Intensive Versus Conventional Treatment in Patients With Primary Amyloidosis

Phase 3

Completed

• Conditions: Primary Systemic Amyloidosis (AL)

• Registration Number: NCT00344526
• Lead Sponsor: University Hospital, Limoges

Brief Summary

AL amyloidosis is caused by a clonal plasma cell dyscrasia and characterized by progressive deposition of amyloid fibrils derived from monoclonal Ig light chains, leading to multisystem organ failure and death. The prognosis for AL amyloidosis with conventional treatment remains poor, Autologous stem cell transplantation (ASCT) for AL amyloidosis produces high hematologic and organ responses. However, treatment-related mortality remains high and reported series are subject to selection bias.

Detailed Description

A prospective randomized trial was conducted to compare in AL amyloidosis ASCT (melphalan 140 or 200 mg/m2 depending on age and clinical status supported with ASCT collected with G-CSF alone) and the oral regimen M-Dex (melphalan 10 mg/m2 and dexamethasone 40 mg for 4 days each months up to 18 months). The objectives were to compare survival and hematologic and clinical responses.

Study Timeline

• Study Start: 2000-01
• Study Completion: 2006-06
• Study First Submitted: 2006-06-22
• Study First Submitted QC: 2006-06-23
• Study First Posted: 2006-06-26
• Status Verified: 2007-06
• Last Update Submitted: 2007-06-27
• Last Update Posted: 2007-06-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

• below 70 years of age
• biopsy proven systemic AL amyloidosis
• no more than 2 prior courses of chemotherapy
• ECOG performance status < 3
• Informed written consent

Exclusion Criteria

• localized amyloidosis
• HIV seropositivity
• previous myelodysplasia
• concomitant serious disease

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
survival

Secondary Outcome Measures

Name	Time	Method
hematologic responses
clinical responses

Trial Locations

Locations (8)

Service d'hématologie clinique; 🇫🇷 Nantes, France

Hématologie Clinique; 🇫🇷 Tours, France

Service d'immuno-hématologie, Hôpital Saint-Louis; 🇫🇷 Paris, France

Service des Maladies du Sang; 🇫🇷 Lille, France

Service d'Hématologie et de Thérapie cellulaire; 🇫🇷 Limoges, France

Service d'hématologie; 🇫🇷 Toulouse, France

Service d'hématologie Clinique, Groupe Hospitalier Pitié-Salpétrière; 🇫🇷 Paris, France

Service d'hématologie clinique, Hôpital Necker; 🇫🇷 Paris, France