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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
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
Secondary ID [1] 0 0
2022-001773-31
Secondary ID [2] 0 0
CVAY736O12301
Universal Trial Number (UTN)
Trial acronym
VAYHIA
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Warm Autoimmune Hemolytic Anemia (wAIHA) 0 0
Condition category
Condition code
Blood 0 0 0 0
Anaemia
Blood 0 0 0 0
Anaemia
Blood 0 0 0 0
Haematological diseases
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Other
Intervention code [2] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Binary variable indicating whether a patient achieves a durable response
Timepoint [1] 0 0
Randomization to Week 25
Secondary outcome [1] 0 0
Duration of response (Key Secondary)
Timepoint [1] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [2] 0 0
Time from randomization to start of durable response in each treatment group
Timepoint [2] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [3] 0 0
Time from randomization to start of first response in each treatment group
Timepoint [3] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [4] 0 0
Time from randomization to start of complete response in each treatment group
Timepoint [4] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [5] 0 0
Response rate
Timepoint [5] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [6] 0 0
Complete response rate
Timepoint [6] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [7] 0 0
Hemoglobine Levels
Timepoint [7] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [8] 0 0
Number of participants who received rescue treatment (overall & by type of rescue treatment)
Timepoint [8] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [9] 0 0
Percentage of participants who received rescue treatment (overall & by type of rescue treatment)
Timepoint [9] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [10] 0 0
Change from baseline in the the frequency and absolute number of CD19+ B cell counts
Timepoint [10] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [11] 0 0
Time to first occurrence of B cell recovery, defined as =80% of baseline or =50 cells/µL
Timepoint [11] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [12] 0 0
Change from baseline in immunoglobulin levels
Timepoint [12] 0 0
Randomization until month 30
Secondary outcome [13] 0 0
Change from baseline in the 8 domain scores and in the summary scores (PCS, MCS) of SF-36 questionnaire
Timepoint [13] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [14] 0 0
Change from baseline in the T-score of PROMIS Fatigue-13a questionnaire
Timepoint [14] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [15] 0 0
Ianalumab PK parameter - AUClast
Timepoint [15] 0 0
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).
Secondary outcome [16] 0 0
Ianalumab PK parameter - AUCtau
Timepoint [16] 0 0
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).
Secondary outcome [17] 0 0
Ianalumab PK parameter - Accumulation ratio Racc
Timepoint [17] 0 0
After last dose (pre-dose, 2 hours, 336 hours, 672 hours, 1344 hours, 2016 hours and 3360 hours post dose).
Secondary outcome [18] 0 0
Ianalumab PK parameter - Cmax
Timepoint [18] 0 0
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).
Secondary outcome [19] 0 0
Ianalumab PK parameter - Tmax
Timepoint [19] 0 0
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).
Secondary outcome [20] 0 0
Immunogenicity of ianalumab
Timepoint [20] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)

