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

This study has been completed.
Sponsor:
Collaborator:
Kjaersgaard, Mimi, M.D.
Information provided by:
University of Aarhus
ClinicalTrials.gov Identifier:
NCT00128882
First received: August 8, 2005
Last updated: February 2, 2009
Last verified: February 2009

August 8, 2005
February 2, 2009
November 2004
January 2008   (final data collection date for primary outcome measure)
Clinical effect evaluated on clinical score scale before and after treatment at specified intervals [ Time Frame: day 0,1,3,6,14,30, 180,360 ] [ Designated as safety issue: No ]
  • Clinical effect evaluated on clinical score scale before and after treatment at specified intervals
  • Side effects (safety)
Complete list of historical versions of study NCT00128882 on ClinicalTrials.gov Archive Site
Platelet count [ Time Frame: day 0, 1, 3, 6, 14, 30, 180, 360 ] [ Designated as safety issue: Yes ]
Platelet count
Not Provided
Not Provided
 
Treatment of Idiopathic Thrombocytopenic Purpura (ITP) With Subcutaneously Administered Anti-D
Treatment of Idiopathic Thrombocytopenic Purpura in Children With Subcutaneously Administered Anti-D

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.

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.

Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Idiopathic Thrombocytopenic Purpura
Drug: Anti-D
Subcutaneous injection
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
45
December 2008
January 2008   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Idiopathic thrombocytopenic purpura (ITP)
Both
1 Year to 14 Years
No
Contact information is only displayed when the study is recruiting subjects
Denmark
 
NCT00128882
MK-2005-1, 2003179, 2612-2447
Yes
Mimi Kjaersgaard, MD, University of Aarhus
University of Aarhus
Kjaersgaard, Mimi, M.D.
Study Director: Mimi Kjaersgaard, MD University of Aarhus, Clinical Institute, Department of Pediatrics
Principal Investigator: Henrik Hasle, MD PhD Skejby Hospital, University of Aarhus, Department of Pediatrics
University of Aarhus
February 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP