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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05653349




Registration number
NCT05653349
Ethics application status
Date submitted
23/11/2022
Date registered
16/12/2022

Titles & IDs
Public title
Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP)
Scientific title
A Phase III, Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (VAYHIT1)
Secondary ID [1] 0 0
CVAY736I12301
Universal Trial Number (UTN)
Trial acronym
VAYHIT1
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Immune Thrombocytopenia (ITP) 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Ianalumab
Treatment: Drugs - Placebo
Treatment: Drugs - Corticosteroids

Experimental: Ianalumab Lower dose - Lower dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)

Experimental: Ianalumab Higher dose - Higher dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)

Placebo comparator: Placebo - Placebo administered intravenously with corticosteroids oral or parental (if clinically justified)


Treatment: Other: Ianalumab
Intravenous infusion, prepared from concentrate solution

Treatment: Drugs: Placebo
Intravenous infusion, prepared from matching placebo

Treatment: Drugs: Corticosteroids
Oral or parental (if clinically justified)
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
Time from randomization to treatment failure (TTF)
Timepoint [1] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [1] 0 0
Complete Response (CR) rate in each treatment group
Timepoint [1] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [2] 0 0
Response (R) rate 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 to complete 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
Duration of 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
Stable response at 6 months
Timepoint [5] 0 0
At 6 months
Secondary outcome [6] 0 0
Stable response at 1 year
Timepoint [6] 0 0
At 1 year
Secondary outcome [7] 0 0
Percentage of participants with bleeding events overall and by World Health Organization (WHO) bleeding scale severity
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 with bleeding events overall and by World Health Organization (WHO) bleeding scale severity
Timepoint [8] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [9] 0 0
Number of participants receiving rescue treatment (cummulative dose/duration of steroids exposure)
Timepoint [9] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [10] 0 0
Percentage of participants receiving rescue treatment (cummulative dose/duration of steroids exposure)
Timepoint [10] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [11] 0 0
Cumulative dose/duration of steroids exposure
Timepoint [11] 0 0
From screening to end of study (up to 39 months after randomization of last patient)
Secondary outcome [12] 0 0
Change from baseline on T scores of the PROMIS SF v1.0 Fatigue 13a
Timepoint [12] 0 0
From screening (baseline) till end of study (up to 39 months after randomization of last patient)
Secondary outcome [13] 0 0
Change from baseline in ITP-PAQ domain scores
Timepoint [13] 0 0
From screening (baseline) till end of study (up to 39 months after randomization of last patient)
Secondary outcome [14] 0 0
Change from baseline in frequency of CD19+ B cell counts
Timepoint [14] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [15] 0 0
Change from baseline in absolute number of CD19+ B cell counts
Timepoint [15] 0 0
Randomization to end of study (up to 39 months after randomization of last patient)
Secondary outcome [16] 0 0
Time to first occurrence of B-cell recovery
Timepoint [16] 0 0
Randomization to end of study (up to 39 months after randomized of last patient)
Secondary outcome [17] 0 0
Change from baseline in inmmunoglobulins
Timepoint [17] 0 0
Randomization to end of study (up to 39 months after last randomized patients)
Secondary outcome [18] 0 0
PK parameters: AUClast
Timepoint [18] 0 0
After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
Secondary outcome [19] 0 0
PK parameter: AUCtau
Timepoint [19] 0 0
After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
Secondary outcome [20] 0 0
PK parameters: Cmax
Timepoint [20] 0 0
After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
Secondary outcome [21] 0 0
PK parameters: Tmax
Timepoint [21] 0 0
After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
Secondary outcome [22] 0 0
PK parameters: Accumulation ratio Racc
Timepoint [22] 0 0
After last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
Secondary outcome [23] 0 0
Incidence of anti-ianalumab antibodies in serum (ADA assay) over time
Timepoint [23] 0 0
Up to Week 33
Secondary outcome [24] 0 0
Titer of anti-ianalumab antibodies in serum (ADA assay) over time
Timepoint [24] 0 0
Up to Week 33

Eligibility
Key inclusion criteria
* Signed informed consent prior to participation in the study.
* Male or female participants aged 18 years and older on the day of signing informed consent
* Primary ITP diagnosed within 3 months before initiating first-line ITP therapy (corticosteroids, IVIG)
* Platelet count below 30 G/L before starting any first-line ITP therapy (corticosteroids, IVIG)
* Response (platelet count >=50 G/L) to corticosteroids (+/- IVIG) at any time prior to randomization. Note: Platelet count measured within 7 days of platelet transfusion will not be considered as response.

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Evans syndrome or any other cytopenia (patients with anemia related to bleeding or iron deficiency are eligible)
* Current life-threatening bleeding
* Previous ITP treatment, including splenectomy, except for corticosteroids and/or IVIG initiated as first-line therapy for up to 28 days before randomization and rescue corticosteroids and/or IVIG given prior to confirmed diagnosis of primary ITP .
* Prior use of B-cell depleting therapy (e.g., rituximab).
* Absolute neutrophil count below 1.0 G/L at randomization
* Participants with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid

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
6/03/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
18/12/2028
Actual
Sample size
Target
225
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
ACT,QLD,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Canberra
Recruitment hospital [2] 0 0
Novartis Investigative Site - Wooloongabba
Recruitment hospital [3] 0 0
Novartis Investigative Site - Clayton
Recruitment hospital [4] 0 0
Novartis Investigative Site - Prahran
Recruitment postcode(s) [1] 0 0
2605 - Canberra
Recruitment postcode(s) [2] 0 0
4102 - Wooloongabba
Recruitment postcode(s) [3] 0 0
3168 - Clayton
Recruitment postcode(s) [4] 0 0
3181 - Prahran
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Indiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Maryland
Country [8] 0 0
United States of America
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Minnesota
Country [9] 0 0
United States of America
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Montana
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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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Ohio
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Oklahoma
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Pennsylvania
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Rhode Island
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United States of America
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Texas
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United States of America
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Utah
Country [18] 0 0
Argentina
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Buenos Aires
Country [19] 0 0
Argentina
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San Miguel De Tucuman
Country [20] 0 0
Argentina
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Buenos aires
Country [21] 0 0
Argentina
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Cordoba
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Austria
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Tyrol
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Austria
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Salzburg
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Austria
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Wien
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Belgium
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Brasschaat
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Belgium
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Brugge
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Belgium
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Leuven
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Belgium
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Roeselare
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Bulgaria
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Plovdiv
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Bulgaria
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Sofia
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Bulgaria
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Varna
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China
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Chongqing
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China
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Guangdong
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China
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China
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China
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China
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Shanxi
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China
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China
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Giessen
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Chiba
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Fukuoka
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Sarawak
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Johor Bahru
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Kuala Lumpur
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Penang
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Selangor
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Mexico CP
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Mexico
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Michoacan
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Mexico
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Nuevo Leon
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Norway
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Gralum
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Romania
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District 2
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Romania
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Bucharest
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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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Andalucia
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Spain
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Castilla Y Leon
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Spain
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Catalunya
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Madrid
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Murcia
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Bangkok
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Thailand
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Chiang Mai
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TUR
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Aydin
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Istanbul
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Izmir
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Turkey
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Samsun
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United Kingdom
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Cornwall
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United Kingdom
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Birmingham
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Glasgow
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Leeds
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Liverpool
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Southampton
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Vietnam
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Hanoi
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Vietnam
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Ho Chi Minh

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 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
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/NCT05653349