Clinical trial NCT00344526: Intensive Versus Conventional Treatment in Patients With Primary Amyloidosis
Official title: Autologous Stem Cell Transplantation (ASCT) Versus Oral Melphalan and High-Dose Dexamethasone in Patients With AL (Primary)Amyloidosis. A Prospective Randomized Trial .
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Primary Systemic Amyloidosis (AL)
Keywords: AL amyloidosis
Autologous stem cell transplantation
Melphalan
Dexamethasone
Lead sponsor: University Hospital, Limoges
Sponsor class: OTHER
Collaborators: name: Ministry of Health, France
First posted: 2006-06-26
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: survival
Secondary outcomes:
measure: hematologic responses
measure: clinical responses
Other outcomes:
Locations:
city: Lille
country: France
facility: Service des Maladies du Sang
city: Limoges
country: France
facility: Service d'Hématologie et de Thérapie cellulaire
city: Nantes
country: France
facility: Service d'hématologie clinique
city: Paris
country: France
facility: Service d'hématologie Clinique, Groupe Hospitalier Pitié-Salpétrière
city: Paris
country: France
facility: Service d'hématologie clinique, Hôpital Necker
city: Paris
country: France
facility: Service d'immuno-hématologie, Hôpital Saint-Louis
city: Toulouse
country: France
facility: Service d'hématologie
city: Tours
country: France
facility: Hématologie Clinique
Study officials:
affiliation: CH Limoges
name: Arnaud Jaccard, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Jaccard A, Moreau P, Leblond V, Leleu X, Benboubker L, Hermine O, Recher C, Asli B, Lioure B, Royer B, Jardin F, Bridoux F, Grosbois B, Jaubert J, Piette JC, Ronco P, Quet F, Cogne M, Fermand JP; Myelome Autogreffe (MAG) and Intergroupe Francophone du Myelome (IFM) Intergroup. High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis. N Engl J Med. 2007 Sep 13;357(11):1083-93. doi: 10.1056/NEJMoa070484.