Allo SCT in Amyloidosis Non-interventional Study

AL Amyloidosis Clinical Trial

Official title:

A Non-interventional Prospective Study on Allogeneic Stem Cell Transplantation in Patients With AL Amyloidosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02257905
• Other study ID #: 42206633
• Status: Completed
• Phase: N/A
• First received: October 2, 2014
• Last updated: July 23, 2015
• Start date: December 2009

Study information

• Verified date: July 2015
• Source: European Group for Blood and Marrow Transplantation
• Contact: n/a
• Is FDA regulated: No
• Health authority: Switzerland: Approval of EBMT registry covers this sub-projectGermany: Approval of EBMT registry covers this sub-projectFrance: Approval of EBMT registry covers this sub-project
• Study type: Observational [Patient Registry]

Clinical 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. 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.

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. primary completion date: July 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 60 Years

Eligibility

Inclusion Criteria:

• AL Amyloidosis, allogeneic HSCT after November 2009, between 18-60 years at time of transplant.

Exclusion Criteria:

• other diagnoses than AL Amyloidosis, auto transplant, transplant before November 2009, younger than 18 or older than 60 at time of transplant.

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• AL Amyloidosis
• Amyloidosis

Locations

Country	Name	City	State
France	CHRU Limoges	Limoges
Germany	University Hospital Eppendorf	Hamburg
Germany	University of Heidelberg	Heidelberg
Switzerland	University Hospital	Basel

Sponsors (1)

Lead Sponsor	Collaborator
European Group for Blood and Marrow Transplantation

Countries where clinical trial is conducted

France,  Germany,  Switzerland, 

References & Publications (2)

Schönland et al. DLI data, Haematologica, 2008.

Schönland et al. EBMT retrospective data, Blood 2005.

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	safety of allo sct in patients with AL Amyloidosis	• To evaluate safety of allogeneic SCT in patients with AL Amyloidosis (TRM, incidence of acute and chronic GvHD, non-haematological toxicity and engraftment).	5 years	Yes
Primary	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).	5 years	No
Secondary	EFS	• To evaluate event-free survival.	5 years	No
Secondary	OS	to evaluate overall survival	5 years	No