Autologous Stem cell Transplantation International Scleroderma (ASTIS) trial
| ISRCTN | ISRCTN54371254 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN54371254 |
| Secondary identifying numbers | NTR338 |
- Submission date
- 21/09/2005
- Registration date
- 19/10/2005
- Last edited
- 25/07/2014
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Musculoskeletal Diseases
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Plain English summary of protocol
Not provided at time of registration
Contact information
Prof Jacob M. van Laar
Scientific
Scientific
Institute of Cellular Medicine
Newcastle University
Newcastle upon Tyne
NE2 4HH
United Kingdom
| Phone | +44 (0)191 222 7139 |
|---|---|
| j.m.van-laar@ncl.ac.uk |
Study information
| Study design | Multicentre randomised active-controlled parallel-group trial |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details below to request a patient information sheet |
| Scientific title | High dose immunoablation and autologous haematopoietic stem cell transplantation versus monthly intravenous pulse therapy |
| Study acronym | ASTIS |
| Study objectives | It is postulated that high dose immunoablation and autologous stem cell transplantation has superior clinical benefit in comparison to intravenous pulse therapy cyclophosphamide, with respect to survival and prevention of major organ failure (referred to as event-free survival which is considered the primary endpoint) and has a greater impact on skin thickening, visceral involvement, functional status and quality of life. On 17/04/2012 the following changes were made to the trial record: 1. Australia has been removed from the countries of recruitment and Belgium added. 2. The target number of participants has been changed from 200 to 156. |
| Ethics approval(s) | Ethics approval received from the local medical ethics committee |
| Health condition(s) or problem(s) studied | Systemic sclerosis |
| Intervention | This multicentre prospective randomised controlled phase III study compares efficacy and safety of high dose immunoablation and autologous haematopoietic stem cell transplantation (HSCT) (considered the investigational treatment), versus monthly intravenous pulse-therapy cyclophosphamide (considered the control treatment). The investigational treatment arm comprises the following consecutive steps: 1. Mobilisation of haematopoietic stem cells with intravenous (IV) cyclophosphamide (2 x 2 g/m^2) and filgrastim (10 mg/kg/day) 2. Leukapheresis and selection of CD34+ stem cells 3. Conditioning with IV cyclophosphamide (200 mg/kg) and rbATG (7.5 mg/kg) 4. HSCT The procedures are normally completed within three to four months after randomisation. The control treatment arm consists of 12 consecutive monthly IV pulses cyclophosphamide (750 mg/m^2). As of 17/04/2012, the sponsor name has been amended from European Group Bone Marrow + Transplantation (EBMT)/European League Against Rheumatism (EULAR) Working Party Autoimmune Diseases to European Group for Bone Marrow Transplantation. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Phase III |
| Drug / device / biological / vaccine name(s) | Cyclophosphamide, filgrastim, rabbit Anti-Thymocyte Globulin (rbATG) |
| Primary outcome measure | Current primary outcome measure(s) as of 29/05/2012 The primary endpoint is event-free survival defined as the time in days from the day of randomisation until the occurrence of death or the development of persistent major organ failure (heart, lung, kidney). Previous primary outcome measure(s) The primary endpoint is event-free survival defined as the time in days from the day of randomisation until the occurrence of death or the development of persistent major organ failure (heart, lung, kidney) during the study period of two years. |
| Secondary outcome measures | Key secondary outcomes include: 1. Treatment related mortality 2. Treatment toxicity 3. Progression-free survival |
| Overall study start date | 22/03/2001 |
| Completion date | 01/01/2008 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Sex | Both |
| Target number of participants | 156 |
| Key inclusion criteria | Patients with diffuse systemic sclerosis, aged 16 to 65 years, and: 1. Disease duration four years or less, plus evidence of heart, lung or kidney involvement, plus skin score of 15 or more, or 2. Disease duration two years or less, plus evidence of an acute phase reaction in blood, plus skin score 20 or more |
| Key exclusion criteria | Patients with concomitant severe disease, extensive pretreatment according to predefined criteria with cyclophosphamide are excluded. |
| Date of first enrolment | 22/03/2001 |
| Date of final enrolment | 01/01/2008 |
Locations
Countries of recruitment
- Austria
- Belgium
- Canada
- England
- France
- Germany
- Greece
- Italy
- Netherlands
- Switzerland
- United Kingdom
Study participating centre
Institute of Cellular Medicine,
Newcastle upon Tyne
NE2 4HH
United Kingdom
NE2 4HH
United Kingdom
Sponsor information
European Group for Bone Marrow Transplantation
Other
Other
EBMT Central Office
Dept. Haematology
MacDonald Buchanan Building
Middlesex Hospital
Mortimer Street
London
W1N 8AA
United Kingdom
| Phone | +44 (0)20 7380 9317 |
|---|---|
| l.clark@ucl.ac.uk | |
| Website | http://www.ebmt.org |
Funders
Funder type
Industry
European League Against Rheumatism (EULAR)
No information available
Amgen Europe
No information available
Sangstat B.V. (The Netherlands)
No information available
Horton Foundation (Switzerland)
No information available
AP - HP
Private sector organisation / Trusts, charities, foundations (both public and private)
Private sector organisation / Trusts, charities, foundations (both public and private)
- Alternative name(s)
- Assistance Publique Hôpitaux de Paris, Assistance Publique–Hôpitaux de Paris, AP-HP
- Location
- France
European Group for Blood and Marrow Transplantation (EBMT)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Protocol article | protocol | 01/10/2005 | Yes | No | |
| Results article | results | 25/06/2014 | Yes | No |
