Treatment of Idiopathic Thrombocytopenic Purpura (ITP) With Subcutaneously Administered Anti-D

Idiopathic Thrombocytopenic Purpura Clinical Trial

Official title:

Treatment of Idiopathic Thrombocytopenic Purpura in Children With Subcutaneously Administered Anti-D

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00128882
• Other study ID #: MK-2005-1
• Secondary ID: 20031792612-2447
• Status: Completed
• Phase: Phase 2
• First received: August 8, 2005
• Last updated: February 2, 2009
• Start date: November 2004
• Est. completion date: December 2008

Study information

• Verified date: February 2009
• Source: University of Aarhus
• Contact: n/a
• Is FDA regulated: No
• Health authority: Denmark: Danish Medicines Agency
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to describe the effect of anti-D on symptoms and platelet count in children suffering from unexplainable low platelet counts, when anti-D is administered as an injection under the skin. The hypothesis is that the injection with anti-D under the skin is as effective as anti-D given in a vein.

Description:

Background:

Idiopathic thrombocytopenic purpura (ITP) in children is considered a benign hematological disease. The incidence is approximately 50 cases a year in Denmark. Approximately 25 % will experience chronic disease. Follow up and treatment of these patients is not centralized.

The drug of choice is intravenous IgG (IVIG) for treatment of ITP. The side effects are flu-like symptoms, and in rare cases aseptic meningitis. Another option is intravenous anti-D, if the child is rhesus positive. Anti-D is primarily used in North America. The effect of Anti-D is comparable with IVIG when considering the time it takes to bring the platelet count above 50,000/μL. Anti-D also causes flu-like symptoms. Establishing an i.v. access is a disadvantage to both IVIG and anti-D. For both treatments mechanism of action is not finally described.

Subcutaneous IgG substitution therapy is used for patients suffering from agammaglobulinaemia. It is therefore known, that immunoglobulin uptake is possible after subcutaneous administration. Subcutaneous anti-D has been tried in few patients suffering from chronic thrombocytopenia with positive results.

IVIG treatment is expensive compared to anti-D. Treatment of a 20 kg child costs approximately 17,000 Dkr for IVIG and 2,500 Dkr. for anti-D.

Hypothesis:

• Subcutaneous administered anti-D is as effective as IVIG/i.v. anti-D;

• Subcutaneous administered anti-D has fewer less severe side effects than IVIG/i.v. anti-D.

Purpose:

• To document the effect of subcutaneous anti-D;

• Describe complications;

• Describe aspects of the mechanism of action.

Material and Methods:

Children are eligible if admitted to a pediatric department in Denmark for diagnosis, observation or treatment of acute or chronic ITP. Examination and diagnostic work up is similar throughout the country, but not identical. No specific tests are required for diagnosis. If treatment is indicated rhesus positive children are treated with subcutaneous anti-D. Rhesus negative children are treated according to local guidelines. Specified follow-up on all children is mandatory. For research purposes one blood sample form all children is collected, and from children, who receive medical treatment, several blood samples are collected. Analysis for changes in immunological signaling peptides will be performed with special attention to the mechanism of action of anti-D.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 45
• Est. completion date: December 2008
• Est. primary completion date: January 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 14 Years

Eligibility

Inclusion Criteria:

• Idiopathic thrombocytopenic purpura (ITP)

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Idiopathic Thrombocytopenic Purpura
• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombocytopenic, Idiopathic

Intervention

Drug:
• Anti-D
Subcutaneous injection

Locations

Country	Name	City	State
Denmark	Aalborg University Hospital, Department of Pediatrics	Aalborg
Denmark	Skejby Hospital, Aarhus University Hospital, Department of Pediatrics	Aarhus N
Denmark	Rigshospitalet, Copenhagen University Hospital, Pediatric Clinic II	Copenhagen Ø
Denmark	Amager Hospital, Department of Pediatrics	Copenhagen S
Denmark	Esbjerg Hospital, Department of Pediatrics	Esbjerg
Denmark	Gentofte Hospital, Department of Pediatrics	Gentofte
Denmark	Herning Hospital, Department of Pediatrics	Herning
Denmark	Hjoerring Hospital, Department of Pediatrics	Hjoerring
Denmark	Holbæk Hospital, Department of Pediatrics	Holbæk
Denmark	Hvidovre Hospital, Department of Pediatrics	Hvidovre
Denmark	Kolding Hospital Department of Pediatrics	Kolding
Denmark	Næstved Hospital, Department of Pediatrics	Næstved
Denmark	Nykøbing F, Department of Pediatrics	Nykøbing F
Denmark	Odense University Hospital	Odense C
Denmark	Randers Hospital, Department of Pediatrics	Randers
Denmark	Sønderborg Hospital, Department of Pediatrics	Sønderborg
Denmark	Viborg Hospital, Department of Pediatrics	Viborg

Sponsors (2)

Lead Sponsor	Collaborator
University of Aarhus	Kjaersgaard, Mimi, M.D.

Country where clinical trial is conducted

Denmark, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Clinical effect evaluated on clinical score scale before and after treatment at specified intervals		day 0,1,3,6,14,30, 180,360	No
Secondary	Platelet count		day 0, 1, 3, 6, 14, 30, 180, 360	Yes