Crizanlizumab for Sickle Cell Disease

Recruiting in Palo Alto (17 mi)

+35 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Recruiting

Sponsor: Novartis Pharmaceuticals

Must be taking: Crizanlizumab

Disqualifiers: Pregnancy, Grade 3 AEs, others

No Placebo Group

Prior Safety Data

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

This is a multi-center multi-national rollover study to allow continued access to crizanlizumab for patients with sickle cell disease (SCD) who are on crizanlizumab treatment in a Novartis-sponsored study (parent study) and are benefiting from the treatment as judged by the investigator.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It is best to discuss this with the trial investigator to understand how your current medications might interact with the study treatment.

What data supports the effectiveness of the drug crizanlizumab for sickle cell disease?

Crizanlizumab has been shown to reduce the frequency of painful episodes in sickle cell disease by 45.3% compared to a placebo in a clinical trial. It is approved for use in patients aged 16 and older to help prevent these painful crises. ^{1 2 3 4 5}

Is crizanlizumab safe for humans?

Crizanlizumab has been studied for safety in patients with sickle cell disease, showing similar rates of adverse events (unwanted effects) compared to a placebo. Common side effects include infusion-related reactions, joint pain, diarrhea, and nausea, but no serious treatment-related adverse events were reported. ^{1 2 4 5 6}

What makes the drug Crizanlizumab unique for treating Sickle Cell Disease?

Crizanlizumab is unique because it targets P-selectin, a protein that plays a key role in the inflammation and blockage of blood vessels in sickle cell disease, helping to reduce pain crises. This mechanism is different from other treatments that primarily focus on managing symptoms or increasing fetal hemoglobin levels. ^{7 8 9 10 11}

Research Team

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Eligibility Criteria

This trial is for patients with Sickle Cell Disease who are already participating in a Novartis-sponsored Crizanlizumab study and have seen benefits. They must have completed the previous study's treatment schedule, shown commitment to visit schedules, and be willing to continue visits. Pregnant or nursing women, those not using effective contraception, or anyone with unresolved severe side effects from prior treatments cannot join.

Inclusion Criteria

I am in a Novartis study for SCD, benefiting from crizanlizumab, and have met all study requirements.

I (or my guardian if I'm under 18) have signed the consent form.

Patient has demonstrated compliance to the planned visit schedule in the parent study, and in the opinion of the investigator has shown willingness and ability to comply with future visit schedules

Exclusion Criteria

Pregnant or nursing women

Concurrent participation in any other investigational clinical trial other than the parent study or plan to participate in any other investigational clinical trial

I have SCD and cannot continue with crizanlizumab as per the main study.

Trial Timeline

Treatment

Participants continue receiving Crizanlizumab treatment at the same dose/schedule as in the parent study

10 years or until treatment becomes commercially available

Safety Follow-up

A safety follow-up visit is conducted 105 days after the last administration of study treatment

105 days

Long-term Follow-up

Participants are monitored for continued access to Crizanlizumab until it becomes commercially available or no longer needed

Up to 10 years

Treatment Details

Interventions

• Crizanlizumab (Monoclonal Antibodies)

Trial Overview The trial provides continued access to Crizanlizumab for individuals with Sickle Cell Disease who benefited from earlier trials. It's designed as a rollover study where participants receive ongoing Crizanlizumab therapy under the supervision of investigators at multiple centers internationally.

Participant Groups

Group I: Crizanlizumab Experimental Treatment 1 Intervention

All participants will receive crizanlizumab (SEG101) at the same dose/schedule as in the parent study.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

University Of Alabama Birmingham, AL

Childrens National Hospital Washington, United States

Augusta University Georgia Patient Treatment Augusta, GA

Childrens Hospital Of Philadelphia Patient Treatment Philadelphia, PA

More Trial Locations

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2963

Patients Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Findings from Research

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-World Data of Crizanlizumab in Sickle Cell Disease: A Single-Center Analysis. Cheplowitz, H., Block, S., Groesbeck, J., et al.[2023]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The European Medicines Agency Review of Crizanlizumab for the Prevention of Recurrent Vaso-Occlusive Crises in Patients With Sickle Cell Disease. Delgado, J., Voltz, C., Stain, M., et al.[2021]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Profile of crizanlizumab and its potential in the prevention of pain crises in sickle cell disease: evidence to date. Riley, TR., Riley, TT.[2020]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Crizanlizumab: First Approval. Blair, HA.[2020]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Crizanlizumab for the Prevention of Vaso-Occlusive Pain Crises in Sickle Cell Disease. Stevens, DL., Hix, M., Gildon, BL.[2022]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacokinetics, pharmacodynamics, safety, and efficacy of crizanlizumab in patients with sickle cell disease. Kanter, J., Brown, RC., Norris, C., et al.[2023]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Current therapy of chronic myeloid leukemia]. Koskela, H., Koskenvesa, P., Mustjoki, S., et al.[2022]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical activity of ponatinib in one patient with chronic myeloid leukemia in chronic phase with e19a2 transcript and T315I mutation. Ferri, CA., Bianchini, M., Bengió, RM., et al.[2015]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intolerance to tyrosine kinase inhibitors in chronic myeloid leukemia: the possible role of ponatinib. Breccia, M., Efficace, F., Iurlo, A., et al.[2019]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Is imatinib still an acceptable first-line treatment for CML in chronic phase? Goldman, JM., Marin, D.[2017]

Dasatinib has been approved by the FDA as a treatment for pediatric chronic myeloid leukemia, providing a new therapeutic option for young patients.

As the second tyrosine kinase inhibitor approved for this condition, dasatinib can be used in both first- and second-line treatment, expanding the choices available for managing this type of leukemia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dasatinib Approved for Pediatric CML. [2019]

References

1. Canada pubmed.ncbi.nlm.nih.gov

Real-World Data of Crizanlizumab in Sickle Cell Disease: A Single-Center Analysis. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

The European Medicines Agency Review of Crizanlizumab for the Prevention of Recurrent Vaso-Occlusive Crises in Patients With Sickle Cell Disease. [2021]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Profile of crizanlizumab and its potential in the prevention of pain crises in sickle cell disease: evidence to date. [2020]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Crizanlizumab: First Approval. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

Crizanlizumab for the Prevention of Vaso-Occlusive Pain Crises in Sickle Cell Disease. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Pharmacokinetics, pharmacodynamics, safety, and efficacy of crizanlizumab in patients with sickle cell disease. [2023]

7. Finland pubmed.ncbi.nlm.nih.gov

[Current therapy of chronic myeloid leukemia]. [2022]

8. England pubmed.ncbi.nlm.nih.gov

Clinical activity of ponatinib in one patient with chronic myeloid leukemia in chronic phase with e19a2 transcript and T315I mutation. [2015]

9. England pubmed.ncbi.nlm.nih.gov

Intolerance to tyrosine kinase inhibitors in chronic myeloid leukemia: the possible role of ponatinib. [2019]

10. United States pubmed.ncbi.nlm.nih.gov

Is imatinib still an acceptable first-line treatment for CML in chronic phase? [2017]

11. United States pubmed.ncbi.nlm.nih.gov

Dasatinib Approved for Pediatric CML. [2019]