Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia

NCT03825744 | Completed Phase 3

• 1 other identifier: HR-TPO-SAA-III
• Study Type: interventional
• Target: 240
• Locations: 1 country
• Sites: 1

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

Keywords

SAA

Outcome Measures

Primary Outcomes (1)

• Rate of complete hematologic response at six months.

  6 months

Study Arms (2)

Hetrombopag Olamine+Standard Therapy

EXPERIMENTAL

Drug: Hetrombopag Olamine+Standard Therapy

Placebo+Standard Therapy

PLACEBO COMPARATOR

Drug: Placebo+Standard Therapy

Interventions

Hetrombopag Olamine+Standard Therapy DRUG

once daily

Hetrombopag Olamine+Standard Therapy

Placebo+Standard Therapy DRUG

once daily

Placebo+Standard Therapy

Eligibility Criteria

• Age: 15 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Unsuitable or unwilling to perform hematopoietic stem cell transplantation (HSCT).
• Signed informed consent.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Jiangsu HengRui Medicine Co., Ltd.: lead

Study Sites (1)

Blood Diseases Hospital, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300041, China

Location

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 30, 2019
• First Posted: January 31, 2019
• Study Start: March 5, 2019
• Primary Completion: July 3, 2024
• Study Completion: July 3, 2024
• Last Updated: November 7, 2024

Record last verified: 2024-01

Locations

CN (1)