Idiopathic Thrombocytopenic PurpuraPhase 2NCT00128882
Treatment of Idiopathic Thrombocytopenic Purpura (ITP) With Subcutaneously Administered Anti-D
A Phase 2 study testing Anti-D in people with Idiopathic Thrombocytopenic Purpura, run by University of Aarhus at 17 sites in Denmark.
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. Read more about this study
- Who is it for?
- Ages 1 year to 14 years
- Conditions the study is for
- Idiopathic Thrombocytopenic Purpura
- What is being tested?
- Anti-D
- Type of treatment
- Injection or infusion
- How many people
- 45 participants
- Where
- 17 sitesDenmark
- Study dates
- Nov 2004to Dec 2008
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Idiopathic thrombocytopenic purpura (ITP)
Who cannot join
None listed.
Trial locations and contacts
17 sites in 1 country.
17 sites in 1 country
Denmark17 sites
Aalborg University Hospital, Department of Pediatrics
Aalborg, 9100
Skejby Hospital, Aarhus University Hospital, Department of Pediatrics
Aarhus N, 8200
Rigshospitalet, Copenhagen University Hospital, Pediatric Clinic II
Copenhagen Ø, 2100
Amager Hospital, Department of Pediatrics
Copenhagen S, 2300
Esbjerg Hospital, Department of Pediatrics
Esbjerg, 6700
Gentofte Hospital, Department of Pediatrics
Gentofte Municipality, 2900
Herning Hospital, Department of Pediatrics
Herning, 7400
Hjoerring Hospital, Department of Pediatrics
Hjørring, 9800
Holbæk Hospital, Department of Pediatrics
Holbæk, 4300
Hvidovre Hospital, Department of Pediatrics
Hvidovre, 2650
Kolding Hospital Department of Pediatrics
Kolding, 6000
Næstved Hospital, Department of Pediatrics
Næstved, 4700
Nykøbing F, Department of Pediatrics
Nykøbing Falster, 4800
Odense University Hospital
Odense C, 5000
Randers Hospital, Department of Pediatrics
Randers, 8900
Sønderborg Hospital, Department of Pediatrics
Sønderborg, 6400
Viborg Hospital, Department of Pediatrics
Viborg, 8800
About this study
Official title: 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.
Read the 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.
- Conditions
- Idiopathic Thrombocytopenic Purpura
- Being tested
- Anti-D
- Keywords
- Idiopathic Thrombocytopenic Purpura, Treatment, Anti-D, Subcutaneous, Clinical evaluation, Platelet count, IVIG
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anti-DDrug
Subcutaneous injection
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical effect evaluated on clinical score scale before and after treatment at specified intervals
Measured: day 0,1,3,6,14,30, 180,360
Secondary outcomes (1)
Platelet count
Measured: day 0, 1, 3, 6, 14, 30, 180, 360
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Aug 2005 (estimated)
- Study startNov 2004
- Primary completionJan 2008
- Study completionDec 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- More trials run by University of AarhusThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Aarhus
- Sponsor type
- University, hospital or other
- Collaborators
- Kjaersgaard, Mimi, M.D.
- Investigators
- Mimi Kjaersgaard, MD, Study director, University of Aarhus, Clinical Institute, Department of PediatricsHenrik Hasle, MD PhD, Principal investigator, Skejby Hospital, University of Aarhus, Department of Pediatrics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00128882
- Sponsor's protocol ID
- MK-2005-1
- Other IDs
- 2003179, 2612-2447
- Status last verified
- Feb 2009
- First submitted
- 8 Aug 2005
- Last updated
- 3 Feb 2009
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Idiopathic Thrombocytopenic Purpura in Children With Subcutaneously Administered Anti-D
- ClinicalTrials.gov ID
- NCT00128882
- Acronym / sponsor ID
- MK-2005-1
- Conditions
- Idiopathic Thrombocytopenic Purpura
- Keywords
- Idiopathic Thrombocytopenic Purpura, Treatment, Anti-D, Subcutaneous, Clinical evaluation, Platelet count, IVIG
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- University of AarhusUniversity, hospital or other
- Collaborators
- Kjaersgaard, Mimi, M.D.
- Study start
- Nov 2004
- Primary completion
- Jan 2008 (actual)
- Study completion
- Dec 2008 (actual)
- First posted
- 10 Aug 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 1 year to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Idiopathic thrombocytopenic purpura (ITP)
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Anti-DSubcutaneous injection | Drug | – |
Primary outcome
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
1 secondary outcome
Platelet count
Time frame: day 0, 1, 3, 6, 14, 30, 180, 360
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Feb 2009.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mimi Kjaersgaard, MD study director
University of Aarhus, Clinical Institute, Department of Pediatrics
Henrik Hasle, MD PhD principal investigator
Skejby Hospital, University of Aarhus, Department of Pediatrics
17 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Anti-D in people with Idiopathic Thrombocytopenic Purpura, run by University of Aarhus at 17 sites in Denmark.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Feb 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Feb 2009.