AL AmyloidosisNCT02257905
Allo SCT in Amyloidosis Non-interventional Study
An observational study of AL Amyloidosis, run by European Society for Blood and Marrow Transplantation at 4 sites in 3 countries.
Trial summary
Light-chain (AL-) amyloidosis is a very rare monoclonal plasma cell disorder with poor prognosis. Rarity of disease has precluded performance of randomized controlled trials comparing various possible treatment modalities. In general, treatment of AL amyloidosis has been adapted from myeloma (MM) therapy. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- AL Amyloidosis
- How many people
- 14 participants
- Where
- 4 sitesin 3 countries
- Study dates
- Dec 2009to Jul 2015
- How long will I take part?
- 5 Years
- 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 60 years
- All sexes
- No healthy volunteers
- Adult
Study population: Male or female subjects, 18 years to 60 years, with AL amyloidosis who will receive allogeneic transplantation
Who may be able to join
- AL Amyloidosis, allogeneic HSCT after November 2009, between 18-60 years at time of transplant.
Who cannot join
- other diagnoses than AL Amyloidosis, auto transplant, transplant before November 2009, younger than 18 or older than 60 at time of transplant.
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
Germany2 sites
University Hospital Eppendorf
Hamburg
University of Heidelberg
Heidelberg
About this study
Official title: A Non-interventional Prospective Study on Allogeneic Stem Cell Transplantation in Patients With AL Amyloidosis
Light-chain (AL-) amyloidosis is a very rare monoclonal plasma cell disorder with poor prognosis. Rarity of disease has precluded performance of randomized controlled trials comparing various possible treatment modalities. In general, treatment of AL amyloidosis has been adapted from myeloma (MM) therapy. There is large experience with allo SCT in MM. Based on small series of patients and case reports allogeneic transplant has emerged as potentially effective. However, more formal proof of concept of using allogeneic hematopoietic transplantation for treatment of AL Amyloidosis is lacking.
Therefore, given the limitations of conventionally collected registry data (dubious follow-up information and extreme heterogeneity), we developed the: "EBMT non-interventional prospective study on allogeneic transplantation in AL Amyloidosis" which means that transplant centers that already do perform allogeneic transplants for AL Amyloidosis will be encouraged to register their patients with AL Amyloidosis very timely with the EBMT, followed by mandatory submission of EBMT MedB and follow-up forms. The diagnosis of AL Amyloidosis would be based on uniform criteria. All EBMT centres performing allogeneic transplants for Amyloidosis will be invited to participate in this study and centres will be asked to report all AL Amyloidosis cases referred for transplantation using a simple registration form and then to submit Amyloidosis MED B forms for each transplanted patient and follow-up forms as necessary. In conclusion, it should be possible to largely improve the usual quality of registry-based data and to generate scientifically sound knowledge on HSCT in an orphan disease such as AL Amyloidosis.
Read the detailed description
Short description of the study:
We plan to select those centres within the EBMT that in the past have performed any allogeneic transplant for amyloidosis and ask them to participate by completing a centre registration form. When they have a patient they want to include in this study, the centre will notify the CLWP data office of the planned allogeneic transplant the day before the transplant takes place. For this, the centre can use a patient registration form. Once a patient is included, MED B and regular follow-up are mandatory. Data entry of MED B forms and follow up will be performed in the way that is usual for the centre (either by the centre itself, by the national registry or by the Paris data office). The CLWP data office will keep track of the data submitted.
Research design:
This study is designed as a non-interventional prospective study.
Study Population:
Male or female subjects, 18 years to 60 years, with AL amyloidosis who will receive allogeneic transplantation.
Data to be collected/analysed:
The primary endpoint of this study is efficacy (best hematological remission and organ response) of allogeneic SCT in patients with AL amyloidosis. The secondary endpoints are acute and chronic GvHD, TRM and event-free and overall survival.
Purpose of the non-interventional prospective study request:
The main purpose of this non-interventional prospective study is the evaluation of the effectiveness of allogeneic transplant for AL Amyloidosis. It is planned that results will be presented on scientific symposia and that they will suffice for at least one publication (original article).
- Conditions
- AL Amyloidosis
- Keywords
- AL Amyloidosis, Amyloidosis, allogeneic, HSCT
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis, Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| AL Amyloidosis patients who received allo HSCT | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
efficacy of allo sct in patients with AL Amyloidosis
- To evaluate the efficacy of allogeneic SCT in patients with AL Amyloidosis (best hematological remission and organ response).
Measured: 5 years
Secondary outcomes (2)
EFS
- To evaluate event-free survival.
Measured: 5 years
OS
to evaluate overall survival
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2014 (estimated)
- Study startDec 2009
- Primary completionJul 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for AL AmyloidosisStudies 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
- 14 participants (actual)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- European Society for Blood and Marrow Transplantation
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Stefan Schoenland, MD, Principal investigator, Medizinische Klinik, University of Heidelberg, GermanyNicolaus Kroeger, MD, Study chair, BMT Centre, University Hospital Eppendorf, Hamburg, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02257905
- Sponsor's protocol ID
- 42206633
- Status last verified
- Jul 2015
- First submitted
- 2 Oct 2014
- Last updated
- 24 Jul 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Schönland et al. EBMT retrospective data, Blood 2005.
Schönland et al. DLI data, Haematologica, 2008.
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Non-interventional Prospective Study on Allogeneic Stem Cell Transplantation in Patients With AL Amyloidosis
- ClinicalTrials.gov ID
- NCT02257905
- Acronym / sponsor ID
- 42206633
- Conditions
- AL Amyloidosis
- Keywords
- AL Amyloidosis, Amyloidosis, allogeneic, HSCT
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- European Society for Blood and Marrow TransplantationResearch network
- Study start
- Dec 2009
- Primary completion
- Jul 2015 (actual)
- First posted
- 7 Oct 2014 (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.
