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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05653349
Other study ID # CVAY736I12301
Secondary ID
Status Recruiting
Phase Phase 3
First received
Last updated
Start date March 6, 2023
Est. completion date February 4, 2028

Study information

Verified date March 2024
Source Novartis
Contact Novartis Pharmaceuticals
Phone 1-888-669-6682
Email novartis.email@novartis.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Recruiting
Enrollment 225
Est. completion date February 4, 2028
Est. primary completion date September 25, 2025
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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 (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


Related Conditions & MeSH terms

  • Primary Immune Thrombocytopenia (ITP)
  • Purpura, Thrombocytopenic, Idiopathic
  • Thrombocytopenia

Intervention

Biological:
Ianalumab
Intravenous infusion, prepared from concentrate solution
Drug:
Placebo
Intravenous infusion, prepared from matching placebo
Corticosteroids
Oral or parental (if clinically justified)

Locations

Country Name City State
Argentina Novartis Investigative Site Buenos aires
Argentina Novartis Investigative Site Caba Buenos Aires
Argentina Novartis Investigative Site Caba Buenos Aires
Argentina Novartis Investigative Site Ciudad Autonoma de Bs As Buenos Aires
Argentina Novartis Investigative Site Tucuman San Miguel De Tucuman
Australia Novartis Investigative Site Canberra Australian Capital Territory
Australia Novartis Investigative Site Clayton Victoria
Australia Novartis Investigative Site Prahran Victoria
Australia Novartis Investigative Site Wooloongabba Queensland
Austria Novartis Investigative Site Innsbruck Tyrol
Austria Novartis Investigative Site Salzburg
Austria Novartis Investigative Site Wien
Belgium Novartis Investigative Site Brugge
Belgium Novartis Investigative Site Leuven
Belgium Novartis Investigative Site Roeselare
Bulgaria Novartis Investigative Site Plovdiv
Bulgaria Novartis Investigative Site Sofia
Bulgaria Novartis Investigative Site Sofia
Bulgaria Novartis Investigative Site Varna
China Novartis Investigative Site Beijing
China Novartis Investigative Site Changsha Hunan
China Novartis Investigative Site Chengdu Sichuan
China Novartis Investigative Site Dalian
China Novartis Investigative Site Guangzhou Guangdong
China Novartis Investigative Site Hangzhou Zhejiang
China Novartis Investigative Site Hangzhou Zhejiang
China Novartis Investigative Site Jinan
China Novartis Investigative Site Kunming Yunnan
China Novartis Investigative Site Nanchang Jiangxi
China Novartis Investigative Site Shenzhen Guangdong
China Novartis Investigative Site Taiyuan Shanxi
China Novartis Investigative Site Tianjin
China Novartis Investigative Site Wuhan Hubei
China Novartis Investigative Site Xian Shaanxi
China Novartis Investigative Site Zhengzhou Henan
Czechia Novartis Investigative Site Brno Bohunice Czech Republic
Czechia Novartis Investigative Site Ostrava Poruba
Czechia Novartis Investigative Site Praha
Czechia Novartis Investigative Site Praha 10
France Novartis Investigative Site Caen Cedex
France Novartis Investigative Site Chambéry cedex
France Novartis Investigative Site Le Mans Cedex 09
France Novartis Investigative Site Lille
France Novartis Investigative Site Paris
France Novartis Investigative Site Rennes
Germany Novartis Investigative Site Dresden
Germany Novartis Investigative Site Essen
Germany Novartis Investigative Site Frankfurt
Germany Novartis Investigative Site Giessen
Germany Novartis Investigative Site Greifswald
Germany Novartis Investigative Site Hannover
Germany Novartis Investigative Site Jena
Hong Kong Novartis Investigative Site Hong Kong
Hong Kong Novartis Investigative Site New Territories
