Trial · NCT03814746
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
NCT03814746, Phase 3, Active not recruiting, Novartis Pharmaceuticals; results first posted 2024-01-08
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients. Trials, record NCT03814746. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03814746
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
- NCT number
- NCT03814746
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 253 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Sickle Cell Disease (SCD)
- Interventions
- Crizanlizumab (SEG101), Placebo
- First posted
- 2019-01-24
- Last updated
- 2026-10-14
- Other registries' IDs
- eudract:2017-001746-10
- Results first posted
- 2024-01-08
Source ClinicalTrials.gov
Registered outcomes24
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Annualized Rate of Vaso-occlusive Crisis (VOC) Events Leading to a Healthcare Visit | 1 year | VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. Healthcare visit is defined as any visit to a medical facility such as emergency room (ER), hospital and/or office visit, which includes pain management of VOC in situ. Annualized rate of corresponding VOC events = (Number of corresponding VOC events \* 365)/(number of days in the observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days). |
| Secondary | Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home Over the First-year Post Randomization (Key Secondary) | 1 year | VOCs are based on documentation by provider following contact with participant. VOC:pain crisis requiring therapy with oral/parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome, priapism and hepatic or splenic sequestration. Healthcare visit:a visit to a medical facility (ER, hospital \&/or office visit resulting in pain management of VOC. Managed at home: no visit to any medical facility \&/or healthcare professional to receive treatment for VOC. Healthcare contact for medical advice is allowed. Annualized rate of corresponding VOC events = (# of corresponding VOC events \* 365)/(# of days in observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor \& erythropoietin therapies to treat SCD \&/or to prevent/reduce VOCs), date of randomization + 365 days) |
| Secondary | Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home Over 5 Years Post Randomization (Key Secondary) | 5 years | To compare the efficacy of 5.0 mg/kg vs placebo \& 7.5 mg/kg vs placebo on the annualized rate of all VOCs based on documentation by provider following contact with participant. VOC is defined as pain crisis (an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) requiring therapy with oral/parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome, priapism and hepatic or splenic sequestration. Healthcare visit is defined as a visit to a medical facility (emergency room, hospital and/or office visit resulting in pain management of VOC. Managed at home is defined as no visit to any medical facility and/or healthcare professional to receive treatment for VOC. Healthcare contact for medical advice is allowed. The annualized rate of VOC leading to healthcare visit is the number of VOC leading to healthcare visit multiplied by 365 \& divided by the number of days in observation period. |
| Secondary | Mean Duration of VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | 1 year | To assess the duration of VOCs leading to healthcare visit in each group. Mean duration of VOC per participant is defined as the average duration of all individual episodes of VOCs leading to healthcare visits of a given participant (a VOC duration is defined as end date of the VOC - start date of the VOC + 1). Participants with no VOC leading to healthcare visits have been excluded. |
| Secondary | Number of Participants Free From VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | 1 year | To assess the number of participants free from VOCs leading to healthcare visit. VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. A participant is free from VOC if they do not have a VOC crisis. |
| Secondary | Percentage of Participants Free From VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | 1 year | To assess the percentage of participants free from VOCs leading to healthcare visit. VOC is defined as pain crisis (defined as an acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) which requires therapy with oral or parenteral opioids or parenteral NSAID as well as other complicated crisis such as acute chest syndrome (ACS), priapism and hepatic or splenic sequestration. A participant is free from VOC if they do not have a VOC crisis. |