Light-chain (AL-) amyloidosis is a very rare monoclonal plasma cell disorder with poor prognosis. Rarity of disease has precluded performance of randomized controlled trials comparing various possible treatment modalities. In general, treatment of AL amyloidosis has been adapted from myeloma (MM) therapy. There is large experience with allo SCT in MM. Based on small series of patients and case reports allogeneic transplant has emerged as potentially effective. However, more formal proof of concept of using allogeneic hematopoietic transplantation for treatment of AL Amyloidosis is lacking.
Therefore, given the limitations of conventionally collected registry data (dubious follow-up information and extreme heterogeneity), we developed the: "EBMT non-interventional prospective study on allogeneic transplantation in AL Amyloidosis" which means that transplant centers that already do perform allogeneic transplants for AL Amyloidosis will be encouraged to register their patients with AL Amyloidosis very timely with the EBMT, followed by mandatory submission of EBMT MedB and follow-up forms. The diagnosis of AL Amyloidosis would be based on uniform criteria. All EBMT centres performing allogeneic transplants for Amyloidosis will be invited to participate in this study and centres will be asked to report all AL Amyloidosis cases referred for transplantation using a simple registration form and then to submit Amyloidosis MED B forms for each transplanted patient and follow-up forms as necessary. In conclusion, it should be possible to largely improve the usual quality of registry-based data and to generate scientifically sound knowledge on HSCT in an orphan disease such as AL Amyloidosis.
Detailed description
Short description of the study:
We plan to select those centres within the EBMT that in the past have performed any allogeneic transplant for amyloidosis and ask them to participate by completing a centre registration form. When they have a patient they want to include in this study, the centre will notify the CLWP data office of the planned allogeneic transplant the day before the transplant takes place. For this, the centre can use a patient registration form. Once a patient is included, MED B and regular follow-up are mandatory. Data entry of MED B forms and follow up will be performed in the way that is usual for the centre (either by the centre itself, by the national registry or by the Paris data office). The CLWP data office will keep track of the data submitted.
Research design:
This study is designed as a non-interventional prospective study.
Study Population:
Male or female subjects, 18 years to 60 years, with AL amyloidosis who will receive allogeneic transplantation.
Data to be collected/analysed:
The primary endpoint of this study is efficacy (best hematological remission and organ response) of allogeneic SCT in patients with AL amyloidosis. The secondary endpoints are acute and chronic GvHD, TRM and event-free and overall survival.
Purpose of the non-interventional prospective study request:
The main purpose of this non-interventional prospective study is the evaluation of the effectiveness of allogeneic transplant for AL Amyloidosis. It is planned that results will be presented on scientific symposia and that they will suffice for at least one publication (original article).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AL Amyloidosis patients who received allo HSCT | – | – |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Male or female subjects, 18 years to 60 years, with AL amyloidosis who will receive allogeneic transplantation
Key inclusion criteria (1 listed)
- AL Amyloidosis, allogeneic HSCT after November 2009, between 18-60 years at time of transplant.
Key exclusion criteria (1 listed)
- other diagnoses than AL Amyloidosis, auto transplant, transplant before November 2009, younger than 18 or older than 60 at time of transplant.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
efficacy of allo sct in patients with AL Amyloidosis
Time frame: 5 years
• To evaluate the efficacy of allogeneic SCT in patients with AL Amyloidosis (best hematological remission and organ response).
2 secondary outcomes
EFS
Time frame: 5 years
OS
Time frame: 5 years
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 24 Jul 2015.
- BackgroundSchönland et al. EBMT retrospective data, Blood 2005.
- BackgroundSchönland et al. DLI data, Haematologica, 2008.
Related trials for AL Amyloidosis
Not yet recruitingEarly Phase 1
A Pilot Study of Anselamimab in Patients With AL Amyloidoma and Measurable Tissue Involvement
AL Amyloidosis
RecruitingObservational study
Screening for AL Amyloidosis in Smoldering Multiple Myeloma
Smoldering Multiple Myeloma
Active, not recruitingObservational study
Infection Prophylaxis and Management in Treating Cytomegalovirus (CMV) Infection in Patients With Hematologic Malignancies Previously Treated With Donor Stem Cell Transplant
Hematopoietic/Lymphoid Cancer, Accelerated Phase Chronic Myelogenous Leukemia
Not yet recruitingPhase 1
Phase 1a/b Study of GPRC5D CART Therapy in Relapsed/Refractory Multiple Myeloma and Light-chain Amyloidosis
Relapsed/Refractory Multiple Myeloma (RRMM), Light-Chain Amyloidosis
Active, not recruitingPhase 1
Study of Captisol Enabled Melphalan and Pharmacokinetics for Patients With Multiple Myeloma or Light Chain Amyloidosis That Are Receiving an Autologous Transplant.
Multiple Myeloma, Amyloidosis
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.
Stefan Schoenland, MD principal investigator
Medizinische Klinik, University of Heidelberg, Germany
Nicolaus Kroeger, MD study chair
BMT Centre, University Hospital Eppendorf, Hamburg, Germany
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of AL Amyloidosis, run by European Society for Blood and Marrow Transplantation at 4 sites in 3 countries.
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 1 inclusion and 1 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 24 Jul 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Germany, France, Switzerland. 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?
efficacy of allo sct in patients with AL Amyloidosis (time frame: 5 years). 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 NCT02257905, which also lists 2 linked publications.
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 24 Jul 2015.