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

• Register: ClinicalTrials.gov
• Last refreshed on: 14 March 2016
• Main ID: NCT01530555
• Date of registration: 24/01/2012
• Prospective Registration: No
• Primary sponsor: King Faisal Specialist Hospital & Research Center
• Public title: Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia
• Scientific title: Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia
• Date of first enrolment: April 2008
• Target sample size: 35
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01530555
• Study type: Interventional
• Study design: Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Contacts

• Name: Ahmed Al Zahrani, MD
• Affiliation: King Faisal Specialist Hospital & Research Center

Key inclusion & exclusion 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



Age minimum: 16 Years
Age maximum: 80 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Acquired Aplastic Anaemia

Intervention(s)

Drug: Rabbit ATG, Thymoglobuline (Genzyme)

Primary Outcome(s)

Response [Time Frame: 2 years]

Secondary Outcome(s)

Over all survival [Time Frame: 2 years]

Secondary ID(s)

2081-005

Source(s) of Monetary Support

Please refer to primary and secondary sponsors