Your session is about to expire
← Back to Search
Crizanlizumab for Pediatric Sickle Cell Disease
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.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Side effects data
From 2021 Phase 2 trial • 54 Patients • NCT04435184Trial Design
Find a Location
Who is running the clinical trial?
Media Library
- 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.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.I do not have a major infection or immune system problem.I have cancer, but any other cancers I had were treated successfully and haven't come back in 2 years.I have had serious heart issues or abnormal heart rhythms recently.I am planning to receive regular transfusions or undergo exchange transfusions/plasmapheresis during the study.I have a bleeding disorder.I am not allergic to the study drug or similar medications.I have not had antibody or immunoglobulin therapy in the last 6 months, nor have I had a bad reaction to such treatments before.I haven't had a stroke, brain bleed, or unexplained neurological symptoms in the last year.I haven't taken strong blood thinners or antiplatelet medications, other than aspirin, in the last 10 days.I was in the hospital within the last week before starting the treatment.I plan to have a major surgery during the study period.I agree to use birth control for 15 weeks after my last dose of the study drug.I have not taken voxelotor in the last 30 days and do not plan to take it during the study.I am between 2 and 18 years old, or between 6 and 24 months old and weigh at least 7 kg for a specific part of the study.My child's brain blood flow test shows a low risk for stroke.I had at least one severe pain crisis in the last year that needed medical attention and strong painkillers.I have received all recommended treatments and vaccines for sickle cell disease.I am not able to have children or have tested negative for pregnancy.I am not currently institutionalized by court or administrative order.I can do most activities but may need help, regardless of my age.I have not taken crizanlizumab or similar medications.I have a history of Hepatitis C.I am not planning to start or stop taking hydroxyurea/hydroxycarbamide or L-glutamine during the study, unless for safety.
- Group 1: Crizanlizumab
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
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"
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."
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."
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."
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."
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."
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."
Does this trial accept participants who are of advanced age?
"This trial is seeking participants between 6 months and 17 years of age."
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."
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger