A phase 2 study of ianalumab in adults with primary Immune Thrombocytopenia (ITP) and Warm-antibody Autoimmune Hemolytic Anemia (wAIHA) who have previously benefited from ianalumab.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma AG

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Decision date (initial)

2025-12-08

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Novartis Pharma AG

External identifiers

EU CT number

2024-518231-11-00

ClinicalTrials.gov

NCT07039422

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Therapy

For participants with primary ITP:
. To assess the benefit of a second course of ianalumab in participants with primary ITP who derived benefit from the initial course and subsequently relapsed
For participants with wAIHA:
. To assess the benefit of a second course of ianalumab in participants with wAIHA who had benefit to the initial course and subsequently relapsed.

Secondary objectives 3

1. Primary ITP participating only: Among all participants who received a second course of ianalumab, and among cohorts and/or group of cohorts defined based on the parent study and ianalumab first course dose): • Evaluate proportion of participants achieving response and complete response • To describe the need of rescue treatment and/or new ITP therapy
2. wAIHA participants only: Among all participants who received a second course of ianalumab and among cohorts and/or group of cohorts defined based on the ianalumab arm in parent study (blinded ianalumab 3 mg/kg, blinded ianalumab 9 mg/kg, cross-over ianalumab 9 mg/kg) • Assess the proportion of participants achieving response and complete response • To describe use of rescue treatment and/or new wAIHA therapy
3. All participants: • To assess the safety profile of ianalumab • To assess the pharmacokinetics of a second course of ianalumab treatment • To assess the immunogenicity of second course of ianalumab treatment

Conditions and MedDRA coding

Immune thrombocytopenia (ITP) Warm autoimmune hemolytic anemia (wAIHA)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Signed informed consent obtained prior to participation in the study
2. Male and female participants aged 18 years and older on the day of signing informed consent
3. Primary ITP patients: 3. 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
4. Rescue medication and/or bridging therapy (See Section 6.6.3 and Section 6.6.4 for further details) 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
5. 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
6. for primary or secondary wAIHA patients: 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
7. Rescue medication and/or bridging therapy (see Section 6.6.3 and Section 6.6.4 for further details) 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
8. Supportive care (see Section 6.6.3 for further details) 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

Exclusion criteria 6

1. Evans syndrome or any cytopenia other than thrombocytopenia (for ITP participants) or anemia (for wAIHA participants), except for grade 1 anemia due to blood loss or iron deficiency
2. Secondary wAIHA with BM involvement for wAIHA patients
3. Current life-threatening bleeding or history of life-threatening bleeding due to thrombocytopenia
4. Therapy for ITP or wAIHA other than ianalumab/placebo, bridging therapies and supportive care prior to the beginning of the screening window
5. After primary analysis of each respective parent trial, participants whose treatment was unblinded and who received placebo only will be excluded
6. 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)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. ITP participants: Treatment failure free (yes, no) by 12 m after start of second course of ianalumab is defined as any of: • platelet count <30G/L later than 8 weeks from start of second course • use of rescue treatment later than 8 weeks from second course • start of new ITP treatment • death • inability to taper TPO-RA by week 24
2. For wAIHA participants Durable response (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.

Secondary endpoints 8

1. Primary ITP participants only: • Response rate and complete response rate
2. Primary ITP participants only: • Number and proportion of participants receiving rescue treatment and/or new ITP therapy
3. wAIHA participants only: • Response rate and complete response rate
4. wAIHA participants only: • Number and proportion of participants who received rescue treatment and/or new wAIHA therapy
5. for all participants: Frequency of adverse events and other safety parameters
6. for all participants: Number of severe infections and proportion of participants with severe infection
7. for all participants: Ianalumab concentration in serum
8. for all participants: Incidence and titer of anti-ianalumab antibodies in serum (ADA assay) over time

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Auxiliary 6

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novartis Pharma AG

Sponsor organisation

Novartis Pharma AG

Address

Lichtstrasse 35

City

Basel

Postcode

4056

Country

Switzerland

Scientific contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Public contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Third parties 9

Locations

9 EU/EEA countries · 20 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 70 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications