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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05885555




Registration number
NCT05885555
Ethics application status
Date submitted
15/05/2023
Date registered
2/06/2023

Titles & IDs
Public title
A Study of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least Two Lines of Therapies
Scientific title
A Phase 2 Study to Evaluate the Efficacy and Safety of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least a Corticosteroid and a Thrombopoietin Receptor Agonist (TPO-RA)
Secondary ID [1] 0 0
2022-503041-21
Secondary ID [2] 0 0
CVAY736Q12201
Universal Trial Number (UTN)
Trial acronym
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

Experimental: Single-arm - All eligible participants will receive ianalumab at the same dose.


Treatment: Other: Ianalumab
Intravenous infusion, prepared from concentrate solution
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Confirmed response
Timepoint [1] 0 0
Between Week 1 Day 1 and Week 25 Day 1
Secondary outcome [1] 0 0
Time to confirmed response
Timepoint [1] 0 0
From Week 1 Day 1 to Week 25 Day 1
Secondary outcome [2] 0 0
Duration of confirmed response
Timepoint [2] 0 0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [3] 0 0
Complete Response rate at each timepoint
Timepoint [3] 0 0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [4] 0 0
Response rate at each timepoint
Timepoint [4] 0 0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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
Bleeding events according to the Modified World Health Organization (WHO) bleeding scale
Timepoint [7] 0 0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [8] 0 0
Percentage of participants with bleeding events according to the Modified World Health Organization (WHO) bleeding scale
Timepoint [8] 0 0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [9] 0 0
Number of participants who received rescue treatment
Timepoint [9] 0 0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [10] 0 0
Percentage of participants who received rescue treatment
Timepoint [10] 0 0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [11] 0 0
Change from baseline in the frequency of CD19+ B-cell counts
Timepoint [11] 0 0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [12] 0 0
Change from baseline in the absolute number of CD19+ B-cell counts
Timepoint [12] 0 0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [13] 0 0
Time to first occurrence of B-cell recovery defined as =80% of baseline =50 cells/µL
Timepoint [13] 0 0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [14] 0 0
Change from baseline in immunoglobulins
Timepoint [14] 0 0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [15] 0 0
Incidence of anti-ianalumab antibodies in serum (ADA assay) over time
Timepoint [15] 0 0
Up to 20 weeks after last dose of ianalumab
Secondary outcome [16] 0 0
Titer of anti-ianalumab antibodies in serum (ADA assay) over time
Timepoint [16] 0 0
Up to 20 weeks after last dose of ianalumab
Secondary outcome [17] 0 0
Ianalumab serum concentrations over time
Timepoint [17] 0 0
First dose (pre-dose, 2, 168, 336, 504, 672 hours post-dose); Subsequent doses (pre-dose and 2 hours post-dose); Last dose (pre-dose, 2 336, 672, 1344, 2016, 3360 hours post-dose)
Secondary outcome [18] 0 0
Confirmed response (CR) in the second course
Timepoint [18] 0 0
Second course Week 1 Day1 to second course Week 25 Day1
Secondary outcome [19] 0 0
Time to confirmed response in the second course
Timepoint [19] 0 0
Second course Week 1 Day 1 to second course Week 25 Day 1
Secondary outcome [20] 0 0
Duration of confirmed response in the second course
Timepoint [20] 0 0
Second course Week 1 Day 1 to second course Week 25 Day 1
Secondary outcome [21] 0 0
Response in the second course
Timepoint [21] 0 0
Second course Week 1 Day 1 to second course Week 25 Day 1
Secondary outcome [22] 0 0
Complete Response in the second course
Timepoint [22] 0 0
Second course Week 1 Day 1 to second course Week 25 Day 1
Secondary outcome [23] 0 0
Bleeding events in the second course according to the Modified World Health Organization (WHO) bleeding scale
Timepoint [23] 0 0
Second course Week 1 Day 1 to second course Week 25 Day 1
Secondary outcome [24] 0 0
Percentage of participants with bleeding events in the retreatment/second courseaccording to the Modified World Health Organization (WHO) bleeding scale
Timepoint [24] 0 0
Second course Week 1 Day 1 to second course Week 25 Day 1
Secondary outcome [25] 0 0
Number of Participants who received rescue treatment after second course
Timepoint [25] 0 0
Second course Week 1 Day 1 to second course Week 25 Day 1
Secondary outcome [26] 0 0
Percentage of participants who received rescue treatment after receiving second course
Timepoint [26] 0 0
Second course Week 1 Day 1 to second course Week 25 Day 1
Secondary outcome [27] 0 0
Titer of anti-ianalumab antibodies in serum (ADA assay) over time for second course
Timepoint [27] 0 0
Second course Week 1 Day 1 until 20 weeks after last dose of ianalumab
Secondary outcome [28] 0 0
Change from start of second course in immunoglobulins
Timepoint [28] 0 0
From second course Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [29] 0 0
Change from start of second course in the absolute number of CD19+ B-cell counts
Timepoint [29] 0 0
From second course Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [30] 0 0
Change from start of second course to end of study in the absolute number of CD19+ B-cell counts
Timepoint [30] 0 0
From second course Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [31] 0 0
Time to first occurrence of B-cell recovery defined as =80% of baseline =50 cells/µL
Timepoint [31] 0 0
From second course Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
Secondary outcome [32] 0 0
Ianalumab serum concentrations over time in the second course
Timepoint [32] 0 0
First dose (pre-dose, 2, 168, 336, 504, 672 hours post-dose); Subsequent doses (pre-dose and 2 hours post-dose); Last dose (pre-dose, 2 336, 672, 1344, 2016, 3360 hours post-dose) in the second course