| Secondary | Time to First and Second VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | 1 year | To assess the time to first and second VOC leading to healthcare visit in each group. Time to first occurrence of VOC leading to a healthcare visit is defined as the time from the date of randomization to the date of the first occurrence of the VOC. Time to second occurrence of VOC leading to a healthcare visit is defined as the time from date of randomization to the date of the second occurrence of VOC. |
| Secondary | Annualized Rate of Visits to Clinic, Emergency Room (ER) and Hospitalizations, Both Overall and VOC-related Over the First-year Post-randomization | 1 year | To assess Healthcare resource utilization (visits to clinic, Emergency room (ER) and hospitalizations) both overall and VOC-related in each group. Annualized rate of corresponding healthcare visits =(Number of corresponding healthcare visits \* 365)/(number of days in the observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days) |
| Secondary | Annualized Days of Visits to Clinic, Emergency Room (ER) and Hospitalizations, Both Overall and VOC-related Over the First-year Post-randomization | 1 year | To assess Healthcare resource utilization (visits to clinic, Emergency room (ER) and hospitalizations) both overall and VOC-related in each group. Annualized days of corresponding healthcare visits =(Number of days =(Number of days of corresponding healthcare visits \* 365)/(number of days in the observation period). Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days). |
| Secondary | Evolution of Albumin Creatinine Ratio (ACR) Over the First-year Post-randomization (Change From Baseline) | Over first year post-randomization (Baseline, Week 27 Day 1, Week 51 Day 1) | Laboratory values for parameters related to renal function (creatinine, estimated glomerular filtration rate, urine microalbumin, and urine albumin/creatinine ratio) were measured at 6-month intervals over time from baseline. |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 registered outcomesOutcomes24
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Annualized Rate of Vaso-occlusive Crisis (VOC) Events Leading to a Healthcare Visit | 1 year | The FAS comprises all participants to whom study treatment has been assigned by randomization. | number of events per year | MEAN |
| SECONDARY | Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home Over the First-year Post Randomization (Key Secondary) | 1 year | The FAS comprises all participants to whom study treatment has been assigned by randomization. | number of events per year | MEAN |
| SECONDARY | Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home Over 5 Years Post Randomization (Key Secondary) | 5 years | |||
| SECONDARY | Mean Duration of VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | 1 year | The FAS comprises all participants to whom study treatment has been assigned by randomization. Participants with no VOC leading to healthcare visits are excluded. | days | MEAN |
| SECONDARY | Number of Participants Free From VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | 1 year | The FAS comprises all participants to whom study treatment has been assigned by randomization. | Participants | NUMBER |
| SECONDARY | Percentage of Participants Free From VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | 1 year | The FAS comprises all participants to whom study treatment has been assigned by randomization. | Percentage of participants | NUMBER |
| SECONDARY | Time to First and Second VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | 1 year | The FAS comprises all participants to whom study treatment has been assigned by randomization. The estimated time to first and second VOC using Kaplan-Meier analyzed all participants, including those who experienced first (n=59, 52, 51) and second (n=41, 33, 33) events in 5mg/kg, 7.5mg/kg and placebo arms, respectively, those who were at risk, and those who were censored. | Months | MEDIAN |
| SECONDARY | Annualized Rate of Visits to Clinic, Emergency Room (ER) and Hospitalizations, Both Overall and VOC-related Over the First-year Post-randomization | 1 year | The FAS comprises all participants to whom study treatment has been assigned by randomization. | number of events per year | MEAN |
| SECONDARY | Annualized Days of Visits to Clinic, Emergency Room (ER) and Hospitalizations, Both Overall and VOC-related Over the First-year Post-randomization | 1 year | The FAS comprises all participants to whom study treatment has been assigned by randomization. | number of days per year | MEAN |
| SECONDARY | Evolution of Albumin Creatinine Ratio (ACR) Over the First-year Post-randomization (Change From Baseline) | Over first year post-randomization (Baseline, Week 27 Day 1, Week 51 Day 1) | The FAS comprises all participants to whom study treatment has been assigned by randomization. | g/mol | MEDIAN |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 outcomesResults by group81
