Autoimmune DiseasesNISSCNCT02516124
EBMT ADWP Prospective Non Interventional Study : AutoHSCT in SSc Patients
An observational study of Autoimmune Diseases, run by European Society for Blood and Marrow Transplantation at 1 site in France.
Trial summary
The purpose of this study is to assess the effectiveness of Autologous Hematopoietic Stem Cell transplantation (AHSCT) for early severe or rapidly progressive Systemic Sclerosis (SSc) as currently performed by different study protocols used across Europe in various EBMT centres through the careful recording and… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Autoimmune Diseases
- What is being tested?
- Autologous HSCT
- Type of treatment
- Surgery or procedure
- How many people
- 82 participants
- Where
- 1 siteFrance
- Study dates
- Dec 2012to Mar 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients treated with autologous HSCT for severe systemic sclerosis
Who may be able to join
- Autologous HSCT
- Age between 18 and 65 years at time of transplant.
- Established diagnosis of progressive systemic sclerosis according to ARA-criteria
Who cannot join
- Pregnancy or inadequate contraception
- Severe concomitant disease
- Reduced lung function
- Previously damaged bone marrow
- Uncontrolled severe infection
- Severe concomitant psychiatric illness
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Autologous Stem Cell Transplantation for Progressive Systemic Sclerosis: a Prospective Non-Interventional Approach Across Europe (NISSC) for the Autoimmune Diseases Working Party of the EBMT
The purpose of this study is to assess the effectiveness of Autologous Hematopoietic Stem Cell transplantation (AHSCT) for early severe or rapidly progressive Systemic Sclerosis (SSc) as currently performed by different study protocols used across Europe in various EBMT centres through the careful recording and analysis of routinely collected clinical and biological data.
Read the detailed description
Different protocols are used in the different centres, it is not yet clear which approach will be the most efficient and the safest. Every centre will follow its own local protocol for AHSCT which usually refers to the recent update of the EBMT Guidelines for HSCT in autoimmune disease. Patient selection for AHSCT treatment technique with regard to the risk/benefit balance has to be carefully addressed by standard patient pretransplant evaluation, whereas treatment local regimen, follow-ups evaluation, supportive medication and prophylaxis will be recorded and analysed.
- Conditions
- Autoimmune Diseases
- Being tested
- Autologous HSCT
- Keywords
- Autoimmune diseases, Systemic sclerosis, Autologous Hematopoietic stem cell transplant
- Medical subject headings
- Autoimmune Diseases, Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous HSCTProcedure or surgery
1st AHSCT
| Study group | Role | What they receive |
|---|---|---|
| NISSC | Autologous HSCT Receives: Procedure: Autologous HSCT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression free survival
Progression free survival (PFS), defined as survival since Baseline (the 1st day of mobilisation) without evidence of progression of SSc.
Measured: 2 year post transplant
Secondary outcomes (6)
Safety assessed by Treatment related toxicity throughout the study period using WHO toxicity parameters (expressed as maximum grade toxicity per organ system, see appendix)
Incidence of Adverse Events (AE) and Serious Adverse Events (SE) Neutrophil and platelet engraftment, defined as first day after transplantation with absolute neutrophil count > 500 cells/μL and >20.000 platelets/μL without platelet transfusion, respectively
Measured: 2 year post transplant
Overall Survival
Overall Survival
Measured: 2 year post transplant
Response to treatment
Response to treatment within 1 year following autologous HSCT, defined as
- 25% improvement in mRSS (modified Rodnan Skin Score) and/or
- ≥10% improvement in Diffuse Capacity for carbon monoxide (DLCO) or Forced Vital Capacity (FVC) as compared to baseline without need of further immunosuppression
Measured: at 1 year post transplant
Improvement in Quality of life
Assessed by SHAQ (Scleroderma Health Assessment Questionnaire) evolution
Measured: 2 year post transplant
Relapse incidence
Defined as any of the following changes after prior response to treatment on quarterly follow up as defined below:
- Worsening of mRSS > 25%
- New/Worsening of organ manifestation: lungs, heart or kidney
Measured: 2 year post transplant
100-day Treatment related mortality
any death during 100 day following transplant that cannot be attributed to progression or relapse of the disease
Measured: 100 days post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2015 (estimated)
- Study startDec 2012
- Primary completionJan 2018
- Study completionMar 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Autoimmune DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 82 participants (actual)
Sponsor and investigators
- Lead sponsor
- European Society for Blood and Marrow Transplantation
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Dominique Farge, PhD, Study chair, EBMT ADWP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02516124
- Sponsor's protocol ID
- NCT02516124
- Acronym
- NISSC
- Status last verified
- Jul 2017
- First submitted
- 3 Aug 2015
- Last updated
- 1 May 2018
- Results posted
- No
- Data monitoring committee
- No
Publications
Henes J, Oliveira MC, Labopin M, Badoglio M, Scherer HU, Del Papa N, Daikeler T, Schmalzing M, Schroers R, Martin T, Pugnet G, Simoes B, Michonneau D, Marijt EWA, Lioure B, Olivier Bay J, Snowden JA, Rovira M, Huynh A, Onida F, Kanz L, Marjanovic Z, Farge D. Autologous stem cell transplantation for progressive systemic sclerosis: a prospective non-interventional study from the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party. Haematologica. 2021 Feb 1;106(2):375-383. doi: 10.3324/haematol.2019.230128.
