Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)Phase 1/2AGRAH003NCT01980030
Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell
A Phase 1/2 study testing Romiplostim in people with Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT), run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
This is a Phase I/II multicenter study which aims to assess the toxicity profile of Romiplostim in patients with transfusion-dependent thrombocytopenia after allogeneic HSCT. A total of 24 patients with transfusion-dependent thrombocytopenia after allogeneic HSCT will be included. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
- What is being tested?
- Romiplostim
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteFrance
- Study dates
- Nov 2013to Nov 2018
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects must be ≥ 18 years, willing and able to sign informed consent
- Patients could have been transplanted for hematological disorder (malignant or non-malignant) excepted myelodysplastic syndromes patients and had received either a myeloablative or a reduced intensity conditioning. All sources of allogeneic stem cells are allowed.
- Prolonged (> 2 months) transfusion-dependent thrombocytopenia
- Screenings mean platelet count≤ 20 x giga/L or screenings mean platelet count ≤ 50 x giga/L with a history of bleeding.
- (ECOG) performance status of 0-2
- Adequate liver function
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- Serum creatinine ≤ 176.8 μmol/L
- Bone marrow aspirate with cytogenetics within 6 days of the first dose of romiplostim
- Written informed consent
Who cannot join
- Relapse/progression of hematological malignancy (marrow examination required)
- Non-controlled acute and/or chronic graft versus host disease (GvHD)
- Active or uncontrolled infections
- Cardiac pathology - Thrombosis
- Pregnancy or breast feeding
- Received interleukin-11 (IL-11) within 4 weeks of screening or previously received any thrombopoietic growth factor
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- Patients on anticoagulant therapy
- Receipt or planned receipt of Pegylated Granulocyte Colony Stimulating Factor (PEG-G-CSF), or Granulocyte macrophage-colony stimulating factor (GM-CSF) within 4 weeks of the first dose of investigational product
- Subject not using adequate contraceptive precautions, in the judgment of the investigator
- Sensitivity to any Escherichia coli-derived product
- Inability to comply with study procedures.
- Subject currently is enrolled in or has not yet completed 30 days since ending other investigational device or drug study
- No medical insurance in the French Health system
Trial locations and contacts
1 site in 1 country.
About this study
This is a Phase I/II multicenter study which aims to assess the toxicity profile of Romiplostim in patients with transfusion-dependent thrombocytopenia after allogeneic HSCT.
A total of 24 patients with transfusion-dependent thrombocytopenia after allogeneic HSCT will be included.
The main endpoint is the incidence and severity of adverse events.
- Conditions
- Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
- Being tested
- Romiplostim
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimDrug
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim | Experimental | Weekly Romiplostim for 12 weeks with intra-patient weekly dose escalation from 1µg/Kg to a maximum dose of 10 µg/Kg with a dose reduction schema in case of platelet overshoot Receives: Drug: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events
Incidence and severity of all adverse events
Measured: 12 months
Secondary outcomes (8)
Dose of Romiplostim
Dose of Romiplostim required to reach a platelet count above 50 x 109/L in absence of platelet transfusion
Measured: 12 months
Durable platelet response after transplant:
platelet count above 50 x 109/L on 8 consecutive weeks independent of platelet transfusions
Measured: 12 months
Relapse rate
Measured: 12 months
Graft versus host disease (GVHD)
Measured: 12 months
Non relapse mortality rate
Measured: 12 months
number of platelet transfusions
Measured: 12 months
Overall number of bleeding events
Measured: 12 months
platelet hematological improvements
Incidence and duration of platelet hematological improvements above 20 x 109/L and above 50 x 109/L , respectively
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2013 (estimated)
- Study startNov 2013
- Primary completionDec 2015
- Study completionNov 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)Studies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01980030
- Sponsor's protocol ID
- P110204
- Acronym
- AGRAH003
- Status last verified
- Feb 2019
- First submitted
- 4 Nov 2013
- Last updated
- 15 Feb 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell
- ClinicalTrials.gov ID
- NCT01980030
- Acronym / sponsor ID
- AGRAH003 / P110204
- Conditions
- Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Nov 2013
- Primary completion
- Dec 2015 (actual)
- Study completion
- Nov 2018 (actual)
- First posted
- 8 Nov 2013 (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.
This is a Phase I/II multicenter study which aims to assess the toxicity profile of Romiplostim in patients with transfusion-dependent thrombocytopenia after allogeneic HSCT.
A total of 24 patients with transfusion-dependent thrombocytopenia after allogeneic HSCT will be included.
The main endpoint is the incidence and severity of adverse events.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Romiplostim | Experimental | Drug: RomiplostimWeekly Romiplostim for 12 weeks with intra-patient weekly dose escalation from 1µg/Kg to a maximum dose of 10 µg/Kg with a dose reduction schema in case of platelet overshoot |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Subjects must be ≥ 18 years, willing and able to sign informed consent
- Patients could have been transplanted for hematological disorder (malignant or non-malignant) excepted myelodysplastic syndromes patients and had received either a myeloablative or a reduced intensity conditioning. All sources of allogeneic stem cells are allowed.
- Prolonged (> 2 months) transfusion-dependent thrombocytopenia
- Screenings mean platelet count≤ 20 x giga/L or screenings mean platelet count ≤ 50 x giga/L with a history of bleeding.
- (ECOG) performance status of 0-2
Key exclusion criteria (13 listed)
- Relapse/progression of hematological malignancy (marrow examination required)
- Non-controlled acute and/or chronic graft versus host disease (GvHD)
- Active or uncontrolled infections
- Cardiac pathology - Thrombosis
- Pregnancy or breast feeding
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 |
|---|---|---|
| Romiplostim | Drug | Romiplostim |
Primary outcome
Adverse events
Time frame: 12 months
Incidence and severity of all adverse events
8 secondary outcomes
Dose of Romiplostim
Time frame: 12 months
Durable platelet response after transplant:
Time frame: 12 months
Relapse rate
Time frame: 12 months
Graft versus host disease (GVHD)
Time frame: 12 months
Non relapse mortality rate
Time frame: 12 months
number of platelet transfusions
Time frame: 12 months
Overall number of bleeding events
Time frame: 12 months
platelet hematological improvements
Time frame: 12 months
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 15 Feb 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Romiplostim in people with Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT), run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 9 inclusion and 13 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 15 Feb 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 France. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Adverse events (time frame: 12 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 15 Feb 2019.