Study of Ianalumab in Adults With Primary Immune Thrombocytopenia (ITP) and Warm-antibody Autoimmune Hemolytic Anemia (wAIHA) Who Have Previously Benefited From Ianalumab

NCT07039422 | Recruiting Phase 2 FDA Drug

• 2 other identifiers: CVAY736Q12202B2024-518231-11-00
• Study Type: interventional
• Target: 60
• Locations: 11 countries
• Sites: 14

Brief Summary

This study is intended to explore the efficacy and safety of a second course of ianalumab after experiencing treatment failure in the pivotal Primary Immune Thrombocytopenia (ITP) trials (CVAY736I12301, CVAY736Q12301) and after loss of durable response in the pivotal Warm Autoimmune Hemolytic Anemia (wAIHA) trial (CVAY736O12301).

Conditions

Primary Immune Thrombocytopenia; Warm Autoimmune Hemolytic Anemia

Keywords

Primary Immune Thrombocytopenia (ITP); ITP; Warm Autoimmune Hemolytic Anemia (wAIHA); wAIHA; VAY736; Ianalumab; B-cell depletion; B-cell Activating Factor Receptor (BAFF-R) blockade

Outcome Measures

Primary Outcomes (2)

• Treatment failure free (yes, no) for participants with Immune Thrombocytopenia (ITP)

  Treatment failure free (yes, no) by 12 months after start of second course of ianalumab at assigned dose.

  From treatment start date until the events indicative of treatment failure (up to 2 years after last dose)

• Durable response for participants with Warm Autoimmune Hemolytic Anemia (wAIHA)

  Durable response (hemoglobin (Hb) ≥10 g/dL and ≥2 g/dL increase from baseline) for a period of at least 8 consecutive weeks, between W9 and W25 in the absence of rescue or prohibited treatment prior to that durable response achievement.

  At least 8 consecutive weeks, between week 9 and week 25

Secondary Outcomes (10)

• Response rate in participants with ITP only

  from treatment start to end of study (up to 2 years after last dose)

• Complete response rate in participants with ITP only

  from treatment start to end of study (up to 2 years after last dose)

• Number and percentage of participants receiving new ITP therapy only

  from treatment start to end of study (up to 2 years after last dose)

• Response and complete response in participants with wAIHA only

  from treatment start to end of study (up to 2 years after last dose)

• Number and percentage of participants receiving new wAIHA therapy only

  from treatment start to end of study (up to 2 years after last dose)

Study Arms (2)

Treatment arm 1

EXPERIMENTAL

Participants will receive ianalumab lower dose

Biological: Ianalumab

Treatment arm 2

EXPERIMENTAL

Participants will receive ianalumab higher dose

Biological: Ianalumab

Interventions

Ianalumab BIOLOGICAL

Concentrate for solution for infusion for intravenous use

Also known as: VAY736

Treatment arm 1; Treatment arm 2

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• Primary ITP patients:
• Previously enrolled and treated either with ianalumab/placebo in addition to first-line corticosteroids on protocol CVAY736I12301 or with ianalumab/placebo in addition to eltrombopag in the second line on protocol CVAY736Q12301, and who experienced treatment failure (TF) by parent trial definition ≥ 2 years after the last infusion of ianalumab/placebo
• Rescue medication and/or bridging therapy are allowed to be started within the 28 days prior to screening; platelet count results obtained prior to the start of the therapy must be used to assess eligibility and have to be collected within 30 days prior to screening
• For Primary or Secondary wAIHA patients:
• Previously documented by a positive direct antiglobulin test (DAT) specific for anti-IgG or anti-IgA, previously enrolled and treated with ianalumab/placebo in blinded cohort or placebo followed by crossover to open label ianalumab in protocol CVAY736O12301, having experienced durable response lasting beyond 2 years from the last infusion of ianalumab/placebo in blinded cohorts or a durable response beyond week 20 from last dose of first course of ianalumab in the crossover arm.
• Relapsed wAIHA with hemoglobin concentration ≥5 g/dL and \<10 g/dL and presence of symptoms related to anemia during screening or within 14 days before screening window or within 28 days before screening window if rescue medication/bridging therapy has been initiated.
• Rescue medication and/or bridging therapy are allowed to be started during the screening and within 28 days prior to screening; hemoglobin level result for eligibility assessment needs to be obtained prior to the start of the treatment within 30 days prior to screening
• Supportive care is allowed in the case the participant received it in the parent trial when the relapse occurred and has remained stable at least 4 weeks prior screening

You may not qualify if:

• Evans syndrome or any cytopenia other than thrombocytopenia (for ITP participants) or anemia (for wAIHA), except for grade 1 anemia due to blood loss or iron deficiency.
• Secondary wAIHA with BM involvement for wAIHA patients
• Current life-threatening bleeding or history of life-threatening bleeding due to thrombocytopenia
• Therapy for ITP or wAIHA other than ianalumab/placebo, bridging/rescue therapies and supportive care prior to the beginning of the screening window
• After primary analysis of each respective parent trial, participants whose treatment was unblinded and who received placebo only will be excluded.
• ITP participants only: Participants with concurrent coagulation disorders and/or receiving anti-platelet or anti-coagulant medication except for low dose of acetylsalicylic acid (≤150 mg per day)

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (14)

Novartis Investigative Site

Roeselare, West-Vlaanderen, 8800, Belgium

RECRUITING

Novartis Investigative Site

Guangzhou, Guangdong, 510515, China

RECRUITING

Novartis Investigative Site

Tianjin, 300020, China

RECRUITING

Novartis Investigative Site

Ostrava, Poruba, 708 52, Czechia

RECRUITING

Novartis Investigative Site

Jena, Thuringia, 07740, Germany

RECRUITING

Novartis Investigative Site

Debrecen, Hajdu Bihar Megye, 4032, Hungary

RECRUITING

Novartis Investigative Site

Florence, FI, 50134, Italy

RECRUITING

Novartis Investigative Site

Trieste, TS, 34129, Italy

RECRUITING

Novartis Investigative Site

Vicenza, VI, 36100, Italy

RECRUITING

Novartis Investigative Site

George Town, 10050, Malaysia

RECRUITING

Novartis Investigative Site

Singapore, 119074, Singapore

RECRUITING

Novartis Investigative Site

Madrid, 28009, Spain

RECRUITING

Novartis Investigative Site

Samsun, Atakum, 55200, Turkey (Türkiye)

RECRUITING

Novartis Investigative Site

London, W12 0HS, United Kingdom

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

ianalumab

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Blinded will be maintained at least until parent studies are unblinded.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: May 5, 2025
• First Posted: June 26, 2025
• Study Start: April 27, 2026
• Primary Completion (Estimated): August 30, 2030
• Study Completion (Estimated): August 30, 2030
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

CZ (1); CN (2); HU (1); MY (1); BE (1); IT (3); SG (1); ES (1); DE (1); TU (1); GB (1)