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

NCT01530555 | Completed Phase 2

• 1 other identifier: 2081-005
• Study Type: interventional
• Target: 35
• Locations: 0 countries
• Sites: N/A

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).

Conditions

Acquired Aplastic Anaemia

Outcome Measures

Primary Outcomes (1)

• Response

  Primary outcome is response at 6 months post ATG treatment

  2 years

Secondary Outcomes (1)

• Over all survival

  2 years

Study Arms (1)

Treatment arm

EXPERIMENTAL

1. Rabbit ATG, Thymoglobuline (Genzyme) 1.5 vials/10kg (3.75mg/kg) daily for 5 days given as an intravenous infusion over 12-18 hours. 2. 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.

Drug: Rabbit ATG, Thymoglobuline (Genzyme)

Interventions

Rabbit ATG, Thymoglobuline (Genzyme) DRUG

1. Rabbit ATG, Thymoglobuline® (Genzyme) 1.5 vials/10kg (3.75mg/kg) daily for 5 days given as an intravenous infusion over 12-18 hours. 2. Ciclosporin (CSA) 5mg/kg/day orally from day +1 for a minimum of 6 months

Treatment arm

Eligibility Criteria

• Age: 16 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• King Faisal Specialist Hospital & Research Center: lead

MeSH Terms

Interventions

thymoglobulin; Antilymphocyte Serum

Intervention Hierarchy (Ancestors)

Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures

Study Officials

• Ahmed Al Zahrani, MD

  King Faisal Specialist Hospital & Research Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 24, 2012
• First Posted: February 10, 2012
• Study Start: April 1, 2008
• Primary Completion: July 1, 2013
• Study Completion: July 1, 2013
• Last Updated: February 25, 2016

Record last verified: 2012-02