Using crizanlizumab to prevent silent brain injuries in sickle cell disease
Study of Crizanlizumab for Prevention of Silent Cerebral Infarcts in SCA Novartis Investigator Initiated Trial: CSEG101AUS12T
This study is testing if crizanlizumab can help prevent hidden brain injuries in people with sickle cell disease who are at higher risk.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 31 (estimated) |
| Ages | 16 Years and up |
| Sex | All |
| Sponsor | Washington University School of Medicine Academic / other |
| Drugs / interventions | crizanlizumab |
| Locations | 1 site (Saint Louis, Missouri) |
| Trial ID | NCT05334576 on ClinicalTrials.gov |
What this trial studies
This study aims to evaluate the effectiveness of crizanlizumab in preventing silent cerebral infarcts in patients with sickle cell disease. Participants aged 16 and older will receive crizanlizumab infusions and undergo brain MRI scans before treatment and at 6 and 30 months after starting the medication. The outcomes of these participants will be compared to a matched group of patients not receiving crizanlizumab to assess the drug's impact on brain health. The study focuses on individuals at increased risk for silent cerebral infarcts due to their sickle cell disease.
Who should consider this trial
Good fit: Ideal candidates include adults aged 16 and older with specific sickle cell disease genotypes and a documented increased risk for silent cerebral infarcts.
Not a fit: Patients without sickle cell disease or those who do not meet the criteria for increased risk of silent cerebral infarcts may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce the risk of silent cerebral infarcts in patients with sickle cell disease, potentially improving their overall neurological health.
How similar studies have performed: While there have been studies on crizanlizumab for sickle cell disease, this specific approach to preventing silent cerebral infarcts is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Adult participants age 16 and older 2. Sickle cell disease with confirmation of HbSS, HbSBthal0, HbSC, or HbS thal+ genotype 3. Per patient's sickle cell provider, patient has an increased risk of a silent cerebral infarcts according to one of the following criteria: 1. Silent cerebral infarcts visualized on FLAIR MRI within previous two years 2. Intracranial or extracranial cervical artery vasculopathy 3. History of overt ischemic or hemorrhagic stroke and Intolerance and/or failure of other therapies to prevent cerebral infarction 4. Increased severity of sickle cell disease including having between 2 and 10 sickle cell-related pain crises within the preceding 12 months as determined by medical history or by patient's recall (crises should include the occurrence of appropriate symptoms, a visit to a specific medical facility and/or health care professional, and receipt of pain medication). 5. Increased risk deemed by other objective laboratory and/or imaging results which have been associated with increased risk of cerebral infarction 4. Provide written informed consent. 5. Normal hematologic function defined as: WBC \> 4x10\^9 / L, ANC \>1.5x10\^9 / L and platelets \> 100x10\^9 / L 6. Females of childbearing potential (FCBP) must agree to refrain from becoming pregnant while on crizanlizumb and for 3 months after discontinuation from crizanlizumab, and must agree to use adequate contraception including hormonal contraception, (i.e. birth control pills, etc.), barrier method contraception (i.e. condoms), or abstinence during the time-frame Exclusion Criteria: 1. Current chronic transfusion therapy 2. Planning for hematopoietic stem cell transplant or cerebral revascularization procedure 3. Use of other investigational drug within one year of study participation 4. Other medical/neurological/social/substance abuse history that would alter brain MRI findings prospectively 5. Inability to return for follow-up 6. Contraindication to MRI 7. Acute bacterial, fungal, or viral infection 8. Known HIV, untreated latent tuberculosis (TB), or active hepatitis B or C infection or zoster 9. Pregnant and/or breastfeeding. Negative pregnancy test required prior to starting study treatment. 10. Known hypersensitivity to one or more of the study agents 11. Currently receiving or has received any investigational drugs within the 14 days prior to the first dose of study drug 12. Liver function tests (LFT) higher than 3x the upper limit of normal 13. Treatment with other monoclonal antibody medications within last 30 days 14. Treatment with various forms of anticoagulation within last 30 days, including but not limited to coumadin or direct thrombin inhibitors
Where this trial is running
Saint Louis, Missouri
- Barnes-Jewish Hospital — Saint Louis, Missouri, United States (Recruiting)
Study contacts
- Study coordinator: Andria Ford, MD
- Email: forda@wustl.edu
- Phone: 314-362-7382
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.