Anemia, AplasticPhase 2NCT02857530
Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia
A Phase 2 study testing rhTPO in people with Anemia, Aplastic, run by Tianjin Medical University General Hospital at 1 site in China.
Trial summary
Efficacy and Safety of Recombinant human thrombopoietin in patients with severe aplastic anemia and very severe aplastic anemia, a randomized, double-blind, placebo-controlled, II phase, multi-center clinical research. Read more about this study
- Who is it for?
- Ages 6 to 60 years
- Conditions the study is for
- Anemia, Aplastic
- What is being tested?
- rhTPO
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 250 participants
- Where
- 1 siteChina
- Study dates
- Aug 2016to Jul 2020
- 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 6 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Able to provide written informed consent
- Have severe or very severe aplastic anemia
Who cannot join
- Have diagnosis of Fanconi anemia
- Have infection not adequately responding to appropriate therapy
- Have Paroxysmal Nocturnal Hemoglobinuria (PNH) clone size in neutrophils of greater than or equal to 50%
- Have creatinine and/or blood urea nitrogen (BUN) ≥2 times the upper limit of normal
- Have serum bilirubin ≥ 1.5 times the upper limit of normal, or ≥4.0 times the upper limit of normal if the patient has been treated with Anti-Thymocytes globulin（ATG） within three weeks of screening.
- Have glutamic-oxaloacetic transaminase （AST） and/or glutamic-pyruvic transaminase （ALT） ≥ 3 times the upper limit of normal
Show all 9Show fewer
- Are female and are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential
- Have an Eastern Cooperative Oncology Group (ECOG) performance status of 3 or greater
- Have had other Thrombopoietin (TPO-R) agonists medication in the previous 4 weeks.
Trial locations and contacts
1 site in 1 country.
China1 site
Zonghong Shao
Tianjin, Tianjin Municipality, 300052
ZONGHONG SHAO, MD., 13702036467, shaozonghong@sina.com
About this study
Efficacy and Safety of Recombinant human thrombopoietin in patients with severe aplastic anemia and very severe aplastic anemia, a randomized, double-blind, placebo-controlled, II phase, multi-center clinical research.
Read the detailed description
rhTPO
- Conditions
- Anemia, Aplastic
- Being tested
- rhTPO, placebo
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhTPODrug
Also called Recombinant Human Thrombopoietin
rhTPO
placeboDrug
Also called without Recombinant Human Thrombopoietin
without rhTPO
| Study group | Role | What they receive |
|---|---|---|
| rhTPO | Experimental | rhTPO injection Receives: Drug: rhTPO |
| control | Placebo comparator | without rhTPO injection Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to platelet transfusion independence in patients at 6 month
Measured: 6 month
Secondary outcomes (10)
Time to patients achieve a platelet count 30×10^9/L, 50×10^9/L or100×10^9/L
Measured: up to 1year
Overall response rate up to 1 year after Immunosupressive therapy.
Measured: up to 1 year
Megakaryocyte, granulocyte and erythrocyte recovery in bone marrow up to 1 year after IST.
Measured: up to 1 year
Time to red blood cell/platelet transfusion independence in patients up to 1 year after IST
Measured: up to 1 year
Percentage of patients achieve red blood cell/platelet transfusion independence at 3, 6, 9 and 12 months after IST.
Measured: up to 1 year
Intervals of red blood cell/platelet transfusion up to 1 year after IST.
Measured: up to 1 year
The reticulocyte count of patients up to 1 year after IST.
Measured: up to 1 year
Hospitalization days of patients.
Measured: up to 1 year
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Measured: up to 1 year
Median hematopoietic response rate up to 1 year after IST.
Measured: up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2016 (estimated)
- Study startAug 2016
- Primary completionJul 2019 (estimated)
- Study completionJul 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anemia, AplasticStudies of the same condition, open and under way.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 250 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tianjin Medical University General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, ZhongHong Shao, Tianjin Medical University General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02857530
- Sponsor's protocol ID
- IRB2013-066-01
- Status last verified
- Aug 2016
- First submitted
- 26 Jul 2016
- Last updated
- 5 Aug 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT02857530
- Acronym / sponsor ID
- IRB2013-066-01
- Conditions
- Anemia, Aplastic
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- Tianjin Medical University General HospitalUniversity, hospital or other
- Study start
- Aug 2016
- Primary completion
- Jul 2019 (estimated)
- Study completion
- Jul 2020 (estimated)
- First posted
- 5 Aug 2016 (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.
Efficacy and Safety of Recombinant human thrombopoietin in patients with severe aplastic anemia and very severe aplastic anemia, a randomized, double-blind, placebo-controlled, II phase, multi-center clinical research.
Detailed description
rhTPO
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| rhTPO | Experimental | Drug: rhTPOrhTPO injection |
| control | Placebo comparator | Drug: placebowithout rhTPO injection |
Who may qualify
- Ages
- 6 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Able to provide written informed consent
- Have severe or very severe aplastic anemia
Key exclusion criteria (9 listed)
- Have diagnosis of Fanconi anemia
- Have infection not adequately responding to appropriate therapy
- Have Paroxysmal Nocturnal Hemoglobinuria (PNH) clone size in neutrophils of greater than or equal to 50%
- Have creatinine and/or blood urea nitrogen (BUN) ≥2 times the upper limit of normal
- Have serum bilirubin ≥ 1.5 times the upper limit of normal, or ≥4.0 times the upper limit of normal if the patient has been treated with Anti-Thymocytes globulin（ATG） within three weeks of screening.
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 |
|---|---|---|
| rhTPOAlso: Recombinant Human ThrombopoietinrhTPO | Drug | rhTPO |
| placeboAlso: without Recombinant Human Thrombopoietinwithout rhTPO | Drug | control |
Primary outcome
Time to platelet transfusion independence in patients at 6 month
Time frame: 6 month
10 secondary outcomes
Time to patients achieve a platelet count 30×10^9/L, 50×10^9/L or100×10^9/L
Time frame: up to 1year
Overall response rate up to 1 year after Immunosupressive therapy.
Time frame: up to 1 year
Megakaryocyte, granulocyte and erythrocyte recovery in bone marrow up to 1 year after IST.
Time frame: up to 1 year
Time to red blood cell/platelet transfusion independence in patients up to 1 year after IST
Time frame: up to 1 year
Percentage of patients achieve red blood cell/platelet transfusion independence at 3, 6, 9 and 12 months after IST.
Time frame: up to 1 year
Intervals of red blood cell/platelet transfusion up to 1 year after IST.
Time frame: up to 1 year
The reticulocyte count of patients up to 1 year after IST.
Time frame: up to 1 year
Hospitalization days of patients.
Time frame: up to 1 year
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: up to 1 year
Median hematopoietic response rate up to 1 year after IST.
Time frame: up to 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2016.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing rhTPO in people with Anemia, Aplastic, run by Tianjin Medical University General Hospital at 1 site in China.
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 2 inclusion and 9 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: status not confirmed, as of the sponsor's last update on 5 Aug 2016. 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 China. 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. Some participants in this study may receive a placebo. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact ZONGHONG SHAO, MD. (8613702036467, shaozonghong@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to platelet transfusion independence in patients at 6 month (time frame: 6 month). 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 5 Aug 2016.