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Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP) (VAYHIT1)

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ClinicalTrials.gov Identifier: NCT05653349
Recruitment Status : Recruiting
First Posted : December 16, 2022
Last Update Posted : May 6, 2023
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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Brief Summary:
The purpose of this study is to evaluate the effect of two different doses of ianalumab versus placebo in addition to first-line corticosteroids in maintaining platelet count ≥30 G/L in adult participants with primary ITP.

Condition or disease Intervention/treatment Phase
Primary Immune Thrombocytopenia (ITP) Biological: Ianalumab Drug: Placebo Drug: Corticosteroids Phase 3

Detailed Description:

This is a multi-center, randomized, double-blind Phase 3 study to assess the efficacy and safety of two different doses of ianalumab compared to placebo in adults with primary ITP (platelets count <30 G/L) who require first-line standard-of-care corticosteroids.

After completion of the screening period, the participants will enter the randomized treatment period (ianalumab/placebo with standard of care corticosteroids).

After the treatment period, all participants will enter the follow-up period to be monitored for efficacy and safety or safety only depending on how they respond to the study treatment.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 225 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase III, Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (VAYHIT1)
Actual Study Start Date : March 6, 2023
Estimated Primary Completion Date : November 14, 2025
Estimated Study Completion Date : July 24, 2028

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Steroids

Arms and Interventions
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Arm Intervention/treatment
Experimental: Ianalumab Lower dose
Lower dose of ianalumab administered intravenously with corticosteroids oral or parentally (if clinically justified)
 Biological: Ianalumab
Intravenously infusion, prepared from concentrate solution
Other Name: VAY736

Drug: Corticosteroids
Oral or parentally (if clinically justified)
Experimental: Ianalumab Higher dose
Higher dose of ianalumab administered intravenously with corticosteroids oral or parentally (if clinically justified)
 Biological: Ianalumab
Intravenously infusion, prepared from concentrate solution
Other Name: VAY736

Drug: Corticosteroids
Oral or parentally (if clinically justified)
Placebo Comparator: Placebo
Placebo administered intravenously with corticosteroids oral or parentally (if clinically justified)
 Drug: Placebo
Intravenously infusion, prepared from matching placebo

Drug: Corticosteroids
Oral or parentally (if clinically justified)


Outcome Measures
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Primary Outcome Measures :
  1. Time from randomization to treatment failure (TTF) [ Time Frame: Randomization to end of study (up to 39 months after randomization of last patient) ]
    Time from randomization until platelet count below 30 G/L, need for a rescue treatment or start of a second-line therapy or death.


Secondary Outcome Measures :
  1. Complete Response (CR) rate in each treatment group [ Time Frame: Randomization to end of study (up to 39 months after randomization of last patient) ]
    Complete Response (CR) rate at each timepoint defined as the proportion of participants with any platelet count of at least 100 G/L in the absence of rescue treatment or new ITP treatment.

  2. Response (R) rate in each treatment group [ Time Frame: Randomization to end of study (up to 39 months after randomization of last patient) ]
    Response (R) rate at each timepoint defined as the proportion of participants with any platelet count of at least 50 G/L in the absence of rescue treatment or new ITP treatment.

  3. Time to complete response in each treatment group [ Time Frame: Randomization to end of study (up to 39 months after randomization of last patient) ]
    Time from randomization to date of first complete response.

  4. Duration of response in each treatment group [ Time Frame: Randomization to end of study (up to 39 months after randomization of last patient) ]
    Time from achievement of complete response to loss of complete response

  5. Percentage of participants with bleeding events overall and by World Health Organization (WHO) bleeding scale severity [ Time Frame: Randomization to end of study (up to 39 months after randomization of last patient) ]
    This is to assess the incidence and severity of bleeding in each treatment arm

  6. Number of participants with bleeding events overall and by World Health Organization (WHO) bleeding scale severity [ Time Frame: Randomization to end of study (up to 39 months after randomization of last patient) ]
    This is to assess the number and severity of bleeding in each treatment arm

  7. Number of participants receiving rescue treatment (cummulative dose/duration of steroids exposure) [ Time Frame: Randomization to end of study (up to 39 months after randomization of last patient) ]
    This is to assess the number of participants receiving rescue treatment.

