Primary Immune Thrombocytopenia (ITP)Phase 2VAYHIT-PNCT07660172
A Study of Ianalumab in Addition to Eltrombopag in Pediatric Patients With Primary ITP Who Failed Corticosteroids.
A Phase 2 study testing Ianalumab and Eltrombopag in people with Primary Immune Thrombocytopenia (ITP), run by Novartis Pharmaceuticals.
Trial summary
The purpose of this study is to assess the efficacy, safety and pharmacokinetics (PK) of ianalumab (VAY736) in addition to eltrombopag treatment; and to inform the dose of ianalumab in pediatric patients (5 to <18 years of age) with primary ITP who have had an insufficient response to or relapsed after first-line… Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Primary Immune Thrombocytopenia (ITP)
- What is being tested?
- Ianalumab and Eltrombopag
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- No sites listed yet
- Study dates
- 8 Feb 2027to 22 Apr 2033
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Signed informed consent and/or assent must be obtained from the participant and/or their parent(s)/legal guardian(s) before any study-related activity or assessment is performed. Informed consent must be obtained from participants once they reach the local legal age of adulthood during the study.
- Male or female patients aged 5 to <18 years on the day of signing informed assent/consent (as appropriate for age).
- A confirmed diagnosis of primary ITP, with insufficient response to, or relapse after a firstline corticosteroid therapy with or without IVIG.
- Patients with platelet count <30 G/L for whom eltrombopag is clinically indicated (per physician's discretion) and with no contraindications to receive eltrombopag.
- Patients who are up-to-date on childhood vaccinations as per the local recommended vaccination schedule.
Who cannot join
- Patients with ITP who received previous second-line ITP treatments (other than corticosteroid therapy with or without IVIG) including splenectomy. However, patients exposed to thrombopoietin receptor agonists (TPO-RAs) for a limited time (maximum one week) before or during screening are eligible.
- Patients with key renal or hepatic laboratory abnormalities.
- Patients with other hematologic diagnosis associated with cytopenias, including Evans Syndrome.
- Patients with current or history of life-threatening bleeding due to thrombocytopenia.
- Patients who are Human Immunodeficiency Virus (HIV), hepatitis C virus (HCV), hepatitis B surface antigen (HBsAg), or hepatitis B core antibody (HBcAb) positive.
- Patients with known active or uncontrolled infections requiring systemic treatment during the screening period or history of recurrent clinically significant infection.
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- Patients with hepatic impairment (Child-Pugh score >5, or equivalent).
- Patients with concurrent bleeding disorders or coagulation disorders and/or receiving antiplatelet or anticoagulant medication.
- Female patients who are nursing or pregnant.
- Other protocol-defined inclusion/exclusion may apply.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
About this study
Official title: A Phase 2, Open-label, Single-arm Study of Ianalumab in Addition to Eltrombopag in Pediatric Patients With Primary Immune Thrombocytopenia Who Had an Insufficient Response to or Relapsed After First-line Corticosteroid Treatment (VAYHIT-P).
The purpose of this study is to assess the efficacy, safety and pharmacokinetics (PK) of ianalumab (VAY736) in addition to eltrombopag treatment; and to inform the dose of ianalumab in pediatric patients (5 to <18 years of age) with primary ITP who have had an insufficient response to or relapsed after first-line treatment with corticosteroids.
Read the detailed description
The study will consist of 3 phases:
- Screening phase
- Treatment phase
- Follow-up phase consisting of Efficacy follow-up phase and Safety follow-up phase
- Conditions
- Primary Immune Thrombocytopenia (ITP)
- Being tested
- Ianalumab, Eltrombopag
- Keywords
- Primary Immune Thrombocytopenia, ITP, VAY736, ianalumab, first-line corticosteroid treatment, eltrombopag, Pediatric patients
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IanalumabBiological
Also called VAY736
Liquid in a vial Concentrate for solution for infusion
EltrombopagDrug
Also called ETB115
Film-coated tablet and also Powder for oral suspension
| Study group | Role | What they receive |
|---|---|---|
| Ianalumab + eltrombopag | Experimental | Participants will receive ianalumab in addition to eltrombopag. Receives: Biological: Ianalumab; Drug: Eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to treatment failure (TTF)
TTF is defined as the time from start of treatment until any of a list of specific events is met.
Measured: enrolled until end of study (up to 39 months from the last patient enrolled)
Secondary outcomes (17)
Stable response rate at 6 months (SR-6)
SR-6 is defined as the percentage of participants with a platelet count of at least 50 G/L at 75% of the measures taken between Study Day 121 and 183, in the absence of rescue or new Immune Thrombocytopenia (ITP) therapy.
Measured: 6 months
Stable response rate at 12 months (SR-12)
SR-12 is defined as the percentage of participants with a platelet count of at least 50 G/L at 66% of the measures taken between Study Day 296 and 379, in the absence of rescue or new ITP therapy.
Measured: 12 months
Complete Response (CR) rate
CR is defined as the percentage of participants with any platelet count of at least 100 G/L in the absence of rescue treatment or new ITP treatment.
Measured: from enrollment until end of study (up to 39 months from the last patient enrolled)
Response Rate (RR)
RR is defined as the percentage of participants with any platelet count of at least 50 G/L in the absence of rescue treatment or new ITP treatment.
Measured: from enrollment until end of study (up to 39 months from the last patient enrolled)
Time to response
Time to response is defined as the time from the start of treatment to the date of the first response.
Measured: from enrollment until end of study (up to 39 months from the last patient enrolled)
Time to complete response
Time to complete response is defined as the time from the start of treatment to the date of complete response.
Measured: from enrollment until end of study (up to 39 months from the last patient enrolled)
Duration of response
Duration of response is defined as the time from achievement of response (platelet count ≥ 50 G/L) to treatment failure.
Measured: from enrollment until end of study (up to 39 months from the last patient enrolled)
Duration of complete response
Duration of complete response is defined as the time from achievement of response to loss of complete response.
Measured: from enrollment until end of study (up to 39 months from the last patient enrolled)
Bleeding Events
Percentage of participants with bleeding events according to WHO Bleeding Scale
Measured: from enrollment until end of study (up to 39 months from the last patient enrolled)
Rescue medication
Number and percentage of participants receiving rescue treatment
Measured: from enrollment until end of study (up to 39 months from the last patient enrolled)
Rate of participants who successfully taper and discontinue eltrombopag - being treatment failure-free
Treatment failure-free (as defined for the primary efficacy endpoint) at the end of the planned Treatment period.
Measured: End of Week 24
Ianalumab serum concentration (Cmax)
Cmax is the maximum (or peak) serum concentration that a drug achieves in a specified compartment or test area of the body.
Measured: at pre-dose of each of the 4 dosing events and end-of-infusion and first and last dose
B cell levels: Change from baseline in the frequency (% within CD45+ cells) and absolute number of CD19+ B cells
Number and percentage of B-cell levels in the blood, and comparison with baseline levels of B-cells at specific timepoints.
Measured: 12 months after the first treatment of the last enrolled participant, final dbl: until end of study (up to 39 months from the last patient enrolled)
B cell levels: Time to first occurrence of B cell recovery
Time to first occurrence of B cell recovery is defined as ≥80% of baseline or ≥50 cells/μL.
Measured: 12 months after the first treatment of the last enrolled participant, final dbl: until end of study (up to 39 months from the last patient enrolled)
Immunoglobulins: Change from baseline in immunoglobulin levels
Change from baseline levels of serum immunoglobulins at specific timepoints.
Measured: 12 months after the first treatment of the last enrolled participant, final dbl: until end of study (up to 39 months from the last patient enrolled)
Platelet counts: Change from baseline in platelet count
Change from baseline levels of platelets at specific timepoints.
Measured: 12 months after the first treatment of the last enrolled participant, final dbl: until end of study (up to 39 months from the last patient enrolled)
Incidence and titer of anti-ianalumab antibodies in serum (anti-drug-antibody (ADA) assay)
Anti-drug antibodies (ADAs) are immune responses developed by a participant's body against biologic or gene therapies.
Measured: over time (up to 39 months from the last patient enrolled)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2026
- Study start8 Feb 2027 (estimated)
- Primary completion22 Apr 2033 (estimated)
- Study completion22 Apr 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune Thrombocytopenia (ITP)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07660172
- Sponsor's protocol ID
- CVAY736T12201
- Other IDs
- 2026-525995-25
- Acronym
- VAYHIT-P
- Status last verified
- Jun 2026
- First submitted
- 15 Jun 2026
- Last updated
- 25 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
- Expanded access
- Available