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

Acquired Aplastic Anaemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01530555
• Other study ID #: 2081-005
• Status: Completed
• Phase: Phase 2
• First received: January 24, 2012
• Last updated: February 24, 2016
• Start date: April 2008
• Est. completion date: July 2013

Study information

• Verified date: February 2012
• Source: King Faisal Specialist Hospital & Research Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal GovernmentSaudi Arabia: Ethics Committee
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 35
• Est. completion date: July 2013
• Est. primary completion date: July 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 16 Years to 80 Years

Eligibility

Inclusion Criteria:

1. 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

2. Time from diagnosis to study registration = 6mths

3. No prior treatment except for haemopoietic growth factors given for no more than 4 weeks, and androgens.

4. Age = 16yrs (= 18yrs in Germany in accordance with German law), with no upper age limit.

Exclusion criteria:

1. Eligibility for an HLA-matched sibling donor transplant for SAA patients

2. Prior therapy with ATG or CSA

3. Haematopoeitic growth factors more than 4 weeks before study enrollment

4. Diagnosis of Fanconi anaemia, dyskeratosis congenita or congenital bone marrow failure syndrome

5. Evidence of myelodysplastic disease

6. Paroxysmal nocturnal haemoglobinuria with evidence of significant haemolysis, history of PNH associated thrombosis or a PNH clone > 50% by flow cytometry

7. Diagnosis or previous history of carcinoma (except local cervical, basal cell, squamous cells, or melanoma)

8. Subject is pregnant (e.g. positive HCG test) or is breast feeding

9. Severe uncontrolled infection or unexplained fever > 38oC

10. Subjects who have hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that life expectancy is less than 3 months

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Acquired Aplastic Anaemia
• Anemia
• Anemia, Aplastic

Intervention

Drug:
• Rabbit ATG, Thymoglobuline (Genzyme)
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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
King Faisal Specialist Hospital & Research Center

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Response	Primary outcome is response at 6 months post ATG treatment	2 years	Yes
Secondary	Over all survival	Secondary outcome variables include overall survival and failure free survival at 2 years post ATG treatment	2 years	Yes