NCT02257905

Brief 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.

Trial Health

85
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for all trials

Geographic Reach
3 countries

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2009

Completed
4.8 years until next milestone

First Submitted

Initial submission to the registry

October 2, 2014

Completed
5 days until next milestone

First Posted

Study publicly available on registry

October 7, 2014

Completed
9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2015

Completed
Last Updated

July 24, 2015

Status Verified

July 1, 2015

Enrollment Period

5.6 years

First QC Date

October 2, 2014

Last Update Submit

July 23, 2015

Conditions

AL Amyloidosis

Keywords

AL AmyloidosisAmyloidosisallogeneicHSCT

Outcome Measures

Primary Outcomes (1)

  • 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

Secondary Outcomes (2)

  • EFS

    5 years

  • OS

    5 years

Other Outcomes (1)

  • safety of allo sct in patients with AL Amyloidosis

    5 years

Study Arms (1)

AL Amyloidosis patients who received allo HSCT

Eligibility Criteria

Age18 Years - 60 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)
Sampling MethodProbability Sample
Study Population

Male or female subjects, 18 years to 60 years, with AL amyloidosis who will receive allogeneic transplantation

You may qualify if:

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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

CHRU Limoges

Limoges, France

Location

University Hospital Eppendorf

Hamburg, Germany

Location

University of Heidelberg

Heidelberg, Germany

Location

University Hospital

Basel, Switzerland

Location

Related Publications (2)

  • Schönland et al. EBMT retrospective data, Blood 2005.

    BACKGROUND

  • Schönland et al. DLI data, Haematologica, 2008.

    BACKGROUND

MeSH Terms

Conditions

Immunoglobulin Light-chain AmyloidosisAmyloidosis

Condition Hierarchy (Ancestors)

Neoplasms, Plasma CellNeoplasms by Histologic TypeNeoplasmsProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesLymphoproliferative DisordersImmunoproliferative DisordersImmune System DiseasesParaproteinemias

Study Officials

  • Stefan Schoenland, MD

    Medizinische Klinik, University of Heidelberg, Germany

    PRINCIPAL INVESTIGATOR

  • Nicolaus Kroeger, MD

    BMT Centre, University Hospital Eppendorf, Hamburg, Germany

    STUDY CHAIR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
5 Years
Sponsor Type
NETWORK
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 2, 2014

First Posted

October 7, 2014

Study Start

December 1, 2009

Primary Completion

July 1, 2015

Last Updated

July 24, 2015

Record last verified: 2015-07

Locations

CH(1)DE(2)FR(1)