Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia

NCT ID: NCT03825744

Last Updated: 2024-11-07

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 240 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-03-05
• Study Completion Date: 2024-07-03

Brief 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.

Conditions

Severe Aplastic Anemia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Hetrombopag Olamine+Standard Therapy

Group Type: EXPERIMENTAL

Hetrombopag Olamine+Standard Therapy

Intervention Type: DRUG

once daily

Placebo+Standard Therapy

Group Type: PLACEBO_COMPARATOR

Placebo+Standard Therapy

Intervention Type: DRUG

once daily

Interventions

Hetrombopag Olamine+Standard Therapy

once daily

Intervention Type: DRUG

Placebo+Standard Therapy

once daily

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 15 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Jiangsu HengRui Medicine Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Blood Diseases Hospital, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, China

Countries

China

Other Identifiers

HR-TPO-SAA-III

Identifier Type: -

Identifier Source: org_study_id