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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05648968
Registration number
NCT05648968
Ethics application status
Date submitted
23/11/2022
Date registered
13/12/2022
Titles & IDs
Public title
A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia
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Scientific title
A Phase 3, Randomized, Double-blind, Study to Assess Efficacy and Safety of Ianalumab (VAY736) Versus Placebo in Warm Autoimmune Hemolytic Anemia (wAIHA) Patients Who Failed at Least One Line of Treatment
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Secondary ID [1]
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2022-001773-31
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Secondary ID [2]
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CVAY736O12301
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Universal Trial Number (UTN)
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Trial acronym
VAYHIA
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Warm Autoimmune Hemolytic Anemia (wAIHA)
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Condition category
Condition code
Blood
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Anaemia
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Blood
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Anaemia
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Blood
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Haematological diseases
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Inflammatory and Immune System
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0
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Autoimmune diseases
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Blood
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0
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0
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Other blood disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Other - Ianalumab
Treatment: Drugs - Placebo
Experimental: Ianalumab low dose - Participants will receive low dose ianalumab intravenously
Experimental: Ianalumab high dose - Participants will receive high dose ianalumab intravenously
Placebo comparator: Placebo - Participants will receive placebo intravenously
Treatment: Other: Ianalumab
i.v. infusion, prepared from concentrate solution
Treatment: Drugs: Placebo
i.v. infusion, prepared from matching placebo
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Intervention code [1]
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Treatment: Other
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Intervention code [2]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Binary variable indicating whether a patient achieves a durable response
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Assessment method [1]
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Durable response: hemoglobin level =10 g/dL and =2 g/dL increase from baseline, for a period of at least eight consecutive weeks between W9 and W25, in the absence of rescue medication or prohibited treatment
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Timepoint [1]
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Randomization to Week 25
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Secondary outcome [1]
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Duration of response (Key Secondary)
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Assessment method [1]
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• For patients who previously reached durable response:
Time from first hemoglobin assessment showing durable response to confirmed loss of durable response, defined as the first of the following events:
* hemoglobin level \<10 g/dL in at least two consecutive weekly assessments,
* start of any rescue medication or prohibited treatment,
* death; • For patients who did not achieve the durable response according to primary endpoint definition: duration will be 0 days
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Timepoint [1]
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Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [2]
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Time from randomization to start of durable response in each treatment group
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Assessment method [2]
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Durable response is defined as in primary endpoint.
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Timepoint [2]
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Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [3]
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Time from randomization to start of first response in each treatment group
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Assessment method [3]
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Response is defined as hemoglobin level of at least 10 g/dL and an increase of at least 2 g/dl from baseline, or normalization of hemoglobin (at least 11 g/dL for women and 12 g/dL for men), without biochemical resolution of hemolysis.
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Timepoint [3]
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Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [4]
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Time from randomization to start of complete response in each treatment group
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Assessment method [4]
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Complete response is defined as normalization of hemoglobin levels and no evidence of hemolysis (normal levels of indirect bilirubin, LDH, haptoglobin and reticulocytes), in the absence of red blood cell transfusions.
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Timepoint [4]
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Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [5]
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Response rate
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Assessment method [5]
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Assessment of quality of response in each treatment group.
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Timepoint [5]
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Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [6]
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Complete response rate
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Assessment method [6]
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Assessment of complete response rate in each treatment group.
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Timepoint [6]
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Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [7]
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Hemoglobine Levels
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Assessment method [7]
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Assessment of hemoglobin levels in each treatment group.
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Timepoint [7]
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Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [8]
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Number of participants who received rescue treatment (overall & by type of rescue treatment)
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Assessment method [8]
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This is to assess the need for rescue treatment in all treatment groups, measured as time-standardized numbers of each type of rescue treatment and as change from baseline in time-standardized number of transfusions.
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Timepoint [8]
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Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [9]
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Percentage of participants who received rescue treatment (overall & by type of rescue treatment)
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Assessment method [9]
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This is to assess the need for rescue treatment in all treatment groups.
