Intensive Versus Conventional Treatment in Patients With Primary Amyloidosis

Primary Systemic Amyloidosis (AL) Clinical Trial

Official title:

Autologous Stem Cell Transplantation (ASCT) Versus Oral Melphalan and High-Dose Dexamethasone in Patients With AL (Primary)Amyloidosis. A Prospective Randomized Trial .

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00344526
• Other study ID #: I00001
• Status: Completed
• Phase: Phase 3
• First received: June 22, 2006
• Last updated: June 27, 2007
• Start date: January 2000
• Est. completion date: June 2006

Study information

• Verified date: June 2007
• Source: University Hospital, Limoges
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 100
• Est. completion date: June 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 70 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyloidosis
• Primary Systemic Amyloidosis (AL)

Intervention

Drug:
• Melphalan

• Dexamethasone

Procedure:
• Autologous stem cell transplantation

Locations

Country	Name	City	State
France	Service des Maladies du Sang	Lille
France	Service d'Hématologie et de Thérapie cellulaire	Limoges
France	Service d'hématologie clinique	Nantes
France	Service d'hématologie clinique, Hôpital Necker	Paris
France	Service d'immuno-hématologie, Hôpital Saint-Louis	Paris
France	Service d’hématologie Clinique, Groupe Hospitalier Pitié-Salpétrière	Paris
France	Service d'hématologie	Toulouse
France	Hématologie Clinique	Tours

Sponsors (2)

Lead Sponsor	Collaborator
University Hospital, Limoges	Ministry of Health, France

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	survival
Secondary	hematologic responses
Secondary	clinical responses