Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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
  Purpose

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.


Condition Intervention Phase
Idiopathic Thrombocytopenic Purpura
Drug: Anti-D
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Treatment of Idiopathic Thrombocytopenic Purpura in Children With Subcutaneously Administered Anti-D

Resource links provided by NLM:


Further study details as provided by University of Aarhus:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • Platelet count [ Time Frame: day 0, 1, 3, 6, 14, 30, 180, 360 ] [ Designated as safety issue: Yes ]

Enrollment: 45
Study Start Date: November 2004
Study Completion Date: December 2008
Primary Completion Date: January 2008 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Anti-D
    Subcutaneous injection
Detailed 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.

  Eligibility

Ages Eligible for Study:   1 Year to 14 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Idiopathic thrombocytopenic purpura (ITP)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00128882

Locations
Denmark
Aalborg University Hospital, Department of Pediatrics
Aalborg, Denmark, 9100
Skejby Hospital, Aarhus University Hospital, Department of Pediatrics
Aarhus N, Denmark, 8200
Amager Hospital, Department of Pediatrics
Copenhagen S, Denmark, 2300
Rigshospitalet, Copenhagen University Hospital, Pediatric Clinic II
Copenhagen Ø, Denmark, 2100
Esbjerg Hospital, Department of Pediatrics
Esbjerg, Denmark, 6700
Gentofte Hospital, Department of Pediatrics
Gentofte, Denmark, 2900
Herning Hospital, Department of Pediatrics
Herning, Denmark, 7400
Hjoerring Hospital, Department of Pediatrics
Hjoerring, Denmark, 9800
Holbæk Hospital, Department of Pediatrics
Holbæk, Denmark, 4300
Hvidovre Hospital, Department of Pediatrics
Hvidovre, Denmark, 2650
Kolding Hospital Department of Pediatrics
Kolding, Denmark, 6000
Nykøbing F, Department of Pediatrics
Nykøbing F, Denmark, 4800
Næstved Hospital, Department of Pediatrics
Næstved, Denmark, 4700
Odense University Hospital
Odense C, Denmark, 5000
Randers Hospital, Department of Pediatrics
Randers, Denmark, 8900
Sønderborg Hospital, Department of Pediatrics
Sønderborg, Denmark, 6400
Viborg Hospital, Department of Pediatrics
Viborg, Denmark, 8800
Sponsors and Collaborators
University of Aarhus
Kjaersgaard, Mimi, M.D.
Investigators
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
  More Information

No publications provided

Responsible Party: Mimi Kjaersgaard, MD, University of Aarhus
ClinicalTrials.gov Identifier: NCT00128882     History of Changes
Other Study ID Numbers: MK-2005-1, 2003179, 2612-2447
Study First Received: August 8, 2005
Last Updated: February 2, 2009
Health Authority: Denmark: Danish Medicines Agency

Keywords provided by University of Aarhus:
Idiopathic Thrombocytopenic Purpura
Treatment
Anti-D
Subcutaneous
Clinical evaluation
Platelet count
IVIG

Additional relevant MeSH terms:
Purpura
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Autoimmune Diseases
Blood Coagulation Disorders
Blood Platelet Disorders
Hematologic Diseases
Hemorrhage
Hemorrhagic Disorders
Immune System Diseases
Pathologic Processes
Signs and Symptoms
Skin Manifestations
Thrombocytopenia
Thrombotic Microangiopathies

ClinicalTrials.gov processed this record on November 20, 2014