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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05653219




Registration number
NCT05653219
Ethics application status
Date submitted
23/11/2022
Date registered
16/12/2022

Titles & IDs
Public title
A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids
Scientific title
A Phase 3 Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP) Who Had an Insufficient Response or Relapsed After First Line Steroid Treatment (VAYHIT2)
Secondary ID [1] 0 0
CVAY736Q12301
Universal Trial Number (UTN)
Trial acronym
VAYHIT2
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Immune Thrombocytopenia 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 - Eltrombopag
Treatment: Drugs - Placebo

Experimental: Treatment arm 1 - Participants will receive eltrombopag and ianalumab lower dose

Experimental: Treatment arm 2 - Participants will receive eltrombopag and ianalumab higher dose

Placebo comparator: Treatment arm 3 - Participants will receive eltrombopag and placebo


Treatment: Other: Ianalumab
Concentrate for solution for infusion for intravenous use

Treatment: Drugs: Eltrombopag
Film-coated tablet for oral use

Treatment: Drugs: Placebo
Concentrate for solution for infusion for intravenous use.

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 until treatment failure
Timepoint [1] 0 0
Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcome [1] 0 0
Complete Response rate at each timepoint
Timepoint [1] 0 0
Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcome [2] 0 0
Response rate at each timepoint
Timepoint [2] 0 0
Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcome [3] 0 0
Best response rate across all timepoints
Timepoint [3] 0 0
Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcome [4] 0 0
Time to first response/time to first complete response
Timepoint [4] 0 0
Time from randomization up to the longest observed treatment period duration
Secondary outcome [5] 0 0
Duration of response
Timepoint [5] 0 0
Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcome [6] 0 0
Stable response at 6 months
Timepoint [6] 0 0
At 6 months
Secondary outcome [7] 0 0
Stable response at 1 year
Timepoint [7] 0 0
At 1 year
Secondary outcome [8] 0 0
Duration of complete response
Timepoint [8] 0 0
Randomization to end of study (up to 39 months after randomization of last participant)
Secondary outcome [9] 0 0
Rate of participants who successfully taper and discontinue eltrombopag in each treatment arm
Timepoint [9] 0 0
up to week 24
Secondary outcome [10] 0 0
Percentage of participants with bleeding events according to World Health Organization (WHO)
Timepoint [10] 0 0
Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcome [11] 0 0
Number of participants receiving rescue treatment
Timepoint [11] 0 0
Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcome [12] 0 0
Percentage of participants receiving rescue treatment
Timepoint [12] 0 0
Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcome [13] 0 0
Change from baseline in the frequency of CD19+ B-cell counts
Timepoint [13] 0 0
Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcome [14] 0 0
Change from baseline in the absolute number of CD19+ B-cell counts
Timepoint [14] 0 0
Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcome [15] 0 0
Change from baseline on T-score of the PROMIS SF v1.0 Fatigue 13a
Timepoint [15] 0 0
From screening (baseline) until end of study (up 39 months after randomization of last participant)
Secondary outcome [16] 0 0
Change from baseline in ITP PAQ domain scores of symptoms, fatigue, bother (uncomfortable), activity
Timepoint [16] 0 0
From screening (baseline) until end of study (up 39 months after randomization of last participant)
Secondary outcome [17] 0 0
Time to first occurence of B-cell recovery defined as =80% of baseline =50 cells/µL
Timepoint [17] 0 0
Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcome [18] 0 0
Change from baseline in immunoglobulins
Timepoint [18] 0 0
Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcome [19] 0 0
PK parameters: AUClast
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: AUCtau
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: Cmax
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: Tmax
Timepoint [22] 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 [23] 0 0
PK parameters: Accumulation ratio Racc
Timepoint [23] 0 0
After last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
Secondary outcome [24] 0 0
Incidence of anti-ianalumab antibodies in serum (ADA assay) over time
Timepoint [24] 0 0
up to week 33
Secondary outcome [25] 0 0
Titer of anti-ianalumab antibodies in serum (ADA assay) over time
Timepoint [25] 0 0
up to week 33

