← Back to Search

Monoclonal Antibodies

Crizanlizumab for Pediatric Sickle Cell Disease

Phase 2
Recruiting
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 6 months, 2 years
Awards & highlights

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.

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.Check my eligibility
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.See study design
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
Select...
I have received all recommended treatments and vaccines for sickle cell disease.
Select...
I am not able to have children or have tested negative for pregnancy.
Select...
My kidney and liver functions are within the required range.
Select...
I can do most activities but may need help, regardless of my age.

Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
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

Side effects data

From 2021 Phase 2 trial • 54 Patients • NCT04435184
8%
Chest pain
4%
Headache
4%
Diarrhea
4%
Dark and Infrequent Urination
4%
Venous thrombembolism
4%
Altered mental status
100%
80%
60%
40%
20%
0%
Study treatment Arm
Crizanlizumab
Placebo Saline

Trial Design

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
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Crizanlizumab
2021
Completed Phase 2
~600

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor
2,855 Previous Clinical Trials
4,197,231 Total Patients Enrolled

Media Library

Crizanlizumab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT03474965 — Phase 2
Sickle Cell Disease Research Study Groups: Crizanlizumab
Sickle Cell Disease Clinical Trial 2023: Crizanlizumab Highlights & Side Effects. Trial Name: NCT03474965 — Phase 2
Crizanlizumab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03474965 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What qualifications must a person possess to participate in this medical experiment?

"This medical trial is recruiting 119 individuals of both genders, between 6 months and 17 years old, who are diagnosed with anemia or sickle cell. To be eligible for participation in the study, prospective patients must comply to a specific set of criteria such as: gender (male/female), age bracket (2-18 year olds; 6 month - 24 month olds provided they weigh over 7 kgs), confirmation their diagnosis through hemoglobin electrophoresis tests or HPLC methods (two accepted forms recommended); if receiving hydroxyurea/hydroxycarbamide medication, l-glutamine supplements or erythropoietin"

Answered by AI

What tangible results is this investigation expected to deliver?

"Novartis Pharmaceuticals, the clinical trial sponsor of this study, reports that its main objective includes measuring pharmacokinetic data (AUCd15) after one dose at Week 1 and Week 15. As for secondary outcomes, researchers are analyzing pre-dose concentrations of medications ahead of each dosage as well as long-term PK/PD effects in participants aged 6 months to 18+ years old. Furthermore, they plan on evaluating VOC events treated from home and those requiring medical assistance in a clinic/emergency room/hospital environment over an annual duration."

Answered by AI

What other clinical experiments have been performed to test the efficacy of Crizanlizumab?

"Crizanlizumab was first investigated in 2017 by East Carolina University. There have been 2 completed trials and 10 ongoing studies, most of which are situated in Bronx, New york."

Answered by AI

Has Crizanlizumab received official sanction by the Food and Drug Administration?

"After careful consideration, our team at Power has assigned a score of 2 to Crizanlizumab's safety; this is due the Phase 2 trial status which indicates that there are some data points supporting its security but none regarding efficacy."

Answered by AI

Is there still an opportunity for prospective participants to enroll in this investigation?

"As per clinicaltrials.gov, this particular medical study is actively recruiting patients. It's inaugural post was on the 1st of October 2018 and the latest update to its information occurred recently on November 9th 2022."

Answered by AI

To what extent is this investigation being conducted in healthcare facilities?

"This trial is currently being conducted at a variety of locations, including Children s National Hospital in Bronx, New york; Ann and Robert H Lurie Childrens Hospital of Chicago CTBM100C2407 in Atlanta, Georgia; and the esteemed Children's Hospital of Philadelphia in Washington D.C., among 42 other sites."

Answered by AI

Has there been precedent for this type of research before?

"The pharmaceutical company Novartis Pharmaceuticals initiated research into Crizanlizumab in 2017 and conducted an initial trial of 57 patients. This drug was subsequently granted Phase 2 approval, leading to 10 active clinical trials spanning 144 cities and 36 nations today."

Answered by AI

Does this trial accept participants who are of advanced age?

"This trial is seeking participants between 6 months and 17 years of age."

Answered by AI

What is the current number of participants engaged in this experiment?

"This experiment necessitates 119 qualified participants. Individuals may take part at various sites, including Children's National Hospital in Bronx and Ann & Robert H Lurie Childrens Hospital of Chicago CTBM100C2407 in Atlanta."

Answered by AI
~13 spots leftby Jan 2025