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

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/12/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
8/02/2029
Actual
Sample size
Target
90
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
ACT,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Canberra
Recruitment hospital [2] 0 0
Novartis Investigative Site - Prahran
Recruitment postcode(s) [1] 0 0
2605 - Canberra
Recruitment postcode(s) [2] 0 0
3181 - Prahran
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
New Jersey
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
Washington
Country [11] 0 0
Argentina
State/province [11] 0 0
Buenos Aires
Country [12] 0 0
Argentina
State/province [12] 0 0
Buenos aires
Country [13] 0 0
China
State/province [13] 0 0
Guangdong
Country [14] 0 0
China
State/province [14] 0 0
Hubei
Country [15] 0 0
China
State/province [15] 0 0
Jiangsu
Country [16] 0 0
China
State/province [16] 0 0
Yunnan
Country [17] 0 0
China
State/province [17] 0 0
Zhejiang
Country [18] 0 0
China
State/province [18] 0 0
Dalian
Country [19] 0 0
China
State/province [19] 0 0
Tianjin
Country [20] 0 0
France
State/province [20] 0 0
Cedex 09
Country [21] 0 0
France
State/province [21] 0 0
Blois Cedex
Country [22] 0 0
France
State/province [22] 0 0
Caen
Country [23] 0 0
France
State/province [23] 0 0
Creteil
Country [24] 0 0
France
State/province [24] 0 0
Lille Cedex
Country [25] 0 0
France
State/province [25] 0 0
Nantes Cedex 1
Country [26] 0 0
France
State/province [26] 0 0
Nice
Country [27] 0 0
France
State/province [27] 0 0
Toulouse
Country [28] 0 0
France
State/province [28] 0 0
Vandoeuvre Les Nancy
Country [29] 0 0
Germany
State/province [29] 0 0
Dresden
Country [30] 0 0
Germany
State/province [30] 0 0
Essen
Country [31] 0 0
Germany
State/province [31] 0 0
Frankfurt
Country [32] 0 0
Germany
State/province [32] 0 0
Giessen
Country [33] 0 0
Germany
State/province [33] 0 0
Greifswald
Country [34] 0 0
Germany
State/province [34] 0 0
Hannover
Country [35] 0 0
Hungary
State/province [35] 0 0
Debrecen
Country [36] 0 0
India
State/province [36] 0 0
TamilNadu
Country [37] 0 0
India
State/province [37] 0 0
Telangana
Country [38] 0 0
India
State/province [38] 0 0
Uttar Pradesh
Country [39] 0 0
India
State/province [39] 0 0
New Delhi
Country [40] 0 0
Israel
State/province [40] 0 0
Afula
Country [41] 0 0
Israel
State/province [41] 0 0
Kfar Saba
Country [42] 0 0
Israel
State/province [42] 0 0
Petach Tikva
Country [43] 0 0
Israel
State/province [43] 0 0
Zerifin
Country [44] 0 0
Italy
State/province [44] 0 0
AV
Country [45] 0 0
Italy
State/province [45] 0 0
BA
Country [46] 0 0
Italy
State/province [46] 0 0
MI
Country [47] 0 0
Italy
State/province [47] 0 0
VI
Country [48] 0 0
Italy
State/province [48] 0 0
Novara
Country [49] 0 0
Japan
State/province [49] 0 0
Chiba
Country [50] 0 0
Japan
State/province [50] 0 0
Ehime
Country [51] 0 0
Japan
State/province [51] 0 0
Fukuoka
Country [52] 0 0
Japan
State/province [52] 0 0
Gifu
Country [53] 0 0
Japan
State/province [53] 0 0
Hyogo
Country [54] 0 0
Japan
State/province [54] 0 0
Kanagawa
Country [55] 0 0
Japan
State/province [55] 0 0
Osaka
Country [56] 0 0
Japan
State/province [56] 0 0
Tokyo
Country [57] 0 0
Japan
State/province [57] 0 0
Aomori
Country [58] 0 0
Japan
State/province [58] 0 0
Yamagata
Country [59] 0 0
Malaysia
State/province [59] 0 0
Sarawak
Country [60] 0 0
Malaysia
State/province [60] 0 0
Johor Bahru
Country [61] 0 0
Malaysia
State/province [61] 0 0
Kuala Lumpur
Country [62] 0 0
Malaysia
State/province [62] 0 0
Penang
Country [63] 0 0
Malaysia
State/province [63] 0 0
Pulau Pinang
Country [64] 0 0
Malaysia
State/province [64] 0 0
Selangor
Country [65] 0 0
Romania
State/province [65] 0 0
Bucuresti
Country [66] 0 0
Singapore
State/province [66] 0 0
Singapore
Country [67] 0 0
Spain
State/province [67] 0 0
Catalunya
Country [68] 0 0
Spain
State/province [68] 0 0
Murcia
Country [69] 0 0
Taiwan
State/province [69] 0 0
Taoyuan
Country [70] 0 0
Thailand
State/province [70] 0 0
Bangkok
Country [71] 0 0
Thailand
State/province [71] 0 0
Chiang Mai
Country [72] 0 0
United Kingdom
State/province [72] 0 0
Birmingham
Country [73] 0 0
United Kingdom
State/province [73] 0 0
Leeds
Country [74] 0 0
United Kingdom
State/province [74] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
1-888-669-6682
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.clinicalstudydatarequest.com


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