceding 12 months prior to screening, as determined by medical history
You must meet the following laboratory values prior to Week 1 Day 1: Absolute Neutrophil Count ≥1.0 x 109/L, Platelets ≥75 x 109/L, Hemoglobin (Hgb) > 5.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 23rd, 2021

Last Reviewed: November 3rd, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.