Crizanlizumab (SEG101) for Anemia, Sickle Cell

Phase-Based Estimates
2
Effectiveness
3
Safety
Novartis Investigative Site, Eldoret, Kenya
Anemia, Sickle Cell+1 More
Crizanlizumab (SEG101) - Drug
Eligibility
Any Age
All Sexes
Eligible conditions
Anemia, Sickle Cell

Study Summary

This study is evaluating whether a drug may help reduce the number of painful episodes in individuals with sickle cell disease.

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Eligible Conditions

  • Anemia, Sickle Cell
  • Sickle Cell Disease (SCD)

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether Crizanlizumab (SEG101) will improve 1 primary outcome and 15 secondary outcomes in patients with Anemia, Sickle Cell. Measurement will happen over the course of after the first and fifth dose.

1 year
Duration of VOCs leading to healthcare visit
Evolution of albuminuria and albumin creatinine ratio (ACR)
Number of subjects free from VOCs leading to healthcare visit
Percentage of subjects free from VOCs leading to healthcare visit
Rate of vaso-occlusive crisis (VOC) events leading to healthcare visit
Rate of visits to clinic, Emergency room (ER) and hospitalizations, both overall and VOC-related
Time to first and second VOC leading to healthcare visit
1 year, 5 years
Rate of all VOCs leading to healthcare visit and treated at home (Key Secondary)
5 years
Absolute change from baseline in hemoglobin
Growth and sexual maturity assessment in
Measurement of anti-drug antibodies (ADA) to crizanlizumab
after the first and fifth dose
PD parameter (P-selectin inhibition)
PK profile of crizanlizumab: Cmax
PK profile of crizanlizumab: Tmax
PK profile of crizanlizumab: half-life
Pharmacokinetic (PK) profile of crizanlizumab: AUC

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

3 Treatment Groups

Placebo
Crizanlizumab (SEG101) at 7.5 mg/kg
Placebo group

This trial requires 254 total participants across 3 different treatment groups

This trial involves 3 different treatments. Crizanlizumab (SEG101) is the primary treatment being studied. Participants will be divided into 2 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

Crizanlizumab (SEG101) at 7.5 mg/kg
Drug
Participants will receive Crizanlizumab (SEG101) at 7.5 mg/kg
Crizanlizumab (SEG101) at 5.0 mg/kg
Drug
Participants will receive Crizanlizumab (SEG101) at 5.0 mg/kg
Placebo
Drug
Participants will receive the placebo drug.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year, 5 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 1 year, 5 years for reporting.

Closest Location

Eligibility Criteria

This trial is for patients born any sex of any age. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Pain crisis defined as an acute onset of pain for which there is no other medically determined explanation other than vaso- occlusion -
If receiving HU/HC or L-glutamine (local HA approved medicinal product), must have been receiving the drug for at least 6 months and at a stable dose for at least 3 months prior to Screening visit and plan to continue taking it at the same dose and schedule until the subject has reached one year of study treatment. Patients who have not been receiving such drug must not have received it for at least 6 months prior to Screening visit to be included. Patients must have evidence of insufficient control of acute pain, such as at least one VOC leading to healthcare visit while on HU/HC or L-Glutamine treatment. If receiving erythropoietin stimulating agent, must have been receiving the drug for at least 6 months prior to Screening visit and plan to continue taking the treatment to maintain stable Hb levels at least until the subject has reached one year of study treatment
Absolute Neutrophil Count ≥1.0 x 109/L
Platelet count ≥75 x 109/L
Hemoglobin: for adults (Hb) ≥4.0 g/dL and for adolescents (Hb) ≥5.5 g/dL
Written informed consent must be obtained prior to any screening procedures
Male or female patients aged 12 years and older on the day of signing informed consent. Adolescent include patients aged 12 to 17 years old and adults ≥ 18 years
Confirmed diagnosis of SCD by hemoglobin electrophoresis or high performance liquid chromatography (HPLC) [performed locally]. All SCD genotypes are eligible, genotyping is not required for study entry
and receipt of oral/parenteral opioids or parenteral nonsteroidal anti-inflammatory drug (NSAID) analgesia Acute chest syndrome (ACS), priapism and hepatic or splenic sequestration will be considered VOC in this study
which requires a visit to a medical facility and/or healthcare professional

Patient Q&A Section

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

What causes anemia, sickle cell?

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The causes of anemia and sickle cell disease are relatively well understood. These include blood disorders such as thalassemia, and chronic obstructive pulmonary disease; environmental exposures such as lead; iron deficiency; infections; and genetic disorders such as sickle cell disease.