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Timepoint [9]
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0
Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [10]
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Change from baseline in the the frequency and absolute number of CD19+ B cell counts
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Assessment method [10]
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Change from baseline in the the frequency and absolute number of CD19+ B cell counts in whole blood
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Timepoint [10]
0
0
Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [11]
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Time to first occurrence of B cell recovery, defined as =80% of baseline or =50 cells/µL
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Assessment method [11]
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Time to first occurrence of B cell recovery, defined as =80% of baseline or =50 cells/µL in whole blood
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Timepoint [11]
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Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [12]
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Change from baseline in immunoglobulin levels
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Assessment method [12]
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Change from baseline in immunoglobulin levels (change in titers of IgG, IgM, IgA)
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Timepoint [12]
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Randomization until month 30
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Secondary outcome [13]
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Change from baseline in the 8 domain scores and in the summary scores (PCS, MCS) of SF-36 questionnaire
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Assessment method [13]
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SF-36 v2.0 (acute) includes 36 items that assess general health related quality of life covering 8 domains: physical functioning, role functioning, bodily pain, general health, vitality, social functioning, role-emotional health and mental health.
Scores for the 8 domains are generated, as well as a physical component summary (PCS) score and a mental component summary (MCS) score.
Responses to items are based on a 5-point Likert scale. Scores below 50 indicate less than average health, while scores above 50 indicate better than average health.
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Timepoint [13]
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Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [14]
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Change from baseline in the T-score of PROMIS Fatigue-13a questionnaire
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Assessment method [14]
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Assessment of quality of life in each treatment group. The PROMIS Short Form v1.0 Fatigue-13a includes 13 items that assess fatigue. All items in the PROMIS-Fatigue-13a utilize a 5-point response scale (e.g., not at all, a little bit, somewhat, quite a bit, very much).
Higher scores on the PROMIS-Fatigue-13a represent greater fatigue.
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Timepoint [14]
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Randomization to end of study (up to 39 months after randomization of last patient)
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Secondary outcome [15]
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Ianalumab PK parameter - AUClast
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Assessment method [15]
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AUClast: area under the curve from time zero to last measurable concentration sampling time (tlast).
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Timepoint [15]
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After first dose of study treatment (pre-dose, 2 hours, 336 hours, 672 hours post dose) and after last dose (pre-dose, 2 hours, 336 hours, 672 hours, 1344 hours, 2016 hours and 3360 hours post dose).
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Secondary outcome [16]
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Ianalumab PK parameter - AUCtau
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Assessment method [16]
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AUCtau: the AUV calculated to the end of a dosing interval (tau).
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Timepoint [16]
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After first dose of study treatment (pre-dose, 2 hours, 336 hours, 672 hours post dose) and after last dose (pre-dose, 2 hours, 336 hours, 672 hours, 1344 hours, 2016 hours and 3360 hours post dose).
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Secondary outcome [17]
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Ianalumab PK parameter - Accumulation ratio Racc
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Assessment method [17]
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Accumulation ratio calculated using AUC values obtained between last and first dose
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Timepoint [17]
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After last dose (pre-dose, 2 hours, 336 hours, 672 hours, 1344 hours, 2016 hours and 3360 hours post dose).
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Secondary outcome [18]
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Ianalumab PK parameter - Cmax
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Assessment method [18]
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Maximum (peak) observed plasma, blood, serum or other body fluid drug concentration
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Timepoint [18]
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After first dose of study treatment (pre-dose, 2 hours, 336 hours, 672 hours post dose) and after last dose (pre-dose, 2 hours, 336 hours, 672 hours, 1344 hours, 2016 hours and 3360 hours post dose).
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Secondary outcome [19]
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Ianalumab PK parameter - Tmax
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Assessment method [19]
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Time to reach maximum (peak) plasma, blood, serum or other body fluid drug concentration
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Timepoint [19]
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After first dose of study treatment (pre-dose, 2 hours, 336 hours, 672 hours post dose) and after last dose (pre-dose, 2 hours, 336 hours, 672 hours, 1344 hours, 2016 hours and 3360 hours post dose).
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Secondary outcome [20]
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Immunogenicity of ianalumab
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Assessment method [20]
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Incidence and titer of anti-ianalumab antibodies in serum (ADA assay) over time. Confirmed anti-drug-antibody positive samples will be further characterized for neutralizing capacity.
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Timepoint [20]
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Randomization to end of study (up to 39 months after randomization of last patient)
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Eligibility
Key inclusion criteria
Key
* 18 years and older at time of signing consent
* Patients with primary or secondary wAIHA documented by positive direct antiglobulin test specific for anti-IgG or anti-IgA, who had an insufficient response to, or relapsed after at least one line of treatment, including patients with steroid resistance, dependence or intolerance
* Hemoglobin concentration at screening and at Week 1 >=5 g/dL and <10 g/dL, associated with presence of symptoms related to anemia
* The dose of supportive care must be stable for at least 4 weeks prior to randomization into the study
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* wAIHA secondary to hematologic disease involving bone marrow (e.g., CLL) or another immunologic disease requiring prohibited medication as per protocol. Patients with autoimmune diseases after wash-out from the treatments are allowed.
