Sickle Cell Disease (SCD)Phase 3STANDNCT03814746
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
A Phase 3 study testing Crizanlizumab (SEG101) in people with Sickle Cell Disease (SCD), run by Novartis Pharmaceuticals at 58 sites in 21 countries.
Trial summary
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis (VOC) leading to healthcare visit. Read more about this study
- Who is it for?
- Ages 12 to 100 years
- Conditions the study is for
- Sickle Cell Disease (SCD)
- What is being tested?
- Crizanlizumab (SEG101)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 253 participants
- Where
- 58 sitesin 21 countries
- Study dates
- 26 Jul 2019to 5 Nov 2026
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
- Experienced at least 2 VOCs leading to healthcare visit within the 12 months prior to screening visit as determined by medical history. Prior VOC leading to healthcare visit must resolve at least 7 days prior to Week 1 Day 1 and must include:
- Pain crisis defined as an acute onset of pain for which there is no other medically determined explanation other than vaso- occlusion -
- which requires a visit to a medical facility and/or healthcare professional,
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- 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
- 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
- Patients must meet the following central laboratory values prior to Week 1 Day 1:
- 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
- Glomerular filtration rate ≥ 45 mL/min/1.73 m2 using CKD-EPI formula in adults, and Shwartz formula in adolescents
- Direct (conjugated) bilirubin < 2.0 x ULN
- Alanine transaminase (ALT) < 3.0 x ULN
- ECOG performance status ≤2.0 for adults and Karnofsky ≥ 50% for adolescents
Who cannot join
- History of stem cell transplant.
- Participating in a chronic transfusion program (pre-planned series of transfusions for prophylactic purposes) and/or planning on undergoing an exchange transfusion during the duration of the study; episodic transfusion in response to worsened anemia or VOC is permitted.
- Contraindication or hypersensitivity to any drug or metabolites from similar class as study drug or to any excipients of the study drug formulation. History of severe hypersensitivity reaction to other monoclonal antibodies, which in the opinion of the investigator may pose an increased risk of serious infusion reaction.
- Received active treatment on another investigational trial within 30 days (or 5 half-lives of that agent, whichever is greater) prior to Screening visit or plans to participate in another investigational drug trial.
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant unless they are using highly effective methods of contraception during dosing and for 15 weeks after stopping treatment.
- Concurrent severe and/or uncontrolled medical conditions which, in the opinion of the Investigator, could cause unacceptable safety risks or compromise participation in the study.
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- History or current diagnosis of ECG abnormalities indicating significant risk of safety such as:
- Concomitant clinically significant cardiac arrhythmias (e.g ventricular tachycardia), and clinically significant second or third degree AV block without a pacemaker
- History of familial long QT syndrome or know family history of Torsades de Pointes
- Not able to understand and to comply with study instructions and requirements.
- Received prior treatment with crizanlizumab or other selectin targeting agent
Trial locations and contacts
58 sites in 21 countries.
