A Study of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least Two Lines of Therapies

NCT05885555 | Active Not Recruiting Phase 2 FDA Drug

• 2 other identifiers: CVAY736Q122012022-503041-21-00
• Study Type: interventional
• Target: 41
• Locations: 13 countries
• Sites: 24

Brief Summary

The purpose of this study is to evaluate the therapeutic efficacy, safety and tolerability of ianalumab in adult patients with primary ITP previously treated with at least one corticosteroid and one TPO-RA.

Conditions

Primary Immune Thrombocytopenia (ITP)

Keywords

Primary immune thrombocytopenia (ITP); ianalumab; VAY736; B-cell depletion; B-cell Activating Factor Receptor (BAFF-R) blockade

Outcome Measures

Primary Outcomes (1)

• Confirmed response

  Confirmed response is defined as a platelet count of equal or above 50 G/L at two (or more) consecutive assessments at least 7 days apart, in the absence of: * Rescue treatment for ≥4 weeks prior to the assessment of the platelet count, and * New immune thrombocytopenia (ITP) treatment before reaching a confirmed response.

  Between Week 1 Day 1 and Week 25 Day 1

Secondary Outcomes (35)

• Time to confirmed response

  From Week 1 Day 1 to Week 25 Day 1

• Duration of confirmed response

  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)

• Complete Response rate at each timepoint

  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)

• Response rate at each timepoint

  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)

• Stable response at 6 months

  At 6 months

Study Arms (1)

Single-arm

EXPERIMENTAL

All eligible participants will receive ianalumab at the same dose.

Biological: Ianalumab

Interventions

Ianalumab BIOLOGICAL

Intravenous infusion, prepared from concentrate solution

Also known as: VAY736

Single-arm

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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (24)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Beth Israel Deaconess Med Center

Boston, Massachusetts, 02215, United States

Location

Novartis Investigative Site

CABA, C1181ACH, Argentina

Location

Novartis Investigative Site

Garran, Australian Capital Territory, 2605, Australia

Location

Novartis Investigative Site

Melbourne, Victoria, 3004, Australia

Location

Novartis Investigative Site

Wuhan, Hubei, 430022, China

Location

Novartis Investigative Site

Beijing, 100730, China

Location

Novartis Investigative Site

Jinan, 250012, China

Location

Novartis Investigative Site

Toulouse, 31059, France

Location

Novartis Investigative Site

Dresden, Saxony, 01307, Germany

Location

Novartis Investigative Site

Jena, Thuringia, 07740, Germany

Location

Novartis Investigative Site

Giessen, 35392, Germany

Location

Novartis Investigative Site

Torino, TO, 10126, Italy

Location

Novartis Investigative Site

Johor Bahru, 80100, Malaysia

Location

Novartis Investigative Site

Katowice, 40-519, Poland

Location

Novartis Investigative Site

Seoul, 06591, South Korea

Location

Novartis Investigative Site

Barcelona, Catalonia, 08003, Spain

Location

Novartis Investigative Site

Aydin, Efeler, 09100, Turkey (Türkiye)

Location

Novartis Investigative Site

Istanbul, Fatih, 34098, Turkey (Türkiye)

Location

Novartis Investigative Site

Edirne, Merkez, 22030, Turkey (Türkiye)

Location

Novartis Investigative Site

Izmir, 35100, Turkey (Türkiye)

Location

Novartis Investigative Site

Kocaeli, 41380, Turkey (Türkiye)

Location

Novartis Investigative Site

Glasgow, G31 2ER, United Kingdom

Location

Novartis Investigative Site

London, W12 0HS, United Kingdom

Location

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: May 15, 2023
• First Posted: June 2, 2023
• Study Start: August 16, 2023
• Primary Completion: February 5, 2025
• Study Completion (Estimated): March 19, 2029
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

CN (3); DE (3); KR (1); AU (2); PL (1); US (2); AR (1); ES (1); TU (5); FR (1); MY (1); GB (2); IT (1)