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Annualized Rate of Vaso-occlusive Crisis (VOC) Events Leading to a Healthcare Visit | Crizanlizumab (SEG101) at 5.0 mg/kg | 2.5 | Standard Deviation 2.98 | number of events per year | |
| Annualized Rate of Vaso-occlusive Crisis (VOC) Events Leading to a Healthcare Visit | Crizanlizumab (SEG101) at 7.5 mg/kg | 1.9 | Standard Deviation 2.30 | number of events per year | |
| Annualized Rate of Vaso-occlusive Crisis (VOC) Events Leading to a Healthcare Visit | Placebo | 2.1 | Standard Deviation 2.81 | number of events per year | |
| Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home Over the First-year Post Randomization (Key Secondary) | Crizanlizumab (SEG101) at 5.0 mg/kg | 4.5 | Standard Deviation 6.49 | number of events per year | |
| Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home Over the First-year Post Randomization (Key Secondary) | Crizanlizumab (SEG101) at 7.5 mg/kg | 3.1 | Standard Deviation 2.89 | number of events per year | |
| Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home Over the First-year Post Randomization (Key Secondary) | Placebo | 3.7 | Standard Deviation 3.78 | number of events per year | |
| Mean Duration of VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | Crizanlizumab (SEG101) at 5.0 mg/kg | 7.7 | Standard Deviation 6.93 | days | |
| Mean Duration of VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | Crizanlizumab (SEG101) at 7.5 mg/kg | 6.0 | Standard Deviation 4.54 | days | |
| Mean Duration of VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | Placebo | 6.6 | Standard Deviation 5.55 | days | |
| Number of Participants Free From VOCs Leading to a Healthcare Visit Over the First-year Post Randomization | Crizanlizumab (SEG101) at 5.0 mg/kg | 25 | Participants |
1 to 10 of 81Source ClinicalTrials.gov
See all 81 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Crizanlizumab (SEG101) at 5.0 mg/kg | 84 | |
| Overall Study | STARTED | Crizanlizumab (SEG101) at 7.5 mg/kg | 83 | |
| Overall Study | STARTED | Placebo | 85 | |
| Overall Study | COMPLETED | Crizanlizumab (SEG101) at 5.0 mg/kg | 66 | |
| Overall Study | COMPLETED | Crizanlizumab (SEG101) at 7.5 mg/kg | 68 | |
| Overall Study | COMPLETED | Placebo | 58 | |
| Overall Study | NOT COMPLETED | Crizanlizumab (SEG101) at 5.0 mg/kg | 18 | |
| Overall Study | NOT COMPLETED | Crizanlizumab (SEG101) at 7.5 mg/kg | 15 | |
| Overall Study | NOT COMPLETED | Placebo | 27 |
Source ClinicalTrials.gov
Adverse events369
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Crizanlizumab (SEG101) at 5.0 mg/kg (On-treatment) | 3 | 84 | |
| other | Anaemia | Blood and lymphatic system disorders | Crizanlizumab (SEG101) at 7.5 mg/kg (On-treatment) | 8 | 83 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo (On-treatment) | 4 | 85 | |
| other | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Crizanlizumab (SEG101) at 5.0 mg/kg (On-treatment) | 5 | 84 | |
| other | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Crizanlizumab (SEG101) at 7.5 mg/kg (On-treatment) | 3 | 83 | |
| other | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Placebo (On-treatment) | 1 | 85 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Crizanlizumab (SEG101) at 5.0 mg/kg (On-treatment) | 5 | 84 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Crizanlizumab (SEG101) at 7.5 mg/kg (On-treatment) | 3 | 83 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Placebo (On-treatment) | 1 | 85 | |
| other | Abdominal pain | Gastrointestinal disorders | Crizanlizumab (SEG101) at 5.0 mg/kg (On-treatment) | 5 | 84 |
1 to 10 of 369Source ClinicalTrials.gov
See all 369 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Crizanlizumab (SEG101) at 5.0 mg/kg (On-treatment) | 2 | 84 |
| Total, all-cause mortality | Crizanlizumab (SEG101) at 7.5 mg/kg (On-treatment) | 2 | 83 |
| Total, all-cause mortality | Placebo (On-treatment) | 2 | 85 |
| Total, other adverse events | Crizanlizumab (SEG101) at 5.0 mg/kg (On-treatment) | 67 | 84 |
| Total, other adverse events | Crizanlizumab (SEG101) at 7.5 mg/kg (On-treatment) | 71 | 83 |
| Total, other adverse events | Placebo (On-treatment) | 66 | 85 |
| Total, serious adverse events | Crizanlizumab (SEG101) at 5.0 mg/kg (On-treatment) | 35 | 84 |
| Total, serious adverse events | Crizanlizumab (SEG101) at 7.5 mg/kg (On-treatment) | 22 | 83 |
| Total, serious adverse events | Placebo (On-treatment) | 26 | 85 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03814746
GET https://conductscience.org/api/trials/v1/NCT03814746/registered?page=2