PubMed 31949011
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Stem Cell Transplantation for Progressive Systemic Sclerosis: a Prospective Non-Interventional Approach Across Europe (NISSC) for the Autoimmune Diseases Working Party of the EBMT
- ClinicalTrials.gov ID
- NCT02516124
- Acronym / sponsor ID
- NISSC / NCT02516124
- Conditions
- Autoimmune Diseases
- Keywords
- Autoimmune diseases, Systemic sclerosis, Autologous Hematopoietic stem cell transplant
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 82 participants (actual)
- Lead sponsor
- European Society for Blood and Marrow TransplantationResearch network
- Study start
- Dec 2012
- Primary completion
- Jan 2018 (actual)
- Study completion
- Mar 2018 (actual)
- First posted
- 5 Aug 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the effectiveness of Autologous Hematopoietic Stem Cell transplantation (AHSCT) for early severe or rapidly progressive Systemic Sclerosis (SSc) as currently performed by different study protocols used across Europe in various EBMT centres through the careful recording and analysis of routinely collected clinical and biological data.
Detailed description
Different protocols are used in the different centres, it is not yet clear which approach will be the most efficient and the safest. Every centre will follow its own local protocol for AHSCT which usually refers to the recent update of the EBMT Guidelines for HSCT in autoimmune disease. Patient selection for AHSCT treatment technique with regard to the risk/benefit balance has to be carefully addressed by standard patient pretransplant evaluation, whereas treatment local regimen, follow-ups evaluation, supportive medication and prophylaxis will be recorded and analysed.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NISSC | – | Procedure: Autologous HSCTAutologous HSCT |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients treated with autologous HSCT for severe systemic sclerosis
Key inclusion criteria (3 listed)
- Autologous HSCT
- Age between 18 and 65 years at time of transplant.
- Established diagnosis of progressive systemic sclerosis according to ARA-criteria
Key exclusion criteria (6 listed)
- Pregnancy or inadequate contraception
- Severe concomitant disease
- Reduced lung function
- Previously damaged bone marrow
- Uncontrolled severe infection
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous HSCT1st AHSCT | Procedure or surgery | NISSC |
Primary outcome
Progression free survival
Time frame: 2 year post transplant
Progression free survival (PFS), defined as survival since Baseline (the 1st day of mobilisation) without evidence of progression of SSc.
6 secondary outcomes
Safety assessed by Treatment related toxicity throughout the study period using WHO toxicity parameters (expressed as maximum grade toxicity per organ system, see appendix)
Time frame: 2 year post transplant
Overall Survival
Time frame: 2 year post transplant
Response to treatment
Time frame: at 1 year post transplant
Improvement in Quality of life
Time frame: 2 year post transplant
Relapse incidence
Time frame: 2 year post transplant
100-day Treatment related mortality
Time frame: 100 days post transplant
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 2018.
- Derived from this study: PubMed 31949011Henes J, Oliveira MC, Labopin M, Badoglio M, Scherer HU, Del Papa N, Daikeler T, Schmalzing M, Schroers R, Martin T, Pugnet G, Simoes B, Michonneau D, Marijt EWA, Lioure B, Olivier Bay J, Snowden JA, Rovira M, Huynh A, Onida F, Kanz L, Marjanovic Z, Farge D. Autologous stem cell transplantation for progressive systemic sclerosis: a prospective non-interventional study from the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party. Haematologica. 2021 Feb 1;106(2):375-383. doi: 10.3324/haematol.2019.230128.
- Organisation websiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dominique Farge, PhD study chair
EBMT ADWP
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Autoimmune Diseases, run by European Society for Blood and Marrow Transplantation at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 May 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Progression free survival (time frame: 2 year post transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02516124, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 May 2018.