Hungary Novartis Investigative Site Budapest
Hungary Novartis Investigative Site Debrecen
India Novartis Investigative Site Kolkata West Bengal
Italy Novartis Investigative Site Bologna BO
Italy Novartis Investigative Site Firenze FI
Italy Novartis Investigative Site Roma RM
Italy Novartis Investigative Site Trieste TS
Italy Novartis Investigative Site Vicenza VI
Japan Novartis Investigative Site Chiba
Japan Novartis Investigative Site Fukuoka
Japan Novartis Investigative Site Gifu shi Gifu
Japan Novartis Investigative Site Iruma-gun Saitama
Japan Novartis Investigative Site Matsumoto-city Nagano
Japan Novartis Investigative Site Okayama city Okayama
Japan Novartis Investigative Site Osaka-city Osaka
Japan Novartis Investigative Site Sapporo Hokkaido
Japan Novartis Investigative Site Shibukawa-city Gunma
Malaysia Novartis Investigative Site Johor Bahru
Malaysia Novartis Investigative Site Kuala Lumpur MYS
Malaysia Novartis Investigative Site Kuching Sarawak
Malaysia Novartis Investigative Site Penang
Malaysia Novartis Investigative Site Selangor
Mexico Novartis Investigative Site Ciudad de Mexico Mexico CP
Mexico Novartis Investigative Site Monterrey Nuevo Leon
Mexico Novartis Investigative Site Morelia Michoacan
Norway Novartis Investigative Site Gralum
Romania Novartis Investigative Site Bucharest District 2
Romania Novartis Investigative Site Bucharest
Romania Novartis Investigative Site Bucuresti
Singapore Novartis Investigative Site Singapore
Singapore Novartis Investigative Site Singapore
Singapore Novartis Investigative Site Singapore
Spain Novartis Investigative Site Barcelona Catalunya
Spain Novartis Investigative Site Madrid
Spain Novartis Investigative Site Madrid
Spain Novartis Investigative Site Murcia
Spain Novartis Investigative Site Salamanca Castilla Y Leon
Spain Novartis Investigative Site Sevilla Andalucia
Thailand Novartis Investigative Site Bangkok
Thailand Novartis Investigative Site Bangkok
Thailand Novartis Investigative Site Chiang Mai
Turkey Novartis Investigative Site Aydin
Turkey Novartis Investigative Site Edirne
Turkey Novartis Investigative Site Istanbul
Turkey Novartis Investigative Site Istanbul TUR
Turkey Novartis Investigative Site Izmir
Turkey Novartis Investigative Site Samsun
United Kingdom Novartis Investigative Site Birmingham
United Kingdom Novartis Investigative Site Glasgow
United Kingdom Novartis Investigative Site Leeds
United Kingdom Novartis Investigative Site London
United Kingdom Novartis Investigative Site London
United Kingdom Novartis Investigative Site Nottingham
United Kingdom Novartis Investigative Site Truro Cornwall
United States Oncology Care Associates Bethesda Maryland
United States St Vincent Frontier Cancer Center . Billings Montana
United States Uni of Chi Medi Ctr Hema and Onco Main Centre Chicago Illinois
United States Cleveland Clinic Foundation . Cleveland Ohio
United States Community Cancer Institute Clovis California
United States Texas Oncology-Baylor Res Ins Dallas Texas
United States STAT Research Inc Premier Clin Res LLC STAT Res Dayton Ohio
United States Compassionate Care Res Group Inc Fountain Valley California
United States Clinical Research Alliance Research Lake Success New York
United States Napa Research Margate Florida
United States Texas Oncology McAllen Texas
United States Inspira Medical Cent Mullica Hill Mullica Hill New Jersey
United States Hematology Oncology Association of Rockland Nyack New York
United States Community Cancer Trials of Utah Ogden Utah
United States INTEGRIS Cancer Institute of Oklahoma Integris South West Med Center Oklahoma City Oklahoma
United States Metro Minnesota CCOP Main Site Address Oncology Saint Louis Park Minnesota
United States New Tampa Health Tampa Florida
United States Yuma Regional Medical Center Yuma Arizona
Vietnam Novartis Investigative Site Hanoi