58 sites in 21 countries
Brazil9 sites
Novartis Investigative Site
Belém, Pará, 66033-000
Novartis Investigative Site
Porto Alegre, Rio Grande do Sul, 90035-003
Novartis Investigative Site
Recife, Pernambuco, 50070-170
Novartis Investigative Site
Ribeirão Preto, São Paulo, 14048-900
Novartis Investigative Site
Rio de Janeiro, Rio de Janeiro, 20211-030
Novartis Investigative Site
Salvador, Estado de Bahia, 41253-190
Novartis Investigative Site
São Paulo, São Paulo, 01232-010
Novartis Investigative Site
São Paulo, São Paulo, 05403-000
Novartis Investigative Site
São Paulo, São Paulo, 08270-070
United States5 sites
Childrens Healthcare of Atlanta
Atlanta, Georgia, 30342
Boston Medical Center
Boston, Massachusetts, 02118
Levine Cancer Insitute Carolinas Healthcare System
Charlotte, North Carolina, 28204
U of TX Health Science Ct
Houston, Texas, 77030
Univ of Tenn Health Sciences Ctr
Memphis, Tennessee, 38163
United Kingdom5 sites
Novartis Investigative Site
Cambridge, CB2 0QQ
Novartis Investigative Site
London, SE1 9RT
Novartis Investigative Site
London, SE5 9RS
Novartis Investigative Site
Sheffield, South Yorkshire, S10 2JF
Novartis Investigative Site
Sheffield, S10 2TH
Germany4 sites
Novartis Investigative Site
Berlin, 13353
Novartis Investigative Site
Cologne, North Rhine-Westphalia, 50937
Novartis Investigative Site
Essen, 45147
Novartis Investigative Site
Stuttgart, Baden-Wurttemberg, 70376
Italy4 sites
Novartis Investigative Site
Genova, GE, 16128
Novartis Investigative Site
Milan, MI, 20122
Novartis Investigative Site
Naples, 80138
Novartis Investigative Site
Verona, VR, 37134
Belgium3 sites
Novartis Investigative Site
Brussels, Brussels Capital, 1070
Novartis Investigative Site
Brussels, 1000
Novartis Investigative Site
Edegem, 2650
Colombia3 sites
Novartis Investigative Site
Barranquilla, Atlántico, 080020
Novartis Investigative Site
Montería, 230004
Novartis Investigative Site
Valledupar, Cesar Department, 200001
France3 sites
Novartis Investigative Site
Créteil, 94010
Novartis Investigative Site
Marseille, 13885
Novartis Investigative Site
Paris, 75015
Greece3 sites
Novartis Investigative Site
Athens, 115 27
Novartis Investigative Site
Pátrai, 265 04
Novartis Investigative Site
Thessaloniki, 546 36
Netherlands3 sites
Novartis Investigative Site
Amsterdam, North Holland, 1105 AZ
Novartis Investigative Site
Rotterdam, South Holland, 3015 GD
Novartis Investigative Site
The Hague, South Holland, 2545 AA
Spain3 sites
Novartis Investigative Site
Barcelona, 08035
Novartis Investigative Site
Madrid, 28034
Novartis Investigative Site
Madrid, 28046
Canada2 sites
Novartis Investigative Site
Montreal, Quebec, H2X 1R9
Novartis Investigative Site
Toronto, Ontario, M5G 2C4
India2 sites
Novartis Investigative Site
Bhubaneswar, Odisha, 751003
Novartis Investigative Site
Hyderabad, Telangana, 500082
Lebanon2 sites
Novartis Investigative Site
Beirut, 113-0236
Novartis Investigative Site
Tripoli, 1434
Turkey (Türkiye)1 site
Novartis Investigative Site
Adana, Saricam, 01330
About this study
Official title: A Phase III, Multicenter, Randomized, Double-blind Study to Assess Efficacy and Safety of Two Doses of Crizanlizumab Versus Placebo, With or Without Hydroxyurea/ Hydroxycarbamide Therapy, in Adolescent and Adult Sickle Cell Disease Patients With Vaso-Occlusive Crises (STAND)
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis (VOC) leading to healthcare visit.
Read the detailed description
Study CSEG101A2301 (STAND) is an ongoing Phase III, multicenter, randomized, double-blind study to assess efficacy and safety of two doses of crizanlizumab (5 mg/kg and 7.5 mg/kg) versus placebo, with or without hydroxyurea/ hydroxycarbamide therapy (HU/HC), in adolescent and adult patients with SCD and history of VOC leading to healthcare visit.
This is a multicenter clinical trial comparing 2 doses of crizanlizumab (5 mg/kg and 7.5 mg/kg) versus placebo in addition to standard of care participants might be taking at the time of study start, in adolescent and adult participants with confirmed diagnosis of sickle cell disease (SCD) and history of vaso-occlusive crisis (VOC) leading to a healthcare visit.
240 participants (including 48 adolescents) were planned to be randomized in a 1:1:1 ratio to either 5 mg/kg, 7.5 mg/kg of crizanlizumab or placebo. Randomized participants were stratified by concomitant HU/HC usage (yes/no) and baseline rate of VOCs leading to a healthcare visit in 12 months prior to screening visit (2-4 vs. ≥ 5 VOCs) at the time of enrollment. In November 2020, a capping of 90 adult participants per strata was implemented to ensure adequate opportunity for enrollment into each of the 4 strata.
