The purpose of the study is to evaluate the safety and efficacy of rituximab in children
ages 18 months to 18 years, who have severe, chronic ITP. Eligible patients with either
primary or secondary ITP are treated with rituximab once a week for 4 doses, and then
followed for up to one year. Response is defined as having a platelet count greater than
or equal to 50,000/mL on four consecutive weekly measures beginning anytime in weeks 9 -
12.

Clinical Trial of Rituximab in Children and Adolescents With Chronic Idiopathic Thrombocytopenic Purpura (ITP)

Idiopathic Thrombocytopenic Purpura (ITP)

Immune Thrombocytopenic Purpura (ITP)

The purpose of this open label, phase I/II study is to evaluate the safety and efficacy
of rituximab in children ages 18 months to 18 years, who have severe, chronic ITP.
Eligible patients with either primary or secondary ITP are treated with rituximab once a
week for 4 doses, and then followed for up to one year. The primary and secondary
objectives for safety and efficacy are as follows:

Primary

  1. Efficacy: To evaluate the effectiveness of rituximab in severe or refractory
     pediatric ITP, with response defined as follows: platelet count greater than or
     equal to 50,000/mL on four consecutive weekly measures beginning anytime in weeks 9
     - 12 (day 57 - day 84), with the first and fourth measure being spaced at least 22
     days apart (i.e., once established during the 9 - 12 week timeframe, the response
     would be defined as beginning at the first one of these measures). All measurements
     must be independent of supportive care, as follows: 1) no IVIG (intravenous
     immunoglobulin) administration within 7 days of the first measure or at any time
     between measures, 2) no initiation of a 4-day corticosteroid "pulse" within 7 days
     of the first measure or at any time between measures, 3) no RHo (D) immunoglobulin
     (WinRHo-SDFTM ) administration within 7 days of the first measure or at any time
     between measures, and 4) no platelet transfusions administered within 7 days of the
     first measure or at any time between measures.

  2. Safety: To obtain further safety information on rituximab in this clinical setting
     using Genentech standard safety monitoring and SAE reporting. In addition, the
     frequency of hypogammaglobulinemia will be assessed as the incidence of IgG levels
     <1/2 the lower limit of normal for age.

Secondary

  1. To evaluate the one-year clinical course of severe or refractory ITP patients
     treated with a single course of rituximab. What fraction of responsive patients
     maintains this response?

  2. To assess the need for salvage therapy (supportive care) in this severely affected
     group of patients during the clinical trial.

  3. To evaluate the rate of "early response," defined as: platelets greater than or
     equal to 50,000/mL at least one week off rescue therapy, BEFORE day 57, and
     continuing for four consecutive weeks.

  4. To follow the trend of bleeding scores from onset of therapy through the duration of
     the trial.

  5. To assess the incidence of hypogammaglobulinemia requiring intervention (IgG level
     <1/2 of lower limit of normal for age) in this setting. IVIG 400 mg/kg monthly will
     be used to treat hypogammaglobulinemia.

  6. To describe the health-related quality of life (HRQL) of children with severe or
     refractory ITP, based on parent report and to assess the impact on the family, using
     standardized questionnaires. This is a pilot-scale objective.

Clinical Trial of Rituximab in Children and Adolescents With Chronic Idiopathic Thrombocytopenic Purpura (ITP)

About this trial

Study ID

Status

Type

Phase

Placebo

Accepting

Not accepting

Trial Timing

What is this trial about?

What are the participation requirements?

Locations

Location

Status