Sponsors (1)

Lead Sponsor Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Vietnam,  Argentina,  Australia,  Austria,  Belgium,  Bulgaria,  China,  Czechia,  France,  Germany,  Hong Kong,  Hungary,  India,  Italy,  Japan,  Malaysia,  Mexico,  Norway,  Romania,  Singapore,  Spain,  Thailand,  Turkey,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Time from randomization to treatment failure (TTF) Time from randomization until platelet count below 30 G/L, need for a rescue treatment or start of a second-line therapy or death. Randomization to end of study (up to 39 months after randomization of last patient)
Secondary Complete Response (CR) rate in each treatment group 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. Randomization to end of study (up to 39 months after randomization of last patient)
Secondary Response (R) rate in each treatment group 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. Randomization to end of study (up to 39 months after randomization of last patient)
Secondary Time to complete response in each treatment group Time from randomization to date of first complete response. Randomization to end of study (up to 39 months after randomization of last patient)
Secondary Duration of response in each treatment group Time from achievement of complete response to loss of complete response Randomization to end of study (up to 39 months after randomization of last patient)
Secondary Stable response at 6 months Percentage of participants with at least 2 platelet count collected at month 6 (between study dates 107 and 183) and at least 66% of platelet counts qualified as a response At 6 months
Secondary Stable response at 1 year Percentage of participants with at least 2 platelet counts collected at year 1 (between study days 296 and 379) and at least 66% of platelet counts qualified as a response At 1 year
Secondary Percentage of participants with bleeding events overall and by World Health Organization (WHO) bleeding scale severity This is to assess the incidence and severity of bleeding in each treatment arm Randomization to end of study (up to 39 months after randomization of last patient)
Secondary Number of participants with bleeding events overall and by World Health Organization (WHO) bleeding scale severity This is to assess the number and severity of bleeding in each treatment arm Randomization to end of study (up to 39 months after randomization of last patient)
Secondary Number of participants receiving rescue treatment (cummulative dose/duration of steroids exposure) This is to assess the number of participants receiving rescue treatment. Randomization to end of study (up to 39 months after randomization of last patient)
Secondary Percentage of participants receiving rescue treatment (cummulative dose/duration of steroids exposure) This is to assess the need of rescue treatment in each treatment group by percentage. Randomization to end of study (up to 39 months after randomization of last patient)
Secondary Cumulative dose/duration of steroids exposure 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). From screening to end of study (up to 39 months after randomization of last patient)
Secondary Change from baseline on T scores of the PROMIS SF v1.0 Fatigue 13a The Patient-Reported Outcomes Measurement Information System (PROMIS) Short Form v1.0 Fatigue 13a includes 13 items that assess fatigue From screening (baseline) till end of study (up to 39 months after randomization of last patient)
Secondary Change from baseline in ITP-PAQ domain scores 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 From screening (baseline) till end of study (up to 39 months after randomization of last patient)
Secondary Change from baseline in frequency of CD19+ B cell counts Post baseline frequency (%within the CD45) of CD19+ B cell counts compare to baseline. Randomization to end of study (up to 39 months after randomization of last patient)
Secondary Change from baseline in absolute number of CD19+ B cell counts Post baseline absolute number of CD19+ B cell counts compare with baseline Randomization to end of study (up to 39 months after randomization of last patient)
Secondary Time to first occurrence of B-cell recovery B-cell recovery, defined as =80% of baseline or =50 cells/µL Randomization to end of study (up to 39 months after randomized of last patient)
Secondary Change from baseline in inmmunoglobulins Change from baseline in immunoglobulin levels Randomization to end of study (up to 39 months after last randomized patients)
Secondary PK parameters: AUClast AUClast: area under the curve from time zero till the last measurable concentration sampling time (tlast) 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 PK parameter: AUCtau Area under the curve calculated to the end of a dosing interval (tau) 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 PK parameters: Cmax Maximum (peak) observed plasma, blood, serum, or other body fluid drug concentration after single dose administration 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 PK parameters: Tmax Time to reach maximum (peak) plasma, blood, serum, or other body fluid drug concentration 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 PK parameters: Accumulation ratio Racc Accumulation ratio calculated using AUC values obtained between the last and first dose After last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
Secondary Incidence of anti-ianalumab antibodies in serum (ADA assay) over time Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants assess the immunogenicity of ianalumab Up to Week 33
Secondary Titer of anti-ianalumab antibodies in serum (ADA assay) over time Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants assess the immunogenicity of ianalumab Up to Week 33
See also
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Completed NCT06408324 - Evaluation of the Use of Thrombopoietin Receptor Agonists in Adults With Primary ITP in Europe
Recruiting NCT05338190 - Efficacy and Safety of Subcutaneous Belimumab or Placebo in Addition of Rituximab in Persistent or Chronic Immune Thrombocytopenia Phase 3
Completed NCT02042560 - Study of Immune Thrombocytopenia Pathogenesis: N/A
Withdrawn NCT04274452 - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP) Phase 3
Recruiting NCT05718856 - TPO-RAs Combining Anti-CD 20 Monoclonal Antibody Versus TPO-RAs in the Management of Pediatric Primary Immune Thrombocytopenia (ITP) Phase 4
Recruiting NCT05885555 - A Study of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least Two Lines of Therapies Phase 2
Recruiting NCT04518475 - Eltrombopag Combining Rituximab Versus Eltrombopag in the Management of Primary Immune Thrombocytopenia (ITP) in Adults Phase 4