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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified February 2012 by King Faisal Specialist Hospital & Research Center.
Recruitment status was  Active, not recruiting
Sponsor:
Information provided by (Responsible Party):
King Faisal Specialist Hospital & Research Center
ClinicalTrials.gov Identifier:
NCT01530555
First received: January 24, 2012
Last updated: February 9, 2012
Last verified: February 2012
  Purpose

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).


Condition Intervention Phase
Acquired Aplastic Anaemia
Drug: Rabbit ATG, Thymoglobuline (Genzyme)
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by King Faisal Specialist Hospital & Research Center:

Primary Outcome Measures:
  • Response [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
    Primary outcome is response at 6 months post ATG treatment


Secondary Outcome Measures:
  • Over all survival [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
    Secondary outcome variables include overall survival and failure free survival at 2 years post ATG treatment


Estimated Enrollment: 35
Study Start Date: April 2008
Estimated Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment arm
  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)
  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

  Eligibility

Ages Eligible for Study:   16 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01530555

Sponsors and Collaborators
King Faisal Specialist Hospital & Research Center
Investigators
Principal Investigator: Ahmed Al Zahrani, MD King Faisal Specialist Hospital & Research Center
  More Information

No publications provided

Responsible Party: King Faisal Specialist Hospital & Research Center
ClinicalTrials.gov Identifier: NCT01530555     History of Changes
Other Study ID Numbers: 2081-005
Study First Received: January 24, 2012
Last Updated: February 9, 2012
Health Authority: United States: Federal Government
Saudi Arabia: Ethics Committee

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases
Antilymphocyte Serum
Cyclosporins
Cyclosporine
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Immunosuppressive Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antifungal Agents
Anti-Infective Agents
Therapeutic Uses
Dermatologic Agents
Antirheumatic Agents

ClinicalTrials.gov processed this record on August 19, 2014