Eligibility
Key inclusion criteria
Key Inclusion criteria

1. Male or female patients aged 18 years and older on the day of signing the informed consent.
2. A signed informed consent must be obtained prior to participation in the study.
3. A diagnosis of primary ITP, with insufficient response to, or relapse after a first-line corticosteroid therapy ± IVIG.
4. Patient with platelet count <30G/L (whom eltrombopag is clinically indicated as per physician's discretion) and with no contraindication to receive eltrombopag

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

1. ITP patients who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy. However, patients exposed to thrombopoietin receptor agonists (TPO-RAs) for a limited time (max one week) before screening are eligible.
2. Patients with key lab abnormalities and patients with Evans syndrome or any other cytopenia, (patients with low grade anemia related to bleeding or iron deficiency are eligible).
3. Patients with history of clinically significant hematological disorders, or with marked altered hematologic parameters
4. Patients with current or history of life-threatening bleeding
5. Patient that are Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B surface Antigen (HBsAg)/ Hepatitis B core antibody (HBcAb)-positive. HBcAb-positive patients can be enrolled if HBsAg negative, HBV DNA negative, no pre-existing liver fibrosis is present and antiviral prophylaxis is given
6. Patients with known active or uncontrolled infection requiring systemic treatment during screening period
7. Patients with hepatic impairment
8. Patients with concurrent coagulation disorders and/or receiving antiplatelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid (=150 mg daily)
9. Female patients who are pregnant or nursing

Other protocol-defined inclusion/exclusion criteria 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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Clayton
Recruitment hospital [2] 0 0
Novartis Investigative Site - Parkville
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment postcode(s) [2] 0 0
3050 - Parkville
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
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Massachusetts
Country [8] 0 0
United States of America
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Missouri
Country [9] 0 0
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Montana
Country [10] 0 0
United States of America
State/province [10] 0 0
New Jersey
Country [11] 0 0
United States of America
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New York
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
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Oklahoma
Country [14] 0 0
United States of America
State/province [14] 0 0
Pennsylvania
Country [15] 0 0
United States of America
State/province [15] 0 0
Texas
Country [16] 0 0
United States of America
State/province [16] 0 0
Utah
Country [17] 0 0
Argentina
State/province [17] 0 0
Buenos Aires
Country [18] 0 0
Austria
State/province [18] 0 0
Linz
Country [19] 0 0
Austria
State/province [19] 0 0
Vienna
Country [20] 0 0
Austria
State/province [20] 0 0
Wels
Country [21] 0 0
Belgium
State/province [21] 0 0
Leuven
Country [22] 0 0
Belgium
State/province [22] 0 0
Roeselare
Country [23] 0 0
Belgium
State/province [23] 0 0
Yvoir
Country [24] 0 0
China
State/province [24] 0 0
Guangdong
Country [25] 0 0
China
State/province [25] 0 0
Hubei
Country [26] 0 0
China
State/province [26] 0 0
Jiangsu
Country [27] 0 0
China
State/province [27] 0 0
Shandong
Country [28] 0 0
China
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Zhejiang
Country [29] 0 0
China
State/province [29] 0 0
Beijing
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China
State/province [30] 0 0
Ji Nan
Country [31] 0 0
China
State/province [31] 0 0
Tianjin
Country [32] 0 0
Czechia
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Czech Republic
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Czechia
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Praha 10
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Czechia
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Praha
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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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Vandoeuvre Les Nancy
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Germany
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Dresden
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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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Germany
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Jena
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Koeln
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Hungary
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Budapest
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Hungary
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Debrecen
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Uttarakhand
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India
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West Bengal
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India
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Chandigarh
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Italy
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BO
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Italy
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RM
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Italy
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Aichi
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Chiba
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Kumamoto
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Nagasaki
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Osaka
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Yamanashi
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Aomori
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Quezon
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Romania
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District 2
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Bucharest
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Craiova
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Sibiu
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Timisoara
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Singapore
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Singapore
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Castilla Y Leon
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Spain
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Catalunya
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Spain
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Galicia
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Spain
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Madrid
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Spain
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Murcia
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Taiwan
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Kaohsiung
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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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Turkey
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TUR
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Turkey
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Ankara
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Turkey
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Aydin
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Turkey
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Edirne
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Turkey
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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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London
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Nottingham
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Oxford
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United Kingdom
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Southampton

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
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.