NCT01527032

Brief Summary

High-dose melphalan (MEL) with autologous stem cell transplant (SCT) is an effective therapy for systemic AL amyloidosis (AL), but treatment-related mortality (TRM) has historically been high. The investigators performed a phase II trial of risk-adapted SCT followed by adjuvant dexamethasone (dex) and thalidomide (thal) in an attempt to reduce TRM and improve response rates. Patients with newly diagnosed AL involving £2 organ systems were assigned to MEL 100, 140, or 200 mg/m2 with SCT, based on age, renal function and cardiac involvement. Patients with persistent clonal plasma cell disease 3 months post-SCT received 9 months of adjuvant thal/dex (or dex if there was a history of deep vein thrombosis or neuropathy). TRM was 4.4%. Thirty-one patients began adjuvant therapy, with 16 (52%) completing 9 months of treatment and 13 (42%) achieving an improvement in hematological response. By intention-to-treat, overall hematological response rate was 71% (36% complete response) with 44% having organ responses. With a median follow-up of 31 months, 2-year survival was 84% (95% confidence interval: 73%, 94%). Risk-adapted SCT with adjuvant thal/ dex is feasible and results in low TRM and high hematological and organ response rates in AL patients.

Trial Health

100
On Track

Trial Health Score

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

Timeline
Completed

Started Sep 2002

Typical duration for phase_2

Status
completed

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

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2002

Completed
3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2005

Completed
5.9 years until next milestone

First Submitted

Initial submission to the registry

July 28, 2011

Completed
6 months until next milestone

First Posted

Study publicly available on registry

February 6, 2012

Completed
Last Updated

March 25, 2015

Status Verified

July 1, 2011

First QC Date

July 28, 2011

Last Update Submit

March 24, 2015

Conditions

Amyloidosis

Keywords

amyloidosisstem cell transplant

Interventions

melphalan, thalidomide and dexamethasoneDRUG

Sponsors & Collaborators

MeSH Terms

Conditions

Amyloidosis

Interventions

MelphalanThalidomideDexamethasone

Condition Hierarchy (Ancestors)

Proteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Nitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhenylalanineAmino Acids, AromaticAmino Acids, CyclicAmino AcidsAmino Acids, Peptides, and ProteinsPhthalimidesPhthalic AcidsAcids, CarbocyclicCarboxylic AcidsPiperidonesPiperidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsIsoindolesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingPregnadienetriolsPregnadienesPregnanesSteroidsFused-Ring CompoundsPolycyclic CompoundsSteroids, Fluorinated

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
FED

Study Record Dates

First Submitted

July 28, 2011

First Posted

February 6, 2012

Study Start

September 1, 2002

Study Completion

September 1, 2005

Last Updated

March 25, 2015

Record last verified: 2011-07