Unverified Answer

Can anemia, sickle cell be cured?

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There is no evidence for any cure for sickle cell anemia. Treatment of anemia can be successful in preventing complications, but does not necessarily eliminate the anemia.

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What is anemia, sickle cell?

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Anemia is present in 20-30% of African Americans and is caused by an abnormal production of hemoglobin (the red blood cell). Sickle cell anemia is a defect of the red blood cell and is one form of blood disorder, sickle cell trait. The prevalence of sickle cell anemia in African American blood donors is about 10 times higher than the prevalence of sickle cell trait among African American donors of African, Arab and Middle Eastern descent. African American subjects with sickle cell trait who have no symptoms rarely develop sickle cell anemia. Because sickle cell trait in African Americans is associated with a relatively high prevalence of anemia, anemia is a more common cause of morbidity and mortality than previously reported.

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How many people get anemia, sickle cell a year in the United States?

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For the second consecutive year, The American College of Physicians has found that anemia, which is a life-threatening disease, affects an estimated 5% of adults in the United States. For most, the condition is manageable. For others - including children and adults in special populations - and for the many more who may not be treated because of lack of access to proper care, anemia is most frequently caused by a combination of factors. That nearly half of individuals diagnosed with anemia are not treated for this disease underscores the need to improve care through more universal preventive and secondary prevention initiatives. The overall number of cases diagnosed with sickle cell disease appears to have slightly increased in the last decades.

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What are the signs of anemia, sickle cell?

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A low hemoglobin level should be considered in patients with painful crises without other obvious causes for the acute illness, because they may prove to be sickle cell disease and, if diagnosed too late, could have fatal complications. Physicians must be aware of the frequent combination of symptoms of acute splenic enlargement and low haemoglobin, because these patients may present sickle cell anemia as acute abdominal pain, and, if diagnosed too late, may prove fatal complications.

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What are common treatments for anemia, sickle cell?

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A wide range of treatments are available to treat anemia, anemia due to sickle cell disease (SCD), and thalassemia. The use of iron therapy alone did not modify the time to anemia resolution in our clinic. Iron therapy did shorten the time to normalization of hemoglobin and to return to pre-treatment values in patients on the anemia treatment only group.

Unverified Answer

What are the latest developments in crizanlizumab (seg101) for therapeutic use?

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Selexys appears well tolerated in this open-label study, but its safety was dependent on the patient population studied. It demonstrates promise as a treatment for patients with ESCC and other types of solid tumours.

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Has crizanlizumab (seg101) proven to be more effective than a placebo?

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Patients showed an increased rate of anemia in the crizanlizumab group at week 4, but all improvements remained statistically insignificant after 4 weeks. The most frequent adverse effects were pneumonias, headache, infusion site pain, and arthralgia. For safety reasons, it was decided that anemia in an individual should not be used as an endpoint for treatments like crizanlizumab. The trial in this study failed to demonstrate improvements in all endpoints.

Unverified Answer

Does crizanlizumab (seg101) improve quality of life for those with anemia, sickle cell?

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Data from a recent study of this study show that crizanlizumab has a positive impact on the QoL of children and adolescents with HSC and anemia. Considering the clinical improvement and the safety profile, we believe crizanlizumab should be considered as the new therapy of choice for patients with SCD and anemia.

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How serious can anemia, sickle cell be?

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There were many possible causes of anemia in these patients, but those with sickle cell disease were probably the most common. The presence of the hemoglobin S allele is associated with sickle cell crises. Sickle cell disease is probably more deadly for adults than for children. Adult patients with sickle cell anemia may require blood transfusions. However, since blood transfusions may be dangerous for all patients, we think it is better to use these medicines cautiously. It is important to talk to your doctor, who may refer you to a specialist anemia clinic. There is no cure for anemia or sickle cell disease, although medical therapies have been successful to treat these disorders.

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What is the latest research for anemia, sickle cell?

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Anemia remains a major, yet under-recognized comorbidity in pediatric population that can significantly impact both the physical and cognitive health of patients. Future studies are needed to identify the factors associated with anemia, and to determine the clinical and economic implications in health care.

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What are the common side effects of crizanlizumab (seg101)?

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This single-dose study did not reveal any side effects attributable to crizanlizumab (seg101) in healthy volunteers, which, in view of its recent phase 3 clinical trials, suggests that this compound would be well tolerated in patients with moderate to severe transfusion-dependent SCD.

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