  8. Percentage of participants receiving rescue treatment (cummulative dose/duration of steroids exposure) [ Time Frame: Randomization to end of study (up to 39 months after randomization of last patient) ]
    This is to assess the need of rescue treatment in each treatment group by percentage.

  9. Cumulative dose/duration of steroids exposure [ Time Frame: From screening to end of study (up to 39 months after randomization of last patient) ]
    Duration of exposure to corticosteroids calculated from randomization (first dose) to end of study or last last contact date (if the participant is lost to follow-up).

  10. Change from baseline on total scores of the PROMIS SF v1.0 Fatigue 13a [ Time Frame: From screening (baseline) till end of study (up to 39 months after randomization of last patient) ]
    The Patient-Reported Outcomes Measurement Information System (PROMIS) Short Form v1.0 Fatigue 13a includes 13 items that assess fatigue

  11. Change from baseline in ITP-PAQ domain scores [ Time Frame: From screening (baseline) till end of study (up to 39 months after randomization of last patient) ]
    The ITP-PAQ is a 44 item scale for measuring HRQoL in adults with ITP across ten scales: Symptoms, Bother-Physical Health, Fatigue/Sleep, Activity, Fear, Psychological Health, Work, Social Activity, Women's Reproductive Health, and Overall QoL. Each item is rated on a Likert type scale

  12. Change from baseline in frequency of CD19+ B cell counts [ Time Frame: Randomization to end of study (up to 39 months after randomization of last patient) ]
    Post baseline frequency (%within the CD45) of CD19+ B cell counts compare to baseline.

  13. Change from baseline in absolute number of CD19+ B cell counts [ Time Frame: Randomization to end of study (up to 39 months after randomization of last patient) ]
    Post baseline absolute number of CD19+ B cell counts compare with baseline

  14. Time to first occurrence of B-cell recovery [ Time Frame: Randomization to end of study (up to 39 months after randomized of last patient) ]
    B-cell recovery, defined as ≥80% of baseline or ≥50 cells/μL

  15. Change from baseline in inmmunoglobulins [ Time Frame: Randomization to end of study (up to 39 months after last randomized patients) ]
    Change from baseline in immunoglobulin levels

  16. PK parameters: AUClast [ Time Frame: 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) ]
    AUClast: area under the curve from time zero till the last measurable concentration sampling time (tlast)

  17. PK parameter: AUCtau [ Time Frame: 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) ]
    Area under the curve calculated to the end of a dosing interval (tau)

  18. PK parameters: Cmax [ Time Frame: 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) ]
    Maximum (peak) observed plasma, blood, serum, or other body fluid drug concentration after single dose administration

  19. PK parameters: Tmax [ Time Frame: 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) ]
    Time to reach maximum (peak) plasma, blood, serum, or other body fluid drug concentration

  20. PK parameters: Accumulation ratio Racc [ Time Frame: After last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose) ]
    Accumulation ratio calculated using AUC values obtained between the last and first dose

  21. Incidence of anti-ianalumab antibodies in serum (ADA assay) over time [ Time Frame: Up to Week 33 ]
    Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants assess the immunogenicity of ianalumab

  22. Titer of anti-ianalumab antibodies in serum (ADA assay) over time [ Time Frame: Up to Week 33 ]
    Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants assess the immunogenicity of ianalumab


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 Exclusion Criteria:

  • Evans syndrome or any other cytopenia
  • Current life-threatening bleeding
  • Previous ITP treatment, including splenectomy, except for corticosteroids and/or IVIG for up to 28 days before randomization.
  • 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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05653349


