NCT01713738

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
36

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started May 2003

Typical duration for phase_1

Geographic Reach
1 country

9 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2003

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2005

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2005

Completed
6.9 years until next milestone

First Submitted

Initial submission to the registry

October 18, 2012

Completed
7 days until next milestone

First Posted

Study publicly available on registry

October 25, 2012

Completed
Last Updated

November 9, 2012

Status Verified

November 1, 2012

Enrollment Period

2.6 years

First QC Date

October 18, 2012

Last Update Submit

November 8, 2012

Conditions

Idiopathic Thrombocytopenic Purpura (ITP)Immune Thrombocytopenic Purpura (ITP)

Keywords

ITPrituximabbleeding scoreplatelet counthealth-related quality of lifeHRQL

Outcome Measures

Primary Outcomes (2)

  • platelet levels

    9 - 12 weeks after 1st dose of rituximab

  • hypogammaglobulinemia

    over one year

Secondary Outcomes (5)

  • fraction of responsive patients maintaining response over 1 year

    week 52

  • assessment of need for salvage therapy

    first 12 weeks of trial

  • rate of early response before day 57

    before day 57, and 4 additional weeks

  • trend of bleeding scores throughout trial

    over one year

  • description of health-related quality of life

    over one year

Study Arms (1)

rituximab

EXPERIMENTAL
Drug: rituximab

Interventions

rituximabDRUG

infusion of 4 weekly doses of 375 mg/m2 rituximab

rituximab

Eligibility Criteria

Age18 Months - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • severe, chronic ITP, including refractory; at least 6 months from diagnosis for refractory; at least 12 months from diagnosis for severe; platelet counts \<10,000/mm3 twice in past 3 months without bleeding; platelet counts \<20,000/mm3 twice in past 3 months with bleeding

You may not qualify if:

  • ever had B or T cell neoplasm; HIV/AIDS; allergy to murine antibodies; treatment with investigational immunosuppressive strategies within past 3 months -

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

UCLA, Mattel Children's Hospital

Los Angeles, California, 90095, United States

Location

Stanford University School of Medicine

Palo Alto, California, 94305, United States

Location

University of California, San Francisco

San Francisco, California, 94143, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30322, United States

Location

Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

Van Eslander Cancer Center, St. John Hospital

Detroit, Michigan, 48236, United States

Location

Weill Medical College at Cornell University

New York, New York, 10021, United States

Location

Southwestern Medical Center at Dallas

Dallas, Texas, 75390, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Rituximab

Condition Hierarchy (Ancestors)

Purpura, ThrombocytopenicPurpuraBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesThrombotic MicroangiopathiesThrombocytopeniaBlood Platelet DisordersCytopeniaHemorrhagic DisordersAutoimmune DiseasesImmune System DiseasesHemorrhagePathologic ProcessesPathological Conditions, Signs and SymptomsSkin ManifestationsSigns and Symptoms

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Murine-DerivedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulins

Study Officials

  • Ellis J Neufeld, MD, PhD

    Boston Children's Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDIV

Study Record Dates

First Submitted

October 18, 2012

First Posted

October 25, 2012

Study Start

May 1, 2003

Primary Completion

December 1, 2005

Study Completion

December 1, 2005

Last Updated

November 9, 2012

Record last verified: 2012-11

Locations

US(9)