* Presence of other forms of AIHA (cold or intermediate forms), Evans Syndrome or other cytopenias
* Prior use of B-cell depleting therapy (e.g., rituximab) within 12 weeks prior to randomization, or without hematological response to the last course of B-cell depleting therapy
* Neutrophils: <1000/mm3
* Serum creatinine >1.5 × upper limit of normal (ULN)
* Immunoglobulin G (IgG) <5g/L
* Active viral, bacterial or other infections (including tuberculosis and SARS-CoV-2) requiring systemic treatment at time of screening, or history of recurrent clinically significant infection
* Positivity for hepatitis C virus, hepatitis B surface antigen (HBsAg), or hepatitis B core antibody (HBcAb). HBcAb positive patients can be enrolled if HBsAg negative, HBV DNA negative, no pre-existing liver fibrosis is present and antiviral prophylaxis is given.
* Known history of primary or secondary immunodeficiency, or a positive human immune deficiency virus (HIV) test result
* Live or live-attenuated vaccination within 4 weeks before randomization
* History of splenectomy
Other protocol-defined Inclusion/Exclusion may apply.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
30/12/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
8/02/2029
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Actual
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Sample size
Target
90
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
ACT,VIC
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Recruitment hospital [1]
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Novartis Investigative Site - Canberra
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Recruitment hospital [2]
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Novartis Investigative Site - Prahran
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Recruitment postcode(s) [1]
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2605 - Canberra
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Recruitment postcode(s) [2]
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3181 - Prahran
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Colorado
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United States of America
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Florida
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United States of America
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Illinois
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United States of America
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Indiana
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United States of America
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Michigan
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United States of America
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Minnesota
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United States of America
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New Jersey
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United States of America
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New York
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United States of America
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Ohio
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United States of America
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Washington
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Argentina
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Buenos Aires
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Argentina
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Buenos aires
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China
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Guangdong
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China
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Hubei
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China
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Jiangsu
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China
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Yunnan
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China
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Zhejiang
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China
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Dalian
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China
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Tianjin
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France
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Cedex 09
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France
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Blois Cedex
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France
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Caen
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France
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Creteil
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France
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Lille Cedex
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France
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Nantes Cedex 1
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France
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Nice
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France
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Toulouse
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France
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Vandoeuvre Les Nancy
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Germany
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Dresden
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Germany
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Essen
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Germany
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Frankfurt
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Germany
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Giessen
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Germany
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Greifswald
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Germany
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Hannover
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Hungary
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Debrecen
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India
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India
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Uttar Pradesh
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India
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New Delhi
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Israel
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Afula
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Israel
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Kfar Saba
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Israel
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Petach Tikva
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Israel
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Zerifin
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Italy
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AV
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Italy
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BA
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Italy
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MI
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Italy
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VI
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Italy
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Novara
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Japan
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Chiba
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Japan
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Ehime
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Japan
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Fukuoka
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Gifu
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Japan
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Hyogo
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Japan
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Kanagawa
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Japan
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Osaka
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Tokyo
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Aomori
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Japan
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Yamagata
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Malaysia
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Sarawak
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Malaysia
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Johor Bahru
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Malaysia
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Kuala Lumpur
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Malaysia
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Penang
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Malaysia
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Pulau Pinang
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Malaysia
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Selangor
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Romania
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Bucuresti
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Singapore
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Singapore
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Spain
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Catalunya
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Spain
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Murcia
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Taiwan
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Taoyuan
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Thailand
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Bangkok
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Thailand
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Chiang Mai
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United Kingdom
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Birmingham
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United Kingdom
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Leeds
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United Kingdom
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Novartis Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of this study is to evaluate efficacy and safety of ianalumab compared to placebo in patients with warm autoimmune hemolytic anemia, who failed at least one line of treatment.
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Trial website
https://clinicaltrials.gov/study/NCT05648968
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
0
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Novartis Pharmaceuticals
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Address
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Novartis Pharmaceuticals
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Country
0
0
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Phone
0
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Fax
0
0
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Email
0
0
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Contact person for public queries
Name
0
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Novartis Pharmaceuticals
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1-888-669-6682
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
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 expert 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 is available according to the process described on www.clinicalstudydatarequest.com.
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://www.clinicalstudydatarequest.com
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT05648968