Randomised Study Comparing Different Dosages of Rabbit ATG in Patients With SAA

Tokai National Higher Education and Research System

Status and phase: Unknown; Phase 3

Conditions: Acquired Aplastic Anemia.

Treatments: Drug: Thymoglobulin

Study type: Interventional

Funder types: Other

Identifiers

NCT01844635

APBMT AAWG-01

Details and patient eligibility

About

The objective of this study is to evaluate the feasibility and effectiveness of immunosuppressive therapy (IST) using rabbit anti-thymocyte globulin (ATG) (Thymoglobuline, Genzyme) for patients with very severe aplastic anemia (VSAA) and severe aplastic anemia (SAA) as a primary therapy. The primary endpoint is the response rate (complete response (CR) + partial response (PR)) at day 180 after the start of IST. Secondary endpoints include evaluation of the presence and frequency of Epstein-Barr virus (EBV)-reactivation and EBV-associated lymphoproliferative disorder (EBV-LPD), Cytomegalovirus(CMV)-reactivation and CMV associated diseases, the response rate (CR+PR) on Day 360 after the start of IST, relapse rate and overall survival.

Enrollment: 320 estimated patients

Sex: All

Ages: Under 69 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Acquired aplastic anemia
• Age: younger than 70 years old
• Severity: SAA, VSAA.
• Interval between diagnosis and registration <6 months.
• Written informed consent from the caretakers and/or whenever possible consent from the patient.

Exclusion criteria

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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320 participants in 2 patient groups

2.5 mg/kg/day of Thymoglobulin for 5 days

Experimental group

Description:

2.5 mg/kg/day of Thymoglobulin for 5 days

Treatment:

Drug: Thymoglobulin

3.5 mg/kg/day of Thymoglobulin for 5 days

Active Comparator group

Description:

3.5 mg/kg/day of Thymoglobulin for 5 days

Treatment:

Drug: Thymoglobulin