Comparison of anti-thymocyte globulin preparations in severe aplastic anaemia

ISRCTN	ISRCTN70251363
DOI	https://doi.org/10.1186/ISRCTN70251363
Protocol serial number	12
Sponsor	Federal Clinical Research Center of Pediatric Hematology, Oncology and Immunology (Russia)
Funder	Federal Clinical Research Center of Pediatric Hematology, Oncology and Immunology (Russia)

Submission date: 11/03/2010
Registration date: 07/04/2010
Last edited: 07/04/2010

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Haematological Disorders

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Record updated in last year

Plain English summary of protocol

Not provided at time of registration

Contact information

Prof Alexei Maschan
Scientific

Leninskii prt 117
Moscow
117997
Russian Federation

Phone	+7 495 936 91 69
Email	amaschan@mail.ru

Study information

Primary study design	Interventional
Study design	Randomised two-period cross-over study
Secondary study design	Randomised controlled trial
Study type	Participant information sheet
Scientific title	Direct randomised comparison of horse anti-thymocyte globulin and rabbit anti-thymocyte globulin in children with severe aplastic anaemia
Study objectives	Rabbit anti-thymocyte globulin (ATG) has equivalent activity compared to standard horse ATG as part of combined immune suppression in children with severe aplastic anaemia.
Ethics approval(s)	Local Ethics Committee of Research Institute of Pediatric Hematology approved on the 11th December 2000 (ref: 1-12-1999)
Health condition(s) or problem(s) studied	Severe aplastic anaemia
Intervention	Combined immunosupressive therapy with cyclosporin A and either horse ATG (ATGAM, Apjohn) - standard arm or rabbit ATG (ATG-Fresenius, Fresenius) - study arm. Horse ATG (ATGAM) was used in a standard 160 mg\kg total dose, given as four consequtive daily 40 mg\kg doses as long I.V. infusion. Rabbit ATG (ATG-Fresenius) was given 40 mg\kg total dose, given as four consequtive daily 10 mg\kg doses as long I.V. infusion. Cyclosporine A was given per os in a 5 mg\kg\day for at least 18 months total duration. Total duration of follow up is 7 years.
Intervention type	Drug
Phase	Phase IV
Drug / device / biological / vaccine name(s)	Rabbit anti-thymocyte globulin (ATG), horse ATG
Primary outcome measure(s)	1. Minimal haematologic response rate at day 180 from therapy start 2. Complete haematologic response rate, assessed at last follow-up (5 years from time of enrolment of the last patient) 3. Overall survival, assessed at last follow-up (5 years from time of enrolment of the last patient)
Key secondary outcome measure(s)	1. Relapse probability in patients who achieved haematologic response, assessed at last follow-up (5 years from time of enrolment of the last patient) 2. Event-free survival, assessed at last follow-up (5 years from time of enrolment of the last patient) 3. Toxicity, assessed at day 30 from therapy start
Completion date	01/02/2003

Eligibility

Participant type(s)	Patient
Age group	Child
Lower age limit	1 Year
Upper age limit	18 Years
Sex	All
Target sample size at registration	40
Key inclusion criteria	1. Aged from 1 - 18 years, either sex 2. Diagnosis of severe aquiered aplastic anaemia
Key exclusion criteria	1. Previous immune supressive therapy with ATG and/or cyclosporin A 2. Previous corticosteroid therapy over 2 weeks 3. Inherited bone marrow failure syndrome 4. Uncontrolled invasive fungal infection
Date of first enrolment	01/12/2000
Date of final enrolment	01/02/2003

Locations

Countries of recruitment

Russian Federation

Study participating centre

Leninskii prt 117

Moscow
117997
Russian Federation

Results and Publications

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Participant information sheet	Participant information sheet	11/11/2025	11/11/2025	No	Yes