Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia

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.

Study Overview

• Status: Completed
• Conditions: Severe Aplastic Anemia
• Intervention / Treatment: Drug: Hetrombopag Olamine+Standard Therapy; Drug: Placebo+Standard Therapy

• Study Type: Interventional
• Enrollment (Actual): 240
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Tianjin
    • Tianjin, Tianjin, China, 300041
      • Blood Diseases Hospital, Chinese Academy of medical Sciences

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 15 years to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Hetrombopag Olamine+Standard Therapy	Drug: Hetrombopag Olamine+Standard Therapy; once daily
Placebo Comparator: Placebo+Standard Therapy	Drug: Placebo+Standard Therapy; once daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Rate of complete hematologic response at six months.	6 months

Collaborators and Investigators

• Sponsor: Jiangsu HengRui Medicine Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): March 5, 2019
• Primary Completion (Actual): July 3, 2024
• Study Completion (Actual): July 3, 2024

Study Registration Dates

• First Submitted: January 30, 2019
• First Submitted That Met QC Criteria: January 30, 2019
• First Posted (Actual): January 31, 2019

Study Record Updates

• Last Update Posted (Actual): November 7, 2024
• Last Update Submitted That Met QC Criteria: November 5, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: SAA
• Additional Relevant MeSH Terms: Bone Marrow Failure Disorders; Hematologic Diseases; Bone Marrow Diseases; Anemia; Anemia, Aplastic
• Other Study ID Numbers: HR-TPO-SAA-III

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No