- Conditions
- Sickle Cell Disease (SCD)
- Being tested
- Crizanlizumab (SEG101), Placebo
- Keywords
- Sickle Cell Disease, SCD, SEG101, Crizanlizumab, Hydroxyurea/ Hydroxycarbamide Therapy, Vaso-Occlusive Crises, SCA, blood disorders, hemoglobin, red blood cells, sickle-like shape, mutation in hemoglobin gene, sickle-cell trait, sickle-cell crisis
- Medical subject headings
- Anemia, Sickle Cell, Vaso-Occlusive Crises, Hematologic Diseases, Sickle Cell Trait
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Crizanlizumab (SEG101)Drug
Also called SEG101
Crizanlizumab was supplied in single use 10 mL glass vials at a concentration of 10 mg/mL. One vial contains 100 mg of crizanlizumab. This is a concentrate for solution for infusion.
IV.
PlaceboDrug
Placebo was supplied in single use 10 mL glass vials at a concentration of 0 mg/mL. This is a concentrate for solution for infusion IV.
| Study group | Role | What they receive |
|---|---|---|
| Crizanlizumab (SEG101) at 5.0 mg/kg | Experimental | Participants received Crizanlizumab (SEG101) at 5.0 mg/kg Receives: Drug: Crizanlizumab (SEG101) |
| Crizanlizumab (SEG101) at 7.5 mg/kg | Experimental | Participants received Crizanlizumab (SEG101) at 7.5 mg/kg Receives: Drug: Crizanlizumab (SEG101) |
| Placebo | Placebo comparator | Participants received the placebo drug. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Rate of Vaso-occlusive Crisis (VOC) Events Leading to a 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.
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).
Measured: 1 year
Secondary outcomes (23)
Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home Over the First-year Post Randomization (Key Secondary)
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)
Measured: 1 year
Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home Over 5 Years Post Randomization (Key Secondary)
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.
Measured: 5 years
Mean Duration of VOCs Leading to a Healthcare Visit Over the First-year Post Randomization
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.
Measured: 1 year
Number of Participants Free From VOCs Leading to a Healthcare Visit Over the First-year Post Randomization
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.
Measured: 1 year
Percentage of Participants Free From VOCs Leading to a Healthcare Visit Over the First-year Post Randomization
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.
Measured: 1 year
Time to First and Second VOCs Leading to a Healthcare Visit Over the First-year Post Randomization
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.
Measured: 1 year
Annualized Rate of Visits to Clinic, Emergency Room (ER) and Hospitalizations, Both Overall and VOC-related Over the First-year Post-randomization
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)
Measured: 1 year
Annualized Days of Visits to Clinic, Emergency Room (ER) and Hospitalizations, Both Overall and VOC-related Over the First-year Post-randomization
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).
Measured: 1 year
Evolution of Albumin Creatinine Ratio (ACR) Over the First-year Post-randomization (Change From Baseline)
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.
Measured: Over first year post-randomization (Baseline, Week 27 Day 1, Week 51 Day 1)
Evolution of Albuminuria (Urine Microalbumin) Over the First-year Post-randomization (Change From Baseline)
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.
Measured: Over first year post-randomization (Baseline, Week 27 Day 1, Week 51 Day 1)
Pharmacokinetic (PK) Profile of Crizanlizumab: AUCd15, AUCtau
To characterize the pharmacokinetic (PK) profile of crizanlizumab at 5.0 and 7.5 mg/kg: Area under the (concentration-time profile) curve.
Measured: AUCd15 (first-dose) was assessed at W1D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose) to W3D1; AUCtau (steady-state) was assessed at W15D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose), W15D2, W15D4, W16D1, W17D1, W18D1, W19D1
PK Profile of Crizanlizumab: Cmax
To characterize the pharmacokinetic (PK) profile of crizanlizumab at 5.0 and 7.5 mg/kg: Maximum concentration.
Measured: first-dose was assessed at W1D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose) through to W3D1; steady-state was assessed at W15D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose), W15D2, W15D4, W16D1, W17D1, W18D1, W19D1
PK Profile of Crizanlizumab: Tmax
To characterize the pharmacokinetic (PK) profile of crizanlizumab at 5.0 and 7.5 mg/kg: Time to maximum concentration.
