Contact information
Type
Scientific
Primary contact
Prof Alexei Maschan
ORCID ID
Contact details
Leninskii prt 117
Moscow
117997
Russian Federation
+7 495 936 91 69
amaschan@mail.ru
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
12
Study information
Scientific title
Direct randomised comparison of horse anti-thymocyte globulin and rabbit anti-thymocyte globulin in children with severe aplastic anaemia
Acronym
Study hypothesis
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
Local Ethics Committee of Research Institute of Pediatric Hematology approved on the 11th December 2000 (ref: 1-12-1999)
Study design
Randomised two-period cross-over study
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Hospitals
Trial type
Treatment
Patient information sheet
Not available in web format, please use the contact details below to request a patient information sheet
Condition
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 names
Rabbit anti-thymocyte globulin (ATG), horse ATG
Primary outcome measures
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)
Secondary outcome measures
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
Overall trial start date
01/12/2000
Overall trial end date
01/02/2003
Reason abandoned
Eligibility
Participant inclusion criteria
1. Aged from 1 - 18 years, either sex
2. Diagnosis of severe aquiered aplastic anaemia
Participant type
Patient
Age group
Child
Gender
Both
Target number of participants
40
Participant 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
Recruitment start date
01/12/2000
Recruitment end date
01/02/2003
Locations
Countries of recruitment
Russian Federation
Trial participating centre
Leninskii prt 117
Moscow
117997
Russian Federation
Sponsor information
Organisation
Federal Clinical Research Center of Pediatric Hematology, Oncology and Immunology (Russia)
Sponsor details
Leninskii prt 117
Moscow
117997
Russian Federation
+7495 936 91 59
info@niidg.ru
Sponsor type
Research organisation
Website
Funders
Funder type
Research organisation
Funder name
Federal Clinical Research Center of Pediatric Hematology, Oncology and Immunology (Russia)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Results - basic reporting
Publication summary