Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia

Tianjin Medical University

Status and phase: Unknown; Phase 2

Conditions: Anemia, Aplastic

Treatments: Drug: rhTPO; Drug: placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT02857530

IRB2013-066-01

Details and patient eligibility

About

Efficacy and Safety of Recombinant human thrombopoietin in patients with severe aplastic anemia and very severe aplastic anemia, a randomized, double-blind, placebo-controlled, II phase, multi-center clinical research.

Full description

rhTPO

Enrollment: 250 estimated patients

Sex: All

Ages: 6 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Able to provide written informed consent
• Have severe or very severe aplastic anemia

Exclusion criteria

• Have diagnosis of Fanconi anemia
• Have infection not adequately responding to appropriate therapy
• Have Paroxysmal Nocturnal Hemoglobinuria (PNH) clone size in neutrophils of greater than or equal to 50%
• Have creatinine and/or blood urea nitrogen (BUN) ≥2 times the upper limit of normal
• Have serum bilirubin ≥ 1.5 times the upper limit of normal, or ≥4.0 times the upper limit of normal if the patient has been treated with Anti-Thymocytes globulin（ATG） within three weeks of screening.
• Have glutamic-oxaloacetic transaminase （AST） and/or glutamic-pyruvic transaminase （ALT） ≥ 3 times the upper limit of normal
• Are female and are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential
• Have an Eastern Cooperative Oncology Group (ECOG) performance status of 3 or greater
• Have had other Thrombopoietin (TPO-R) agonists medication in the previous 4 weeks.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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250 participants in 2 patient groups, including a placebo group

rhTPO

Experimental group

Description:

rhTPO injection

Treatment:

Drug: rhTPO

control

Placebo Comparator group

Description:

without rhTPO injection

Treatment:

Drug: placebo