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Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia

Primary Purpose

Acquired Aplastic Anaemia

Status

Completed

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

Rabbit ATG, Thymoglobuline (Genzyme)

About this trial

This is an interventional treatment trial for Acquired Aplastic Anaemia

Eligibility Criteria

16 Years - 80 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

To define aplastic anaemia there must be at least two of the following: (1) haemoglobin < 10g/dl; (2) platelet count < 50 x 109/l; (3) neutrophil count < 1.5 x 109/l, and a hypocellular bone marrow on bone marrow biopsy
Time from diagnosis to study registration ≤ 6mths
No prior treatment except for haemopoietic growth factors given for no more than 4 weeks, and androgens.
Age ≥ 16yrs (≥ 18yrs in Germany in accordance with German law), with no upper age limit.

Exclusion criteria:

Eligibility for an HLA-matched sibling donor transplant for SAA patients
Prior therapy with ATG or CSA
Haematopoeitic growth factors more than 4 weeks before study enrollment
Diagnosis of Fanconi anaemia, dyskeratosis congenita or congenital bone marrow failure syndrome
Evidence of myelodysplastic disease
Paroxysmal nocturnal haemoglobinuria with evidence of significant haemolysis, history of PNH associated thrombosis or a PNH clone > 50% by flow cytometry
Diagnosis or previous history of carcinoma (except local cervical, basal cell, squamous cells, or melanoma)
Subject is pregnant (e.g. positive HCG test) or is breast feeding
Severe uncontrolled infection or unexplained fever > 38oC
Subjects who have hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that life expectancy is less than 3 months

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment arm

Arm Description

Rabbit ATG, Thymoglobuline (Genzyme) 1.5 vials/10kg (3.75mg/kg) daily for 5 days given as an intravenous infusion over 12-18 hours. Ciclosporin (CSA) 5mg/kg/day orally from day +1 for a minimum of 6 months, with later tailing according to individual patient response. Aim to maintain trough whole blood CSA levels between 150 and 250 ng/ml.

Outcomes

Primary Outcome Measures

Response

Primary outcome is response at 6 months post ATG treatment

Secondary Outcome Measures

Over all survival

Secondary outcome variables include overall survival and failure free survival at 2 years post ATG treatment

Full Information

NCT ID

NCT01530555

First Posted

January 24, 2012

Last Updated

February 24, 2016

Sponsor

King Faisal Specialist Hospital & Research Center

1. Study Identification

Unique Protocol Identification Number

NCT01530555

Brief Title

Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia

Official Title

Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia

Study Type

Interventional

2. Study Status

Record Verification Date

February 2012

Overall Recruitment Status

Completed

Study Start Date

April 2008 (undefined)

Primary Completion Date

July 2013 (Actual)

Study Completion Date

July 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

King Faisal Specialist Hospital & Research Center

4. Oversight

5. Study Description

Brief Summary

To assess the tolerability and efficacy of rabbit antithymocyte globulin (ATG, Thymoglobuline®) with ciclosporin (CSA) in the first line treatment of patients with acquired severe aplastic anaemia (SAA), and patients with non-severe aplastic anaemia (NSAA) and who are transfusion dependent. To compare the response rate of the combination of rabbit ATG (Thymoglobuline® and CSA from this pilot study with the response rate observed in a series of matched AA patients; treated after 1994 with the combination of horse ATG (Lymphoglobuline®) and CSA; obtained from the EBMT database (comparative study).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Acquired Aplastic Anaemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

35 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Treatment arm

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Rabbit ATG, Thymoglobuline (Genzyme)

Intervention Description

Rabbit ATG, Thymoglobuline® (Genzyme) 1.5 vials/10kg (3.75mg/kg) daily for 5 days given as an intravenous infusion over 12-18 hours. Ciclosporin (CSA) 5mg/kg/day orally from day +1 for a minimum of 6 months

Primary Outcome Measure Information:

Title

Response

Description

Primary outcome is response at 6 months post ATG treatment

Time Frame

2 years

Secondary Outcome Measure Information:

Title

Over all survival

Description

Secondary outcome variables include overall survival and failure free survival at 2 years post ATG treatment

Time Frame

2 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

16 Years

Maximum Age & Unit of Time

80 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: To define aplastic anaemia there must be at least two of the following: (1) haemoglobin < 10g/dl; (2) platelet count < 50 x 109/l; (3) neutrophil count < 1.5 x 109/l, and a hypocellular bone marrow on bone marrow biopsy Time from diagnosis to study registration ≤ 6mths No prior treatment except for haemopoietic growth factors given for no more than 4 weeks, and androgens. Age ≥ 16yrs (≥ 18yrs in Germany in accordance with German law), with no upper age limit. Exclusion criteria: Eligibility for an HLA-matched sibling donor transplant for SAA patients Prior therapy with ATG or CSA Haematopoeitic growth factors more than 4 weeks before study enrollment Diagnosis of Fanconi anaemia, dyskeratosis congenita or congenital bone marrow failure syndrome Evidence of myelodysplastic disease Paroxysmal nocturnal haemoglobinuria with evidence of significant haemolysis, history of PNH associated thrombosis or a PNH clone > 50% by flow cytometry Diagnosis or previous history of carcinoma (except local cervical, basal cell, squamous cells, or melanoma) Subject is pregnant (e.g. positive HCG test) or is breast feeding Severe uncontrolled infection or unexplained fever > 38oC Subjects who have hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that life expectancy is less than 3 months

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Ahmed Al Zahrani, MD

Organizational Affiliation

King Faisal Specialist Hospital & Research Center

Official's Role

Principal Investigator

12. IPD Sharing Statement

Learn more about this trial

Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia

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