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A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids

PHASE3RECRUITING

The purpose of this study is to evaluate the effect of two different doses of ianalumab added to eltrombopag to prolong Time to Treatment Failure (TTF) in adults with primary ITP who failed previous first-line treatment with steroids.

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Study details:

This is a multicenter, randomized, double-blinded phase 3 study to assess efficacy and safety of two different doses of ianalumab versus placebo in addition to eltrombopag in adults with primary ITP (platelet count \<30 G/L) who failed previous first-line treatment with corticosteroids. After completion of the screening period, the participants will enter the randomized treatment period (ianalumab/placebo with eltrombopag) followed by the eltrombopag tapering period. Afterwards, all participants will enter the follow-up period to be monitored for efficacy and safety or safety only depending on how the participants responded to the study treatment.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

Male or female patients aged 18 years and older on the day of signing the informed consent.

A signed informed consent must be obtained prior to participation in the study.

A diagnosis of primary ITP, with insufficient response to, or relapse after a first-line corticosteroid therapy ± IVIG.

Patient with platelet count <30G/L (whom eltrombopag is clinically indicated as per physician's discretion) and with no contraindication to receive eltrombopag

Exclusion criteria

ITP patients who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy. However, patients exposed to thrombopoietin receptor agonists (TPO-RAs) for a limited time (max one week) before screening are eligible.

Patients with key lab abnormalities and patients with Evans syndrome or any other cytopenia, (patients with low grade anemia related to bleeding or iron deficiency are eligible).

Patients with history of clinically significant hematological disorders, or with marked altered hematologic parameters

Patients with current or history of life-threatening bleeding

Patient that are Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B surface Antigen (HBsAg)/ Hepatitis B core antibody (HBcAb)-positive. HBcAb-positive patients can be enrolled if HBsAg negative, HBV DNA negative, no pre-existing liver fibrosis is present and antiviral prophylaxis is given

Patients with known active or uncontrolled infection requiring systemic treatment during screening period

Patients with hepatic impairment

Patients with concurrent coagulation disorders and/or receiving antiplatelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid (≤150 mg daily)

Nursing (breast feeding) or pregnant women

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Eligibility

Age eligible for study : 18 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2023-02-02

Primary completion: 2025-08-07

Study completion finish: 2028-05-19

Study type

TREATMENT

Phase

PHASE3

Trial ID

NCT05653219

Intervention or treatment

BIOLOGICAL: Ianalumab

DRUG: Eltrombopag

DRUG: Placebo

Conditions

• Primary Immune Thrombocytopenia

Image related to Primary Immune Thrombocytopenia

Condition: Primary Immune Thrombocytopenia
BIOLOGICAL: Ianalumab and other drugs
Clayton, Victoria, Australia
Sponsor: Novartis Pharmaceuticals

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Find a site

Closest Location:

Novartis Investigative Site

Research sites nearby

Select from list below to view details:

Novartis Investigative Site
Clayton, Victoria, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: Treatment arm 1 Participants will receive eltrombopag and ianalumab lower dose	BIOLOGICAL: Ianalumab Concentrate for solution for infusion for intravenous use
EXPERIMENTAL: Treatment arm 2 Participants will receive eltrombopag and ianalumab higher dose	BIOLOGICAL: Ianalumab Concentrate for solution for infusion for intravenous use
PLACEBO_COMPARATOR: Treatment arm 3 Participants will receive eltrombopag and placebo	DRUG: Eltrombopag Film-coated tablet for oral use

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
Time from randomization until treatment failure	Time from randomization until treatment failure is defined as the time from randomization date until the first of the following events indicative of treatment failure: * platelet count below 30 G/L * start of a new ITP treatment * need for a rescue treatment * ineligibility to taper or inability to discontinue eltrombopag * death	Randomization to until end of study (up to 39 months after randomization of last participant)

