Idiopathic Thrombocytopenic PurpuraNCT01443351
Long-term Safety Study of Treatment With the Thrombopoietin Agonists Eltrombopag and Romiplostim in Patients With Immune Thrombocytopenia (ITP)
An observational study of Idiopathic Thrombocytopenic Purpura, run by Zealand University Hospital at 1 site in Denmark.
Trial summary
Thrombopoietin Receptor Agonists (TPO-ra) are novel treatment modalities for patients with refractory Primary Immune Thrombocytopenia (ITP), but only few data are available for long-term effects of these drugs. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Idiopathic Thrombocytopenic Purpura
- How many people
- 0 participants
- Where
- 1 siteDenmark
- Study dates
- Mar 2015to Aug 2019
- Phase
- Observational
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patinets will be selected from Departments of Hematology
Who may be able to join
- Adult patients with ITP
- platelet counts < 25 x10*9/L or < 50 x10*9/L with bleeding symptoms
- meets criteria for treatment with TPO-ra
- Females must use contraceptives when applicable for at least three months before inclusion
Who cannot join
- Pregnancy or nursing
- Former thromboembolic events excluding one incidence of deep venous thrombosis as complication to surgery or pregnancy or one incidence of cerebral embolism as complication to atrial fibrillation
- Liver insufficiency (for eltrombopag only)
- TPO-ra contraindications (e.g. allergy)
- TPO-ra treatments less than 6 months prior to inclusion
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Long-term Safety Study of Treatment With the Thrombopoietin Agonists Eltrombopag and Romiplostim in Patients With Primary Immune Thrombocytopenia (ITP)
Thrombopoietin Receptor Agonists (TPO-ra) are novel treatment modalities for patients with refractory Primary Immune Thrombocytopenia (ITP), but only few data are available for long-term effects of these drugs. In this observational study, effects and adverse effects including evaluation of bone marrow biopsies done at fixed intervals will be recorded from ITP patients treated with TPO-ra. For some patients, blood samples will be collected for research use.
- Conditions
- Idiopathic Thrombocytopenic Purpura
- Keywords
- ITP, romiplostim, eltrombopag, bone marrow fibrosis, long-term safety
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| ITP patients | Patients with refractory ITP eligible for treatment with TPO-ra |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with sustained response at 6 months follow-up
Response is defined as platelet count > 30.000 mio/L and no bleeding
Measured: 6 months
Secondary outcomes (4)
Yearly response rates
Number of patients with response to treatment at 1, 2, 3, 4, and 5-year follow-up
Measured: 5 years
Frequency of relapse
Measured: 5 years
Rate of splenectomy
Measured: 5 years
Development of reticulin fibrosis in bone marrow biopsies
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2011 (estimated)
- Study startMar 2015
- Primary completionAug 2019
- Study completionAug 2019
Why it stopped: failure to recruit patients
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 Zealand University HospitalThe 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zealand University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sif Gudbrandsdottir, Zealand University Hospital
- Investigators
- Hans Hasselbalch, Prof., MD, Principal investigator, Department of Hematology, Copenhagen University Hospital RoskildeHenrik Frederiksen, Dr., MD, Principal investigator, Odense University HospitalSif Gudbrandsdottir, MD, Principal investigator, Copenhagen University Hospital Roskilde
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01443351
- Sponsor's protocol ID
- TPO-SJ-227
- Status last verified
- Sep 2019
- First submitted
- 13 Sep 2011
- Last updated
- 11 Sep 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Safety Study of Treatment With the Thrombopoietin Agonists Eltrombopag and Romiplostim in Patients With Primary Immune Thrombocytopenia (ITP)
- ClinicalTrials.gov ID
- NCT01443351
- Acronym / sponsor ID
- TPO-SJ-227
- Conditions
- Idiopathic Thrombocytopenic Purpura
- Keywords
- ITP, romiplostim, eltrombopag, bone marrow fibrosis, long-term safety
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: failure to recruit patients
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Zealand University HospitalUniversity, hospital or other
- Collaborators
- Copenhagen University Hospital, Rigshospitalet, Odense University Hospital
- Study start
- Mar 2015 (actual)
- Primary completion
- Aug 2019 (actual)
- Study completion
- Aug 2019 (actual)
- First posted
- 29 Sep 2011 (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.
Thrombopoietin Receptor Agonists (TPO-ra) are novel treatment modalities for patients with refractory Primary Immune Thrombocytopenia (ITP), but only few data are available for long-term effects of these drugs. In this observational study, effects and adverse effects including evaluation of bone marrow biopsies done at fixed intervals will be recorded from ITP patients treated with TPO-ra. For some patients, blood samples will be collected for research use.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ITP patients | – | –Patients with refractory ITP eligible for treatment with TPO-ra |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patinets will be selected from Departments of Hematology
Key inclusion criteria (4 listed)
- Adult patients with ITP
- platelet counts < 25 x10*9/L or < 50 x10*9/L with bleeding symptoms
- meets criteria for treatment with TPO-ra
- Females must use contraceptives when applicable for at least three months before inclusion
Key exclusion criteria (5 listed)
- Pregnancy or nursing
- Former thromboembolic events excluding one incidence of deep venous thrombosis as complication to surgery or pregnancy or one incidence of cerebral embolism as complication to atrial fibrillation
- Liver insufficiency (for eltrombopag only)
- TPO-ra contraindications (e.g. allergy)
- TPO-ra treatments less than 6 months prior to inclusion
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Number of patients with sustained response at 6 months follow-up
Time frame: 6 months
Response is defined as platelet count > 30.000 mio/L and no bleeding
4 secondary outcomes
Yearly response rates
Time frame: 5 years
Frequency of relapse
Time frame: 5 years
Rate of splenectomy
Time frame: 5 years
Development of reticulin fibrosis in bone marrow biopsies
Time frame: 5 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2019.
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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.
Hans Hasselbalch, Prof., MD principal investigator
Department of Hematology, Copenhagen University Hospital Roskilde
Henrik Frederiksen, Dr., MD principal investigator
Odense University Hospital
Sif Gudbrandsdottir, MD principal investigator
Copenhagen University Hospital Roskilde
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Idiopathic Thrombocytopenic Purpura, run by Zealand University Hospital at 1 site 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 4 inclusion and 5 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: withdrawn, as of the sponsor's last update on 11 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Number of patients with sustained response at 6 months follow-up (time frame: 6 months). 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 11 Sep 2019.