Measured: first-dose was assessed at W1D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose) through to W3D1; steady-state was assessed at W15D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose), W15D2, W15D4, W16D1, W17D1, W18D1, W19D1
PK Profile of Crizanlizumab: Half-life
To characterize the pharmacokinetic (PK) profile of crizanlizumab at 5.0 and 7.5 mg/kg: half life.
Measured: steady-state was assessed at W15D1 (pre-dose, 0.5 hr, 2 hrs and 4 hrs post-dose), W15D2, W15D4, W16D1, W17D1, W18D1, W19D1
PD Parameter (P-selectin Inhibition)
To characterize the pharmacodynamic (PD) of crizanlizumab at 5.0 and 7.5 mg/kg: P-selectin inhibition (% inhibition multipled by hr)
Measured: AUCd15 (first dose): W1D1, W1D2, W1D4, W2D1 and W3D1; steady state: W15D1, W15D2, W15D4, W16D1, W17D1 W18D1 and W19D1
Annualized Rate of Various Subtypes of VOCs Leading to a Healthcare Visit at 1 Year
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the annualized rate of VOCs leading to healthcare visit. VOC is defined as pain crisis 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: any visit to a medical facility such as 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).
Measured: 1 year
Annualized Rate of Various Subtypes of VOCs Leading to a Healthcare Visit at 5 Years
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the annualized rate of 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.
Healthcare visit is defined as any visit to a medical facility such as emergency room, hospital and/or office visit, which includes pain management of VOC in situ.
The annualized rate of VOC leading to healthcare visit is the number of VOC leading to healthcare visit multiplied by 365 and divided by the number of days in the observation period.
Measured: 5 years
Annualized Rate of All VOCs Leading to a Healthcare Visit and Treated at Home at 5 Years
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the rates of all VOCs (managed at home + leading to healthcare visit).
Measured: 5 years
Number of VOCs Managed at Home at Year 1
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the number of VOC events that were managed at home.
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. 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 was allowed.
Measured: 1 year
Number of VOCs Managed at Home at 5 Years
To compare the efficacy of 5.0 mg/kg versus placebo and 7.5 mg/kg of crizanlizumab versus placebo on the number of VOC events that were managed at home.
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. 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 was allowed.
Measured: 5 years
Absolute Change From Baseline in Hemoglobin
To assess safety of crizanlizumab over the study period.
Measured: 5 years
Growth and Sexual Maturity Assessment in Adolescents (Tanner Stage)
To assess safety of crizanlizumab over the study period.
Measured: 5 years
Immunogenicity: Measurement of Anti-drug Antibodies (ADA) to Crizanlizumab
To assess immunogenicity of crizanlizumab over the study period.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2019
- Study start26 Jul 2019
- Primary completion31 Aug 2022
- Study completion5 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell Disease (SCD)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 253 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03814746
- Sponsor's protocol ID
- CSEG101A2301
- Other IDs
- 2017-001746-10, 2017
- Acronym
- STAND
- Status last verified
- Sep 2026
- First submitted
- 21 Jan 2019
- Last updated
- 14 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
- Expanded access
- Available
Publications
Abboud MR, Cancado RD, De Montalembert M, Smith WR, Rimawi H, Voskaridou E, Guvenc B, Ataga KI, Keefe D, Grosch K, Watson J, Reshetnyak E, Nassin ML, Dei-Adomakoh Y. Crizanlizumab with or without hydroxyurea in patients with sickle cell disease (STAND): primary analyses from a placebo-controlled, randomised, double-blind, phase 3 trial. Lancet Haematol. 2025 Apr;12(4):e248-e257. doi: 10.1016/S2352-3026(24)00384-3. Epub 2025 Mar 12.
PubMed 40088922Obadina M, Wilson S, Derebail VK, Little J. Emerging Therapies and Advances in Sickle Cell Disease with a Focus on Renal Manifestations. Kidney360. 2023 Jul 1;4(7):997-1005. doi: 10.34067/KID.0000000000000162. Epub 2023 May 31.
PubMed 37254256