A phase II, open-label, prospective, single-arm, study to assess ability of eltrombopag to induce sustained remission in subjects with ITP who are refractory or relapsed after first-line steroids (TAPER)

JRCT ID: jRCT2080225175

Registered date:24/04/2020

Basic Information

• Recruitment status: completed
• Health condition(s) or Problem(s) studied: Immune Thrombocytopenia
• Date of first enrollment: 28/02/2020
• Target sample size: 101
• Countries of recruitment: Japan,Asia except Japan,North America,South America,Europe
• Study type: Interventional

Intervention(s)

investigational material(s)
Generic name etc :
INN of investigational material : Eltrombopag
Therapeutic category code : 399 Agents affecting metabolism, n.e.c.
Dosage and Administration for Investigational material :

control material(s)
Generic name etc :
INN of investigational material : -
Therapeutic category code : --- Other
Dosage and Administration for Investigational material :

Outcome(s)

Primary Outcome

efficacy
To assess ability of eltrombopag to induce sustained remission by month 12 in ITP patients who relapsed or failed to respond to first-line steroid treatment

Secondary Outcome

Safety
Efficacy
Exploratory
Pharmacokinetics
Efficacy/efficacy
To assess the duration of sustained remission after discontinuation of investigational product.
To evaluate the effect of eltrombopag in inducing early response by 1 month.
To evaluate the effect of eltrombopag in inducing re-response during or after eltrombopag taper when efficacy is lost.
Platelet count will be quantified from baseline to 3, 6, 9, and 12 months.
To evaluate the effect of eltrombopag in maintaining a platelet count > 30 x 109/L within 12 months.

Safety/Safety
The overall impact of side effects associated with investigational product treatment will be explored using GP5 at baseline and at study completion (EOS).
To evaluate the safety and tolerability of eltrombopag.

Exploratory
To assess the relationship between the immunomodulatory effects of eltrombopag and clinical response.
To determine the effect of investigational product treatment on immunoglobulin levels.
Effects on iron parameters and effects on treatment response and immune modulation will be assessed at 12 months and through the end of treatment.
To be assessed at Month 12 and through end of treatment based on the bleeding scale.

Pharmacokinetics/pharmacokinetics
To evaluate the pharmacokinetics of eltrombopag

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1.Patients >18 years old
2.Patients with a confirmed di(gnosis of primary ITP, who are not responsive or in relapse after a first line of steroid therapy intravenous immunoglobulin (IVIG)(used as a rescue therapy)
First line of steroid therapy will be defined as: prednisone/prednisolone 0.5 to 1 mg/kg/day for a minimum of 2 weeks, or minimum of 1 course of high-dose dexamethasone 20-40 mg/day for consecutive 4 days with or without IVIG (used as rescue therapy). Maximum exposure to high-dose steroids treatment (steroids tapering time excluded) should be limited to: 4 weeks of high dose prednisone/prednisolone or 3 courses of high-dose dexamethasone. Overall exposure to steroids must not be longer than 3 months, including period of dose tapering.
3. Platelet count < 30x109/L and assessed as needing treatment (per physicians discretion)
Other criteria specified in the protocol also apply.

Exclude criteria

1.ITP patients previously treated with any ITP second-line therapies, thrombopoietin receptor (TPO-R) agonists for ITP, except steroids / IVIG
2.Patients who relapsed more than one year after the end of first-line full course of steroid therapy
3.Patients with a diagnosis of secondary thrombocytopenia
4.Patients who have life threatening bleeding complications per investigator discretion
5.Patients who had a deep vein thrombosis or arterial thrombosis in the 6 months preceding enrollment
Other criteria specified in the protocol also apply.

Related Information

• Primary Sponsor: Novartis Pharma. K.K.
• Secondary ID(s): NCT03524612,JapicCTI-205271