Eligibility
Key inclusion criteria
* Signed informed consent obtained prior to participation in the study.
* Male or female participants aged 18 years and older on the day of signing informed consent.
* Confirmed diagnosis of primary ITP.

* Prior treatment with at least a corticosteroid (±IVIG) and a TPO-RA:
* Prior additional therapies are allowed; the corticosteroid or the TPO-RA do not need to be the last treatment.
* Prior response to IVIG/anti-D or a corticosteroid (platelet count =50 G/L) that was not maintained.
* At last ITP treatment, loss of response, insufficient response, no response or intolerance.
* Platelet count <30 G/L and assessed as needing treatment (per physician's discretion) at screening. If concomitant ITP medication is clinically indicated, the platelet assessment showing a value <30 G/L must be performed after at least 14 days on a stable dose of a corticosteroid or/and a TPO-RA (less than 10% variation from current dose) and continue stable thereafter.

Key exclusion criteria:

* Diagnosis of secondary thrombocytopenia.
* Platelet or whole blood transfusion, plasmapheresis, or use of any other rescue medications within 14 days before first ianalumab infusion.
* Participants with the following conditions at screening:

* Neutrophils <1000/mm3.
* Immunoglobulin G (IgG) <5 g/L
* Treatment with a B-cell depleting therapy (e.g., rituximab) or anti-B-cell Activating Factor of the TNF Family (BAFF) (e.g., belimumab) within 12 weeks prior to the first administration of ianalumab.
* Immunosuppressant drugs other than corticosteroids within 5 times the elimination half-life of the drug or 14 days before first ianalumab infusion, whichever is longer.
* Prior splenectomy.

Other protocol-defined inclusion/exclusion criteria may apply.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
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
16/08/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
19/03/2029
Actual
Sample size
Target
40
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
District of Columbia
Country [2] 0 0
United States of America
State/province [2] 0 0
Massachusetts
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
United States of America
State/province [5] 0 0
Virginia
Country [6] 0 0
Argentina
State/province [6] 0 0
Buenos Aires
Country [7] 0 0
China
State/province [7] 0 0
Hubei
Country [8] 0 0
China
State/province [8] 0 0
Beijing
Country [9] 0 0
China
State/province [9] 0 0
Jinan
Country [10] 0 0
Czechia
State/province [10] 0 0
Czech Republic
Country [11] 0 0
France
State/province [11] 0 0
Dijon
Country [12] 0 0
France
State/province [12] 0 0
Toulouse
Country [13] 0 0
Germany
State/province [13] 0 0
Dresden
Country [14] 0 0
Germany
State/province [14] 0 0
Giessen
Country [15] 0 0
Germany
State/province [15] 0 0
Jena
Country [16] 0 0
Italy
State/province [16] 0 0
FI
Country [17] 0 0
Italy
State/province [17] 0 0
TO
Country [18] 0 0
Italy
State/province [18] 0 0
TS
Country [19] 0 0
Korea, Republic of
State/province [19] 0 0
Seocho Gu
Country [20] 0 0
Korea, Republic of
State/province [20] 0 0
Seoul
Country [21] 0 0
Malaysia
State/province [21] 0 0
Sabah
Country [22] 0 0
Malaysia
State/province [22] 0 0
Sarawak
Country [23] 0 0
Malaysia
State/province [23] 0 0
Johor Bahru
Country [24] 0 0
Poland
State/province [24] 0 0
Katowice
Country [25] 0 0
Poland
State/province [25] 0 0
Krakow
Country [26] 0 0
Spain
State/province [26] 0 0
Andalucia
Country [27] 0 0
Spain
State/province [27] 0 0
Catalunya
Country [28] 0 0
Spain
State/province [28] 0 0
Madrid
Country [29] 0 0
Turkey
State/province [29] 0 0
TUR
Country [30] 0 0
Turkey
State/province [30] 0 0
Aydin
Country [31] 0 0
Turkey
State/province [31] 0 0
Edirne
Country [32] 0 0
Turkey
State/province [32] 0 0
Izmir
Country [33] 0 0
Turkey
State/province [33] 0 0
Kocaeli
Country [34] 0 0
United Kingdom
State/province [34] 0 0
Glasgow
Country [35] 0 0
United Kingdom
State/province [35] 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 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/NCT05885555