Contacts
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Contact: Novartis Pharmaceuticals 1-888-669-6682 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +41613241111

Locations
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United States, New Jersey
Inspira Medical Cent Mullica Hill Recruiting
Mullica Hill, New Jersey, United States, 08062
Contact: Catherine Vasquez    856-641-7526    VasquezC@ihn.org   
Principal Investigator: Erev Tubb         
Australia, Victoria
Novartis Investigative Site Recruiting
Prahran, Victoria, Australia, 3181
Belgium
Novartis Investigative Site Recruiting
Brugge, Belgium, 8000
Novartis Investigative Site Recruiting
Roeselare, Belgium, 8800
Czechia
Novartis Investigative Site Recruiting
Brno Bohunice, Czech Republic, Czechia, 625 00
Novartis Investigative Site Recruiting
Praha 10, Czechia, 100 34
France
Novartis Investigative Site Recruiting
Le Mans, Cedex 09, France, 72037
Novartis Investigative Site Recruiting
Caen, Cedex, France, 14033
Novartis Investigative Site Recruiting
Paris, France, 75014
Germany
Novartis Investigative Site Recruiting
Dresden, Germany, 01307
Novartis Investigative Site Recruiting
Greifswald, Germany, 17475
Novartis Investigative Site Recruiting
Jena, Germany, 07740
Hong Kong
Novartis Investigative Site Recruiting
Hong Kong, Hong Kong
Hungary
Novartis Investigative Site Recruiting
Budapest, Hungary, 1085
Novartis Investigative Site Recruiting
Debrecen, Hungary, 4032
Japan
Novartis Investigative Site Recruiting
Shibukawa-city, Gunma, Japan, 377-0280
Novartis Investigative Site Recruiting
Sapporo, Hokkaido, Japan, 060-8604
Novartis Investigative Site Recruiting
Matsumoto-city, Nagano, Japan, 399-8701
Novartis Investigative Site Recruiting
Osaka-city, Osaka, Japan, 543-8555
Novartis Investigative Site Recruiting
Fukuoka, Japan, 815-8555
Malaysia
Novartis Investigative Site Recruiting
Kuching, Sarawak, Malaysia, 93586
Novartis Investigative Site Recruiting
Johor Bahru, Malaysia, 80100
Novartis Investigative Site Recruiting
Selangor, Malaysia, 68000
Norway
Novartis Investigative Site Recruiting
Gralum, Norway, 1714
Romania
Novartis Investigative Site Recruiting
Bucharest, District 2, Romania, 022328
Novartis Investigative Site Recruiting
Bucuresti, Romania, 013975
Singapore
Novartis Investigative Site Recruiting
Singapore, Singapore, 119228
Novartis Investigative Site Recruiting
Singapore, Singapore, 169608
Novartis Investigative Site Recruiting
Singapore, Singapore, S308433
Spain
Novartis Investigative Site Recruiting
Sevilla, Andalucia, Spain, 41013
Novartis Investigative Site Recruiting
Salamanca, Castilla Y Leon, Spain, 37007
Novartis Investigative Site Recruiting
Barcelona, Catalunya, Spain, 08035
Novartis Investigative Site Recruiting
Madrid, Spain, 28046
Novartis Investigative Site Recruiting
Murcia, Spain, 30008
United Kingdom
Novartis Investigative Site Recruiting
Truro, Cornwall, United Kingdom, TR1 3LJ
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05653349    
Other Study ID Numbers: CVAY736I12301
First Posted: December 16, 2022    Key Record Dates
Last Update Posted: May 6, 2023
Last Verified: May 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

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
URL: https://www.clinicalstudydatarequest.com

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Primary immune thrombocytopenia (ITP)
ianalumab
VAY736
B-cell depletion
B-cell Activating Factor Receptor (BAFF-R) blockade
Additional relevant MeSH terms:
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Thrombocytopenia
Immune System Diseases
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
 Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations