A Study of Ianalumab in Addition to Eltrombopag in Pediatric Patients With Primary ITP Who Failed Corticosteroids.
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age5-17
SponsorNovartis Pharmaceuticals
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
Ianalumab
Biological/VaccineLiquid in a vial Concentrate for solution for infusion
Eltrombopag
DrugFilm-coated tablet and also Powder for oral suspension
Treatment groups
36 Participants
are divided into 1 treatment groupGroup A: Ianalumab + eltrombopagExperimental treatment 2 interventions
Trial outcomes
Primary outcomes
1
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.
Time frame
enrolled until end of study (up to 39 months from the last patient enrolled)Secondary outcomes
1
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.
Time frame
6 months2
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.
Time frame
12 months3
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.
Time frame
from enrollment until end of study (up to 39 months from the last patient enrolled)4
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.
Time frame
from enrollment until end of study (up to 39 months from the last patient enrolled)Locations
Sponsors and contacts
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Source ClinicalTrials.gov
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