Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 15 January 2024
• Main ID: NCT03825744
• Date of registration: 30/01/2019
• Prospective Registration: Yes
• Primary sponsor: Jiangsu HengRui Medicine Co., Ltd.
• Public title: Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
• Scientific title: A Phase III Multicenter Randomized Study of Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
• Date of first enrolment: March 5, 2019
• Target sample size: 240
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03825744
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 3

Countries of recruitment

China

Key inclusion & exclusion criteria

Inclusion Criteria:

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.

Exclusion Criteria:

1. Diagnosis of whole blood cell reduction due to other causes or bone marrow
hypoproliferative diseases.

2. 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.

3. Previous history of hematopoietic stem cell transplantation.

4. Subjects who is known or suspected of contraindications or hypersensitivity to
Hetrombopag's API (Active Pharmaceutical Ingredient).

5. Evidence of clonal cytogenetic abnormalities at the time of screening.

6. Bleeding and/or Infection not adequately responding to appropriate therapy.

7. 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.

8. 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).

9. 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.

10. Subjects diagnosed with cirrhosis or portal hypertension.

11. 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.

12. Subjects with deep vein thrombosis, myocardial infarction, stroke or peripheral
arterial embolization within 12 months prior to randomization.

13. Female subjects who are nursing or pregnant.

14. Subjects cannot comply with effective contraception.

15. Subjects have participated in other clinical trial within the 3 months prior to study
entry.



Age minimum: 15 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Severe Aplastic Anemia

Intervention(s)

Drug: Placebo+Standard Therapy

Drug: Hetrombopag Olamine+Standard Therapy

Primary Outcome(s)

Rate of complete hematologic response at six months. [Time Frame: 6 months]

Secondary ID(s)

HR-TPO-SAA-III

Source(s) of Monetary Support

Please refer to primary and secondary sponsors