Severe Aplastic AnemiaPhase 3NCT03825744
Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
A Phase 3 study testing Hetrombopag Olamine+Standard Therapy in people with Severe Aplastic Anemia, run by Jiangsu HengRui Medicine Co., Ltd. at 1 site in China.
Trial summary
This is a multicenter, randomized, placebo-control, phase III study to investigate hetrombopag in subjects with severe AA who are treated naive. 180 treated naive patients with SAA will be enrolled in the study. The primary objective of the study will be the rate of complete hematologic response at six months. Read more about this study
- Who is it for?
- Ages 15 to 75 years
- Conditions the study is for
- Severe Aplastic Anemia
- What is being tested?
- Hetrombopag Olamine+Standard Therapy
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 240 participants
- Where
- 1 siteChina
- Study dates
- 5 Mar 2019to 3 Jul 2024
- Phase
- Phase 3
- 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 15 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 1.15 Years to 75 Years (weight greater than 50 kg if Age < 18 years old). 2.Severe aplastic anemia characterized by Bone marrow cell proliferation less than 25 percent (If ≥25% but <50%, the remaining hematopoietic cells should be <30%) AND At least two of the following: Absolute neutrophil count <0.5×109/L; Platelet count <20×109/L; Absolute reticulocyte count <20×109/L.
- 3.Unsuitable or unwilling to perform hematopoietic stem cell transplantation (HSCT).
- 4.Signed informed consent.
Who cannot join
- Diagnosis of whole blood cell reduction due to other causes or bone marrow hypoproliferative diseases.
- Subjects who have previously received immunosuppressive therapy with mycophenolate mofetil, sirolimus, high dose cyclophosphamide (≥45mg/kg/d), alemtuzumab, etc；or have treated with thrombopoietin receptor agonist (eg, eltrombopag, romiplostim, rhTPO, etc.) prior to randomization.
- Previous history of hematopoietic stem cell transplantation.
- Subjects who is known or suspected of contraindications or hypersensitivity to Hetrombopag's API (Active Pharmaceutical Ingredient).
- Evidence of clonal cytogenetic abnormalities at the time of screening.
- Bleeding and/or Infection not adequately responding to appropriate therapy.
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- Any laboratory or clinical evidence for HIV infection. Any clinical history for hepatitis C infection; chronic hepatitis B infection; or any evidence for active hepatitis at the time of subjects screening.
- ALT> 2.5 x upper limit of normal (ULN), AST> 2.5 x upper limit of normal (ULN) DBLI> 1.5 x upper limit of normal (ULN), Scr> upper limit of normal (ULN).
- Subjects with uncontrolled hypertension (>180/100mmHg), severe arrhythmia (such as complete left bundle branch block, QT interval prolongation (Bazetts formula), torsade ventricular tachycardia, etc.), unstable angina, pulmonary hypertension at the time of screening.
- Subjects diagnosed with cirrhosis or portal hypertension.
- Subjects with malignant solid tumors of any organ system within 5 years prior to screening, with or without treatment, metastasis or recurrence, except for local cutaneous basal cell carcinoma; subjects with hematological tumors found previously or during screening.
- Subjects with deep vein thrombosis, myocardial infarction, stroke or peripheral arterial embolization within 12 months prior to randomization.
- Female subjects who are nursing or pregnant.
- Subjects cannot comply with effective contraception.
- Subjects have participated in other clinical trial within the 3 months prior to study entry.
Trial locations and contacts
1 site in 1 country.
China1 site
Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300041
About this study
Official title: A Phase III Multicenter Randomized Study of Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
This is a multicenter, randomized, placebo-control, phase III study to investigate hetrombopag in subjects with severe AA who are treated naive.
180 treated naive patients with SAA will be enrolled in the study. The primary objective of the study will be the rate of complete hematologic response at six months.
- Conditions
- Severe Aplastic Anemia
- Being tested
- Hetrombopag Olamine+Standard Therapy, Placebo+Standard Therapy
- Keywords
- SAA
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hetrombopag Olamine+Standard TherapyDrug
once daily
Placebo+Standard TherapyDrug
once daily
| Study group | Role | What they receive |
|---|---|---|
| Hetrombopag Olamine+Standard Therapy | Experimental | Not described Receives: Drug: Hetrombopag Olamine+Standard Therapy |
| Placebo+Standard Therapy | Placebo comparator | Not described Receives: Drug: Placebo+Standard Therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of complete hematologic response at six months.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2019
- Study start5 Mar 2019
- Primary completion3 Jul 2024
- Study completion3 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Jiangsu HengRui Medicine Co., Ltd.The 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 240 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jiangsu HengRui Medicine Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03825744
- Sponsor's protocol ID
- HR-TPO-SAA-III
- Status last verified
- Jan 2024
- First submitted
- 30 Jan 2019
- Last updated
- 7 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III Multicenter Randomized Study of Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT03825744
- Acronym / sponsor ID
- HR-TPO-SAA-III
- Conditions
- Severe Aplastic Anemia
- Keywords
- SAA
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 240 participants (actual)
- Lead sponsor
- Jiangsu HengRui Medicine Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 5 Mar 2019 (actual)
- Primary completion
- 3 Jul 2024 (actual)
- Study completion
- 3 Jul 2024 (actual)
- First posted
- 31 Jan 2019 (actual)
- 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 multicenter, randomized, placebo-control, phase III study to investigate hetrombopag in subjects with severe AA who are treated naive.
180 treated naive patients with SAA will be enrolled in the study. The primary objective of the study will be the rate of complete hematologic response at six months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hetrombopag Olamine+Standard Therapy | Experimental | Drug: Hetrombopag Olamine+Standard Therapy |
| Placebo+Standard Therapy | Placebo comparator | Drug: Placebo+Standard Therapy |
Who may qualify
- Ages
- 15 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 1.15 Years to 75 Years (weight greater than 50 kg if Age < 18 years old). 2.Severe aplastic anemia characterized by Bone marrow cell proliferation less than 25 percent (If ≥25% but <50%, the remaining hematopoietic cells should be <30%) AND At least two of the following: Absolute neutrophil count <0.5×109/L; Platelet count <20×109/L; Absolute reticulocyte count <20×109/L.
- 3.Unsuitable or unwilling to perform hematopoietic stem cell transplantation (HSCT).
- 4.Signed informed consent.
Key exclusion criteria (15 listed)
- Diagnosis of whole blood cell reduction due to other causes or bone marrow hypoproliferative diseases.
- Subjects who have previously received immunosuppressive therapy with mycophenolate mofetil, sirolimus, high dose cyclophosphamide (≥45mg/kg/d), alemtuzumab, etc；or have treated with thrombopoietin receptor agonist (eg, eltrombopag, romiplostim, rhTPO, etc.) prior to randomization.
- Previous history of hematopoietic stem cell transplantation.
- Subjects who is known or suspected of contraindications or hypersensitivity to Hetrombopag's API (Active Pharmaceutical Ingredient).
- Evidence of clonal cytogenetic abnormalities at the time 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 |
|---|---|---|
| Hetrombopag Olamine+Standard Therapyonce daily | Drug | Hetrombopag Olamine+Standard Therapy |
| Placebo+Standard Therapyonce daily | Drug | Placebo+Standard Therapy |
Primary outcome
Rate of complete hematologic response at six months.
Time frame: 6 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 7 Nov 2024.
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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 3 study testing Hetrombopag Olamine+Standard Therapy in people with Severe Aplastic Anemia, run by Jiangsu HengRui Medicine Co., Ltd. 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 3 inclusion and 15 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 7 Nov 2024. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Rate of complete hematologic response at six months. (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 7 Nov 2024.