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
Complete Response rate at each timepoint	Percentage of participants with any platelet count of at least 100 G/L in the absence of rescue treatment or new ITP treatment	Randomization to until end of study (up to 39 months after randomization of last participant)
Response rate at each timepoint	Percentage of participants with any platelet count of at least 50 G/L in the absence of rescue treatment or new ITP treatment	Randomization to until end of study (up to 39 months after randomization of last participant)
Best response rate across all timepoints	Percentage of participants with a best response rate of either response or complete response	Randomization to until end of study (up to 39 months after randomization of last participant)
Time to first response/time to first complete response	Time from randomization to date of first response and time from randomization to date of first complete response	Time from randomization up to the longest observed treatment period duration
Duration of response	Time from achievement of response to treatment failure Stable response at 6 months	Randomization to until end of study (up to 39 months after randomization of last participant)
Stable response at 6 months	Percentage of participants with at least 3 platelet count collected at month 6 between (study days 121 and 183 and at least 75% of platelet counts qualified as a response	At 6 months
Stable response at 1 year	Percentage of participants with at least 2 platelet count collected at year 1 between (study days 296 and 379 and at least 66% of platelet counts qualified as a response	At 1 year
Duration of complete response	Time from achievement of complete response to loss of complete response stable response at 1 year period	Randomization to end of study (up to 39 months after randomization of last participant)
Rate of participants who successfully taper and discontinue eltrombopag in each treatment arm	Probability to be treatment failure-free (as defined for the primary efficacy endpoint)	up to week 24
Percentage of participants with bleeding events according to World Health Organization (WHO)	Percentage of participants reporting bleeding events according to WHO bleeding scale	Randomization to until end of study (up to 39 months after randomization of last participant)
Number of participants receiving rescue treatment	Number of participants who are in need of rescue treatment in each treatment arm	Randomization to until end of study (up to 39 months after randomization of last participant)
Percentage of participants receiving rescue treatment	Percentage of participants who are in need of rescue treatment	Randomization to until end of study (up to 39 months after randomization of last participant)
Change from baseline in the frequency of CD19+ B-cell counts	Post-baseline frequency of CD19+ B-cell counts (percentage within CD45) compared to baseline	Randomization to until end of study (up to 39 months after randomization of last participant)
Change from baseline in the absolute number of CD19+ B-cell counts	Post-baseline absolute number of CD19+ B-cell counts compared to baseline	Randomization to until end of study (up to 39 months after randomization of last participant)
Change from baseline on T-score of the PROMIS SF v1.0 Fatigue 13a	The Patient-Reported Outcomes Measurement Information System (PROMIS) Short Form v1.0 Fatigue 13a includes 13 items that assess fatigue in adults.	From screening (baseline) until end of study (up 39 months after randomization of last participant)
Change from baseline in ITP PAQ domain scores of symptoms, fatigue, bother (uncomfortable), activity	The ITP-PAQ is a 44 item scale for measuring HRQoL in adults with ITP across ten scales: Symptoms, Bother-Physical Health, Fatigue/Sleep, Activity, Fear, Psychological Health, Work, Social Activity, Women´s Reproductive Health, overall QoL. Each item is rated on a Likert type scale. Each scale is scored from 0 to 100. Higher scores represent better HRQoL.	From screening (baseline) until end of study (up 39 months after randomization of last participant)
Time to first occurence of B-cell recovery defined as ≥80% of baseline ≥50 cells/µL	Time to B-cell recovery defined as ≥80% of baseline or ≥50 cells/µL	Randomization to until end of study (up to 39 months after randomization of last participant)
Change from baseline in immunoglobulins	Change from baseline in immunoglobulin levels	Randomization to until end of study (up to 39 months after randomization of last participant)
PK parameters: AUClast	AUClast: Area under the curve from time zero to the last measurable concentration sampling time (tlast)	After first dose (pre-dose, EOI, 168, 336 and 504 hours post dose) and after last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
PK parameters: AUCtau	AUCtau: Area under the curve calculated to the end of a dosing interval (tau)	After first dose (pre-dose, EOI, 168, 336 and 504 hours post dose) and after last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
PK parameters: Cmax	Maximum (peak) observed plasma, blood, serum or other body fluid drug concentration	After first dose (pre-dose, EOI, 168, 336 and 504 hours post dose) and after last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
PK parameters: Tmax	Time to reach maximum (peak) observed plasma, blood, serum or other body fluid drug concentration	After first dose (pre-dose, EOI, 168, 336 and 504 hours post dose) and after last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
PK parameters: Accumulation ratio Racc	Accumulation ratio calculated using AUC values obtained after the last and first dose	After last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
Incidence of anti-ianalumab antibodies in serum (ADA assay) over time	Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants assessing the immunogenicity of ianalumab	up to week 33
Titer of anti-ianalumab antibodies in serum (ADA assay) over time	Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants assessing the immunogenicity of ianalumab	up to week 33

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References

Clinical Trials Gov: A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids